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Celebrating Diversity Within the Sickle Cell Community: Commitment, Innovation, Practice

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Wednesday, October 10
 

12:00pm

Novartis Advisory Board
Wednesday October 10, 2018 12:00pm - 5:00pm
Pratt/Calvert

12:00pm

HRSA Grant Team Meeting
Invitation Only

Wednesday October 10, 2018 12:00pm - 5:30pm
Pisces

4:00pm

Prolong Focus Group
Wednesday October 10, 2018 4:00pm - 8:00pm
President

5:30pm

Executive Leadership Council Meeting/Member Training
Invitation Only

Wednesday October 10, 2018 5:30pm - 8:00pm
Piesces

7:00pm

Get Connected Kiosk
Wednesday October 10, 2018 7:00pm - 9:00pm
Broadcast Lounge B 300 Light Street, baltimore, MD 21202

7:00pm

Registration
Registering Speakers

Wednesday October 10, 2018 7:00pm - 9:00pm
Constellation Foyer

8:00pm

SCDAA Board Meeting
Wednesday October 10, 2018 8:00pm - 10:00pm
Chesapeake A 300 Light Street, baltimore, MD 21202
 
Thursday, October 11
 

7:00am

General Evaluation Form
Thursday October 11, 2018 7:00am - 9:00pm

7:00am

Nursing Evaluation Form
Thursday October 11, 2018 7:00am - 9:00pm

7:00am

Social Worker Evaluation Form
Thursday October 11, 2018 7:00am - 9:00pm

7:30am

Breakfast
Thursday October 11, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Speed Networking
Thursday October 11, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Super Power Hour
Thursday October 11, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

8:00am

SCDAA Business Session
8:00AM-8:10AM             Genice Nelson- Roll Call Secretary
8:10AM-8:20AM             Voting
8:20AM-8:30AM             David Braxton, PhD- Board Chair Report
8:30AM-8:40AM             Beverley Francis-Gibson - President's Report
8:40AM-8:50AM             Biree Andemariam, MD- CMO Report
8:50AM-9:00AM             JaKela Parker - Treasure Report
9:00AM-9:15AM              Q&A

Thursday October 11, 2018 8:00am - 9:15am
Constellation A

8:00am

Prolong Focus Group
Thursday October 11, 2018 8:00am - 7:00pm
Pratt/Calvert

9:15am

10:00am

Break
Intermission

Thursday October 11, 2018 10:00am - 10:30am
Constellation EF

10:30am

Treatment Demonstration Update
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Speakers
RB

Rosemary Britt

Executive Director, Sickle Cell Association


Thursday October 11, 2018 10:30am - 11:00am
Constellation A

10:30am

Plenary Session I Community Based Organization Highlights
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Thursday October 11, 2018 10:30am - 12:00pm
Constellation AB

11:00am

Newborn Screening Grant Update
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Speakers
avatar for Kathy Norcott

Kathy Norcott

Executive Director, Piedmont Health Services and Sickle Cell Agency
Kathy M. Norcott presently serves as the Executive Director of Piedmont Health Services and Sickle Cell Agency. While she has served in this role for only 14 months, she has been employed with the agency for 41 years. Ms. Norcott earned her undergraduate degree in Managerial Psychology... Read More →


Thursday October 11, 2018 11:00am - 11:30am
Constellation A

11:30am

PCORI Grant Update
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Speakers
avatar for Mary Lamar

Mary Lamar

Founder & Executive Director, The Sickle Cell Association of New Jersey, Inc. (SCANJ) A 501c3 Charitable Organization
Founder and Executive Director of the Sickle Cell Association of New Jersey, Inc., is an award winning leader with a reputation for successfully “making it happen.” This graduate of Indiana University in Bloomington Indiana is dedicated to improving conditions for those living... Read More →


Thursday October 11, 2018 11:30am - 12:00pm
Constellation A

12:00pm

Executive Leadership Luncheon
Invitation Only

Thursday October 11, 2018 12:00pm - 1:10pm
President

12:00pm

Hemolytic Anemia in Sickle Cell Disease: What is it and Why Does it Matter?
Sponsored by GBT.

Lunch and Learn.  Lunch provided for everyone.


Thursday October 11, 2018 12:00pm - 1:10pm
Constellation AB

1:15pm

Developing My Healthcare Passport
Moderators
avatar for Caterina Minnitti, MD

Caterina Minnitti, MD

Director, The Children’s Hospital of Montefiore
Dr. Minniti is Professor of Medicine and Pediatrics at Einstein College of Medicine.  She is the Director of the Sickle Cell Center for Adults at Montefiore Hospital, whose mission is to provide exceptional, seamless, comprehensive, compassionate and individualized care, education... Read More →

Speakers
avatar for André Marcel Harris, CHW

André Marcel Harris, CHW

Social Worker, Student
André Marcel Harris is a Bachelor of Social Work (BSW) student and a Ronald E. McNair Scholar at Fayetteville State University in Fayetteville, North Carolina. Upon completion of his BSW, Mr. Harris aspires to pursue a Master of Social Work/Master of Public Health (MSW/MPH) and a... Read More →


Thursday October 11, 2018 1:15pm - 1:45pm
Frederick/Columbia

1:15pm

Telementoring
Moderators
Speakers
avatar for Sophie Lanzkron, MD, MHS

Sophie Lanzkron, MD, MHS

Associate Professor, Associate Professor of Medicine and Oncology
Dr. Sophie Lanzkron is an associate professor of medicine at the Johns Hopkins University School of Medicine. Her areas of clinical expertise include hematology and sickle cell disease. Dr. Lanzkron is director of the Sickle Cell Center for Adults at Johns Hopkins.Dr. Lanzkron received... Read More →


Thursday October 11, 2018 1:15pm - 1:45pm
Constellation D

1:15pm

Using Implementation Science to Develop and Evaluate Psychosocial Interventions in SCD
Speakers
avatar for Lori Crosby, PsyD

Lori Crosby, PsyD

Professor of Pediatrics, Cincinnati Children’s Hospital Medical Center
Lori E. Crosby, PsyD, is a Professor in the Division of Behavioral Medicine and Clinical Psychology at Cincinnati Children’s Hospital Medical Center (CCHMC) and Department of Pediatrics at the University of Cincinnati College of Medicine. Dr. Crosby is also Co-Director of the Cincinnati... Read More →


Thursday October 11, 2018 1:15pm - 1:45pm
Baltimore/ Annapolis

1:15pm

Virtual Reality as an Adjunct Pain Management Therapy
Moderators
avatar for Genice T. Nelson, DNP, APRN, ANP-BC

Genice T. Nelson, DNP, APRN, ANP-BC

Nurse Practitioner, UT Southwestern Medical Center Comprehensive Sickle Cell Disease Program

Speakers
avatar for Latika Puri, MD

Latika Puri, MD

Assitant Member St. Jude Faculty, St. Jude Children's Research Hospital


Thursday October 11, 2018 1:15pm - 1:55pm
Constellation C

1:15pm

Community Health Worker Training
Pre-registration Required

Thursday October 11, 2018 1:15pm - 3:15pm
Camden/Lombard

1:15pm

Concurrent Workshop
Thursday October 11, 2018 1:15pm - 3:15pm

1:15pm

Sickle Cell Basics
Speakers
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →


Thursday October 11, 2018 1:15pm - 3:15pm
Constellation AB

1:45pm

Hydroxyurea and Transfusions
Moderators
avatar for Caterina Minnitti, MD

Caterina Minnitti, MD

Director, The Children’s Hospital of Montefiore
Dr. Minniti is Professor of Medicine and Pediatrics at Einstein College of Medicine.  She is the Director of the Sickle Cell Center for Adults at Montefiore Hospital, whose mission is to provide exceptional, seamless, comprehensive, compassionate and individualized care, education... Read More →

Speakers
avatar for Andrew Campbell, MD

Andrew Campbell, MD

Associate Professor, Children’s National
Dr. Andrew Campbell's distinguished training and career path began at Morehouse College. He continued medical school at Case Western Reserve University and completed post graduate training at Massachusetts General Hospital (Harvard) and Lurie Children's Hospital (Northwestern University... Read More →


Thursday October 11, 2018 1:45pm - 2:15pm
Frederick/Columbia

1:45pm

Chronic Pain
Moderators
Speakers
avatar for Deepika Darbari, MD

Deepika Darbari, MD

Attending Physician, Associate Professor, Childrens National


Thursday October 11, 2018 1:45pm - 2:15pm
Constellation D

1:45pm

Examining Spiritual Well Being During Health Care Transition for Adolescents and Emerging Adults with Sickle Cell Disease
Moderators
Speakers
avatar for Dora Clayton-Jones, RN, PhD, CPNP-PC

Dora Clayton-Jones, RN, PhD, CPNP-PC

Assitant Professor/Adjunct Faculty, College of Nursing Marquette University, Medical College of Wisconsin
Dr. Dora Clayton-Jones, PhD, RN, CPNP-PC is an assistant professor at Marquette University in the College of Nursing and an adjunct faculty at the Medical College of Wisconsin. She completed her PhD and MS degrees in nursing at Marquette University. As a board-certified pediatric... Read More →


Thursday October 11, 2018 1:45pm - 2:15pm
Baltimore/ Annapolis

1:55pm

The Opioid Crisis and Nursing Ramifications
Moderators
avatar for Genice T. Nelson, DNP, APRN, ANP-BC

Genice T. Nelson, DNP, APRN, ANP-BC

Nurse Practitioner, UT Southwestern Medical Center Comprehensive Sickle Cell Disease Program

Speakers
avatar for Yvonne Carroll, RN, JD

Yvonne Carroll, RN, JD

Director of Patient Services in the Department of hematology at St. Jude Childrens Research Hospital, St. Jude Children's Research Hospital
Yvonne M. Carroll, RN, JD is the Director of Patient Services in the Department of Hematology at St. Jude Children’s Research Hospital.  Yvonne graduated with a Bachelors of Nursing Degree from the University of Tennessee and a Law degree from the University of Cincinnati. She... Read More →


Thursday October 11, 2018 1:55pm - 2:35pm
Constellation C

2:15pm

Effective Transition
Moderators
avatar for Caterina Minnitti, MD

Caterina Minnitti, MD

Director, The Children’s Hospital of Montefiore
Dr. Minniti is Professor of Medicine and Pediatrics at Einstein College of Medicine.  She is the Director of the Sickle Cell Center for Adults at Montefiore Hospital, whose mission is to provide exceptional, seamless, comprehensive, compassionate and individualized care, education... Read More →

Speakers
avatar for Leila Jerome Clay, MD, MCTS

Leila Jerome Clay, MD, MCTS

Director, Johns Hopkins ALL Children's Hospital
Dr. Clay is the Director of the Sickle Cell Disease Program at John Hopkins All Children’s Hospital and Assistant Professor of Pediatrics, Section of Hematology at Johns Hopkins University School of MedicineDr. Clay received her Bachelor’s degree in Physical Anthropology from... Read More →


Thursday October 11, 2018 2:15pm - 2:45pm
Frederick/Columbia

2:15pm

Transplantation Updates
Moderators
Speakers
avatar for Courtney Fitzhugh, MD

Courtney Fitzhugh, MD

Attending Physician, The National Institutes of Health


Thursday October 11, 2018 2:15pm - 2:45pm
Constellation D

2:15pm

Peer Mentoring vs. Structured Education to Improve Transition to Adult Care in SCD
Moderators
Speakers
avatar for Ify Osunkwo, MD, MPH

Ify Osunkwo, MD, MPH

Medical Director,Clinical Associate Professor, Carolinas Health Care System, Levine Cancer Institute
Ifeyinwa Osunkwo, MD, MPH, is medical director of the sickle cell program at Levine Cancer Institute/Atrium Health in Charlotte, North Carolina. She has over 2 decades of clinical and research experience in sickle cell disease (SCD). She is board certified in pediatrics and pediatric... Read More →


Thursday October 11, 2018 2:15pm - 2:45pm
Baltimore/ Annapolis

2:35pm

The Opioid Crisis- Non opioid Pain Management Treatment Options
Moderators
avatar for Genice T. Nelson, DNP, APRN, ANP-BC

Genice T. Nelson, DNP, APRN, ANP-BC

Nurse Practitioner, UT Southwestern Medical Center Comprehensive Sickle Cell Disease Program

Thursday October 11, 2018 2:35pm - 3:15pm
Constellation C

2:45pm

Chronic Organ Damage
Moderators
avatar for Caterina Minnitti, MD

Caterina Minnitti, MD

Director, The Children’s Hospital of Montefiore
Dr. Minniti is Professor of Medicine and Pediatrics at Einstein College of Medicine.  She is the Director of the Sickle Cell Center for Adults at Montefiore Hospital, whose mission is to provide exceptional, seamless, comprehensive, compassionate and individualized care, education... Read More →

Thursday October 11, 2018 2:45pm - 3:15pm
Frederick/Columbia

2:45pm

Managing Sickle Cell Disease in Older Adults
Moderators
Speakers
avatar for Swee Lay Thein, MD

Swee Lay Thein, MD

Chief, Sickle Cell Branch, National Heart, Lung and Blood Insitute
Swee Lay Thein was educated in both Malaysia and the United Kingdom. She completed her specialist training in hematology at the U.K. Royal Postgraduate Medical School, Hammersmith, and the Royal Free Hospital, London. In 1982, she joined the U.K. Medical Research Council Molecular... Read More →


Thursday October 11, 2018 2:45pm - 3:15pm
Constellation D

2:45pm

Community Health Workers vs. A Mobile App to Improve Transition to Adult Care in SCD
Moderators
Speakers
avatar for Kim Smith-Whitley, MD

Kim Smith-Whitley, MD

Director, Clinical Director, The Children’s Hospital of Philadelphia
Dr. Smith-Whitley is associate professor of the Sickle Cell Clinical Program and clinical director of the Division of Hematology and the director of the Comprehensive Sickle Cell Center at The Children's Hospital of Philadelphia (CHOP). .Dr. Smith-Whitley received her medical degree... Read More →


Thursday October 11, 2018 2:45pm - 3:15pm
Baltimore/ Annapolis

3:15pm

Break
Intermission

Thursday October 11, 2018 3:15pm - 3:30pm
Constellation EF

3:30pm

PLENARY II Clinical Trial Update
3:30PM-3:45PM                  Dr. Biree Andemariam
3:45PM-4:00PM                 GBT
4:00PM-4:15PM                 Emmaus
4:15PM-4:30PM                 Imara

 4:30PM-4:45PM                Pfizer
4:45PM-5:00PM                 Novartis
5:00PM-5:15PM                 Bluebird bio
5:15PM-5:30PM                 Bioverativ
5:30PM-5:45PM                 Ironwood Pharmaceuticals 




Thursday October 11, 2018 3:30pm - 5:30pm
Constellation A

4:00pm

PCORI
Speakers
avatar for Lori Crosby, PsyD

Lori Crosby, PsyD

Professor of Pediatrics, Cincinnati Children’s Hospital Medical Center
Lori E. Crosby, PsyD, is a Professor in the Division of Behavioral Medicine and Clinical Psychology at Cincinnati Children’s Hospital Medical Center (CCHMC) and Department of Pediatrics at the University of Cincinnati College of Medicine. Dr. Crosby is also Co-Director of the Cincinnati... Read More →


Thursday October 11, 2018 4:00pm - 5:00pm
Camden/Lombard

6:00pm

Poster Session
FOR INFORMATION ON POSTER PRESENTERS, SEE THE DIRECTORY

Thursday October 11, 2018 6:00pm - 7:00pm
Atrium

7:00pm

 
Friday, October 12
 

7:00am

General Evaluation Form
Friday October 12, 2018 7:00am - 9:00pm

7:00am

Nursing Evaluation Form
Friday October 12, 2018 7:00am - 9:00pm

7:00am

Social Worker Evaluation Form
Friday October 12, 2018 7:00am - 9:00pm

7:30am

Breakfast/ Poster Viewing
Friday October 12, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Super Power Hour
Friday October 12, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

8:30am

8:30am

Real Life Experiece wiith Transplantation: Patient and Family Perspective
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Friday October 12, 2018 8:30am - 9:30am
Constellation A

9:30am

Charles F. Whitten, MD Memorial Lecture
Moderators
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →

Speakers
avatar for M. Dawn Nelson, Ph.D, CCC-A

M. Dawn Nelson, Ph.D, CCC-A

Associate Professor, Central Michigan University
M. Dawn Nelson, Ph.D., CCC-AAssociate professorCentral Michigan UniversityDr. M. Dawn Nelson is an Associate Professor at Central Michigan University in the Department of Communication Sciences and Disorders, Doctor of Audiology (Au.D) Program.  She received her Masters degree from... Read More →


Friday October 12, 2018 9:30am - 10:00am
Constellation A

10:00am

Diversity Panel LaTonya Kittles, Pfizer
Friday October 12, 2018 10:00am - 11:30am
Constellation A

11:30am

11:30am

Community Health Worker Training
Friday October 12, 2018 11:30am - 12:30pm
President

11:30am

Healing the Healer Provider Session
Sponsored by Bluebird Bio

Speakers
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →


Friday October 12, 2018 11:30am - 1:15pm
Constellation C

11:30am

Prolong Focus Group
Friday October 12, 2018 11:30am - 2:30pm
Pratt/Calvert

11:30am

Patient Family Session I
Friday October 12, 2018 11:30am - 3:15pm
Pratt/Calvert

12:00pm

Finding your Voice between the Patient and Caregiver



Speakers
avatar for Velvet Brown Watts, MSW, CM

Velvet Brown Watts, MSW, CM

Master Level Social Worker, Oklahoma Healthcare Authority (Supporters of Families with Sickle Cell Disease, Inc.)
Velvet Brown-Watts is the Founder and Chairperson of Supporters of Families with Sickle Cell Disease, Inc. Supporters (as it is affectionately known) is a community-based Oklahoma nonprofit organization that has assisted families living with sickle cell and its inherited diseases... Read More →
avatar for Trudy Tchume-Johnson, LSW

Trudy Tchume-Johnson, LSW

Social Work Manager, Children's Hospital of Philadelphia Division of Hematology-Comprhensive Sickle Cell Center
Trudy Tchume-Johnson, MSW, LSW, is the social work Manager in the Division of Hematology and a social worker in the Comprehensive Sickle Cell Center at Children's Hospital of Philadelphia.Areas of Expertise: Providing psychosocial support for chronically ill children with sickle cell... Read More →


Friday October 12, 2018 12:00pm - 12:30pm
Baltimore/ Annapolis

12:00pm

American Red Cross (Luncheon)
Lunch Break

Friday October 12, 2018 12:00pm - 1:20pm
Frederick/Columbia

12:00pm

Prolong Focus Group Luncheon
Friday October 12, 2018 12:00pm - 1:20pm
Camden/Lombard

12:15pm

Lunch on Your Own
Lunch Break

Friday October 12, 2018 12:15pm - 1:15pm

12:15pm

IASCHAA
Friday October 12, 2018 12:15pm - 1:30pm
Constellation D

1:30pm

3:00pm

Break
Intermission

Friday October 12, 2018 3:00pm - 4:00pm
Constellation EF

3:15pm

Effect Of Long Term Treatment With Sc411 On Sickle Cell Crisis Rate In Children With Sickle Cell Disease
Abstract 

