Haploidentical Hematopoietic Stem Cell Transplantation with Post-Transplantation Cyclophosphamide in Children with High-Risk Leukemia Using a Reduced-Intensity Conditioning Regimen and Peripheral Blood as the Stem Cell Source

The use of haploidentical donor hematopoietic stem cell transplantation with post-transplantation cyclophosphamide (Haplo-PTCy) in children is increasing; however, it is still not clear which preparative regimen is best in this setting. We present the long-term results of 42 patients age <18 years with high-risk leukemia who underwent this procedure using a reduced-intensity conditioning regimen (RIC) and peripheral blood as the stem cell source. Twenty-six patients had acute lymphoblastic leukemia, 13 had acute myelogenous leukemia, 2 had juvenile myelomonocytic leukemia, and 1 had blast crisis of chronic myelogenous leukemia. One-third of the patients were in first remission, 50% were in second remission, 14% were in third remission, and 3% had refractory disease. Neutrophil recovery occurred in 100% of the 40 patients alive at day +30, and transplantation-related mortality at 1 year was 14%. The incidence of acute graft-versus-disease (GVHD) grade III-IV was 17%, and the cumulative incidence of moderate to severe chronic GVHD at 1 year was 29%. The median duration of follow-up for surviving patients was 45 months; overall survival and event-free survival at 36 months were 56% and 46%, respectively. Long-term results of this series show that the use of an RIC regimen with peripheral blood stem cells as the cell source, in children with high-risk leukemia who underwent haplo-PTCy has tolerable toxicity, universal engraftment, and good survival rates.