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.
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.
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.
The WeDecide study includes 480 pediatric patients with sickle cell disease (SCD) from two sub-studies: Project Sickle Cure (PSC), focusing on patients receiving transplant through the Sickle Cell Transplant Advocacy Research Alliance (STAR), and the Globin Research Network for Data and Discovery (GRNDaD), a registry of patients on non-transplant disease-modifying therapies (NT-DMT) run by the National Association of Sickle Cell Centers (NASCC). MRD HCT Group: 160 children receiving matched related donor hematopoietic stem cell transplantation (MRD HCT) will be assessed before and at multiple post-transplant time points to evaluate long-term effects. NT-DMT Group: 320 children receiving non-transplant disease-modifying therapies (NT-DMT) will be assessed at study start and annually for three years to track treatment effects.
Cohort
Prospective
Not listed
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Parent-Proxy Report)
Measures physical, emotional, social, and school functioning as reported by parents.
Cognitive Function as Measured by the NIH Toolbox Cognitive Battery
Assesses the following cognitive domains: attention, executive function, memory, language, processing speed, and working memory.
Health-Related Quality of Life as Measured by the Pediatric Quality of Life Inventory (PedsQL) Generic Core Scale (Patient Reported)
Description: Assesses self-reported physical, emotional, social, and school functioning (for age-eligible participants).
Health Related Quality of Life Using the PedsQL Sickle Cell Disease Module (Patient and Parent-Proxy Reported)
Description: The Peds QL Sickle Cell Module assesses pain and hurt, pain impact, pain management and control, worry, emotions, treatment and communication.
Peds QL Family Impact Module (Parent Reported)
Assess the burden a parent experiences. It assesses: Physical, Emotional, Social and Cognitive functioning; communication, worry, family daily activities, and family relationships.
Health Quality of Life as Measured by the Patient Reported Outcomes Measurement Information System (PROMIS, Patient and Parent-Proxy Reports)
Assesses physical, mental and social health
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University of Rochester
Lead sponsor
Patient-Centered Outcomes Research Institute
Collaborator