Authors:
Dr. Ofelia Alvarez- University of Miami, Dr. Ahmed Daak- SCI, Dr. Matthew Heeney- Dana-Farber / Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Dr. Beng Fuh- East Carolina University Department of Pediatrics, Dr. Carlton Dampier-Emory University School of Medicine, Dr. Julie Kanter- Division of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA, Dr. Vandy Black- University of Florida College of Medicine, Dr. Melissa Mcnaull- University of Mississippi Medical Center, Dr. Michael Callaghan- Children's Hospital of Michigan, Dr. Alex George- Texas Children’s Hospital

Background:
Docosahexaenoic acid (DHA) is a bioactive omega-3 fatty acid known to have potent anti-inflammatory, anti-blood cell adhesion and anti-oxidant properties. There is evidence that treatment with DHA reduces the rate of sickle cell crises (SCC) and ameliorates the chronic inflammation, oxidative stress and hypercoagulable state observed in patients with SCD. SC411 is a novel DHA ethyl ester formulation with a proprietary delivery platform (Advanced Lipid Technology® (ALT®)) that enhances DHA bioavailability. The SCOT trial is a phase 2 randomized placebo-controlled double-blinded study followed by an open label extension phase (OLE).
Methods:
Children with SCD (HbSS, HbSC or S/β°‑thalassemia) aged 5-17 years (n=67), who had ≥2 - ≤10 documented Sickle Cell Crises (SCC) during the 12 months prior to screening, and either had not received or who were on hydroxyurea (HU) for at least six months prior to screening were enrolled. Subjects received either placebo or one of three doses of SC411 (20 [12-26]; 36 [26-48] or 60 [51-72] mg/kg/day DHA) for two months. Subjects who consented to the OLE were rolled into the lowest dose level (20 mg/Kg). The annualized rates of SCC against baseline were analyzed using a Poisson regression model with a log-link and with treatment, baseline disease severity, age group, and hydroxyurea use as covariates, and subject as a repeated measure. The model uses the log of (days in the study / 365) as the offset variable to produce the annualized rates. The percentage of subjects who remained crisis-free for at least 300 days post-randomization at the time of this analysis, was also reported. Safety and tolerability are reported.
Results:
A total of 67 subjects, 34 of whom are female (50.7%), were randomized to one of the three active groups (n=50) or placebo (n=17). Subjects on HU treatment constituted 76% of total study population. Sixty-two subjects (93%) completed the blinded portion of the study. Forty-one subjects (66%) opted into the OLE. Of all subjects in the OLE, seven (17.07%) received placebo during the blinded part of the study. At the time of this analysis, thirty-eight subjects have completed at least 300 days in the study and thirteen patients have completed one year. No unexpected or treatment-related adverse events were reported.
The median annualized rate of SCC at baseline for the subjects who enrolled into the study (n=67) or the OLE (n=41) was 3.00. The rate ratios of SCC of the year post versus the year prior to randomization were 0.560 (95% confidence interval [CI]: 0.39 to 0.80; p=0.0016) for the group who was on active drug during the blinded part (n=34), and 0.53 (CI: 0.66 to 0.79, p=0.0017) for patients who were on active treatment for a period of at least 6 months (n=32).
300 days after randomization, 40% of the patients (15/38) remained crisis-free. Of the 13 patients that have completed over one year of treatment, five (38.46%) are crisis free.
Conclusions:
The SCOT OLE study provides additional evidence that SC411 is safe and well-tolerated. Subjects on a dose of 20 mg/Kg DHA of SC411 experienced a significantly lower rate of sickle cell crises compared to the year prior to enrollment.

Speakers
avatar for Beng Fuh

Beng Fuh

Associate Professor, East Carolina University Department of Pediatrics
Dr. Fuh is an Associate Professor of Pediatrics at East Carolina University in Greenville, NC.He was born in Cameroon, and attended Medical School at the Philipps University in Marburg, Germany. He completed his residency in Pediatrics at Howard University and Children’s National... Read More →


Friday October 12, 2018 3:15pm - 3:30pm
Constellation C

3:15pm

Patient and Caregiver Perspectives on Areas of Burden In Sickle Cell Disease Management
Abstract

Authors:
Dr. Tilicia Mayo-Gamble- Georgia Southern University, Dr. Velma McBride Murry- Vanderbilt University, Ms. Alexis Gorden- Sickle Cell Foundation of Tennessee, Ms. Delores Quasie-Woode- Georgia Southern University

Objective: Sickle cell disease (SCD) has a substantial impact on both the health of individuals affected and healthcare utilization in the United States. Successful disease self-management is shown to positively affect health outcomes and decreases overall healthcare utilization. Yet, many SCD patients and caregivers are faced with external barriers that limit their ability to self-manage their disease. As part of a patient-centered outcomes research study, we partnered with the Sickle Cell Foundation of Tennessee to determine areas of greatest need in disease management among individuals with SCD and caregivers residing urban and rural communities.  
   
Methods: Individuals with SCD and their caregivers (N=43) completed open-ended questionnaires on barriers and challenges to SCD management. Participants were asked questions on: (1) issues of concern regarding sickle cell health care management; (2) conditions which place the greatest burden when trying to effectively manage care; (3) possible solutions to lessen these burdens; and (4) thoughts about ways of providing health care to increase optimal health functioning for patients with SCD. Content analysis was conducted on responses to identify important emerging themes. Responses were analyzed collectively noting differences in urban and rural groups.  
   
Results: Six themes emerged illustrating areas of burden in SCD management for patients and caregivers residing in urban and rural communities: (1) pain management; (2) communicating with healthcare providers; (3) stigma as a drug-seeker; (4) limited SCD knowledge among providers outside of hematology; (5) absence of treatment protocols; and (6) a need for enhanced disease management resources for both patients and caregivers. Two additional themes were central to patients and caregivers residing in rural communities: (1) the need for satellite clinics; and (2) distance to a hematologist.  
   
Conclusions: Findings revealed patient-centered priorities that need to be addressed to facilitate successful management of SCD. Patients and caregivers living in rural communities have similar concerns as those living in urban areas; yet, their distance from a sickle cell clinic and/or hematologist creates additional barriers to care. Areas of burden identified will be useful for increasing self-management resources that are tailored according to patient-centered preferences, as well as developing provider interventions to enhance care for patients living in urban and underserved rural communities.


Speakers
avatar for Tilicia Mayo-Gamble, PhD, MPH, MA, CHES

Tilicia Mayo-Gamble, PhD, MPH, MA, CHES

Assistant Professor, Department of Community Health Behavior and Education
Dr. Mayo-Gamble is an Assistant Professor in Jiann-Ping Hsu College of Public Health, Department of Community Health Education and Behavior at Georgia Southern University. She conducts research on community engaged approaches to increasing access to care and facilitating self-management... Read More →


Friday October 12, 2018 3:15pm - 3:30pm
Baltimore/ Annapolis

3:15pm

Discovering And Breaking Down Barriers: Improving Compliance with Appointments in the Pediatric Sickle Cell Clinic
Abstract

Authors:
Mrs. carla Jones, Atrium Health: Levine Children's Hospital,Mr. Kenneth Mitten, Levine Children's Hospital Charlotte, NC    

Objective: Information from our pediatric sickle cell disease (SCD) clinic indicates that we have an average noncompliance rate for attendance of clinic appointments of 30%. Our objective was to determine barriers to clinic attendance and strategies to overcome these barriers to improve compliance for 1 month clinic appointments for pediatric patients (birth-21 years of age) with sickle cell disease (SCD) on hydroxyurea.
 
Method: Caregivers of Sickle Cell patients indicated via a survey 5 main barriers that prevented them from consistently attending their 1 month clinic follow ups. They were  transportation, inflexibility of employers, personal family issues, inconsistent contact information (frequent phone number and address changes) and even forgetfulness (no barriers) as primary reasons for missed clinic appointments. Pareto chart analysis revealed patients scheduled for 1 month appointments were most likely to miss appointments. A multidisciplinary team was formed and created an aim statement, current and ideal process flow maps, and tested multiple change ideas with small tests of change.  Changes tested include offering offsite lab visits, posting signage to assist with transportation, mailing letters after missed appointments, assisting with reminders in phones, reminder calls to patients with a history of missed appointments, and giving out patient expectation forms to new patients and at yearly appointments.  


Speakers
KM

Kenneth Mitten, MSW

Pediatric Sickle Cell Social Worker, Levine Childrens Hospitals
I am a Pediatric Sickle Cell Social Worker in the Pediatric Hematology, Oncology, and Transplant Program at Levine Children’s Hospital in Charlotte, NC. I specialize in treating the psychosocial needs of children, adolescents and young adults and families living with Sickle Cell... Read More →
avatar for Carla Jones, MSN, RN

Carla Jones, MSN, RN

Pediatric Sickle Cell Nurse Navigator, Levine Children's Hematology Oncology Bone Marrow Transplant Clinic
I am currently serving as the Pediatric Sickle Cell Nurse Navigator at Levine Children's Hematology Oncology Bone Marrow Transplant Clinic in Charlotte, NC. I have been serving in this capacity for 3.5 years. I specialize in providing education as well as navigating the newly diagnosed... Read More →


Friday October 12, 2018 3:15pm - 3:30pm
Constellation D

3:15pm

Strategies And Next Steps For Improving Sickle Cell Disease Care: Highlights From The Sickle Cell Disease Treatment Demonstration Program (SCDTDP)
Abstract

Authors: 
Dr. Suzette O. Oyeku- National Institute for Children’s Health Quality (NICHQ) and Division of Academic General Pediatrics, Children’s Hospital at Montefiore/Department of Pediatrics, Albert Einstein College of Medicine, Dr. Ifeyinwa Osunkwo- Levine Cancer Institute, Carolinas HealthCare System, Dr. Allison King- Department of Pediatrics, Division of Pediatric Hematology/Oncology and Program in Occupational Therapy, Washington University School of Medicine, Ms. Lisa Shook- Division of Hematology, Department of Pediatrics, Cincinnati, OH Cincinnati Children’s Hospital Medical Center; Division of Pediatrics, University of Cincinnati, College of Medicine, Dr. Rosalyn W. Stewart- Departments of Internal Medicine and Pediatrics, Johns Hopkins University School of Medicine, Dr. Marsha Treadwell- UCSF Benioff Children's Hospital Oakland, Department of Hematology/Oncology; Center for Inherited Blood Disorders,
Dr. Julie Kanter- Division of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA, Dr. John Strouse- Division of Hematology, Department of Medicine, Duke University
 
OBJECTIVE:
Since the Sickle Cell Treatment Act of 2003, the Sickle Cell Disease Treatment Demonstration Program (SCDTDP), funded by the Health Resources and Services Administration (HRSA) has promoted best practice treatments, offered provider training, and increased access to high-quality care for individuals living with sickle cell disease in the US.
This session will highlight the impact and accomplishments of the 2013-2017 SCDTDP, lessons learned and recommendations as highlighted in programs 2017 Congressional Report to Congress. A panel including presenters from the National Coordinating Center and the five current Regional Coordinating Centers will share their collective results and describe their regional efforts to improve:
  • access to high quality knowledgeable care,
  • the use of disease modifying therapies (Hydroxyurea), and
  • and broaden knowledge base among providers for caring for patients, from childhood to adulthood with this disease.
Recommendations for SCD programs and initiatives, clinical care, and policy will be shared to bridge the program into its current iteration, introducing the additional 5th Regional Coordinating Center to join the project, covering the Southeast region of the United States.
METHODS
Over the past three years, a regional model was established to increase: access to Hydroxyurea (a disease-modifying therapy); knowledgeable sickle-cell providers; and access to quality care. Regional learning collaboratives tracked progress through data collection and quality improvement efforts. Some brief highlights include:
Heartland Regional Coordinating Center (IA, KS, MO, NE) established telementoring programs for healthcare providers to address geographic disparities in care access.
Midwest Regional Coordinating Center (IL, IN, MI, MN, OH, WI) addressed gaps that affect care, including setting up satellite clinics that connected patients and local providers to staff and resources at larger medical systems with SCD expertise.
Northeast Regional Coordinating Center (DC, MD, NJ, NY, VA, PA, DE, WV, Virgin Islands, Puerto Rico) developed strong relationships with community-based organizations both in individual states and at the regional level to increase patient access to SCD care.
Pacific Regional Coordinating Center (AK, AZ, CA, ID, HI, OR, NV, WA, Guam) increased patient access to care with the opening of two new comprehensive centers for SCD care in key urban areas targeting larger concentrations of patients with SCD.
Southeast Regional Coordinating Center (AL, FL, GA, KY MS, NC, SC, TN), has been newly-funded for the 2017-2022 program cycle will be working to implement many of the lessons learned from their RCC colleagues as well as share innovation they are driving in the southeast region of the country.
RESULTS
The concerted efforts of SCDTDP have improved access to care, increased the use of Hydroxyurea among patients with SCD, and increased the number of providers knowledgeable about SCD. These efforts have led to the following accomplishments. Within this time, across the four regional learning collaboratives:
  • Nearly 11,000 patients with SCD are receiving care through SCDTDP regional networks, reflecting an increase of more than 3,000 from baseline
  • Nearly 7,000 patients with SCD are contributing information to electronic health registries
  • The number of patients reached increased by more than 3,000, hydroxyurea use increased across all regions, and
  • over 200 providers accessed telementoring services and strategies to improve care. The continuing national initiative builds off this previous work and increases the focus on ensuring the highest quality of care for patients living with sickle cell disease.
CONCLUSIONS 
The regional collaborative approach has modest impact on meeting the overarching SCDTDP program objectives. Spread of innovative approaches identified in the SCDTDP to educate providers and enhance access to care are necessary to ensure continued improvements in the delivery of high quality care to individuals with sickle cell disease.

Speakers
MB

Mary Brown

President & CEO, PSCRC, Sickle Cell Disease Foundation of California
SO

Suzette Oyeku MD, MPH, FAAP

Director, Academic General Pediatrics Fellowship, The Children’s Hospital at Montefiore
RW

Roslyn W. Stewart, MD, MS, MBA

SiNERGe, Johns Hopkins University, School of Medicine
LM

Lisa M. Shook, MA, MCHES

Instructor, STORM, Cincinnati Children’s Hospital Medical Center
IO

Ifeyinwa Osunkwo, MD, MPH

EMBRACE, Levine Cancer Institute Division of Hematologic Oncology, Atrium Health
ED

E. Donnell Ivy, MD, MPH

eivy@hrsa.gov, Health Resources and Services Administration
TV

Taniya Varughese, MSOT, OTR/L

Heartland Sickle Cell Disease Network, Washington University School of Medicine


Friday October 12, 2018 3:15pm - 4:15pm
Frederick/Columbia

3:15pm

Striving in the Workforce with Sickle Cell
Speakers
avatar for Carla Lewis

Carla Lewis

Executive Director, Atrium Health: Levine Childrens Institute
Carla Lewis is a dedicated advocate for those living with sickle cell disease. Carla’s advocacy journey began in April 2002 when she gave birth to her son diagnosed with sickle cell beta – thalassemia. In 2004 Carla started the first Sickle Cell Support group at or Arnold Palmer... Read More →


Friday October 12, 2018 3:15pm - 5:15pm
Pratt/Calvert

3:15pm

Simultaneous Session I
Friday October 12, 2018 3:15pm - 5:30pm
Baltimore/ Annapolis

3:30pm

Compassionate-Use Voxelotor (GBT440) For Up To 2 Years In Patients With Severe Sickle Cell Disease And Life-Threatening Comorbidities
Abstract 

Authors:
Dr. Gershwin T. Blyden- Foundation for Sickle Cell Disease Research,
Dr. Kenneth R. Bridges- Global Blood Therapeutics, Dr. Lanetta Bronté- Foundation for Sickle Cell Disease Research
 
Objective: Voxelotor (GBT440) is a first-in-class, oral, once-daily therapy designed to modulate the affinity of hemoglobin (Hb) for oxygen and delay sickle Hb polymerization, thereby reducing consequent red blood cell damage and life-threatening complications from sickle cell disease (SCD). Seven patients with severe SCD ineligible for ongoing clinical trials because of high mortality risk from comorbidities, including renal dysfunction, frequent hospitalizations for vaso-occlusive crisis (VOC), extreme anemia, and multiorgan failure were provided compassionate-use access to voxelotor by US Food and Drug Administration single-patient international new drug application.
Methods: Once-daily voxelotor began at 900 mg with possible increase to 1500 mg. Baseline echocardiograms were performed. Initial assessments at 2 and 4 weeks were followed by routine evaluations at least monthly. Key data captured included Hb levels, reticulocyte count, indirect bilirubin, resting oxygen saturation, pain level on 0-10 scale, transfusions, and hospitalizations for VOC. Patient Health Questionnaire 9-item (PHQ-9) assessed depression.
Results: Four female and 3 male patients aged 22-67 years had severe comorbidities at baseline including iron overload in all patients, frequent transfusions in 5/7, severe fatigue in 4/7, chronic oxygen supplementation in 2/7, and progressive severe renal dysfunction or multiorgan failure in 1 each. In 5/7 patients, baseline Hb was ≤6.4 g/dL, with three <6 g/dL. Hb level increased in all 7 patients by 24 weeks of voxelotor, with 5 reaching ≥1.0 g/dL. Hb increase due to voxelotor was clearest in 3 patients who experienced 1.0 to 5.4 g/dL Hb rises without transfusion. The number of transfusions fell by 60% in the 7 patients. VOC hospitalizations fell by 67%. Baseline oxygen saturation improved to 98%-99% from <95% in 4 patients following voxelotor treatment, permitting 2 patients to discontinue long-term supplemental oxygen. Overall well-being improved, typically 2-3 weeks after treatment initiation, for all patients, and the PHQ-9 score registered reduced depression. Voxelotor was well-tolerated with no discontinuations during treatment periods of up to 2 years. There was 1 dose reduction because of grade 2 diarrhea. Two patients with advanced end-stage organ injury died of SCD complications unrelated to treatment.
Conclusions: Seven patients given compassionate-use voxelotor after exhausting all treatment options achieved substantial improvements in clinical and mental health parameters for as long as 2 years. Controlled clinical trials will be needed to confirm the benefit of voxelotor in patients with severe SCD and multiple comorbidities.

