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
Inclusion Criteria6
- 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.
- Participants (or their guardians) must provide informed consent to be part of the study.
- Participants must be willing to undergo the necessary assessments and follow-up visits over the 3-year study period.
Exclusion Criteria5
- 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.
- Children who are unable to adhere to the study protocol or follow-up requirements.
Interested in this trial?
Get notified about updates and connect with the research team.
Locations(37)
View Full Details on ClinicalTrials.gov
For the most up-to-date information, visit the official listing.
NCT06941389