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

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

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.

Who May Be Eligible (Plain English)

Who May Qualify: - 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 willing to sign a consent form 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. Who Should NOT Join This Trial: - 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. Always talk to your doctor about whether this trial is right for you.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria: * 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 Criteria: * 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.

Locations (20)

Children's of Alabama (MRD-HCT)
Birmingham, Alabama, United States
Children's of Alabama (NT-DMT)
Birmingham, Alabama, United States
Nemours Children's Hospital, Delaware (MRD-HCT)
Wilmington, Delaware, United States
Children's National Hospital (MRD-HCT)
Washington D.C., District of Columbia, United States
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia, United States
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois, United States
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana, United States
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana, United States
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts, United States
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts, United States
Wahington Univ in St Louis (NT-DMT)
St Louis, Missouri, United States
Washington University in St Louis (MRD-HCT)
St Louis, Missouri, United States
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey, United States
Roswell Park (MRD-HCT)
Buffalo, New York, United States
Columbia Presbytarian (NT-DMT)
New York, New York, United States
Cohen's Children Hospital (NT-DMT)
Queens, New York, United States
University of Rochester (MRD-HCT)
Rochester, New York, United States
University of Rochester (NT-DMT)
Rochester, New York, United States
Children's Hospital at Montefiore (MRD HCT)
The Bronx, New York, United States
Children's Hospital at Montefiore (NT-DMT)
The Bronx, New York, United States