Speakers
avatar for Kenneth R. Bridges

Kenneth R. Bridges

Clinical Scientist, Global Blood Therapeutics
Dr. Kenneth R. Bridges received the MD degree from Harvard Medical School, and subsequently trainedin internal medicine and hematology in Boston, at Massachusetts General and Brigham and Women’sHospitals, respectively. Following medical subspecialty training, Dr. Bridges worked... Read More →


Friday October 12, 2018 3:30pm - 3:45pm
Constellation C

3:30pm

Finding SCD Patients In Michigan - A Case Study
Abstract 

Authors:
Ms. Jeanne M. Loboda- Global Blood Therapeutics, Dr. Kartik S. Pappu- Global Blood Therapeutics, Ms. Robin C. Howard- Global Blood Therapeutics, Mr. Vincent Y. Siu- Global Blood Therapeutics, Ms. Wanda Whitten-Shurney- Sickle Cell Disease Association of America–Michigan Chapter
 

Objective: Sickle cell disease (SCD) is a genetic, lifelong blood disorder that can cause significant, long-term, severely disabling, and costly clinical complications. Many individuals with SCD, especially adults, lack access to hematologists with significant experience treating SCD. Many providers in the primary care setting have limited knowledge of SCD and available therapies. Provider and patient disease education is a vital component of quality care delivery in this setting.
We describe the results of a recent collaboration between the Michigan Chapter of the Sickle Cell Disease Association of America (SCDAA-MI) and Global Blood Therapeutics, a biotechnology company dedicated to developing novel disease-modifying therapies for SCD, to better target community providers and patients for educational outreach programs in the state of Michigan. A special focus was placed on federally qualified healthcare centers (FQHCs) near areas with moderate to high densities of affected individuals.
The ability to target outreach efforts is particularly relevant to the SCDAA-MI’s role as the subrecipient of 2 health resource and service administration (HRSA) grants. One of the major objectives of the Sickle Cell Treatment Demonstration Grant is to educate physicians about the management of SCD. The Newborn Screening Grant is focused on identifying patients previously lost to follow-up, placing them in a medical home, and providing care coordination.
Methods: Pharmacy and medical claims for patients with at least 2 SCD specific health care claims (using SCD specific ICD9/10 codes) from Symphony Health’s Integrated Dataverse™ were analyzed. This longitudinal patient dataset captures claims across the United States and covers all payment types, including Medicaid, Medicare, commercial plans, cash, and assistance programs. All data collected from the database is deidentified in compliance with the patient confidentiality requirement of the Health Insurance Portability and Accountability Act.
We first identified individuals residing in the state of Michigan with at least 1 ICD9/10 code for SCD in each of the 2 most recent years (2016 and 2017). We then analyzed each individual’s age (derived from birth year), geographic location (using the first 3 digits of the zip code of residence [zip3]), and healthcare payer (derived from payment type). Visits to a hematologist during 2016 and 2017 were quantified through the National Provider Identifier number associated with each claim. Finally, we overlaid the locations of the latest list of FQHCs in Michigan on an SCD population density map to identify the FQHCs with the largest local SCD populations.
Results: A total of 1628 individuals were identified in Michigan with at least 1 SCD-related claim in both 2016 and 2017. Of these, 83% were older than age 18 years, and 60% had Medicaid as their primary payer (of those that do have payer information provided). The distribution of these individuals with SCD was geographically concentrated: 69% resided in 3 zip3 areas (Figure 1). A majority of pediatric patients (64%) and of adult patients (71%) had not seen a hematologist at least once a year in 2016 and 2017. We identified 52 FQHCs that serviced the 3 zip codes with the largest share of individuals affected by SCD.
 Conclusions: Individuals with SCD in Michigan are geographically concentrated. Most individuals with SCD in Michigan have Medicaid as their primary medical insurance provider. Although it is likely that some patient visits are not captured in this dataset, this analysis nevertheless demonstrates that most individuals with SCD in Michigan have reduced access to hematologists. Therefore, it is crucial to identify and provide disease education to community providers that service the Medicaid population and have the potential to treat significant volumes of individuals with SCD. To this end, we have identified several FQHCs that might serve as recipients of initial outreach by the SCDAA-MI. This establishes a case study for biotechnology and pharmaceutical companies to work in concert with local advocacy organizations to better focus outreach efforts on improving disease education and care delivery. Making similar data available to other HRSA grantees as well as other community-based organizations could prove to be of great benefit in targeting efforts to maximize all aspects of care.

Speakers
JL

Jeanne Loboda

Senior Director, Global Blood Therapeutics
Accomplished healthcare marketer and strategist with oncology and rare disease experience. Proven builder of brand and product launch teams. Skilled leader recognized for developing functional and team visions and translating them into achievements. Keen ability to identify unmet... Read More →
avatar for Wanda Whitten-Shurney, MD

Wanda Whitten-Shurney, MD

Pediatrician, Sickle Cell Disease Associtaion, Michigan Chapter Inc.
Dr. Shurney is a pediatrician at Comprehensive Sickle Cell Clinic at Children’s Hospital of Michigan where she has provided out – patient care for children who have sickle cell disease for the past 19 years.  Her interest in sickle cell disease is “genetic”.  She inherited... Read More →


Friday October 12, 2018 3:30pm - 3:45pm
Baltimore/ Annapolis

3:30pm

The Opioid Epidemic And CDC Guidelines: A Case Of An Unfortunate Consequences Paved By Good Intentions
Abstracts

Authors: 
Dr. Cynthia Sinha- Emory University School of Medicine, Dr. Nitya Bakshi- Emory University School of Medicine, Ms. Diana Ross- Emory University School of Medicine, Dr. Lakshmanan Krishnamurti- Emory University School of Medicine

Objective 
Chronic pain is a debilitating complication in adults with sickle cell disease (SCD), and is associated with substantial morbidity, healthcare utilization, and risk of premature mortality. While chronic pain is most commonly treated with opioid analgesics, it is not clear whether opioids are safe or effective in the treatment of this condition. In the past two decades, the incidence of opioid abuse has soared in the general population, and opioid abuse was associated with over 64,000 deaths in the US in 2016. Recognizing the public health importance of the “opioid epidemic,” the CDC has published recommendations for prescribing opioids for chronic pain. The guidelines do not cover the use of opioids in patients with cancer and end-of-life care. The guidelines provide 12 detailed recommendations for initiating and continuing opioids for chronic pain, determining how to develop an opioid therapy plan, and assessing risk and harm of opioid use. The guidelines also propose restrictions on the dosage and frequency of “as needed” prescriptions. While the guidelines do not specifically address SCD chronic pain, there is a concern that implementation may exceed the scope of the guidelines, that there may be unintended medical consequences for the SCD patient with chronic pain, and that patients may feel stigmatized or discriminated against. To date, no research has investigated the perspective of adult patients on the impact of the heightened focus on opioid abuse as a result of the CDC guidelines. We, therefore, sought to interrogate the standpoint of adult patients on the effect of the implementation of the CDC guidelines by healthcare professionals at various levels.
Methods
We conducted semi-structured qualitative interviews of adults living with SCD to learn about their experiences with chronic pain, pain management, and physician interactions. We specifically asked participants thoughts on how the concern about the opioid epidemic among public health and medical communities has impacted their individual experiences and the SCD community as a whole. We used a semi-structured interview guide to collect data. Participants were recruited from SCD conferences and symposiums. From this first wave of recruitment, we utilized snowball sampling. Participants shared our study with friends, support groups, and posted the survey on social media sites that support SCD. We also published details of the study at two sickle cell clinics. Individuals 18 years old or older and diagnosed with SCD were eligible. We conducted interviews over the phone, but a few took place face-to-face. Audio recordings were transcribed verbatim. Data were analyzed using Grounded Theory Methodology.
Results
Twenty adults living with SCD were interviewed, of whom 17 were female. All but one participant reported experiencing pain on 3 or more days a week. The participants reported a mean daily pain intensity range of 4.8 on a scale of 10. Results were grouped into three emerging themes. The first theme, was the consistency of implementation of the CDC guidelines. The experience of the subjects suggest that implementation of the guidelines is variable. Patients had their doses decreased variably and had decreased access to opioids but were not offered alternative approaches to pain management. The second theme was the adverse impact of the guideline implementation on the health of patients. Restrictive access to pain medications leads to patients rationing their opioid usage and seeking alternatives such as the use of cannabis. Participants also felt that the exclusive focus on pain by their physicians also seemed to adversely impact their attention to the other aspects of their care. The third theme was the unintended stigmatization related to the implementation of the guidelines. Many patients felt stigmatized even though they did not feel like they were abusing opioids and that their opioid use for pain was unrelated to the opioid epidemic. Many patients were required for the first time to complete opioid contracts.
Conclusion
These data indicate that in the perspective of adult patients the CDC guidelines are implemented inconsistently, have adversely impacted care and resulted in the unjustified stigmatization of patients with SCD with chronic pain

Speakers
avatar for Cynthia Sinha, PhD

Cynthia Sinha, PhD

Associate Scientist, Emory University School of Medicine
Cynthia B. Sinha, PhD is a sociologist with a focus on families, culture, race-ethnicity, and more recently, medical sociology. She has over 10 years of experience in qualitative research methodology. Areas of research interest for Sickle Cell Disease include the experiences of older... Read More →


Friday October 12, 2018 3:30pm - 3:45pm
Constellation D

3:45pm

Crizanlizumab 5.0 Mg/Kg Increased the Time to First On-Treatment Sickel Cell Pain Crisis (SCPC) and the Likelihood of not Experiencing SCPC While on Treatment: Subgroup Analyses of the Phase 2 Sustain Study
Abstract

Authors:
Dr. Julie Kanter, Division of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA , Dr. Abdullah Kutlar, Sickle Cell Center, Medical College of Georgia, Augusta University, Augusta, Georgia, US, Dr. Darla Liles, Division of Hematology–Oncology, East Carolina University, Greenville, North Carolina, USA , Dr. Rodolfo Cancado, Department of Hematology–Oncology, Santa Casa Medical School of São Paulo, São Paulo, Brazil, Dr. Andreas Bruederle,  Novartis Pharma AG, Basel, Switzerland, Dr. Michael Shi, Novartis Pharma AG, Basel, Switzerland, Dr. Zewen Zhu, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA, Dr. Kenneth Ataga, Division of Hematology–Oncology, University of North Carolina, Chapel Hill, North Carolina, USA , Dr. Menaka Bhor, Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA        

Objective: In the 52-week SUSTAIN study, crizanlizumab 5.0 mg/kg significantly reduced frequency of SCPCs versus placebo (1.6 vs 3.0, P=0.01) and increased time to first on-treatment SCPC (4.1 vs 1.4 months, P=0.001) in patients with sickle cell disease (SCD). Objectives of this study included evaluating time to first SCPC in patient subgroups and likelihood of not experiencing SCPC for the duration of the trial using post hoc analyses.
Methods: SUSTAIN was a randomized, double-blind, placebo-controlled, phase 2 study (NCT01895361). Inclusion criteria were: SCD patients aged 16-65 years; 2-10 SCPCs in previous 12 months; concomitant hydroxyurea use permitted if ≥6 months and stable dose for ≥3 months. Patients were randomized 1:1:1 to receive intravenous crizanlizumab 5.0 mg/kg, 2.5 mg/kg, or placebo. Study treatments were administered on days 1 and 15, then every 4 weeks to week 50, with the final assessment at week 52. Median time to first SCPC after first dose was summarized for crizanlizumab 5.0 mg/kg or placebo in these subgroups: 2-4 or 5-10 SCPCs in previous 12 months; SCD genotype; and hydroxyurea use at baseline. Hazard ratios (HRs) for crizanlizumab 5.0 mg/kg versus placebo were calculated based on Cox regression analysis, with treatment as a covariate. Descriptive statistics were used to summarize frequency of patients who were SCPC event-free for the duration of the study by prior SCPC events, SCD genotype, and hydroxyurea use at baseline.
Results: 67 patients received crizanlizumab 5.0 mg/kg and 65 received placebo. There was a meaningful delay in time to first SCPC with crizanlizumab 5.0 mg/kg versus placebo observed in the entire study population. The effect was present in both SCPC subgroups, and the largest treatment difference was observed in HbSS SCD versus other genotypes (4.1 vs 1.1 months; HR: 0.50). In patients taking hydroxyurea who experienced 2-10 SCPCs in the previous year, time to first on-study SCPC was longer with crizanlizumab 5.0 mg/kg versus placebo (2.4 vs 1.2 months; HR: 0.58). A greater proportion of patients treated with crizanlizumab 5.0 mg/kg were SCPC event-free versus placebo in each of the analyzed subgroups. One third of patients who were taking hydroxyurea and treated with crizanlizumab 5.0 mg/kg were SCPC event-free during the study versus 17.5% with placebo, possibly suggesting an additive effect.  
Conclusions: With crizanlizumab 5.0 mg/kg, there was a clinically meaningful delay in time to first SCPC and an increased likelihood of being SCPC-free versus placebo in all subgroups investigated, including patients taking hydroxyurea who had still experienced 2-10 SCPCs in the previous year, indicating that P-selectin inhibition provided additional beneficial treatment effect compared with hydroxyurea alone.



         
       
       
     
         
     
       
       


Speakers
avatar for Julie Kanter, MD

Julie Kanter, MD

Lifespan Hematologist, Medical University of South Carolina
Dr. Julie Kanter is a lifespan hematologist specializing in sickle cell disease.  She is an associate professor of Pediatrics at the Medical University of South Carolina and runs the first all-ages, Lifespan Comprehensive Sickle Cell Center.  Dr. Kanter works closely with national... Read More →


Friday October 12, 2018 3:45pm - 4:00pm
Constellation C

3:45pm

The Communication, Awareness, Relationship and Empowerment (C.A.R.E.) Model: An Effective Tool for Engaging Urban Communities in Community-Based Participatory Research
Abstract 

Authors:
Ms. Marlene Peters-Lawrence- National Heart, Lung, and Blood Institute/National Institutes of Health, Ms. Joniqua Ceasar- National Heart, Lung, and Blood Institute/National Institutes of Health, Dr. Tiffany Powell-Wiley- National Heart, Lung, and Blood Institute/National Institutes of Health, Mrs. Valerie Mitchell- National Heart, Lung, and Blood Institute/National Institutes of Health

Objective:
Little is known about recruitment methods for racial/ethnic minority populations from resource-limited areas for community-based health and needs assessments, particularly assessments that incorporate mobile health (mHealth) technology for characterizing physical activity and dietary intake. We examined whether the Communication, Awareness, Relationships and Empowerment (C.A.R.E.) model could reduce challenges recruiting and retaining participants from faith-based organizations in predominantly African American Washington, D.C. communities for a community-based assessment. Employing C.A.R.E. model elements, our diverse research team developed partnerships with churches, health organizations, academic institutions and governmental agencies. Through these partnerships, we cultivated a visible presence at community events, provided cardiovascular health education and remained accessible throughout the research process. Additionally, these relationships led to the creation of a community advisory board (CAB), which influenced the study's design, implementation, and dissemination.
Methods:
Each part of the C.A.R.E. model involved specific strategies that were implemented throughout various stages of the study.
Results:
Over thirteen months, 159 individuals were recruited for the study, 99 completed the initial assessment, and 81 used mHealth technology to self-monitor physical activity over 30 days.
Conclusion:
The culturally and historically sensitive C.A.R.E. model strategically engaged CAB members and study participants. The C.A.R.E. model was essential for the success in recruitment and retention of an at-risk, African American population and may be an effective model for researchers hoping to engage racial/ethnic minority populations living in urban communities. The C.A.R.E research recruitment model was implemented in a randomized clinical trial for sickle cell disease. (figure attached publication pending).

Speakers
avatar for Marlene Peters-Lawrence, BSN, RN, RRT

Marlene Peters-Lawrence, BSN, RN, RRT

Clinical Trial Specialist, National Heart Lung and Blood Institute
Marlene Peters -Lawrence is a Clinical Trials Specialist in the Division of Blood Diseases and Resources, Blood (DBDR), Epidemiology and Clinical Therapeutics Branch at the National Heart Lung and Blood Institute (NHLBI). Ms. Peters-Lawrence has been with NIH for over fifteen years... Read More →


Friday October 12, 2018 3:45pm - 4:00pm
Baltimore/ Annapolis

3:45pm

Availability of Information on Blood and Marrow Transplantation (BMT) Coverage Benefits for Patients with Sickle Cell Disease (SCD) Enrolled in Medicaid
Abstract

Authors: 

Dr. Tatenda Mupfudze- National Marrow Donor Program, Ms. Lensa Idossa, MPH -National Marrow Donor Program, Dr. Lih-Wen Mau - PhD, MPH, National Marrow Donor Program, Ms. Aisha Mehmud, MPH - National Marrow Donor Program, Ms. Jaime Preussler, MS -National Marrow Donor Program, Ms. Rachel Glissmann, MPH- National Marrow Donor Program, Ms. Alicia Silver, MPP –National Marrow Donor Program, Ms. Susan Leppke, MPH -National Marrow Donor Program, Dr. Linda Burns, MD - National Marrow Donor Program, Ms. Ellen Denzen, MS -National Marrow Donor Program

Background:
Sickle cell disease (SCD) is the most common inherited hemoglobin disorder affecting approximately 100,000 people in the United States (U.S.) [1]. The Centers for Disease Control and Prevention (CDC) report SCD incidence rates of 1:365 and 1:16,300 for Black/African Americans and Hispanics in the United States (U.S.), respectively [2]. Nearly 2,000 new diagnoses are reported each year via newborn screening [2]. Painful crises are the hallmark of SCD, coupled with an unpredictable clinical course that can lead to organ dysfunction, poor quality of life and premature death [3-4].    
Allogeneic blood and marrow transplantation (BMT) is a complex and resource-intense therapy that uses donor cells from either a closely matched family member, unrelated donor, or cord blood to restore the patient’s bone marrow, giving rise to healthy red blood cells [3-4]. It is the only potentially curative treatment option for SCD with a 5-year disease-free survival rate of 92% in children [3-4].  
The timing of when a patient with SCD should undergo BMT is evolving based on data from clinical trials. Currently, experts recommend that children with severe SCD, including overt strokes, recurrent acute chest syndrome (ACS), or recurrent vaso-occlusive crisis despite adherence to hydroxyurea therapy and/or regular blood transfusion therapy, be referred for BMT consultation [3-4]. Some experts recommend earlier referral due to the progressive nature of the disease.  
Lack of appropriate insurance coverage can be a significant barrier to accessing advanced therapies such as BMT. Many patients with SCD are covered by Medicaid, a federal program administered at the state level [5]. BMT is an optional benefit under Medicaid, which leads to significant variation in Medicaid policy over time and between states, further exacerbating disparities in access [5]. There is a dearth of information on the impact of: 1) Medicaid coverage and 2) easy-to-access policy information for patients with SCD.   
Objective:
To describe the availability of information on Medicaid coverage benefits for BMT for patients with SCD  
 Methods:
 SCD incidence estimates were calculated using the number of births in 2016 [6-7] multiplied by the CDC SCD incidence rates by race [2].  States estimated to have more than 75 newborns diagnosed with SCD were identified and information on Medicaid BMT coverage benefits was collected. Where available, information was obtained from Medicaid plan websites for the calendar year 2018. When information was not available online, state Medicaid offices were contacted. Lastly, if contact with the state office could not be made, transplant centers within that state were contacted for information. Data on the number of BMTs performed annually for patients with SCD was extracted from the Center for International Blood and Marrow Transplant (CIBMTR) outcomes registry for 2016 and 2017 (there is 1-year lag in data submission to the CIBMTR by transplant centers, hence 2016-2017 data was included in this analysis).  
   
   
Results:
Six states were estimated to have more than 75 newborns diagnosed with SCD in 2016 (Table 1). Although Medicaid programs determine BMT coverage for SCD by reviewing individual patient characteristics and clinical indications, general requirements differed by state. Information on BMT coverage for patients with SCD was publicly available only on the Texas, North Carolina and Californian plan websites [8-11]. The absence of information on plan websites necessitated contacting state Medicaid offices. Contact with Medicaid office representatives revealed that BMT may be covered with prior approval in California. A Georgia Medicaid representative reported that although BMT may be covered for patients with SCD under 21, after obtaining prior approval, BMT was not covered for patients with SCD over 21 years. Data collection for New York and Florida is currently ongoing. Data collection through state Medicaid offices and transplant centers on comprehensive benefits including BMT-related travel, housing, and medications is also currently ongoing and will be completed by September 2018 [8].    
   
