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

Studying stem cell transplant vs. therapy for kids with sickle cell.

Recruiting
3-20 years
All
Phase N/A
  • Simplified description (AI rewritten)
  • Technical description (Original)

The WeDecide study explores two treatments for children with sickle cell disease (SCD): matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT). SCD is a blood disorder where red blood cells are misshaped, leading to health problems. MRD HCT involves replacing the patient's bone marrow with healthy cells from a matching donor. NT-DMT are treatments that help manage SCD symptoms without a transplant. This study aims to understand how these treatments affect the quality of life, cognitive function, survival, and health risks. It involves 480 children aged 3 to 20.9, followed for three years. Researchers will look at each child's disease severity, treatment history, and family situation to understand treatment outcomes better. The study's findings will guide doctors and families in making informed treatment choices for SCD.

  • Duration: The study lasts for three years.
  • Participants: 480 children aged 3 to 20.9 years.
  • Assessments: Regular check-ups to track health and treatment effects.
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Study details
    Sickle Cell Disease (SCD)

NCT06941389

University of Rochester

17 April 2026

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