Bone marrow transplantation using HLA-matched unrelated donors for patients suffering from severe combined immunodeficiency

Abstract

Severe combined immunodeficiency (SCID) is fatal in infancy unless corrected with allogeneic bone marrow transplants (BMT), preferably from a family-related genotypically HLA-identical donor (RID) or phenotypically HLA-matched family donor (PMD). For the majority of SCID patients, such donors are not available; Therefore, parents who are HLA-haploidentical donors (HID) or HLA-matched unrelated donors (MUD) have been used. MUD BMT are associated with increased frequency of acute graft versus host disease, which can be controlled by high doses of steroids. HID BMT are associated with increased frequency of short- and long-term graft failure, need for repeated transplants, fatal pneumonitis, impaired immune reconstitution, and long-term complications, contributing to lower survival. In conclusion, the excellent long-term survival, immune reconstitution, and normal quality of life after MUD BMT suggests that in the absence of RID or PMD, MUD BMT should be offered for patients suffering from SCID.