Successful Haploidentical Stem Cell Transplantation with Post-Transplant Cyclophosphamide in a Severe Combined Immune Deficiency Patient: a First Report

Excerpt

Allogeneic hematopoietic stem cell transplantation (HSCT) is still the mainstay of treatment in the majority of severe combined immune deficiency (SCID) patients [1]. In the absence of a matched related donor (MRD), several alternative stem cell sources are available, including unrelated volunteer donors (UD), umbilical cord blood (UCB) units, or haploidentical transplantation (HT) [2]. Although overall survival rates after HT have improved significantly over time for SCID patients, problems with ex vivo T cell depletion still exist, including graft manipulation, high technology cost, slow immune recovery, and high rate of infections [1, 2]. T cell replete grafts with post-transplant cyclophosphamide (PT-CY) is an emerging recent approach to HT in adult patients with malignant hematological conditions. It seems to overcome many of the obstacles associated with ex vivo T-depleted HT [3‐5]. We report, to our knowledge, the first successful haploidentical transplantation using T cell replete graft and PT-CY in a SCID patient. …