Conclusion:
There is disparate information available online regarding Medicaid coverage of BMT for patients with SCD across states with high numbers of SCD patients. Limited publicly available information on BMT coverage for patients with SCD may contribute to disparities in access. Coverage for resources beyond the procedure itself needed to undergo BMT, such as travel, housing, and medications, may also affect patient access to BMT (data collection is ongoing). Although Medicaid programs may grant prior approval for BMT on a case-by-case basis, making general requirements/guidelines on patient and clinical indications that would be considered for BMT and covered by Medicaid publicly available would better support patient-provider informed treatment decision-making. Provision of easy-to-access information on current Medicaid coverage for BMT in SCD is needed to address informational barriers and help reduce disparities in access. Expanded awareness of available Medicaid coverage benefits may also increase engagement among health professionals and the SCD community around a potentially lifesaving therapy.   
References:
  1. National Heart Lung and Blood Institute. Evidence-Based Management of Sickle Cell Disease Expert Panel Report 2014.  
  2. Centers for Disease Control and Prevention (CDC). Data & Statistics. https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed Feb, 2018.  
  3.  Kassim AA, Sharma D. Hematopoietic stem cell transplantation for sickle cell disease: The changing landscape. Hematol Oncol Stem Cell Ther. 2017.  
  4. Walters MC, De Castro LM, Sullivan KM, et al. Indications and Results of HLA-Identical Sibling Hematopoietic Cell Transplantation for Sickle Cell Disease. Biol Blood Marrow Transplant. 2016;22:207-211  
  5. Medicaid.gov. Medicaid & CHIP Enrollment Data Highlights. https://www.medicaid.gov/medicaid/program-information/medicaid-and-chip-enrollment-data/report-highlights/index.html, Accessed Apr, 2018.  
  6. Kaiser Family Foundation. Number of Biths by Race, https://www.kff.org/other/state-indicator/births-by-raceethnicity/?currentTimeframe=0&sortModel=%7B%22colId%22:%22Black%22,%22sort%22:%22desc%22%7D. Accessed Apr, 2018  
  7. The Centers for Disease Control and Prevention (CDC), National Vital Statistics Reports (NVSR), Vol. 67, No. 1: Births: Final Data for 2016. Accessed Jan 2018.  
  8. Be the Match. Transplant Benefits & Coverage. https://payer.bethematchclinical.org/transplant-benefits-and-coverage/. Accessed Apr, 2018.  
  9. Florida Medicaid. Transplant Services Coverage Policy Agency for Health Care Administration. http://www.fdhc.state.fl.us/medicaid/review/Specific/59G-4.360_Transplant_Coverage_Policy.pdf. Accessed Feb 2018  
  10. Texas Medicaid Provider Procedure Manual December 2015 Volume 1 & 2.  http://www.tmhp.com/TMHP_File_Library/Provider_Manuals/TMPPM/2015/Dec_2015%20TMPPM.pdf. Accessed Jun 2018  
  11. NC Division of Medical Assistance. Medicaid and Health Choice Allogeneic Hematopoietic & Bone Clinical Co

Speakers
TM

Tatenda Mupfudze, PhD

Health Services Research Analyst II, Center for International Blood and Marrow Transplant Research (CIBMTR)
Tatenda Mupfudze is a Health Services Research Analyst at the National Marrow Donor Program. Her area of research interests center on anemia of inflammation, hepcidin, sickle cell anemia and blood and marrow transplantation.


Friday October 12, 2018 3:45pm - 4:00pm
Constellation D

4:00pm

Parent and Youth Barriers to Hydroxyurea Adherence in Adolescents with Sickle Cell Disease
Abstract

Authors:
Dr. Arlene Smaldone, Columbia University School of Nursing, Dr. Deepa Manwani, The Children's Hospital at Montefiore,  Dr. Nancy S. Green, Columbia University Medical Center  

Objective:  The purpose of this study was to examine the effect of a community health worker (CHW) intervention on reduction of self-reported barriers to hydroxyurea (HU) adherence in a sample of poorly adherent youth ages 10-17 years who participated with their parent in the Hydroxyurea Adherence for Personal Best in Sickle Cell Disease "HABIT" feasibility trial. Dyads randomized to the intervention worked with CHWs to develop a HU habit reinforced by daily personized text messages.
Methods: We modified the Adolescent Medication Barriers Scale (AMBS) (17 items) and its parent counterpart, Parent Medication Barriers Scale (PMBS) (16 items) for use with HU by adding 9 items regarding HU beliefs.  Three subscales (Disease frustration, Regimen adaptation, Ingestion Issues) are common to both scales; the parent scale has an additional subscale (Reliance on parent reminders). Youth and parents completed the measure at 0, 3 and 6 months in either Spanish or English. Youth and parent proxy reported generic (PedsQL) and disease specific (PedsQL Sickle Cell module) HRQL were measured at the same intervals. Data were analyzed using Cronbach's alpha, descriptive statistics, spearman correlation coefficients and linear growth models controlling for group assignment and time.
Results: 28 parent youth dyads (50% Latino, youth 43% female, age 14.3 ± 2.6 years) participated. Internal reliability was high for both parent (alpha=0.88) and youth (alpha=0.91) total scales and all subscales except the parent ingestion subscale (alpha=0.30). On average, parents and youth acknowledged 3.5±3.2 and 5.0±3.9 total barriers (possible score 0-25 youth; 0-26 parents) respectively. Adherence barriers most frequently reported by parents were child overreliance on parent reminders [42.9%]; adolescent frustration (child tired of living with SCD [35.7%]); regimen adaptation problems (finding it hard to stick to a medication schedule [28.6%]); and HU beliefs (parent concern about possible adverse HU effects on fertility or on a fetus [25%]). Youth most frequently reported barriers were related to adolescent frustration (tired of living with SCD [57.1%]; being forgetful and not always remembering to take HU [53.6%], tired of taking HU [39.3%], not wanting to take HU at school [28.6%], and not wanting to be seen taking HU [25%],); ingestion issues (HU hard to swallow [25%], too many pills to take [39.3%], dislikes HU taste [35.7%]); and regimen adaptation problems (not organized about when and how to take HU [28.6%]). Parent and youth responses were moderately correlated for the total scale (r=0.50, p<0.001) and all subscales except HU beliefs (r=0.08, p=0.51). Greater number of barriers were associated with lower generic (parent r=-0.43, p=0.03; youth r=-0.44, p<0.001) and disease specific (parent r=-0.53, p=0.005; youth r=-0.53, p<0.001) HRQL. Controlling for group assignment and time, intervention parents demonstrated a trend in less reported adolescent frustration (p=0.18) at 6 months whereas for adolescents assigned to the intervention group, ingestion related barriers significantly declined (0.17/month, p=0.02) over 6 months. Total barriers and other subscales did not significantly change. 
Conclusions: Barriers to adherence are prevalent, multi-faceted and perceived differently by youth prescribed HU and their parents. Greater barriers were associated with poorer youth and parent-proxy reported generic and disease specific HRQL. Some barriers, e.g. problems with ingestion, are potentially modifiable. A CHW delivered intervention helped youth reduce their ingestion barriers to HU use. To derive the full value of HU in youth with SCD, barriers to its use must be addressed with both youth and parents.
       
     
       


Speakers
avatar for Arlene Smaldone, PhD, CPNP, CDE

Arlene Smaldone, PhD, CPNP, CDE

Professor of Nursing, New York Academy of Medicine: Section on Evidence Based Health Care Columbia University School of Nursing
Dr. Smaldone is a Professor of Nursing and Dental Behavioral Sciences at Columbia University Medical Center. Dr. Smaldone is a behavioral researcher whose research focuses on self-management for youth with chronic health conditions. Dr. Smaldone's research in youth with sickle cell... Read More →


Friday October 12, 2018 4:00pm - 4:15pm
Constellation C

4:00pm

Technology Access And Preferences For Smartphone Application Content Among Adults With Sickle Cell Disease And Their Caregivers
Abstract

Authors:
Dr. Tilicia Mayo-Gamble- Georgia Southern University, Dr. Velma McBride Murry- Vanderbilt University, Ms. Alexis Gorden- Sickle Cell Foundation of Tennessee, Ms. Delores Quasie-Woode- Georgia Southern University
 
Objective: Sickle cell disease (SCD) is a chronic disease that affects over 100,000 individuals in the United States, of whom many are from underserved communities with limited access to disease management resources. The purpose of this study was to determine access to resources in the form of technology and preferences for self-care management and social support in adults with SCD and their caregivers residing in urban and rural communities.
Methods: Community Health Ambassadors led community listening sessions (n=4) with adults with SCD and their caregivers (n=43) in two urban and two rural communities throughout Tennessee. Community listening sessions are a qualitative method of obtaining feedback that allows individuals to express their views on an issue in a more wide-ranging manner. Participants were asked questions on: (1) methods of finding information about SCD self-care; (2) satisfaction with current methods for finding SCD management information; (3) support for SCD management; 4) important features for development of a smartphone application (app) SCD; and (5) areas of benefit for using a smartphone app for SCD self-care. Content analysis identified emerging themes.
Results: Five critical themes emerged: (1) desired information; (2) types of support for SCD self-care; (3) recommendations for disseminating information to the sickle cell community; (4) barriers and facilitators to using an app; and (5) desirable features for a SCD mobile app. All participants had a smartphone but most did not have an iphone. Most participants would prefer to receive regular emails on SCD management from a credible source. Participants also expressed a range of comfort levels with using a mobile app but were open to trying an app for SCD self -care and SCD social support.
 Conclusions: Through our discussions we learned more about how to reduce barriers to accessing information by offering a technology platform based on patient and caregiver preferences. Findings can be used to develop a patient-centered, user-friendly smartphone application to facilitate disease self-management among individuals with SCD and their caregivers; thus, increasing access to resources for families residing in rural communities.

Speakers
avatar for Tilicia Mayo-Gamble, PhD, MPH, MA, CHES

Tilicia Mayo-Gamble, PhD, MPH, MA, CHES

Assistant Professor, Department of Community Health Behavior and Education
Dr. Mayo-Gamble is an Assistant Professor in Jiann-Ping Hsu College of Public Health, Department of Community Health Education and Behavior at Georgia Southern University. She conducts research on community engaged approaches to increasing access to care and facilitating self-management... Read More →


Friday October 12, 2018 4:00pm - 4:15pm
Baltimore/ Annapolis

4:00pm

A Training Approach for Newborn Screening Personnel in Nigeria
Abstract

Authors:
Dr. Baba Inusa- Guys and St. Thomas Hospital, Dr. Lewis Hsu- University of Illinois at Chicago,
Dr. Wale Atoyebi- Oxford University, Dr. Dogara Livingstone-Barau Dikko Teaching Hospital / Kaduna State University, Mrs. Bola Ibilola- SCORE Charity, Dr. Andrea Lamont- University of South Carolina, Dr. Juliana Olufunke Lawson- SCORE Charity, Mrs. Olukemi Ajamufua- Guys and St. Thomas Hospital, Dr. Kofi Anie- Imperial College, London

BACKGROUND: Nigeria leads the world in births with sickle cell disease (SCD), and
effective management should incorporate newborn screening. Learning from past
mistakes, we are applying the implementation science approach "Getting To Outcomes (GTO)" to incorporate stakeholder goals, capacity, and motivation to plan for pilot programs for SCD newborn screening in Nigeria's Kaduna and Niger States. Needs assessment and Training are needed for personnel in the whole process: key policymakers, laboratory technicians, health care workers (primary care and specialist care), and community workers like Community Health Extension Workers (CHEWs), and Traditional Birth Attendants (TBAs). Community awareness can be built up by SCD community groups and volunteer community mobilizers (VCM). Education theory highlights that the role-playing format is a good match for adult learners. Scenarios also permit rehearsal and planning for actual problems before the trainees encounter them.
GOAL: Develop the training content and format for SCD newborn screening across
these stakeholder groups.
METHODS: Needs assessment was conducted with stakeholder focus groups for 2
days in Kaduna in November 2017, followed by a 4 day training workshop in Kaduna in February 2018 to plan for newborn screening. Participants were invited: key policymakers, doctors, nurses, midwives, primary healthcare workers, CHEWs, TBAs, VCM, and SCD non-governmental organizations (NGOs). This was facilitated by a faculty of international and local experts, including one by e-conference. An interactive training approach was developed that included case discussions and role-playing, plus visual aids and a video drama.
RESULTS:
Role-playing was extremely well-received by the CHEW, TBA, VCM, nurse/midwives, and even legislators. Team-building occurred, as people started to refer to each other smilingly by the catch-phrases they had used during role-playing. Additional information emerged during the role-playing as experienced clinicians added rich details: the TBA are pivotal for follow-up; traditional healers prefer not to combine treatments with Western medicine; "difficult-to-reach" areas are served by an infrastructure of TBA, VCM, Emergency Transport (ETS), junior CHEW. Policy-makers using role-playing to apply the GTO approach expressed understanding about the value of stakeholder engagement to mold a plan to their local context. Legislators and medical personnel asked for additional case scenarios with local newborn screening issues, so that they could use the GTO framework more effectively.
CONCLUSION:
Role-playing can be a key approach for future training for SCD newborn screening and care in Nigeria. Role-playing can particularly be powerful for remembering key concepts, facilitating adaptation to local needs, and seeing the needs of other stakeholders. A recording of the case scenarios could be used to disseminate the learning and further raise awareness of SCD in Nigeria. This training approach also aligned well with the implementation science framework "Getting To Outcomes" for involving stakeholders in the newborn screening project.

Speakers
avatar for Lewis L. Hsu, MD, Ph.D

Lewis L. Hsu, MD, Ph.D

Professor Pediatric Hem-Onc, University of Illinois Hospital
Lewis Hsu is a pediatric hematologist dedicated to finding more cures for sickle cell disease, and improving treatment and education until cures can be found. He has experience with clinical and translational research in sickle cell disease, including clinical research on pain assessment... Read More →


Friday October 12, 2018 4:00pm - 4:15pm
Constellation D

4:15pm

Sickle Cell Patients with Leg Ulcers have a Pro-Thrombotic Profile
Abstract

Authors:
Dr. Ugochi Ogu, Montefiore Medical Center, Ms. Ashley Buscetta, National Human Genome Research Institute/National Institutes of Health, Ms. Elena Crouch, Montefiore Medical Center, Dr. Shuo You, Montefiore Medical Center, Prof. Moonseong Heo, Montefiore Medical Center, Ms. Khadijah Abdallah, National Human Genome Research Institute/National Institutes of Health, Dr. Giacomo Vinces, ontefiore Medical Center,Mr. Vence Bonham, National Human Genome Research Institute/National Institutes of Health, Prof. Caterina Minniti, Montefiore Medical Center

OBJECTIVE: 
- To review coagulation profile in sickle cell disease (SCD). 
- To describe coagulation profile in SCD patients with leg ulcers. 
BACKGROUND:
Leg ulcers are a serious complication of Sickle Cell Disease, often linked to a hemolytic phenotype that includes, priapism, pulmonary hypertension and renal disease. In the SCD pathogenesis, hemolysis and ischemia-reperfusion injuries lead to WBC, platelet and endothelial cell activation, pro inflammatory cytokine production and subsequent activation of coagulation that tips the balance towards a prothrombotic state. Decreased levels of the naturally occurring anticoagulants (protein C, protein S, antithrombin III), have been observed in patients with SCD in steady state, with some reports stating no differences between steady state and crisis. These decreases occur due to decreased production, increased consumption, or a combination of both. However, the definite mechanisms have not yet been elucidated. Factor V Leiden and prothrombin G20210A mutations are rare in African Americans and in patients with SCD. We speculated that patients with leg ulcers would have a more pronounced pro-thrombotic phenotype than patients who never developed them. 
METHODS: 
127 adults with and without SCD leg ulcers were evaluated at steady state in the prospective cross-sectional INSIGHTS study - Insights into Microbiome and Environmental Contributions to Sickle Cell Disease and Leg Ulcers (ClinicalTrials.gov Identifier NCT02156102). At the time of enrollment, a clinical examination and a complete past medical history were completed. Routine blood work including a full coagulation profile and analysis of blood clotting genetic mutations (thrombophilia genetic tests) were obtained. Laboratory results were compared between groups with and without leg ulcers using Chi-square or Fisher exact tests.
RESULTS: 
The study cohort consisted of 109 adults with Hb SS, 10 with Hb SC, 5 with Hb S beta thalassemia zero, and 3 with Hb S beta thalassemia plus. Of these, there were 35 subjects with leg ulcers (33 with Hb SS, 1 with Hb S beta thalassemia zero, 1 with Hb SC disease). Naturally occurring anticoagulants were found to be decreased in the sub-group of subjects with leg ulcers. In SCD patients with leg ulcers, 49% had functional protein C levels that were below the lower limit of normal, compared to 27% in SCD patients without leg ulcers (*p=0.0220); 74% had functional protein S levels that were below the lower limit of normal, compared to 51% in SCD patients without leg ulcers (*p=0.0181); 23% had antithrombin III (AT III) levels that were below the lower limit of normal, compared to 17% in SCD patients without leg ulcers (p=0.4820); and 29% tested positive for lupus anticoagulant, compared to 25% in SCD patients without leg ulcers (p=0.6818). In SCD patients with leg ulcers, none of the subjects had the prothrombin gene mutation, while 3 subjects without leg ulcers were heterozygous for the prothrombin mutation. None of the subjects in our cohort had factor V Leiden mutation.
CONCLUSIONS: 
In SCD patients with leg ulcers, protein C and protein S levels are significantly lower than in SCD patients without leg ulcers. Additionally, in SCD patients with leg ulcers, there is a trend towards decreased levels of AT III, and higher percentage of patients that are positive for lupus anticoagulant. Factor V Leiden and prothrombin gene mutations were rare in our cohort, consistent with the general population. 
Our results of decreased naturally anticoagulants confirm previous reports from our group that demonstrated similar findings. Of greater significance, however, is our finding that sickle cell patients with leg ulcers have further decreased levels of the naturally occurring anticoagulants, compared to their counterparts without leg ulcers. This finding lends credence to the hypothesis that sickle cell patients with leg ulcers have a procoagulant phenotype.1,2 In such patients, the clinical index of suspicion for thromboembolic events should be high. It is important to consider and evaluate for venous clots in such patients. A deep venous thrombus (DVT) found in a sickle cell patient with leg ulcer, could be the primary inciting factor for the ulcer or deterrent for wound healing.  
Future directions of our study involve correlating these laboratory values with clinical history and findings in these patients. History of thromboembolic events such as DVT and pulmonary embolism (PE) will be evaluated to detect if the laboratory findings clinically correlate to a procoagulant phenotype. 
References: 
1-Am J Hematol. 2011 Aug; 86(8): 705–708. 
2-Am J Hematol. 2014 Jan; 89(1): 1–6



Speakers
avatar for Ugochi Ogu, M.D.

Ugochi Ogu, M.D.

