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)
- Registration Number
- NCT06941389
- Lead Sponsor
- University of Rochester
- Brief 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.
- Detailed Description
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.
Recruitment & Eligibility
- Status
- RECRUITING
- Sex
- All
- Target Recruitment
- 480
- 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.
- Children younger than 3 years or older than 20.9 years.
- Children with significant comorbidities or other health conditions that would interfere with the study's outcomes.
- 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.
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Health-Related Quality of Life as measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale at Baseline and Follow-Up Time Points Baseline, 1-year, 2-year, 3-year follow-up The Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale measures the physical, emotional, social, and school functioning in children and adolescents. Possible scores range from 0 to 100, with higher scores indicating better health-related quality of life (HRQoL).
Health-Related Quality of Life as Measured by PROMIS Pediatric Profile 25 (Self-Report and Parent-Proxy Report) at Baseline and Follow-Up Time Points Baseline, 1-year, 2-year, 3-year follow-up The PROMIS Pediatric Profile 25 measures the health-related quality of life across domains including physical functioning, emotional functioning, social functioning, and pain behavior. Scores range from 0 to 100, with higher scores indicating better health-related quality of life.
Cognitive Function as Measured by the NIH Toolbox Cognitive Battery at Baseline and Follow-Up Time Points Baseline, 1-year, 2-year, 3-year follow-up The NIH Toolbox Cognitive Battery assesses domains of cognitive function including executive function, attention, episodic memory, language, processing speed, and working memory. Scores vary by subtest, and each domain is standardized with a mean of 50 and standard deviation of 10. Higher scores indicate better cognitive function.
- Secondary Outcome Measures
Name Time Method Perceived Discrimination as Measured by the Everyday Discrimination Scale at Baseline and Follow-Up Time Points Baseline, 1-year, 2-year, 3-year follow-up The Everyday Discrimination Scale is a 9-item questionnaire assessing perceived discrimination experienced in everyday life. Scores range from 0 to 36, with higher scores indicating more frequent or intense experiences of discrimination.
Financial Wellness as Measured by the Personal Financial Wellness Survey at Baseline and Follow-Up Time Points Baseline, 1-year, 2-year, 3-year follow-up The Personal Financial Wellness Survey is an 8-item measure assessing financial distress and economic stress. Scores range from 0 to 32, with higher scores indicating greater financial distress
Trial Locations
- Locations (33)
University of Alabama (MRD-HCT)
🇺🇸Birmingham, Alabama, United States
University of Alabama (NT-DMT)
🇺🇸Birmingham, Alabama, United States
Nemours Children's Hospital (MRD-HCT)
🇺🇸Wilmington, Delaware, United States
Children's National Hospital (MRD-HCT)
🇺🇸Washington, 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)
🇺🇸Saint Louis, Missouri, United States
Hackensack University Hospital (MRD-HCT)
🇺🇸Hackensack, New Jersey, United States
Children's Hospital at Montefiore (MRD HCT)
🇺🇸Bronx, New York, United States
Children's Hospital at Montefiore (NT-DMT)
🇺🇸Bronx, New York, 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
UNC Chapel Hill (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
Children's Hospital of Winnipeg (MRD-HCT)
🇨🇦Winnipeg, Manitoba, Canada
The Hospital for Sick Children (MRD-HCT)
🇨🇦Toronto, Ontario, Canada