The WeDecide study is a large observational study comparing the long-term effects of matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT) for pediatric patients with sickle cell disease (SCD). The study aims to assess health-related quality of life (HRQoL), cognitive function, risks, and benefits of both treatments, including survival rates, chronic complications, and organ damage prevention. With 160 children in the MRD HCT group and 320 in the NT-DMT group, aged 3-20.9 years, the study will follow participants for three years, examining factors like disease severity, treatment history, and social determinants of health. By providing a comprehensive comparison, the study seeks to inform clinical decisions and improve understanding of SCD treatment outcomes, ultimately supporting families and healthcare providers in choosing the best treatment options.
Pediatric patients aged between 3 and 20.9 years.
Children diagnosed with sickle cell Anemia (HB SS or HBSB0 Thalassemia)
For the MRD HCT group, children who are candidates for matched related donor hematopoietic stem cell transplantation (MRD HCT).
For the NT-DMT group, children who are receiving non-transplant disease-modifying therapies (NT-DMT) for SCD.
Children younger than 3 years or older than 20.9 years.
Children who do not have sickle cell anemia or related conditions.
For the MRD HCT group, children who are not eligible for the transplant or do not have a matched related donor.
Children who are currently enrolled in other clinical trials that might interfere with the WeDecide study.
University of Rochester
Lead sponsor
Patient-Centered Outcomes Research Institute
Collaborator