Single-Agent Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis after Human Leukocyte Antigen Matched Related Bone Marrow Transplantation for Pediatric and Young Adult Patients with Hematologic Malignancies

Single-Agent Post-Transplantation Cyclophosphamide as Graft-versus-Host Disease Prophylaxis after Human Leukocyte Antigen Matched Related Bone Marrow Transplantation for Pediatric and Young Adult Patients with Hematologic Malignancies

Jacoby E (2016), Biology of Blood and Marrow Transplantation (Link to abstract)    

                              

Post transplant cyclophosphamide (PTC) has been shown to successfully modulate GVHD in preclinical models and in the adult population, enabling the patients to be completely off immune suppression on day +5. In this study, 11 pediatric patients were followed prospectively and received PTC for GVHD prophylaxis; they were compared to a retrospectively analyzed control group of 18 patients who received the standard therapy with calcineurin inhibitors. No acute nor chronic GVHD were seen in the PTC group, overall survival and event free survival were similar between the groups, and no significant difference was found with respect to relapse.


* This is the first study in pediatric patients for treatment with single agent post transplant cyclophosphamide for GVHD prophylaxis after matched related SCT for hematological malignancies, showing promising results on a small group of patients that warrant further study.