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Saturday, October 13 • 9:30am - 9:45am
A Systematic Literature Review Of Patient-Reported Outcome Instruments Used In Sickle Cell Disease

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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.

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 EDT
Constellation B