“…Use of PT/Cy as GVHD prophylaxis, with or without additional immunosuppression, has been used with alternative donor sources in hematologic malignancies, leading to successful engraftment, as well as rates of GVHD, TRM, and graft failure comparable to HLA-matched related donors [10-12], without increased risk of posttransplant lymphoproliferative disorder [13]. Importantly, HSCT using alternative donors and PT/Cy has been performed for hemoglobinopathies, with GVHD and TRM comparable to that seen with HLA-matched related donors [14, 15], but reports using this strategy for other nonmalignant pediatric disorders, such as immunodeficiencies and bone marrow failure syndromes, are limited [16, 17]. Herein, we describe our institutional experience treating eleven pediatric patients with life-threatening, nonmalignant conditions using alternative donor sources, RIC, and PT/Cy for GVHD prophylaxis.…”