Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease
Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
University of Rochester
480 participants
Jun 1, 2024
OBSERVATIONAL
Conditions
Summary
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.
Eligibility
Plain Language Summary
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This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.
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NCT06941389