Clinical Trial
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.
Study Overview
The WeDecide study is a large observational study comparing the long-term effects of two treatment options for pediatric patients with sickle cell disease (SCD): matched related donor hematopoietic stem cell transplantation (MRD HCT) and non-transplant disease-modifying therapies (NT-DMT). The main goal is to understand how these treatments affect health-related quality of life (HRQoL) and cognitive function, using standard tools to measure both physical and mental health. The study also looks at risks and benefits of MRD HCT, such as the potential for chronic complications, improved survival, and prevention of organ damage.
The study includes two groups: 160 children receiving MRD HCT and 320 children receiving NT-DMT. Participants, aged 3-20.9 years, are being followed for three years. The MRD HCT group will be assessed before the transplant and then at several points post-transplant. The NT-DMT group will be assessed at the start of the study and then annually for three years.
The research also considers factors like disease severity, treatment history, and social determinants of health (such as family finances and caregiver health literacy) to better understand how these elements might influence treatment outcomes. The study tracks the use of disease-modifying therapies, as well as hospital visits and other care events, throughout the three years. It will also monitor survival rates and other important health outcomes.
This study is significant because it is the first large-scale research comparing these two treatment options for SCD in children. The results will provide essential insights into how these treatments impact long-term health and help guide clinical decisions and treatment recommendations. The goal is to help families and healthcare providers make informed decisions about the best treatment options for SCD.
The study uses advanced methods to ensure fair comparisons between the two groups by accounting for differences in their characteristics. It will also adjust for any factors that could influence the results, helping to identify meaningful differences in health outcomes between the two treatments. Ultimately, the WeDecide study aims to improve our understanding of sickle cell disease treatment and provide a foundation for future research into new therapies.
Contacts and Locations
Children's of Alabama (MRD-HCT)
Birmingham, Alabama 35294
United States
Children's of Alabama (NT-DMT)
Birmingham, Alabama 35294
United States
Nemours Children's Hospital, Delaware (MRD-HCT)
Wilmington, Delaware 19803
United States
Children's National Hospital (MRD-HCT)
Washington D.C., District of Columbia 20010
United States
Children's Healthcare of Atlanta (MRD-HCT)
Atlanta, Georgia 30329
United States
Comer Children's Hospital (MRD-HCT)
Chicago, Illinois 60637
United States
Riley Children's Hospital (MRD-HCT)
Indianapolis, Indiana 46202
United States
Riley's Children Hospital (NT-DMT)
Indianapolis, Indiana 46202
United States
Boston Children's Hospital (MRD-HCT)
Boston, Massachusetts 02115
United States
Boston Children's Hospital (NT-DMT)
Boston, Massachusetts 02115
United States
Wahington Univ in St Louis (NT-DMT)
St Louis, Missouri 63130
United States
Washington University in St Louis (MRD-HCT)
St Louis, Missouri 63130
United States
Hackensack University Hospital (MRD-HCT)
Hackensack, New Jersey 07601
United States
Roswell Park (MRD-HCT)
Buffalo, New York 14203
United States
Columbia Presbytarian (NT-DMT)
New York, New York 10032
United States
Cohen's Children Hospital (NT-DMT)
Queens, New York 11040
United States
University of Rochester (MRD-HCT)
Rochester, New York 14642
United States
University of Rochester (NT-DMT)
Rochester, New York 14642
United States
Children's Hospital at Montefiore (MRD HCT)
The Bronx, New York 10467
United States
Children's Hospital at Montefiore (NT-DMT)
The Bronx, New York 10467
United States
UNC Children's Hospital (MRD HCT)
Chapel Hill, North Carolina 27514
United States
UNC Children's Hospital (NT-DMT)
Chapel Hill, North Carolina 27599.
United States
Atrium Health (MRD-HCT)
Charlotte, North Carolina 28203
United States
Nationwide Children's Hospital (NT-DMT)
Columbus, Ohio 43205
United States
Oklahoma Children's Hospital (MRD-HCT)
Oklahoma City, Oklahoma 73104
United States
Children's Hospital of Philadelphia (MRD-HCT)
Philadelphia, Pennsylvania 19104
United States
St Jude Children Hospital (NT-DMT)
Memphis, Tennessee 38105
United States
St. Jude Children's Research Hospital (MRD-HCT)
Memphis, Tennessee 38105
United States
Texas Children's Hopsital (NT-DMT)
Houston, Texas 77030
United States
Texas Children's Hospital (MRD-HCT)
Houston, Texas 77030
United States
UT San Antonio (NT-DMT)
San Antonio, Texas 78229
United States
Alberta Children's Hospital (NT-DMT)
Calgary Alberta T3B 6A8
Canada
Alberta Children's Hospital (MRD-HCT)
Calgary Alberta T3B6A8
Canada
British Columbia Children's Hosptial (MRD HCT)
Vancouver British Columbia
Canada
HSC Winnipeg Children's Hospital/University of Manitoba (MRD-HCT)
Winnipeg Manitoba R3E 0V9
Canada
The Hospital for Sick Children (MRD-HCT)
Toronto Ontario ON M5G 1E8
Canada
Universite de Montreal / Ste Justine (MRD HCT)
Montreal Quebec
Canada