Hematopoietic cell transplant compared with standard care in adolescents and young adults with sickle cell disease.

ABSTRACT:

Abstract

Disease-modifying therapies are standard of care (SOC) for sickle cell disease (SCD), but hematopoietic cell transplantation (HCT) has curative potential. We compared outcomes prospectively through 2 years after biologic assignment to a donor or no donor (SOC) arm based on the availability of an HLA-matched sibling or unrelated donor (BMT CTN 1503). A donor search was commenced after eligibility confirmation. The primary end point was a comparison of survival between the treatment arms 2 years after biologic assignment. Power calculations required 60 participants in the donor arm and 140 in the no donor arm to determine if early transplant-related mortality might be balanced by disease-related mortality over a longer period of follow-up. Secondary objectives were a compari

SUBMITTER: Walters MC 

PROVIDER: S-EPMC11907447 | biostudies-literature | 2025 Mar

REPOSITORIES: biostudies-literature