Allogeneic hematopoietic stem cell transplantation (allo-SCT) is a curative therapy for chronic granulomatous disease (CGD). Although non-myeloablative conditioning is desirable, an optimal conditioning regimen is yet to be determined, especially in hematopoietic stem cell transplantation (HSCT) from alternative donors. In 2014, the European Society for Blood and Marrow Transplantation (EBMT) working group performed HSCT with a targeted busulfan (BU)-based regimen to treat CGD, and reported reduced intensity allo-SCT. This combination has the potential to be a standard regimen for CGD, however no such cases have been reported in Japan. Here, we report two patients with CGD who received HSCT from unrelated donors with targeted BU-based regimens. The first patient received allo-SCT from one locus antigen-mismatched unrelated donor, and the second received allo-SCT from a serologically HLA-matched unrelated donor. In both patients, superoxide production recovered to normal levels. This conditioning can be applied as a standard regimen for CGD, even in HSCT from unrelated donors.
2017 The Japan Society for Hematopoietic Stem Cell Transplantation