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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

Comparing two treatments for kids with sickle cell disease.

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

The WeDecide study is looking at two treatments for kids with sickle cell disease (SCD): matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT). MRD HCT is a procedure where healthy blood-making cells from a family member are given to the patient. NT-DMT includes medicines that help manage the disease without a transplant. The study focuses on the quality of life, brain function, and health risks over three years. It involves 480 children aged 3 to 20.9 years. By comparing these treatments, the study aims to help families and doctors choose the best care. They will track health, hospital visits, and any complications.

  • The study lasts for three years with multiple health check-ups.
  • Participants are divided into two groups: 160 for MRD HCT and 320 for NT-DMT.
  • Information from this study will help guide future treatment decisions for SCD.
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Study details
    Sickle Cell Disease (SCD)

NCT06941389

University of Rochester

27 April 2025

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