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

Launched by UNIVERSITY OF ROCHESTER · Apr 15, 2025

Keywords

ClinConnect Summary

The WeDecide study is looking at how well two different treatments work for children with sickle cell disease (SCD), which is a condition that affects the blood and can cause pain and other serious health issues. Specifically, the study compares matched related donor hematopoietic stem cell transplantation (MRD HCT) — a type of treatment where healthy stem cells from a family member are used to help the body make new blood cells — with non-transplant disease-modifying therapies (NT-DMT), which are medicines that help manage the disease without a transplant. The goal is to see how these treatments affect the children's health, quality of life, and overall well-being over three years.

To participate, children must be between 3 and 20.9 years old and have been diagnosed with sickle cell anemia. They should either be eligible for the MRD HCT treatment or currently receiving NT-DMT. Parents or guardians will need to give their consent for the child to join the study, and participants will have regular check-ups and assessments during the three years. This study is important because it aims to provide valuable information that can help doctors and families make the best choices for treating sickle cell disease.

Gender

ALL

Eligibility criteria

• Inclusion 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.
• Exclusion Criteria:
• • 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.
• • Children who are unable to adhere to the study protocol or follow-up requirements.