Post-Transplant Cyclophosphamide for Graft-Versus-Host Disease Prophylaxis in Pediatric Stem Cell Transplantation

Graft-Versus-Host disease (GVHD) is a leading contributor to morbidity and mortality following stem cell transplantation (SCT). Current GVHD prevention methods utilize a combination of immunosuppressive drugs to prevent injury to host tissues. Post-Transplant cyclophosphamide (PTCy) for GVHD prophylaxis initially was shown to be effective in the adult haploidentical SCT setting and later in matched donor SCTs. Implementation of PTCy has increased the utilization of alternative donors in curative SCT. In pediatrics, PTCy for GVHD prophylaxis has recently demonstrated safety and efficacy. However, its widespread use still lags compared with adult use due to variability between institutions and the lack of prospective pediatric trials. In this review, we summarize the pathophysiology of GVHD and PTCy and synthesize studies in adults and children of PTCy for GVHD prophylaxis, which support its use. We focus on using PTCy in haploidentical, matched donor, and umbilical cord blood transplantation settings, and discuss the current and future role of PTCy in pediatrics. Our goal is to inform pediatric SCT physicians regarding the use of PTCy to help reduce morbidity and mortality following transplantation.