Science Highlights
Published on June 18, 2026
Reduced Intensity Conditioning for Adults With Sickle Cell Disease
by Bone Marrow Transplantation
Alasbali R, Alzahrani M, Wilkerson K, et al. Excellent Outcomes Using a Novel Reduced Intensity Conditioning With Thiotepa and Post-Transplant Cyclophosphamide for HLA-Matched Donor Transplant in Adolescents and Adults With Sickle Cell Disease. Bone Marrow Transplantation. 2026; (doi: 10.1038/s41409-026-02876-3).
Researchers describe a novel reduced-intensity conditioning protocol that could potentially resolve a common treatment barrier for adults with sickle cell disease (SCD). Children with SCD overwhelmingly benefit from myeloablative HLA-matched related donor hematopoietic stem cell transplantation, but myeloablative conditioning regimens are associated with treatment-related mortality in adults with SCD. To address this issue, investigators developed a reduced intensity conditioning regimen that combines thymoglobulin, thiotepa, cyclophosphamide, fludarabine, and total-body irradiation. The regimen resulted in zero graft failures and favorable survival outcomes in a group of 25 adults, ages 18-32 years, with severe SCD who took part in a multisite trial. The researchers report day +28 median whole blood chimerism of 100% and day +365 of 97%. The overall survival rate at 2-year follow-up was 100%, while 5-year event-free survival was estimated at 96%. Acute graft-versus-host disease (GVHD) was documented in one patient in the year following transplantation, with no cases of chronic GVHD.
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Bone Marrow Transplantation