Phase II trial demonstrates 2-year EFS of 79.3% in children with severe sickle cell disease, indicating the efficacy of haploidentical bone marrow transplant.
Key Points
Graft-versus-host disease occurred in 15.5% of cases, impacting overall treatment outcomes in children with severe sickle cell disease.
Thiotepa-based conditioning combined with total body irradiation showed promising effects, contributing to a 2-year event-free survival of 79.3%.
Bone marrow transplant procedures were performed on 39 out of 41 enrolled participants, indicating strong engagement in this phase II trial.
The study highlights the need for ongoing research due to challenges like graft failure and GVHD in haploidentical bone marrow transplant.