Posttransplant cyclophosphamide as graft-versus-host-disease prophylaxis in children with β-thalassemia major undergoing hematopoietic stem cell transplantation

Background Graft-versus-host disease (GVHD) remains a major complication in hematopoietic stem cell transplantation. The aim of this work is to evaluate the impact of post-transplant cyclophosphamide in conjunction with other immunosuppressive medications based on a conditioning regimen as GVHD prophylaxis in children with beta thalassemia major undergoing stem cell transplantation. Patients and methods This study was carried out on 30 patients with β-thalassemia major (Pesaro class I and II), who were candidates for allogeneic stem cell transplantation from a matched sibling donor. Our patients were categorized into two groups: group I (Bu/Cy/Low-dose antithymocyte globulin-30 mg, Methotrexate) and group II (Bu/Cy/post Cy). Results In both studied groups, no differences were found regarding the clinical features. There were no differences as regards the duration of hospital stay, transplant-related complications, and mortality in both groups. Patients in group 1 achieved faster engraftment of neutrophils with a median time of 13 days and had higher platelet transfusion requirements during transplant. No differences were observed in the incidence of post-transplant viral reactivation. There was a statistically significant difference between both groups as regards the incidence of acute GVHD, which was more in group 1 with P =0.020*. First-year survival and disease-free survival were comparable in both groups. Conclusion Pesaro class I and II thalassemia patients can safely undergo hematopoietic stem cell transplantation using the post-transplant cyclophosphamide regimen and achieve the same excellent outcomes with a partial decrease in GVHD incidence compared with patients receiving the standard low-dose antithymocyte globulin-30 mg/kg plus methotrexate regimen.