Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease

The WeDecide study is a large observational study comparing the long-term effects of two treatment options for pediatric patients with sickle cell disease (SCD): matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT). The main goal is to understand how these treatments affect health-related quality of life (HRQoL) and cognitive function, using standard tools to measure both physical and mental health. The study also looks at risks and benefits of MRD HCT, such as the potential for chronic complications, improved survival, and prevention of organ damage.

The study includes two groups: 160 children receiving MRD HCT and 320 children receiving NT-DMT. Participants, aged 3-20.9 years, are being followed for three years. The MRD HCT group will be assessed before the transplant and then at several points post-transplant. The NT-DMT group will be assessed at the start of the study and then annually for three years.

The research also considers factors like disease severity, treatment history, and social determinants of health (such as family finances and caregiver health literacy) to better understand how these elements might influence treatment outcomes. The study tracks the use of disease-modifying therapies, as well as hospital visits and other care events, throughout the three years. It will also monitor survival rates and other important health outcomes.

This study is significant because it is the first large-scale research comparing these two treatment options for SCD in children. The results will provide essential insights into how these treatments impact long-term health and help guide clinical decisions and treatment recommendations. The goal is to help families and healthcare providers make informed decisions about the best treatment options for SCD.

The study uses advanced methods to ensure fair comparisons between the two groups by accounting for differences in their characteristics. It will also adjust for any factors that could influence the results, helping to identify meaningful differences in health outcomes between the two treatments. Ultimately, the WeDecide study aims to improve our understanding of sickle cell disease treatment and provide a foundation for future research into new therapies.

• Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Patient Reported) [Time frame: Baseline, 1-year, 2-year, 3-year follow-up]
• Health Related Quality of Life Using the PedsQL Sickle Cell Disease Module (Patient and Parent-Proxy Reported) [Time frame: Baseline, 1-year, 2-year, 3-year follow-up]
• Peds QL Family Impact Module (Parent Reported) [Time frame: Baseline, 1-, 2- and 3-year follow-up]
• Health Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS, Patient and Parent-Proxy Reports) [Time frame: Baseline, 1-year, 2-year, 3-year follow-up]
• The Patient Reported Outcomes Measure System (PROMIS) Pain Behavior subscale (Patient and Parent-Proxy Reports) [Time frame: Baseline, 1-year, 2-year and 3-year follow-up]
• Number of Acute Care Visits (ED, Hospitalizations, Infusion/Clinic) [Time frame: Time Points: Year 1, Year 2, Year 3]
• Incidence and Severity of Chronic Graft-Versus-Host Disease [Time frame: Day 100, 6 months, Year 1, Year 2, Year 3]