Assistant Professor/ Asssitant Director, Montefiore Medical Center Division of Hematology, Department of Oncology
Dr. Ogu is an Assistant Professor of Clinical Medicine and Pediatrics at the Albert Einstein College of Medicine. She obtained her medical degree at Windsor University School of Medicine in the West Indies, completed residency in Pediatrics at the University of Iowa, and fellowship... Read More →


Friday October 12, 2018 4:15pm - 4:30pm
Constellation C

4:15pm

Digital Engagement In The Sickle Cell Community
Abstract

Authors:
Ms. Robin C. Howard- Global Blood Therapeutics, Mr. Daniel C. Reinhardt- Return on Focus,
Ms. Jeanne M. Loboda- Global Blood Therapeutics, Ms. Abigail M. Manning- Return on Focus

Objective: Sickle cell disease (SCD) is a genetic, lifelong blood disorder. Many individuals with SCD lack access to adequate resources and care because of the small number of hematology specialists and primary care physicians who have experience treating the disease.
Connecting families with SCD with educational resources to help them better understand the disease, available treatment options, and sources for support is crucial to promote health literacy, improve communication with health care providers, and foster dialogue with the broader community. The goal of this study was to understand how caregivers and individuals with SCD engage digitally to find, use, and share information related to SCD.
Methods: In December 2017, a 25-question online survey was fielded by Return on Focus to address the study goals. Eligible participants included primary caregivers of children aged 6-17 years and adults aged 18-60 years with SCD. Participants were required to reside in the US. Participants were recruited via an online panel.
Results: A total of 61 participants completed the survey. Participants included 51 adults with SCD and 10 primary caregivers of children with SCD.
In the adult participant cohort, 65% were male and the average age was 37 (range 22-55 years); 84% reported current or past use of hydroxyurea; 56% reported seeing a primary care physician (PCP) for the treatment of SCD, and 33% reported seeing a hematologist; 49% reported attaining a bachelor’s degree, and 25% reported attaining an advanced degree.
In the primary caregiver cohort, 60% reported caring for a male child, and the average age of the child cared for was 10 (range 6-15 years); 70% reported current or past use of hydroxyurea; 50% reported seeing a PCP for the treatment of SCD, and 30% reported seeing a hematologist; 60% reported attaining a bachelor’s degree, and 30% reported completing some college.
More than half of the participants reported that they had good or very good knowledge of SCD and its treatments overall. About three-quarters of adults and half of caregivers reported searching digitally for information related to SCD at least weekly. Approximately 40% of adults and caregivers reported using a mobile or smart phone to search for information related to SCD almost daily. Desktops, laptops, and tablets were also used but less frequently, and they were more likely to be used weekly.
Adults and caregivers were very likely to seek information via general searches (eg, Google), as well as by browsing their treatment centers, professional societies, and general health and medicine websites, with treatment centers and professional societies rated as highly influential sources. Participants highly valued information provided by health care providers, but information provided by family and friends was also very valuable when making treatment or medication decisions related to SCD. SCDAA, Sickle Cell 101, WebMD, and Mayo Clinic were top visited sites overall, with more than 25% of participants visiting these sites in the past 6 months. For caregivers, KidsHealth was also a top site, with half of participants visiting within the same time period.
Over 60% of participants reported taking information that they found online to their healthcare provider for further discussion. About one-third of participants reported sharing information they found online with family and friends, and about 20% reported using information they found online to advise others about SCD.
These results compare favorably when compared with normative data for the same questions that have been asked of respondents living with other diseases (including migraine, idiopathic pulmonary fibrosis, cardiovascular disease, and cancer) indicating that individuals with SCD are very active and highly amenable to receiving disease education online.
Conclusions: Individuals with SCD and primary caregivers of children with SCD are active seekers of information online, and many are using information that they find digitally to help inform discussions with their healthcare providers. Various devices are used for online searches. Mobile devices are used most often, but laptop and desktop computers are also used by the same individuals, though less frequently. Content should be formatted across a variety of screen sizes, to ensure readability and an overall good user experience. Materials should be developed for ease of printing or sharing, so patients can easily bring them to their healthcare provider visits to drive productive conversations. Given the high reliance on Internet searches to seek information, it is important for providers of disease education content to consider search engine optimization in their strategies.

Speakers
RC

Robin C. Howard

Market Strategist, Global Blood Therapeutics


Friday October 12, 2018 4:15pm - 4:30pm
Baltimore/ Annapolis

4:15pm

The Importance of Social Work In Complex SCD Care: A Model For Clinical Intervention
Abstract

Author: 
Prof. Teresa Works- New England Sickle Cell Institute, UCONN Health

Teresa Works, LCSW, ACSW, Ph.D. Candidate, New England Sickle Cell Institute, UCONN Health:
Objective: To understand the importance of the role of social worker as part of a multidisciplinary SCD team to improve SCD care while reducing inpatient length of stay, 30 day re-admissions and improving financial solvency of institutions providing SCD care as well as the financial burden of chronic disease on patients.
Methods: Sickle cell disease (SCD) is a common genetic blood disorder in the United States. It is estimated to affect approximately 100,000 African Americans and other people of color in the U.S. SCD causes the oxygen carrying hemoglobin to have an irregular “sickled” shape, which occludes small blood vessels and organs and reduces oxygen supply to cells, organs and bones. These acute vaso-occlusive episodes result in chronic anemia, organ dysfunction and causes unpredictable acute painful crisis which are the primary causes of hospitalizations in persons with SCD. Many SCD patients develop chronic pain and complex co-morbidities requiring specialized and expensive medical care. “The estimated cost of medical care for the individuals with sickle cell disease in the US exceeds $1.1 billion at about $16,000 annually or $1,341 per patient per month. This figure does not include the impact on reduced quality of life, lost productivity, un-compensated care and premature mortality. If these were included the full economic costs of SCD are likely to be much higher”. [1]
A retrospective analysis of the implementation and outcomes of the New England sickle Cell Institute was performed as it relates to several national benchmarks of care was performed for the period of 2012-2017. In 2012, UCONN Health developed the New England Sickle Cell Institute (NESCI): a comprehensive, multidisciplinary team to navigate patient care across both inpatient and outpatient settings. Integral to this team, was the designated, full time licensed clinical social worker (LCSW). The role of the social worker at the NESCI is unique, in that it covers SCD patients in both inpatient and outpatient settings providing a variety of clinical services focused on the unique needs of each SCD patient. The role includes daily rounding with the inpatient multidisciplinary team on the SCD inpatients and facilitating discharge planning, NESCI follow up, and coordination of aftercare. The social worker works within the NESCI clinic having scheduled (and unscheduled) outpatient appointments with SCD patients to provide psychosocial and behavioral health services. In addition, the social worker assists SCD patients with transportation, medical protection for utilities, insurance and entitlements, grant funding for patient unpaid medical bills, referral for employment services, housing and a myriad of other needs. These psychosocial, discharge planning and concrete service needs, if unmet, can cause non-adherence with treatment, longer inpatient lengths of stay, difficulty accessing needed medical care, as well as protracted illness, pain and suffering.
Results:  Over a 5 year period, the use of reporting such as (30) day readmission reports, inpatient length of stay (LOS), Emergency department utilization, and financial reporting assisted in clinical and programmatic developments to improve NESCI SCD care and demonstrate improved patient care and programmatic financial solvency. Clinical and programmatic goals were established and implemented over this period corresponding with the development of the multidisciplinary NESCI team including the LCSW. The social workers unique role proved invaluable in terms of reducing inpatient bed days, average length of stay and (30) days re-admissions. UCONN NESCI demonstrated that by expanding multidisciplinary SCD services, they were able to improve quality clinical care and generate revenue, while decreasing inpatient LOS and (30) day readmissions. These changes saved the institution significant financial savings, more than covering the cost of the clinical social worker’s salary and fringe, while improving patient access to social work clinical and behavioral health services, which decreased the financial burden of chronic SCD care to the patients as well.
Conclusion: SCD is a complex and expensive disease to treat and manage. Social workers, with their unique training in a “person in environment” approach, have a distinct and beneficial role with all chronic diseases, including SCD. Utilization of a dedicated SCD social worker can improve health care outcomes, reduce institutional costs and financial burdens to patients. Through these multidisciplinary interventions, SCD patients can receive improved quality clinical care, while simultaneously reducing the institutional costs of providing SCD care as demonstrated in the 5 year retrospective analysis at the NESCI at UCONN Health. These changes can be implemented systematically at adult SCD comprehensive centers across the country to expand access to SCD care and reduce financial burdens to institutions and patients.

Speakers
avatar for Teresa Works, MSW, LCSW

Teresa Works, MSW, LCSW

Clinical Social Worker, Property Management, Psychotherapist, UCONN Health
Teresa Works, LCSW, ACSW is a social worker with 25 years experience in medical and behavioral health settings. She is a doctoral candidate in social work research at the UCONN School of Social Work, where she also is an adjunct faculty member.  Teresa has been employed full time... Read More →


Friday October 12, 2018 4:15pm - 4:30pm
Constellation D

4:30pm

Crizanlizumab Treatment is Not Asoociated with the Development of Proteinuria and Hematuria in Patients with Sickle Cell Disease: Sustain Study Safety Analysis
Abstract 

Authors:
Dr. Regina Graul- Ironwood pharmaceuticals, Dr. Bina Tejura- Ironwood pharmaceuticals, Dr. José Trevejo- Ironwood pharmaceuticals, Dr. Courtney Walls- Ironwood pharmaceuticals, Ms. Meghan Bratton- Ironwood pharmaceuticals, Dr. Jennifer Chickering- Ironwood pharmaceuticals, Ms. Oluwafunke Ojo- Ironwood pharmaceuticals,Dr. Joshua Field- Froedtert and Medical College of Wisconsin

BACKGROUND: STRONG-SCD is an ongoing randomized, double-blind, placebo-controlled, Phase 2 study of the sGC stimulator, olinciguat (IW-1701), in patients with stable sickle cell disease. Olinciguat is an oral sGC stimulator that enhances NO-sGC-cGMP signaling. Intracellular cyclic guanosine monophosphate (cGMP) produced by soluble guanylate cyclase (sGC) in response to nitric oxide (NO) binding plays an important role in regulating vascular tone, smooth-muscle proliferation, fibrosis, and inflammation. Low NO bioavailability in SCD has been proposed to contribute to marked vascular inflammation, cellular adhesion, and vasoconstriction, which may propagate vaso-occlusive crisis and other complications of SCD. In preclinical studies in relevant animal models, olinciguat decreased biomarkers of intravascular inflammation, leukocyte-endothelial cell interactions, and neutrophil extravasation both alone and in combination with hydroxyurea.
METHODS: The clinical program with olinciguat includes completed Phase 1 single ascending and multiple ascending dose (MAD) studies in healthy volunteers and STRONG-SCD, the ongoing Phase 2 study. STRONG-SCD is a multicenter, randomized, double-blind, placebo-controlled study evaluating 12 weeks of treatment with olinciguat in SCD patients (NCT03285178). Its primary objective is to assess the safety and tolerability of 3 dose levels of olinciguat. Exploratory objectives include evaluation of plasma biomarkers of disease progression, as well as health-related patient-reported outcomes. In particular, daily symptoms of SCD will be assessed via eDiary using the Sickle Cell Disease Symptom Assessment Form (SCD-SAF). The SCD-SAF is a fit-for-purpose instrument designed to reflect the most important and relevant SCD symptoms that impact patients with SCD. The STRONG-SCD study is enrolling patients 16-70 years old with HbSS, HbSC, HbSβ0-thalassemia, or HbSβ+-thalassemia who have had 1 to 10 pain crises in the past year and who meet all protocol defined eligibility criteria. Patients may be on stable therapy of hydroxyurea and other approved SCD medications. An independent data monitoring committee will review safety data. Target enrollment is 88 patients across sites in the US.
RESULTS: Multiple doses of olinciguat were well tolerated across the dose levels tested in the Phase 1 single and multiple ascending dose studies, with olinciguat showing target engagement as evidenced by reductions in blood pressure and increases in circulating cGMP concentrations. The most common adverse event (AE) reported was headache (22% in pooled treatment groups compared to 5% in placebo). Patients for the STRONG-SCD are still being enrolled.
CONCLUSION: Results of preclinical and clinical data supported the clinical development of olinciguat in SCD, with the STRONG SCD study initiated in initiated in 2017. Completion is expected in 2019.

Speakers
avatar for Julie Kanter, MD

Julie Kanter, MD

Lifespan Hematologist, Medical University of South Carolina
Dr. Julie Kanter is a lifespan hematologist specializing in sickle cell disease.  She is an associate professor of Pediatrics at the Medical University of South Carolina and runs the first all-ages, Lifespan Comprehensive Sickle Cell Center.  Dr. Kanter works closely with national... Read More →


Friday October 12, 2018 4:30pm - 4:45pm
Constellation C

4:30pm

Novel Ways To Recruit New Health Care Professionals Into The Sickle Cell Arena
Abstract

Authors:
Dr. Dr. Majorie Dejoie-Brewer- SCDAA/PDVC, Mrs. Donna Pennington Monroe, MS- SCDAA/PDVC

Increasing the number of qualified health care professionals who are knowledgeable, qualified and interested in sickle cell disease has been a primary initiative of the SCDAA national organization and the community at large. Recruiting future providers through nontraditional partnerships has been a focus of the SCDAA /PDVC chapter. One partnership formed with an initiative called "Bridging The Gaps" has done exactly that. Through this partnership are able to expose future physicians, nurses, social workers, dentists and veterinarians to the needs of the sickle cell community and their potential role as a member of their health care team. This program allows for a complete immersion of interns into the lives of individuals living with sickle cell disease. Through structured programming, education and exposure they are able to gain a true understanding of the disease process, the health care needs of the population , the bio-psychosocial paradigm, the social isolation, health care barriers and the daily struggle faced by these individuals. They are then challenged to develop an initiative that would benefit this population, develop a heart healthy program and develop a presentation on sickle cell disease for a community health symposium. This forum gives them an opportunity to see sickle cell disease through a different lense, become passionate about the population, communicate that compassion and enthusiasm to other providers and spark a true interest in wanting to care for the sickle cell population. The impact of this partnership has been significant and the individuals living with the illness have been the primary beneficiaries.
Objective: expose and recruit future HCP to sickle cell disease.
Methods: formal partnership with The Bridging the Gaps program. Training two interns for seven weeks, open forums with approximately 200 future providers, and four health awarness events.
Results: Improve understanding and increase the interest among future providers who may not exposed to SCD at all until later in their career. Increased awareness among HCPS, advocates, activists, other universities and community organizations.
Conclusion: More alliances such as these need to be formed so that our growing population will have capable, knowledgeable and caring providers in the future.

Speakers
DP

Donna Pennington Monroe, MS

Community Health Worker/ Research Coordinator, Sickle Cell Association
Donna Pennington Monroe has knowledge, skills, and experience in healthcare policy and delivery focusing on developing programs that educate and empower patients to improve their quality of life while reducing inefficiency, cost and improving access to care. Skilled in developing... Read More →


Friday October 12, 2018 4:30pm - 4:45pm
Baltimore/ Annapolis

4:30pm

The Forgotten Vital Sign: Use of A Distress Thermometer with Children, Adolescents and Young Adults Diagnosed with Sickle Cell Disease
Abstract

Authors : Mrs. carla Jones- Atrium Health: Levine Children's Hospital, Mr. Kenneth Mitten- Levine Children's Hospital Charlotte, NC

Objective: Information from our pediatric sickle cell disease (SCD) clinic indicates that we have an average noncompliance rate for attendance of clinic appointments of 30%. Our objective was to determine barriers to clinic attendance and strategies to overcome these barriers to improve compliance for 1 month clinic appointments for pediatric patients (birth-21 years of age) with sickle cell disease (SCD) on hydroxyurea.
Method: Caregivers of Sickle Cell patients indicated via a survey 5 main barriers that prevented them from consistently attending their 1 month clinic follow ups. They were transportation, inflexibility of employers, personal family issues, inconsistent contact information (frequent phone number and address changes) and even forgetfulness (no barriers) as primary reasons for missed clinic appointments. Pareto chart analysis revealed patients scheduled for 1 month appointments were most likely to miss appointments. A multidisciplinary team was formed and created an aim statement, current and ideal process flow maps, and tested multiple change ideas with small tests of change. Changes tested include offering offsite lab visits, posting signage to assist with transportation, mailing letters after missed appointments, assisting with reminders in phones, reminder calls to patients with a history of missed appointments, and giving out patient expectation forms to new patients and at yearly appointments.
Results: Weekly show percentage of patients with SC disease on hydroxyurea attending their 4wk follow up appointment was evaluated using a p Shewhart chart. Upper control limit is 100%. Lower control limit is 40%. Mean percentage of target population attending scheduled appointments is 85%.
Implementing personal reminder phone calls to patients/families with a history of missing appointments was a successful strategy to improve show rate. along with Offering offsite lab visits and mailings of letters sharing the number of no show appointments with parents of children with only minimally did not significantly improved compliance rate for appointments. The letters did increase the number of families calling ahead to reschedule appointments.
Conclusions: Attending regular clinic appointments is an important component in positively impacting continuity of care and to maintain health for patients with SCD
By developing a well-defined process, effectively communicating expectations, and using data to guide next steps, this project demonstrates an effective approach to increasing clinic attendance in a vulnerable population.

Speakers
KM

Kenneth Mitten, MSW

Pediatric Sickle Cell Social Worker, Levine Childrens Hospitals
I am a Pediatric Sickle Cell Social Worker in the Pediatric Hematology, Oncology, and Transplant Program at Levine Children’s Hospital in Charlotte, NC. I specialize in treating the psychosocial needs of children, adolescents and young adults and families living with Sickle Cell... Read More →


Friday October 12, 2018 4:30pm - 4:45pm
Constellation D

4:45pm

Genome Editing to Fix the Sickle Cell Disease Causing Variant in Sickle Cell Patient Hematopoietic Stem Cells
Abstract 

Authors:
Dr. Matthew Porteus- StandFord University, Dr. Daniel Dever- StandFord University, Dr. Annalisa Lattanzi- StandFord University, Mr. Joab Camarena- StandFord University,Mr. Carsten Charlesworth- StandFord University, Dr. Helen Segal- StandFord University, Dr. Neehar Bhatia- StandFord University, Dr. David Digiusto- StandFord University

Objective: The objective our research program is to complete the pre-clinical work demonstrating the safety and efficacy of genome editing of somatic hematopoietic stem cells to correct the sickle cell disease causing variant in order to develop a strategy to potentially cure sickle cell disease.
Methods: We have developed a genome editing approach that utilizes the CRISPR/As9 nuclease system that directly corrects the sickle cell disease causing mutation in hematopoietic stem cells using homologous recombination. We isolate both healthy donor and patient derived CD34+ hematopoietic stem and progenitor cells (HSPCs). We then genetically modify these cells ex vivo using a combination of CRISPR/Cas9 nuclease and AAV6 transduction. We evaluate the efficacy of the approach by measuring the percent hemoglobin A in red blood cells derived from genome edited sickle cell patient HSPCs. We evaluate the safety and efficacy by measuring the how well the edited HSPCs engraft and generate blood cells following transplantation of the human cells into immunodeficient (NSG) mice. We measure safety by determining the location of potential off-target INDELs created by the CRISPR/Cas9 nuclease the frequency that INDELs are created at these sites in CD34+ HSPCs.
Results: In CD34+ HSPCs from sickle cell patients, we are able to achieve 60-80% allele correction of the sickle cell mutation. These edited cells give rise to red blood cells that express ~92% Hgb A and 8% Hgb S. When transplanted into mice, the percent allele correction remains above 50% in the human cells thus demonstrating that the modified cells retain their key stem cell properties--the ability to give rise to long-term hematopoiesis in an animal model. After transplantation there is no evidence of abnormal hematopoiesis from the human cells thus providing evidence of safety. When we use a high-fidelity version of Cas9, we detect off-target mutations at only a single site and this site is 95 kb away from the nearest protein coding gene and of no known functional significance. We have submitted a pre-IND application to the FDA and based on FDA feedback are performing the key experiments to file a successful IND to initiate a phase I/II clinical trial testing the strategy in patients. These experiments including scaling up the process to a clinical scale and performing more complete testing of safety in an animal model. These studies are ongoing and we will present ongoing results at the meeting.
Conclusions: Genome editing provides a potentially precise mechanism to genetically correct the disease causing variant that causes sickle cell disease. Our preclinical results are very promising and if we are able to complete our IND enabling experiments we hope to launch a phase I/II clinical trial testing the strategy in 2019.

