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

Conditions

• Sickle Cell Disease (SCD)

Eligibility Criteria

Sex

ALL

Age

3 Years - 20 Years

Healthy Volunteers

Not accepted

Study Details

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.

Key Dates

Start date

Jun 1, 2024

Status verified

Jun 2026

Primary completion

Oct 1, 2030

Completion

Oct 1, 2030

Study Design

Enrollment

480 participants (estimated)

Arms

• Arm: NT-DMT Cohort
  This group includes 320 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.
• Arm: MRD HCT Cohort
  This group includes 160 participants from U.S. and Canadian sites receiving non-transplant disease-modifying therapies, monitored annually over three years to assess health-related quality of life, cognitive function, disease progression, and healthcare utilization.

Primary Outcome Measure

Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Parent-Proxy Report) [ Time Frame: Baseline, 1-year, 2-year, 3-year follow-up ]

Central Contacts

• John Horan, MD, MPH
  5852742345
  [email protected]
• Savannah Pangrazio, BS
  5852750932
  [email protected]

Locations (31)

Find similar trials in Birmingham, AL

Related Studies

• Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
  PHASE4 · Recruiting · University of Pennsylvania · Atlanta, Georgia
• Sickle Cell Kidney Biorepository
  Recruiting · University of Texas Southwestern Medical Center · Dallas, Texas
• Development of a Provider-Focused Intervention to Improve Health Outcomes in Pediatric Sickle Cell Disease
  Not Yet Recruiting · Connecticut Children's Medical Center · Hartford, Connecticut