Abstract

Authors:
Ms. claudia cobianchi- Terumo BCT, Ms. Dessie Fafoutis- Terumo BCT, Mr. Joseph Roig-Terumo BCT, Mr. Koenraad Dierick- Terumo BCT

Background:
Red blood cell (RBC) transfusion is one of the mainstays of long-term Sickle Cell Disease (SCD) therapy. It is supported by multiple RCTs for stroke prevention. The STOP trial randomized children with elevated blood flow velocity as determined by transcranial Doppler (TCD) to standard care without transfusion (control) versus chronic monthly transfusion for primary stroke Red prevention . The trial was terminated prematurely due to the marked (90%) stroke risk reduction by chronic transfusion. Additionally, there is also evidence on its value for other indications such as recurrent acute chest syndrome or recurrent painful crises. Red Cell exchange is preferred to simple transfusion (also known as “top-up” transfusion) as it has many advantages, the main advantage being the avoidance or minimization of costly iron chelation therapy to treat iron overload. Red cell exchange is thought to be an iron neutral procedure that can rapidly lower HgbS percentage without increasing blood viscosity and creating volume overload. Transfusion withdrawal is associated with an increased risk of recurrent stroke or premature death . Currently there are only 3 FDA cleared therapies to treat Sickle Cell Disease: Hydroxyurea (DOXIAâ, HYDREAâ, Bristol Myers Squibb), Automated Red Cell Exchange (Spectra Optiaâ Apheresis System, Terumo BCT) and L-glutamine oral powder (Endari™, Emmaus Medical Inc.). When this study was performed, no published research had been completed that compared patient outcomes in terms of Health-Related Quality of Life (HRQoL) for individuals treated with automated red blood cell exchange versus simple transfusion.
Objectives:
The objectives covered within the scope of this study:
• To quantify HRQoL as experienced by individuals with SCD treated with aRBCX versus simple transfusion.
• To determine the drivers of HRQoL in this group
Methodology:
A cross-sectional study was performed amongst 40 individuals with SCD, 20 from the USA, 10 from France and 10 from the UK each with prior simple transfusion treatment and current treatment aRBCX.
Results
Individuals undergoing aRBCX reported an HRQoL that was 25% higher compared to the period where they were treated with simple transfusion (0.70 vs. 0.55; p<0.01). The main drivers of HRQoL identified were (correlation efficient): pain reduction (0.57), improved social live (0.49), autonomy in terms of all day living activities and being independent from others (0.56), feeling energetic and physical functioning (0.57) and lastly emotional worry and mental health (0.56), all with p-values < 0.01. Together these variables explain 39% of the HRQoL experienced by study participants (R² = 0.39, p < 0.01). 80% of those in the study preferred aRBCX over simple transfusion.
Conclusion:
In this study individuals with sickle cell disease that require chronic blood transfusions experience better health-related quality of life when they are treated with automated red blood cell exchange versus simple transfusions.