Speakers
avatar for Matthew Porteus, MD

Matthew Porteus, MD

Clinical Trial Specialist, Stanford University
Dr. Porteus is physician-scientist trained in Pediatric Hematology/Oncology who is an Associate Professor at Stanford University. His research program has focused on genome editing of hematopoietic stem and progenitor cells to develop cell based autologous cures for patients with... Read More →


Friday October 12, 2018 4:45pm - 5:00pm
Constellation C

4:45pm

Geography Seems To Be A Factor That Determines The Outcome Of Transition For Children With Sickle Cell Disease Into Adult Programs
Abstract 

Authors:
Dr. Nina Anderson, RN, DNP- TOVA Healthcare, Dr. Samir Ballas- Cardeza Foundation for Hematologic Research

The transition of patients with sickle cell disease (SCD) from pediatric to adult care providers represents a milestone in their lives. Major concerns among adolescents and young adults about transition include taking responsibility for self, making own decisions, cost of medical care, fear of suboptimal pain management, and reluctance to leave known providers. The reasons that cause this surge in mortality after transition are not well known. The role of geographical variations in the same country that affect the outcome of transition is examined in two major metropolitan cities include Philadelphia, PA and Atlanta, GA. In Philadelphia, 90 adolescents and young adults with SCD were followed prospectively for 10 years (1994-2004) after transition from the pediatric sickle cell program at St. Christopher Hospital to the adult sickle cell center at Thomas Jefferson University Hospital. The rate of death in Philadelphia was highest in patients with SCD-SS type where 17 out of 65 patients (26%) died within the 10 years after transition. Complications of SCD after transition included stroke, acute chest syndrome, avascular necrosis, leg ulcers, anxiety, depression, priapism and multi-organ failure. About one-third of the patients developed persistent pain between vaso-occlusive crises.
In Atlanta, 387 adolescents and young adults records were reviewed retrospectively for 10 years (1996-2006). Most important among these is that the rate of death in Atlanta was overall much lower than that in Philadelphia: 5.8% in Atlanta versus 22.2% in Philadelphia during the 10 years after transition. The major cause of death in Atlanta was iron overload due chronic organ damage. The care systems available to the youth in the two cities differ because the Georgia Comprehensive Sickle Center at Grady provides emergency care 24 h a day 7 days a week and has had an active transition program since 1985. Other possible causes include the warmer weather in Atlanta, the cultural and psychosocial community fabric in Atlanta may be more attentive to the problems associated with SCD and the genetic types of SCD in Atlanta may include those that are known to be associated with milder disease. These types include the Senegalese haplotype that is more common in Southern USA. In the Philadelphia area the Benin haplotype, known to be associated with more severe disease, is most common.

Speakers
avatar for Nina Anderson, RN, DNP

Nina Anderson, RN, DNP

Executive Director & Founder, Tova Community Health
Nina F. Anderson, DNP, RN, FNP-BC is executive director and founder of Tova Community Health, Inc., and an advanced practice nurse care coordinator and nurse Consultant at Tova Healthcare in Wilmington, DE. She previously served as director of the Adolescent and Young Adult Interdisciplinary... Read More →


Friday October 12, 2018 4:45pm - 5:00pm
Baltimore/ Annapolis

5:00pm

Correction of the Sickle Mutation in Hematopoietic Stem/Progenitor Cells with CRISPR/Cas9: The Berkeley/UCSF/UCLA Sickle Cell Clinical Translation Project
Abstract

Authors: 
Dr. Mark Dewitt-UC Berkeley, Mrs. Wendy Magis-CHORI, Dr. Stacia Wyman-UC Berkeley, Dr. Zulema Romero-UCLA, Mr. Jonathan Vu- UC Berkeley, Prof. Dario Boffelli- CHORI, Dr. Mark Walters- USCF Benioff Children’s Hospital Oakland, Dpt. Of Hematology/Oncology: Center for Inherited Blood Disorders, Dr. Donald Kohn-UCLA, Prof. Jacob Corn –UC Berkeley, Prof. David Martin- CHORI

Sickle Cell Disease (SCD) is a recessive genetic disorder caused by a single nucleotide polymorphism (SNP) in the ß-globin gene (HBB). CRISPR/Cas9 is a gene editing technology that can be used to correct the sickle mutation directly. Here we report progress towards clinical translation of a CRISPR/Cas9 gene editing approach to treat SCD. Our focus is on direct correction of the disease mutation itself. Our approach is based on electroporation of a Cas9 ribnucleoprotein (purified Cas9 protein and a synthetic protected sgRNA) with an optimized single-stranded DNA donor which programs correction of the sickle mutation by homology-directed repair (HDR) in hematopoietic stem/progenitor cells (HSPCs) from sickle cell disease donors. Our approach relies on animal-free synthetic reagents, avoiding use of viral vectors. By avoiding viral HDR donors, our approach promises to reduce manufacturing costs, and ultimately improve accessibility. We obtained surplus HSPC from participants in a sickle cell disease gene therapy trial after mobilization with plerixafor, the same source of cells in our planned clinical protocol. Using an optimized gene correction protocol, we routinely achieved >30% correction of the mutation in these cells. When differentiated into erythroblasts, 70% of hemoglobin is non-sickle, of which 40% is corrected adult hemoglobin. When transplanted into immune-deficient mice, corrected cells maintain engraftment ~10-fold better than cells edited with a AAV6 viral HDR donor. Most importantly, the corrected allele is maintained in 22% of alleles in bone marrow, a substantial improvement over previous studies in SCD. In glycophorin-expressing erythroblasts in circulating blood, the corrected allele is further enriched to 30%, evidence for ineffective erythropoiesis in sickle- or indel-containing cells.
To define the off-target genotoxicity of our sickle correction protocol, we identified off-target sites using a combination of GUIDE-seq and bioinformatics. Using a pooled primer PCR approach, we find that the vast majority of these off-targets have no detectable mutagenesis. We found that a commercially-available high-fidelity Cas9 variant can reduce off-target activity 20-fold or more in HSPCs, with no loss in on-target activity. Only one single off-target site is targeted in >0.2% of the cells. Together, these studies suggest a manufacturing protocol to correct CD34+ HSPC from SCD patients that is both safe and effective in delivering a long-term clinical benefit. We have started IND-enabling studies, and anticipate filing an IND to commence a Phase I clinical trial in 2019.

Speakers
avatar for Mark Dewitt

Mark Dewitt

Project Scientist, Berkeley University
"Mark DeWitt is project manager for a late-stage preclinical development project in sickle cell disease, funded through a grant from the California Institute for Regenerative Medicine (CIRM). The project is a collaboration between UCLA, UC Berkeley, and UC San Francisco. Our approach... Read More →


Friday October 12, 2018 5:00pm - 5:15pm
Constellation C

5:15pm

The Clinical-Stage SGC Stimulator Olinciguat Prevents Increase Of Plasma Biomarkers Of Intravascular Inflammation And Suppresses Leukocyte-Endothelial Interactions In Tnfa-Treated Mice
Abstract

Authors:Dr. Regina Graul- IronWood Pharmaceuticals, Dr. Boris Tchernychev- IronWood Pharmaceuticals, Ms. Susan Feil- University of Tubingen, Prof. Robert Feil- University of Tubingen, Ms. Elisabeth Lonie –IronWood Pharmaceuticals, Dr. Peter Germano- IronWood Pharmaceuticals, Dr. G. Todd Milne- IronWood Pharmaceuticals, Mr. John Hadcock- IronWood Pharmaceuticals, Dr. Yueh-Tyng Chen- IronWood Pharmaceuticals, Dr. Mark Currie- IronWood Pharmaceuticals    

 Objective: Olinciguat is a soluble guanylate (sGC) stimulator in clinical development for the treatment of patients with sickle cell disease (SCD). sGC is a signaling enzyme that increases the production of cyclic guanosine 3’,5’-monophosphate (cGMP) from guanosine triphosphate in response to nitric oxide (NO) binding. The sGC stimulator olinciguat binds to sGC and enhances its NO-dependent activity. Conditions of low NO bioavailability, as seen in hemoglobinopathies such as sickle cell disease, are proposed to lead to increased vascular inflammation, cellular adhesion, and reduced blood flow due to vasoconstriction. By enhancing NO signaling through sGC, olinciguat is hypothesized to reduce vascular inflammation and restore blood flow and tissue oxygenation. Preclinically, modulation of the NO-sGC-cGMP pathway via treatment with NO or with inhibitors of the cGMP-selective phosphodiesterase 9 has been demonstrated to significantly attenuate TNFa-induced intravascular inflammation in C57BL/6 and sickle cell mice. We evaluated the effect of olinciguat alone and with hydroxyurea (HU, current SCD standard of care) on biomarkers of intravascular inflammation, leukocyte-endothelial cell interactions, and neutrophil trafficking in C57BL/6 mice.
Methods and Results: Treatment of C57BL/6 mice with TNFa (50 ng/mouse, ip) increased the plasma levels of biomarkers of endothelial cell activation (sP-selectin, sE-selectin, sICAM-1) and leukocyte activation (MIP-2, sL-selectin). Pretreatment of mice with olinciguat (10 mg/kg, po) attenuated the increase in plasma concentrations of MIP-2 and sL-selectin following TNFa challenge by 81% and 58%, respectively. The TNFa-induced increase in plasma levels of sP-selectin, sE-selectin, and sICAM-1 were attenuated by 31%, 37%, and 34%, respectively, in olinciguat-treated mice. Co-treatment with HU augmented the effect of olinciguat on plasma concentrations of sE-selectin and sICAM-1 in TNFa-treated mice.
The effects of olinciguat, HU, and the combination on leukocyte endothelial cell interactions were assessed using intravital microscopy of post-capillary venules of the mouse cremaster muscle. Compared to controls, TNFa-treated C57BL/6 mice had slower leukocyte velocity (5.5±0.7 vs 26.6±3.1 µm/sec in controls) and leukocyte rolling flux (16.1±2.7 vs 43.3±7.1 cells/min in controls). Pretreatment with olinciguat (10 mg/kg) alone resulted in faster leukocyte velocity and rolling flux (10.3±1.1 µm/sec and 24.1±2.3 cells/min, respectively) compared to TNFa-treated controls. Similarly, pretreatment with HU (100 mg/kg, po) alone led to faster leukocyte velocity and leukocyte rolling flux (5.5±1.7 µm/sec and 50.2±5.9 cells/min, respectively). Co-administration of olinciguat and HU led to even faster leukocyte velocity and leukocyte rolling flux (19.7±1.9 µm/sec and 64.5±5.5 cells/min, respectively). Treatment with either olinciguat, HU, or the combination did not change leukocyte adherence following TNFa stimulation. To further evaluate the effect of olinciguat on neutrophil extravasation and trafficking we used a peritoneal recruitment model. Intraperitoneal injection of TNFa induced accumulation of Gr.1 polymorphonuclear leukocytes (PMN) in the mouse peritoneal cavity (4±0.4×105 PMNs/peritoneal lavage). Trafficking of PMNs into mouse peritoneum was attenuated by pretreatment of mice with HU (2.7±0.5×105 PMNs/peritoneal lavage). Co-administration of olinciguat and HU further attenuated the number of neutrophils extravasated into the peritoneum of TNFa-challenged mice (1.4±0.3×105 PMNs/peritoneal lavage) relative to TNFa-treated controls.
 Conclusion: In summary, prophylactic treatment with olinciguat inhibited the TNFa-induced increase in plasma levels of biomarkers of intravascular inflammation and attenuated the effect of TNFa on transient adhesive interactions between leukocytes and endothelial cells in mice. Co-administration with HU led to significantly reduced neutrophil trafficking and augmented the effect of olinciguat on biomarkers of endothelial cell activation and transient adhesive interactions in the mouse cremaster muscle. This study supports evaluation of the sGC stimulator olinciguat in patients with SCD.

Speakers
BT

Boris Tchernychev

Principal Investigator, IRONWOOD PHARMACEUTICALS


Friday October 12, 2018 5:15pm - 5:30pm
Constellation C

6:00pm

Red Carpet Reception and Photos
Friday October 12, 2018 6:00pm - 6:45pm
Constellation Foyer

7:00pm

UNITY SOIREE
Friday October 12, 2018 7:00pm - 10:30pm
Constellation AB
 
Saturday, October 13
 

7:00am

General Evaluation Form
Saturday October 13, 2018 7:00am - 9:00pm

7:00am

Nursing Evaluation Form
Saturday October 13, 2018 7:00am - 9:00pm

7:00am

Social Worker Evaluation Form
Saturday October 13, 2018 7:00am - 9:00pm

7:30am

Breakfast
Saturday October 13, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Speed Networking
Saturday October 13, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Super Power Hour
Saturday October 13, 2018 7:30am - 8:30am
Harborview 300 Light Street, baltimore, MD 21202

7:30am

Exhibits Open
Saturday October 13, 2018 7:30am - 1:00pm
Constellation EF

7:30am

Get Connected Kiosk
Saturday October 13, 2018 7:30am - 3:00pm
Broadcast Lounge B 300 Light Street, baltimore, MD 21202

8:00am

SCDAA Board Meeting
Saturday October 13, 2018 8:00am - 10:00am
Chesapeake A 300 Light Street, baltimore, MD 21202

8:30am

Pravastatin-Mediated Modulation Of Sickle Red Cell Adhesion Using Mouse Endothelial Cells In Vitro
Abstract 

Authors:
Dr. Ogechukwu Egini- Interfaith Medical Center, Dr. Edouard Guillaume- Interfaith Medical Center, Dr. Eric Jaffe- Interfaith Medical Center, Dr. Xian-Cheng Jiang- SUNY Downstate Medical Center, Mrs. Stephanie Egini- Caters Plan for Healthy Living, Dr. Ugochi Ogu- Montefiore Medical Center, Ms. Miren Blackwood- Interfaith Medical Center, Dr. Antony Lixon- Interfaith medical Center

Background - Beside lipid-lowering effects of statins, they have been shown to decrease inflammatory markers in many trials. The PRINCE (Pravastatin inflammation/CRP evaluation) trial was a 24 week study in which volunteers were treated with Pravastatin and inflammatory markers were measured at end of study. A significant reduction in serum CRP levels in subjects was noted, independently of any changes in LDL cholesterol. Furthermore, simvastatin reduced vaso-occlusive pain and oral analgesic use in patients treated over months.
Objective - To determine whether Pravastatin reduced sickle cell adhesion to endothelial cells using mouse endothelial cells in culture.
Design - Randomized experimental study
Study subjects - 4 sickle cell anemia patients from Interfaith Medical Center sickle cell outpatient program and 3 normal controls were recruited from the outpatient clinic. Subject recruitment was approved by the Institutional Review Board.
Interventions - Demographic information and EDTA blood samples were collected. Red cells were centrifuged, washed and stained with Vybrant Dil fluorescent cell labeling solution. Mouse endothelial cells were cultured on well plates. They were then treated with either Pravastatin or placebo for 1 hour. Both normal and sickle red cells were then activated with 20nM epinephrine and then introduced into the endothelial cell culture medium treated with either Pravastatin or placebo. Incubation was done for 30 minutes.
Main measures - Comparison of the number of red cells adherent to the mouse endothelial cell membrane in culture.
Results - In the placebo and treatment groups, there was significant sickle red cell adhesion compared to normal cells (P value < 0.05). When adherent sickle red cells in both placebo and treatment groups are compared, there was significant sickle red cell adhesion to mouse endothelial cell membrane in the placebo group (p<0.05).
Conclusion - Pravastatin reduced sickle red adhesion to mouse endothelial cell membrane.

