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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
Sponsor: University of Rochester
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.
Official title: Comparing the Effectiveness of Matched Related Donor Hematopoietic Stem Cell Transplantation to Disease Modifying Therapy in Pediatric Patients With Sickle Cell Disease.
Key Details
Gender
All
Age Range
3 Years - 20 Years
Study Type
OBSERVATIONAL
Enrollment
480
Start Date
2024-06-01
Completion Date
2030-10-01
Last Updated
2025-06-19
Healthy Volunteers
No
Conditions
Locations (37)
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
UNC Children's Hospital (MRD HCT)
Chapel Hill, North Carolina, United States
UNC Children's Hospital (NT-DMT)
Chapel Hill, North Carolina, United States
Atrium Health (MRD-HCT)
Charlotte, North Carolina, United States
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio, United States
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma, United States
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania, United States
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee, United States
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee, United States
Texas Children's Hopsital (NT-DMT)
Houston, Texas, United States
Texas Children's Hospital (MRD-HCT)
Houston, Texas, United States
UT San Antonio (NT-DMT)
San Antonio, Texas, United States
Alberta Children's Hospital (NT-DMT)
Calgary, Alberta, Canada
Alberta Children's Hospital (MRD-HCT)
Calgary, Alberta, Canada
British Columbia Children's Hosptial (MRD HCT)
Vancouver, British Columbia, Canada
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
Winnipeg, Manitoba, Canada
The Hospital for Sick Children (MRD-HCT)
Toronto, Ontario, Canada
Universite de Montreal / Ste Justine (MRD HCT)
Montreal, Quebec, Canada