HCT Shows Promising Safety and Efficacy in SCD

HLA-matched sibling donor (MSD) hematopoietic cell transplantation (HCT) done prior to adolescence demonstrated promising safety and efficacy outcomes among patients with sickle cell disease (SCD), according to results of an interim analysis presented at the 2026 ASPHO Conference.

In the multicenter study (ClinicalTrials.gov Identifier: NCT04018937) conducted by the Sickle Transplant, Advocacy, and Research Alliance (STAR), researchers treated 41 patients aged 2 to 12.99 years with an HLA-MSD HCT. Patients received a reduced intensity conditioning regimen that included alemtuzumab, melphalan, and fludarabine. A calcineurin inhibitor and short course of methotrexate were used for graft vs host disease (GVHD) prophylaxis.

The primary endpoint was immune suppression-free, rejection-free survival at 2 years post-HCT. This interim analysis includes data for 1 year post HCT.