Speakers
OE

Ogechukwu Egini, MD

Medical Resident, Interfaith Medical Center


Saturday October 13, 2018 8:30am - 8:45am
Constellation B

8:30am

Rate Of Vaso-Occlusive Crisis Episodes Among Sickle Cell Disease Patients In The US Medicaid Population
Abstract

Authors:
Dr. Menaka Bhor- Novartis, Dr. Lin Xie- SIMR, Inc, Dr. Jincy Paulose- Novartis, Dr. Huseyin Yuce- New York City College of Technology, Dr. Nirmish Shah- Duke University

OBJECTIVE: Vaso-occlusive crisis (VOC) is the hallmark of patients with sickle-cell disease (SCD), and cause patients severe pain, life-threatening complications, hospitalization, decreased quality of life, and early death. This study evaluated the rate of VOC episodes by place of service among adult patients with SCD in the US Medicaid population.
Methods: A retrospective study was conducted using the Medicaid Analytic Extracts Database from 01JAN2009-31DEC2013. Adult patients with SCD were identified using ICD-9-CM diagnosis codes (282.41-282.42, 282.60-282.69). The first observed SCD diagnosis date during the identification period (01JUL2009-31DEC2012) was designated as the index date. Patients were required to have continuous Medicaid health plan enrollment for ≥6 months pre-index date (baseline period) and ≥12 months post-index date (follow-up period). VOC episodes were examined for various follow-up periods, until the earliest occurrence of health plan disenrollment, death, or end of the study period (31DEC2013). Patient demographics and baseline clinical characteristics were examined. VOC claims within a 3-day gap were combined and considered as one VOC episode. The rate of VOC episodes per person-year during the follow-up period was calculated as total number of VOC episodes divided by the total follow-up length to adjust for different patient follow-up periods. VOC episodes in the first month of follow-up was also examined and stratified by setting care, including hospitalization, emergency room (ER), outpatient, office, and other. All variables were analyzed descriptively.
 RESULTS: The study included 8,521 patients; mean (standard deviation [SD]) age was 33 (12) years; 74.0% were identified as black, and 67.3% were female. The most common comorbidities during the baseline period were infectious and parasitic disease (19.7%), asthma (10.9%), and fever (9.3%). Mean (SD) length of follow-up period was 2.7 (1.0) years. The rate of VOC episodes was 3.31 per person-year for the overall adult SCD patient population during the entire follow-up period. During the first month of follow-up, 35.2% of patients had ≥1 VOC episode. Mean (SD) number of pain crisis episodes in the first month was 0.5 (0.8) among the adult SCD patients. Of those, 38% were treated during the hospitalization, 31% in the ER, 20% in the outpatient setting, 8% in a physician office, and 3% were treated in other settings (Figure 1). ConclusionS: Adult SCD patients are burdened with a high rate of VOC episodes, with 35% of the patients having at least one VOC episode in 1 month. In addition, SCD patients utilized a substantial amount of health care resources, with the majority of VOC care (70%) occurring within the hospital and ER

Speakers
avatar for Menaka Bhor, PhD

Menaka Bhor, PhD

Associate Director, Novartis
Currently serving as an Associate Director, HEOR at Novartis Oncology and responsible forSickle Cell Disease portfolio. Previously as a Director at Cardinal Health generated compellingproposals for new sales opportunities and served as scientific lead on retrospective and prospectivestudies... Read More →


Saturday October 13, 2018 8:30am - 8:45am
Baltimore/Annapolis

8:30am

Improving Provider Readiness For Young Adult Transitions: The (8) Behavioral Changes Providers Can Make To Improve Transition Outcomes
Abstract 

Authors:
Prof. Teresa Works- New England Sickle Cell Institute, UCONN Health

Objective: Sickle cell disease (SCD) is a genetic blood disorder estimated to impact about 1 in 400 individuals of African American decent in the United States. SCD can typically cause both acute and chronic pain crisis. Secondary co-morbidities of SCD include risk(s) of infection, strokes, splenic infarct, pulmonary embolus, acute chest syndrome and other acute medical conditions, which can dramatically increase risk of death. Historically, children with SCD did not live until adulthood. However, beginning in 1990’s, the utilization of antibiotics with children, dramatically decreased mortality in children with SCD. As a result, most now live until adulthood. Due to more adults living with SCD there is an increasing need for comprehensive teams of adult sickle cell providers to provide care to these young adults, who are aging out of pediatric care. Ideally, adult and children’s comprehensive SCD programs collaborate around effective and efficient transition programs as they facilitate young SCD adults transitioning to adult SCD care. Studies indicate that those young adults not transitioning to an adult provider are at higher risk of mortality.
Much of the research focused on the transition process has been on young adult readiness for transition, including the readiness of their families. However, there are behavioral and structural changes that can be made through coordination of care between adult and pediatric providers, which can dramatically improve transition outcomes regardless of adolescent readiness. These successful transition outcomes are demonstrated by the collaboration between pediatric providers at Connecticut Children’s Medical Center (CCMC) and the adult New England Sickle Cell Institute (NESCI) at UCONN Health and the changes implemented to their transition program over the past six years.
Methods: A retrospective analysis of CCMC patients seen in transition clinic and referred to the NESCI was performed from 2012- present by the CCMC registered nurse and the UCONN Health social worker, to assess how many patients had successfully transitioned to UCONN of those referred. For those patients who had not connected with a UCONN provider, CCMC staff attempted to contact them to ascertain if they had been connected to another hematologist or if they still needed an adult hematology provider. If a provider was needed, and the patient was willing, an appointment was scheduled at UCONN. A spread sheet was created to track where referred CCMC transitioning young adults were in the transition process. Clients who missed transition appointments were contacted by CCMC and the importance of transitioning was re-iterated. UCONN SW staff apprised CCMC of patient attendance, no-shows or cancellations at UCONN tour and initial appointments, who would then reinforce transition to UCONN and / or schedule a termination appointment with the transitioning young adult to celebrate their “graduation” to the adult provider. In this process, consistent behavioral changes on the part of the providers to ensure transition were implemented with excellent results.
Results: Use of this close tracking system and improved communication between the two institutions caused a 96% success rate in those young adult patients being referred to the adult provider successfully transitioning to care, from the period of 2012-2018 regardless of adolescent “readiness”. Provider focus on adolescent and young adult readiness for transition is important in terms of long term implementation of good disease management, however, it need not be a negating factor in the successful transition of young adults from pediatric to adult provider where disease management skills can continue to be worked on and develop over time under the care of the adult SCD comprehensive clinic.
Conclusions: Goals of adolescent “readiness” for transition are often abstract, difficult to define and vague in terms of pediatric and adult SCD provider team agreement on when patients are ready to transition. Changes on the part of the providers are in many ways easier to implement and can improve communication, provide consistent messages to patients and can lead to a smoother transition process. Improved success with transitioning pediatric SCD patients to SCD adult providers, can lead to better SCD health care and health outcomes.

Speakers
avatar for Teresa Works, MSW, LCSW

Teresa Works, MSW, LCSW

Clinical Social Worker, Property Management, Psychotherapist, UCONN Health
Teresa Works, LCSW, ACSW is a social worker with 25 years experience in medical and behavioral health settings. She is a doctoral candidate in social work research at the UCONN School of Social Work, where she also is an adjunct faculty member.  Teresa has been employed full time... Read More →


Saturday October 13, 2018 8:30am - 8:45am
Constellation CD

8:30am

Mental Health and Sickle Cell Disease
Speakers
avatar for Selena Webster-Bass, MPH

Selena Webster-Bass, MPH

CEO/Lead Innovator, Voices Institute, LLC
Selena Webster-Bass, M.P.H., is a Jacksonville, Florida native with more than 20 years of experience in cultural competency, health equity and community health. Selena has an undergraduate degree in Biology from the University of North Florida and a Masters of Public Health from the... Read More →


Saturday October 13, 2018 8:30am - 9:30am
Frederick/Columbia

8:30am

Registration
Saturday October 13, 2018 8:30am - 3:00pm
Constellation Foyer

8:45am

The Strong-SCD Trial: A Phase 2 Study Ofolinciguat (IW-1701), A Stimulator Of Soluble Guanylate Cyclase (sGC) Inpatients With Sickle Cell Disease (SCD)
Abstract


Authors:
Dr. Regina Graul- Ironwood pharmaceuticals, Dr. Bina Tejura- Ironwood pharmaceuticals, Dr. José Trevejo- Ironwood pharmaceuticals, Dr. Courtney Walls- Ironwood pharmaceuticals, Ms. Meghan Bratton- Ironwood pharmaceuticals, Dr. Jennifer Chickering- Ironwood pharmaceuticals, Ms. Oluwafunke Ojo- Ironwood pharmaceuticals,Dr. Joshua Field- Froedtert and Medical College of Wisconsin
BACKGROUND: STRONG-SCD is an ongoing randomized, double-blind, placebo-controlled, Phase 2 study of the sGC stimulator, olinciguat (IW-1701), in patients with stable sickle cell disease. Olinciguat is an oral sGC stimulator that enhances NO-sGC-cGMP signaling. Intracellular cyclic guanosine monophosphate (cGMP) produced by soluble guanylate cyclase (sGC) in response to nitric oxide (NO) binding plays an important role in regulating vascular tone, smooth-muscle proliferation, fibrosis, and inflammation. Low NO bioavailability in SCD has been proposed to contribute to marked vascular inflammation, cellular adhesion, and vasoconstriction, which may propagate vaso-occlusive crisis and other complications of SCD. In preclinical studies in relevant animal models, olinciguat decreased biomarkers of intravascular inflammation, leukocyte-endothelial cell interactions, and neutrophil extravasation both alone and in combination with hydroxyurea.
METHODS: The clinical program with olinciguat includes completed Phase 1 single ascending and multiple ascending dose (MAD) studies in healthy volunteers and STRONG-SCD, the ongoing Phase 2 study. STRONG-SCD is a multicenter, randomized, double-blind, placebo-controlled study evaluating 12 weeks of treatment with olinciguat in SCD patients (NCT03285178). Its primary objective is to assess the safety and tolerability of 3 dose levels of olinciguat. Exploratory objectives include evaluation of plasma biomarkers of disease progression, as well as health-related patient-reported outcomes. In particular, daily symptoms of SCD will be assessed via eDiary using the Sickle Cell Disease Symptom Assessment Form (SCD-SAF). The SCD-SAF is a fit-for-purpose instrument designed to reflect the most important and relevant SCD symptoms that impact patients with SCD. The STRONG-SCD study is enrolling patients 16-70 years old with HbSS, HbSC, HbSβ0-thalassemia, or HbSβ+-thalassemia who have had 1 to 10 pain crises in the past year and who meet all protocol defined eligibility criteria. Patients may be on stable therapy of hydroxyurea and other approved SCD medications. An independent data monitoring committee will review safety data. Target enrollment is 88 patients across sites in the US.
RESULTS: Multiple doses of olinciguat were well tolerated across the dose levels tested in the Phase 1 single and multiple ascending dose studies, with olinciguat showing target engagement as evidenced by reductions in blood pressure and increases in circulating cGMP concentrations. The most common adverse event (AE) reported was headache (22% in pooled treatment groups compared to 5% in placebo). Patients for the STRONG-SCD are still being enrolled.
CONCLUSION: Results of preclinical and clinical data supported the clinical development of olinciguat in SCD, with the STRONG SCD study initiated in initiated in 2017. Completion is expected in 2019.

Speakers
avatar for Regina Graul, PhD

Regina Graul, PhD

SCD Program Leader Director, IronWood Pharmaceuticals
Regina Graul is the Director of R&D at Ironwood pharmaceuticals with more than 15 years of industrial experience. She is the founder and leader of the internal innovation group charged with identifying and validating new targets and indication opportunities. Additionally, Regina leads... Read More →


Saturday October 13, 2018 8:45am - 9:00am
Constellation B

8:45am

Pilot Group For Middle Schoolers With SCD
Abstract

Authors:
Ms. Jennifer Brereton- Children’s Hospital of Philadelphia, Ms. Trudy Tchume-johnson- The Children’s Hospital of Philadelphia, Ms. Tahira Austin- The Children’s Hospital of Philadelphia

Background: The Comprehensive Sickle Cell Center at Children’s Hospital of Philadelphia (CHOP) provides care for over 1,000 children diagnosed with sickle cell disease (SCD). Children with chronic illnesses, such as SCD, are at a higher risk for developing social/emotional issues. Currently, individual and family therapy is offered in our clinic by our psychologist, but there has not been any recent attempts to offer services in a group format.
Objective: To establish and offer a group program to our patients with SCD. The group was designed to target patients in the middle school age range (11-14), and to provide these patients with psychoeducation about SCD, support around their illness, an opportunity to connect with others living with SCD, and to teach coping and alternative pain management strategies (e.g. yoga). We also wanted to look at various ways to inform families about the groups and to increase participation and engagement from the community.
Methods: Approximately 2 months prior to the start of the group, families with patients in the targeted age range were approached in clinic about the group. A flyer was developed for the group which was distributed in clinic and was sent to families via standard mail as well as emailed through the Center’s list serve. Reminders were sent out on a weekly basis. The groups were run by the Center’s pediatric psychologist, licensed social worker, and community health worker. Group participants were provided with dinner, reimbursement for parking/transportation, yoga mats, water bottles, and gift cards for their participation. Although parents were not included in the group, they were provided with a handout for each group outlining the contents of what was covered, as well as tips for parents on how to continue related discussions at home with their children. Pre- and post-assessments on SCD knowledge and coping strategies were also obtained. Group format was as follows:
Session 1: SCD Basics & Living with SCD
Session 2: SCD Psychoeducation & School/Peer Issues
Session 3: Pain and Coping with SCD
Session 4: Coping with Pain Continued & Introduction to Yoga
Results: The group was held for 4 evening sessions on consecutive weeks in June. The initial session consisted of 5 participants, sessions 2 & 3 had7 participants and the final session had 9 participants. The pre- & post questionnaires were administered to 7 of the 9 participants. The questionnaire revealed the following; 5 out of 7 participants improved their scores, 1 remained the same and 1 decreased.
The participants were engaged and appeared to enjoy the interactive ways of learning about their illness. Positive feedback was received from all group members, as well as their parents. Families expressed interest in having additional group sessions.
The Center is going to send out a survey to all patient families inquiring about desired times and frequencies of groups in the future in order to try and establish an ongoing group program during the academic year.
Conclusions:
Addressing some of the barriers related to attending group (e.g. transportation, parking, food), as well as providing participants with some rewards, may have helped with group attendance and engagement. Some families who did not participate expressed interest in the group, but were not able to make this particular group session due to scheduling conflicts. In addition, caregivers of children in other ages also expressed interest and inquired about groups for younger and older patients. However, the majority of the families in the targeted age group did not respond to the invites so additional methods of increasing participation and engagement need to be explored. The patients and families that did participate, expressed positive feedback about the group, and the majority of the participants who completed pre- and post-assessment measures related to SCD knowledge improved their scores. Due to the interest and engagement of this pilot group, the Center will be offering additional groups in the fall, as well as a yoga group (4 sessions) during the month of August 2018.

Speakers
avatar for Jennifer Brereton, PsyD

Jennifer Brereton, PsyD

Pediatric Psychologist, The Children's Hospital of Philadelphia
Jennifer Leigh Brereton, PsyD, is a psychologist in the Department of Child and Adolescent Psychiatry and Behavioral Sciences at Children's Hospital of Philadelphia.


Saturday October 13, 2018 8:45am - 9:00am
Constellation CD

8:45am

Psychosocial Impact, Health Beliefs And Stigma Of Leg Ulcers In Adults With Sickle Cell Disease In A National Sickle Cell Center
Abstract

Authors:
Ms. MARY AKUA AMPOMAH- GHANA INSTITUTE OF CLINICAL GENETICS/UNIVERSITY OF GHANA, LEGON, Dr. Kofi Anie- Imperial College, London,
Dr. Edeghonghon Olayemi- GHANA INSTITUTE OF CLINICAL GENETICS/UNIVERSITY OF GHANA, LEGON, Dr. Adote Anum- University of Ghana, Legon, Dr. Yvonne Dei- GHANA INSTITUTE OF CLINICAL GENETICS/UNIVERSITY OF GHANA, LEGON, Mr. David Nana Adjei- University of Ghana, Legon, Prof. Solomon Ofori-acquah- University of Ghana, Legon

Background: Sickle Cell Disease (SCD) is recognized as the most predominant hereditary hemoglobinopathy in Africa with a significant public health impact. Currently, owing to the early detection through newborn screening, advancement in medicine, nutrition and patient education, people with SCD are now living longer into adulthood. However, clinical and psychosocial concerns remain on many persistent chronic manifestations such as leg ulcers. Hitherto, the psychosocial, health beliefs, and stigma-related problems that are experienced by SCD patients with chronic leg ulcers remain poorly defined.
Objective: This study examined the psychopathology, psychosocial impact, stigma and health beliefs experienced by adult SCD patients with and without leg ulcers attending a National Sickle Cell Centre in Ghana.
Methods: A total of 84 adults with SCD participated in the cross-sectional study: 36 had chronic leg ulcers and 48 without leg ulcers. Participants were administered Symptom Checklist-90-R, Sickle Cell Illness Impact Measurement, Health Belief Model Questionnaire and an adapted version of the Stigma of Mental Illness Scale. Factorial Multivariate Analyses of Covariance (MANCOVA) were conducted to determine the influence of leg ulcer, gender, and age of diagnosis of SCD on psychopathology, psychosocial, stigma and health beliefs of SCD patients. Age and education measured in years were covariates in the analyses.
Results: Leg ulcer had no impact on psychopathology of SCD patients. There was a significant effect of age of diagnosis of SCD on social functioning in patients [(3, 55) = 3.09, p < .035, Wilks' Λ = .86, partial η2 = .14]. Adult patients diagnosed with SCD after early childhood (aged 11years and above) experienced poor social functioning compared to patients diagnosed during early childhood (aged below11 years) [Social functioning (mean difference) 5.95; 95% confidence interval (CI), .1.04-10.85; p < .02)]. Leg ulcer and age of diagnosis influenced patients’ perception of stigma (discrimination, disclosure and positive aspects sub-scales) [(3, 50) = 4.75, p < .005, Wilks' Λ = .78, partial η2 = .22; (3, 50) = 3.23, p < .03, Wilks' Λ = .84, partial η2 = .16]. Adult patients with leg ulcer reported more frequency of discrimination associated with SCD compared to patients without leg ulcer [discrimination (mean difference 6.52; 95% CI, 2.03-11.02]; p < .005]. In addition, adult patients diagnosed with SCD after early childhood also reported more discrimination than adult patients diagnosed with SCD during early childhood [discrimination (mean difference 5.80; 95% CI, 1.15-10.44]; p < .015]. In addition, leg ulcer influenced SCD health beliefs (perceived severity, vulnerability, benefits and barriers sub-scales) [(4, 48) = 4.37, p < .004, Wilks' Λ = .73, partial η2 = .27]. Adult patients with leg ulcer reported more perceived severity, perceived barriers and negative health beliefs relating to SCD compared to those without leg ulcers [perceived severity (mean difference 2.15; 95% CI, 0.4-3.9]; p < .02), p < .003), perceived barriers (mean difference 3.52; 95% CI, 1.38 -5.67] ; p < .002; negative health beliefs (mean difference 7.88; 95% CI, 3.38-12.8].
Conclusion: Overall, our findings show that early identification and assessment of leg ulcers and their related SCD health beliefs and stigma may provide avenues for enhancing the quality of life of adults with SCD and living with chronic leg ulcers in Ghana.

