Phase 2 trial of HLA-mismatched unrelated donor bone marrow transplantation using PTCy

Hematopoietic cell transplantation is curative for hematologic disorders, but outcomes are inferior with HLA-mismatched donors. Finding a donor is challenging, especially for ethnic minority patients. A multicenter phase 2 study in the USA demonstrated the feasibility and effectiveness of HCT with a HLA-mismatched unrelated donor in the setting of post-transplant cyclophosphamide. The one-year OS was 76% (90% CI 67.3-83.3). Nearly half of the study participants belonged to an ethnic minority population, suggesting this approach may significantly expand access to HCT.