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

Description

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.

Conditions

Sickle Cell Disease (SCD)

Talk to us

Study Overview

On this page

Study Details

Study overview

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.

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

Condition
Sickle Cell Disease (SCD)
Intervention / Treatment

-

Contacts and Locations

Birmingham

University of Alabama (MRD-HCT), Birmingham, Alabama, United States, 35294

Birmingham

University of Alabama (NT-DMT), Birmingham, Alabama, United States, 35294

Wilmington

Nemours Children's Hospital (MRD-HCT), Wilmington, Delaware, United States, 19803

Washington

Children's National Hospital (MRD-HCT), Washington, District of Columbia, United States, 20010

Atlanta

Children's Healthcare of Atlanta (MRD-HCT), Atlanta, Georgia, United States, 30329

Chicago

Comer Children's Hospital (MRD-HCT), Chicago, Illinois, United States, 60637

Indianapolis

Riley Children's Hospital (MRD-HCT), Indianapolis, Indiana, United States, 46202

Indianapolis

Riley's Children Hospital (NT-DMT), Indianapolis, Indiana, United States, 46202

Boston

Boston Children's Hospital (MRD-HCT), Boston, Massachusetts, United States, 02115

Boston

Boston Children's Hospital (NT-DMT), Boston, Massachusetts, United States, 02115

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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.
  • * 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.

Ages Eligible for Study

3 Years to 20 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

University of Rochester,

Study Record Dates

2030-12-01