Hematopoietic stem cell transplantation in patients with severe congenital neutropenia: An analysis of 18 Japanese cases

We studied the outcome of allogeneic HSCT in patients with SCN. Between 1989 and 2005, 18 patients with SCN in Japan received HSCT for reasons other than malignant transformation, i.e., because of the lack of or a partial response to treatment with r-HuG-CSF. The median age of the patients at the first HSCT was three and a half yr (range 0.2-16.7 yr). Nine patients received stem cells from an HLA-identical sibling donor and nine from an alternative donor. Twelve and six patients received myeloablative and non-myeloablative conditioning regimens, respectively. Engraftment occurred at the first HSCT in 12 patients, four patients received a second HSCT for graft failure, and two patients died. The cause of death was renal failure and graft failure at the first and second HSCT, respectively. The cumulative incidence of grade II-IV acute GVHD and TRM at the first transplantation was 11% and 5.6%, respectively. Of our patients, 16 are alive and in complete remission, with a median follow-up of six and a half yr. Our results suggest that HSCT is beneficial for patients with SCN refractory to r-HuG-CSF treatment.