Speakers
avatar for Mary Akua Ampomah

Mary Akua Ampomah

Senior Clinical psychologist, Ghana Institute of Clinical Genetics
I am a Clinical Psychologist at the Ghana Institute of Clinical Genetics where the adolescent and adult sickle cell clinic is located. I have also served as a part-time lecturer at the Department of Psychology of the University of Ghana, Legon and currently teaching at the Regent... Read More →


Saturday October 13, 2018 8:45am - 9:00am
Baltimore/ Annapolis

9:00am

SC411 For Treatment Of Children With Sickle Cell Disease: A Phase 2, Randomized, Double-Blind, Dose-Finding, Placebo-Controlled, Parallel-Group, Multi-Center Study (Scot Trial)
Abstract 

Authors:
Dr. Ahmed Daak- SCI, Dr. Matthew Heeney- Dana-Farber / Boston Children's Cancer and Blood Disorders Center, Harvard Medical School, Dr. Carlton Dampier- Emory University School of Medicine, Dr. Beng Fuh- East Carolina University Department of Pediatrics, Dr. Julie Kanter- Division of Pediatrics, Medical University of South Carolina, Charleston, South Carolina, USA, Dr. Ofelia Alvarez- University of Miami, Dr. Vandy Black- University of Florida College of Medicine, Dr. Melissa Mcnaull- University of Mississippi Medical Center, Dr. Michael Callaghan- Children's Hospital of Michigan, Dr. Alex George- Texas Children’s Hospital

Introduction:
Blood cell membranes of patients with sickle cell disease (SCD) have low docosahexaenoic acid (DHA) levels. DHA is a bioactive fatty acid known to have potent anti-inflammatory, anti- blood cell adhesion and anti-oxidant properties. There is evidence that treatment with DHA reduces the rate of sickle cell crises (SCC) and ameliorates the chronic inflammation, oxidative stress and hypercoagulable state observed in SCD patients. SC411 is a novel DHA ethyl ester formulation with a proprietary delivery platform (Advanced Lipid Technology® (ALT®)) that enhances DHA bioavailability. The SCOT trial investigated three different doses of SC411 in children with sickle cell disease.
Methods:
Children with SCD (HbSS, HbSC or S/β°‑thalassemia) aged 5-17 years (n=67), who had ≥2≤10 documented Sickle Cell Crises (SCC) during the 12 months prior to screening, and either had not received or who were on hydroxyurea (HU) for at least 6 months prior to screening, were enrolled. Subjects received either placebo or one of three increasing doses of SC411 (20; 36 or 60 mg/kg/day DHA, respectively) for two months. The effect of treatment with three dose levels of SC411 on selected clinical and biochemical parameters was investigated. Analysis was conducted under the Intention-To-Treat (ITT) population.
Results:
Sixty-seven (n=67) subjects were randomized, 50 to active treatment. Fifty-one (76%) were on HU treatment. Sixty-two patients (93%) completed the double-blinded phase of the study.
The analysis of the primary endpoint showed a mean percent increase from baseline in DHA and EPA concentration of 108.0 [CI: (49.5, 166.6)], 194.1 [CI: 145.8, 242.5)], 179.2 [CI (126.7, 231.6) and 15.4 [-42.8, 73.6)] in 20, 36 and 60 DHA mg/kg day and placebo, respectively, after 4 weeks of treatment.
After 8 weeks post-treatment, significant changes against placebo were observed in D-dimer (p=0.025) and sE-sel (p=0.0219) on patients exposed to 36 mg/kg. Significant increase in hemoglobin (Hb) against placebo was observed in those who received 20 mg/kg (p=0.039). Positive non-significant directional changes against placebo were observed in plasma hs-CRP, LDH, sVCAM and WBC count.
The adjusted eDiary-recorded incidence rate ratios (IRRs) of pooled active versus placebo were 0.65 (95% CI: 0.46, 0.93; p=0.02) for sickle cell crises, 0.75 (95% CI: 0.62, 0.92; p=0.0054) for analgesic use at home and 0.41 (95% CI: 0.31, 0.56; p=0.0001) for opioid use at home. The odds ratio of days absent from school because of sickle cell pain of active groups pooled versus placebo was 0.28 (95% CI: 0.19, 0.39; p=0.0002).
The rate of clinical SCC was 53% lower in the active groups pooled against placebo (rate ratio, 0.47; 95% confidence interval [CI]: 0.20 to 1.11; p=0.07).
Conclusions:
SC411 significantly reduced eDiary recorded sickle cell crises, analgesic and opioid use at home for sickle cell pain, and days absent from school due to sickle cell pain after two months of treatment. In addition, a non-statistically significant reduction of pain episodes and improvements in markers of inflammation, adhesion and coagulation were observed. These findings support further evaluation of the safety and efficacy of SC411 for SCD treatment in a phase 3 clinical trial


Saturday October 13, 2018 9:00am - 9:15am
Constellation B

9:00am

The Beats Program: Sickle Cell Health Education Through Music Therapy
Abstract

Authors:
Mr. Samuel Rodgers-Melnick- University Hospitals Connor Integrative Health Network

Objective: At University Hospitals Seidman Cancer Center, a music therapist working with the Adult Sickle Cell Disease (SCD) team developed the BEATS (Build, Educate, Advance, Transition, in Sickle cell disease) Music Therapy Program as part of a multidisciplinary SCD clinic to address self-efficacy, SCD knowledge, and adherence in young adults with SCD. Music therapy is the clinical and evidence-based use of music interventions to accomplish individualized goals within a therapeutic relationship by a credentialed professional who has completed an approved music therapy program. Music therapy is an established health profession in which music is used within a therapeutic relationship to address physical, emotional, cognitive, and social needs of individuals (American Music Therapy Association [AMTA], 2018, para 1 and 2). Preliminary research in young adults with SCD age 18-23 revealed that participants in the BEATS program demonstrated significant improvements in SCD knowledge as well as slight improvements in adherence to Adult SCD Clinic appointments as compared to baseline (Rodgers-Melnick et al., 2017). Participants’ feedback from this preliminary study also indicated areas for improvement. These areas included more flexible scheduling options, providing multiple small groups per month instead of one large group, administering study measures electronically, and coordinating sessions with patients’ existing clinic appointments. This study sought to implement these improvements and further investigate the effects of the BEATS program on the self-efficacy, trust, disease knowledge, and adherence in young adults with SCD.
Methods: 50 young adult patients age 18-35 with SCD, including homozygous HbSS and compound heterozygous HbSC, HbSb+thal, or HbSb0thal, were recruited from the Adult SCD Clinic at UH Seidman Cancer Center/UH Cleveland Medical Center. Participants were scheduled to come to the hospital once every 2-3 weeks to participate in BEATS sessions, sometimes in conjunction with prescheduled SCD clinic appointments. Multiple small group sessions of up to 5 participants were scheduled every week to allow participants the flexibility to choose the sessions that best fit their availability. Participants were asked to participate in eight BEATS visits over an 8-month period. BEATS sessions included instrumental improvisation and educational music therapy interventions targeting the following topics: medication management skills, SCD pathophysiology, understanding severe symptoms of SCD, pain management, communication in the emergency room, blood transfusions, and reproductive health. The educational interventions included video presentations from hematologists, nurses, psychiatrists, physical therapists, transfusion coordinators, and other staff who work with adults with SCD. Additionally, the music therapist led practical hands-on demonstrations of health concepts and fill-in-the-blank songwriting exercises to reinforce the health information presented in the session.
Self-efficacy (Sickle Cell Self-Efficacy Scale), trust (Wake Forest Trust in the Medical Profession Scale), and SCD knowledge (Seidman Sickle Cell Knowledge Quiz) were measured via an electronic REDCap survey at five time points: 1) prior to BEATS sessions at baseline, 2) immediately following the fourth session, 3) 4 weeks after the fourth session, 4) immediately following the last session, and 5) 4 weeks after the last session. Health care utilization and adherence to scheduled Adult SCD Clinic appointments and certain medications were measured regularly throughout the study via medical record review. Participants also provided written evaluations of the BEATS program after the fourth and last sessions.
Results: Preliminary results suggest that the BEATS program improved SCD knowledge in the cohort of young adults that participated in this study. The modifications implemented as a result of participants’ feedback from an earlier study of the BEATS program led to improved attendance, coordination with clinic appointments, group cohesion, and study measure completion as compared to the previous study. Participants’ evaluations revealed positive responses to the BEATS program focusing on the importance of learning from others, finding social support, learning more about SCD, and learning new ways to manage pain. Responses also indicated further ways to improve the BEATS program that include having guest speakers, holding more groups, inviting more patients, and providing food and assistance with transportation/parking.
Final data on changes from baseline in self-efficacy, trust, SCD knowledge, and adherence are currently pending completion of follow up data in September 2018. These results will be shared in publications and presentations at upcoming conferences.
Conclusions: For individuals with SCD, young adulthood presents multiple physical, psychological, social, and economic challenges associated with living with the acute and chronic symptoms of SCD and navigating the adult health care environment. In response to these real and perceived needs of young adults with SCD, we designed a multidisciplinary program to engage patients in music therapy as a means of improving self-efficacy, trust, and knowledge as well as assisting patients in navigating the health care system. Preliminary data from this study continue to support the utility of the BEATS program as a means of improving SCD knowledge and the experience of care. Modifications implemented in response to participants’ feedback on previous programming were effective in improving session attendance and program feasibility. New educational interventions developed for this study were well received by participants. Ultimately, the BEATS program is a valuable tool for providers and community-based organizations seeking engaging and culturally-relevant interventions to improve the quality of care for young adults with SCD.

Speakers
avatar for Samuel Rodgers-Melnick

Samuel Rodgers-Melnick

Music Therapist, University Hospitals Connor Integrative Health Network


Saturday October 13, 2018 9:00am - 9:15am
Constellation CD

9:15am

Clinical Development Of Rivipansel (GMI-1070) For The Treatment Of Acute Vaso-Occlusive Crisis In Sickle Cell Disease
Abstract

Authors:
Dr. Lori Luck- Pfizer Inc., Collegeville, Pennsylvania, Dr. Krupa Sivamurthy- Pfizer Inc., Collegeville, Pennsylvania, Dr. Frank Shafer-Pfizer Inc., Collegeville, Pennsylvania, Dr. Carl Kollmer- Pfizer Inc., Collegeville, Pennsylvania, Ms. Amanda O’brien- Pfizer Inc., Collegeville, Pennsylvania, Mr. David Readett- Pfizer Inc., Collegeville, Pennsylvania

Objectives: Acute therapy for a sickle cell vaso-occlusive crisis (SCD-VOC) remains limited to symptomatic relief predominantly with opioids, which fails to address the underlying pathophysiology of VOC. Preclinical studies have demonstrated a reduction in white blood cell (WBC)-endothelial cell adhesion, in addition to a decrease in secondary capture of sickle red blood cells (sRBCs) through selectin inhibition (Wun T, et al. PLoS One. 2014;9[7]:e101301). This suggests that inhibition of selectin-mediated cell adhesion to vascular endothelium may provide a therapeutic approach that would target the underlying cause of VOC. We describe the clinical development of rivipansel, a pan-selectin inhibitor, for the acute treatment of SCD-VOC.
Methods: We report on the clinical development of rivipansel from Phase 1 to Phase 3 investigation. Phase 1 studies were performed in healthy volunteers and individuals with SCD not experiencing a VOC. The Phase 2 multicenter, randomized, double-blind, placebo-controlled trial that followed enrolled and treated 76 individuals with SCD who were 12 – 60 years of age and hospitalized for a VOC. Subjects received a loading dose and a maximum of 14 maintenance doses of rivipansel or placebo. The primary efficacy endpoint was resolution of VOC and secondary endpoints included cumulative opioid use, length of hospital stay and adverse events (Telen MJ, et al. Blood. 2015;125[17]:2656-64). The subsequent ongoing randomized, double-blind Phase 3 clinical trial will include approximately 350 SCD subjects, 6 years of age and older, admitted to the hospital for VOC. The study design largely mirrors the Phase 2 study but the primary endpoint is time to readiness-for-discharge, defined as the difference between the time of readiness-for-discharge and the start time of the first infusion of study drug. Secondary endpoints include time to discharge, cumulative IV opioid consumption, time to discontinuation of IV opioids and adverse events. Subjects in the Phase 3 study also have the option of entering an open label extension study following their participation in the double blind study (ClinicalTrials.gov identifier: NCT02187003).
Results: The Phase 1 investigations demonstrated safety and tolerability in addition to identifying a Phase 2 starting dose for intravenous rivipansel. The subsequent Phase 2 trial revealed shorter median time to resolution of VOC and time to hospital discharge, as well as lower mean cumulative IV opioid (mg/kg morphine equivalent units) use in the rivipansel group compared to the placebo group. (Table 1) All causality and treatment-related adverse event (AE) rates, including serious AEs in the rivipansel group were comparable to the placebo group (Telen MJ, et al. Blood. 2015;125[17]:2656-64). These encouraging results prompted further investigation of rivipansel in a randomized, double-blind Phase 3 study (ClinicalTrials.gov identifier: NCT02187003) which is ongoing.
Conclusions: The clinical development of rivipansel as an acute therapy for SCD-VOC is based on selectin inhibition that is expected to decrease the adhesion between WBCs, endothelial cells and sRBCs, a key event in the evolution of sickle cell vaso-occlusion. Initial clinical studies have shown encouraging results leading to the ongoing randomized, double-blind Phase 3 study.

Speakers
avatar for Lori Luck, MD

Lori Luck, MD

Medical Director, PFIZER Inc, Collegeville Pennsylvania Global Medical Affairs, Sickle Cell Disease
Lori Luck, M.D. is a pediatric hematologist and joined Pfizer in January, 2017 as the Medical Director, Global Medical Affairs, Sickle Cell Disease. Prior to Pfizer, Dr. Luck worked in Oncology at Genzyme and Leadiant (formally Sigma Tau) in Field Medical Affairs. She completed her... Read More →


Saturday October 13, 2018 9:15am - 9:30am
Constellation B

9:30am

A Systematic Literature Review Of Patient-Reported Outcome Instruments Used In Sickle Cell Disease
Abstract 

Authors:
Dr. Grammati Sarri- Evidera, London, United Kingdom, Dr. Menaka Bhor- Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA, Mr. Seye Abogunrin- Evidera, London, United Kingdom, Dr. Caroline Farmer- Evidera, London, United Kingdom, Dr. Savita Nandal- Novartis Pharmaceuticals Corporation, East Hanover, New Jersey, USA, Dr. Dennis A. Revicki- Evidera, Bethesda, Maryland, USA

Objective: Patients with sickle cell disease (SCD) have vaso-occlusive crises (VOCs) that result in repeated hospital admissions and can negatively impact patients’ health-related quality of life (HRQL). Most SCD randomized clinical trials (RCTs) are designed to evaluate efficacy relative to changes in the number of VOCs and their impact on HRQL. The objectives of this study were to conduct a systematic literature review (SLR) to identify patient-reported outcome (PRO) and health care resource utilization (HCRU) instruments used in RCTs and real-world studies and to evaluate their psychometric properties.
Methods: A SLR was conducted to identify and summarize validated PRO instruments used by patients with SCD in the United States and their caregivers. MEDLINE and EMBASE were searched using terms synonymous with SCD and psychometric properties of PRO instruments for articles published in English between 1997 and March 2017, focusing on pediatric patients, their caregivers, and adults. Studies were included if results assessed psychometric properties of PRO instruments and if the studies were interventional or observational in design. The quality of PRO instruments in the included studies was assessed using the COSMIN checklist.
Results: 21 studies assessing 10 child and 15 adult PRO instruments in SCD were included in the review. No validated PRO instruments measured caregiver burden in SCD and no validated HCRU instruments for any SCD population were identified. The most commonly reported instrument was the Pediatric Quality of Life Inventory; most of the adult instruments (9/15) evaluated self-efficacy/self-esteem and pain in relation to SCD. Only 1 instrument, the Adult Sickle Cell Quality of Life Measurement, evaluated HRQL in adults. Of the 25 PRO instruments reviewed for both children and adults, 3/10 and 5/15 instruments, respectively, were specifically developed for these age groups with SCD, and minimally important differences were not assessed for any of the reviewed instruments. Most instruments (9/15) identified for adult populations had strong internal reliability; however, there was often insufficient information to assess the content or construct validity, responsiveness, and test-retest reliability of the instruments identified for both populations.
Conclusions: Limited evidence on the psychometric properties of PRO instruments used in patients with SCD was identified. None of the reviewed instruments were found to sufficiently capture all the PRO aspects of SCD and their complications on patients’ HRQL for use as key trial endpoints. Further research should validate, or develop if no instrument is in existence, PRO instruments to assess HRQL of patients with SCD, PRO instruments for caregivers, and HCRU instruments.

Speakers
avatar for Menaka Bhor, PhD

Menaka Bhor, PhD

Associate Director, Novartis
Currently serving as an Associate Director, HEOR at Novartis Oncology and responsible forSickle Cell Disease portfolio. Previously as a Director at Cardinal Health generated compellingproposals for new sales opportunities and served as scientific lead on retrospective and prospectivestudies... Read More →


Saturday October 13, 2018 9:30am - 9:45am
Constellation B

9:30am

Break
Saturday October 13, 2018 9:30am - 10:00am
Constellation EF

10:00am

Gene Therapy for Sickle Cell Disease:  Fact or Fiction?
Moderators
avatar for Lewis L. Hsu, MD, Ph.D

Lewis L. Hsu, MD, Ph.D

Professor Pediatric Hem-Onc, University of Illinois Hospital
Lewis Hsu is a pediatric hematologist dedicated to finding more cures for sickle cell disease, and improving treatment and education until cures can be found. He has experience with clinical and translational research in sickle cell disease, including clinical research on pain assessment... Read More →

Speakers
avatar for Julie Kanter, MD

Julie Kanter, MD

Lifespan Hematologist, Medical University of South Carolina
Dr. Julie Kanter is a lifespan hematologist specializing in sickle cell disease.  She is an associate professor of Pediatrics at the Medical University of South Carolina and runs the first all-ages, Lifespan Comprehensive Sickle Cell Center.  Dr. Kanter works closely with national... Read More →


Saturday October 13, 2018 10:00am - 10:30am
Constellation CD

10:00am

PLENARY SESSION IV
Speakers
avatar for Lewis L. Hsu, MD, Ph.D

Lewis L. Hsu, MD, Ph.D

Professor Pediatric Hem-Onc, University of Illinois Hospital
Lewis Hsu is a pediatric hematologist dedicated to finding more cures for sickle cell disease, and improving treatment and education until cures can be found. He has experience with clinical and translational research in sickle cell disease, including clinical research on pain assessment... Read More →


Saturday October 13, 2018 10:00am - 2:00pm
Constellation A

10:30am

Clarice D. Reid, MD Lecture – Global Strategies for Sickle Cell Disease
Moderators
avatar for Lewis L. Hsu, MD, Ph.D

Lewis L. Hsu, MD, Ph.D

Professor Pediatric Hem-Onc, University of Illinois Hospital
Lewis Hsu is a pediatric hematologist dedicated to finding more cures for sickle cell disease, and improving treatment and education until cures can be found. He has experience with clinical and translational research in sickle cell disease, including clinical research on pain assessment... Read More →

Speakers
avatar for Russell Ware, MD

Russell Ware, MD

Director, Professor, Cincinati Children's Hospital Medical Center Cancer and Blood Disease Institute/ Division
Dr. Russell Ware obtained his MD and PhD degrees at Duke University, completed his Pediatric Hematology/Oncology fellowship at Duke, and served as Director of the Duke Pediatric Sickle Cell Program until 2004.  He then moved to St. Jude Children’s Research Hospital, serving as... Read More →


Saturday October 13, 2018 10:30am - 11:00am
Constellation CD

11:00am

National Partner Updates
American Society of Hematology - Robert Brodsky, MD
National Heart, Lung, and Blood Institute - W. Keith Hoots, MD
Health Resources and Services Administration - Donnell Ivy, MD, MPH
Center for Disease Control - Craig Hooper, PhD
Food and Drug Administration - Patricia Oneal, MD

Saturday October 13, 2018 11:00am - 12:30pm
Constellation CD

12:30pm

Lunch On Your Own
Saturday October 13, 2018 12:30pm - 1:30pm

12:30pm

Patient Empowerment Luncheon
Saturday October 13, 2018 12:30pm - 1:30pm
Chesapeake AB

12:30pm

NIH SCDIC Session – Overview of NIH SCDIC
Saturday October 13, 2018 12:30pm - 2:00pm
Constellation CD

12:30pm

12:30pm

Youth and Young adult Forum
Saturday October 13, 2018 12:30pm - 4:30pm
Pratt/Calvert

1:00pm

Emmaus Focus Group
Speakers
CS

Charles Stark

Sr. VP of Research and Development, emmaus


Saturday October 13, 2018 1:00pm - 2:00pm
Baltimore/Annapolis

1:00pm

Hydroxyurea and Pregnancy
Speakers
avatar for Russell Ware, MD

Russell Ware, MD

Director, Professor, Cincinati Children's Hospital Medical Center Cancer and Blood Disease Institute/ Division
Dr. Russell Ware obtained his MD and PhD degrees at Duke University, completed his Pediatric Hematology/Oncology fellowship at Duke, and served as Director of the Duke Pediatric Sickle Cell Program until 2004.  He then moved to St. Jude Children’s Research Hospital, serving as... Read More →


Saturday October 13, 2018 1:00pm - 2:00pm
Frederick/Columbia

1:00pm

Exhibitor Breakdown
Saturday October 13, 2018 1:00pm - 3:00pm
Constellation EF

2:00pm

Closing Assembly & Adjournment
Saturday October 13, 2018 2:00pm - 2:30pm
Constellation CD