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

Abstract

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.

Allogeneic hematopoietic stem cell transplantation for inherited bone marrow failure syndromes

“…Allogeneic hematopoietic stem cell transplantation (HSCT) is still the only curative treatment for selected patients. Validated indications of transplantation include G-CSF resistance (>20 μg/kg/day) even in the absence of infections and clonal evolution [39][40][41][42].…”

Hematopoietic stem cell transplantation for severe congenital neutropenia

“…There are currently 18 published reports of HCT in patients with SCN for a total of 65 patients transplanted without malignant transformation (Tables 1 and 2)[8 ⍰ , 40, 41 ⍰ , 42 ⍰ , 43–51, 52 ⍰ , 53, 54] and 18 patients transplanted with development of MDS or leukemia (Table 3)[8 ⍰ , 41 ⍰ , 43, 55, 56]. Indications for proceeding to transplant have included poor response to G-CSF (most common), development of G-CSFR mutation(s), inability to tolerate G-CSF injections, infection unresponsive to GCSF and anti-microbials, development of cytogenetic changes consistent with MDS, and morphologic evidence of MDS or leukemia.…”

Outcome of invasive mechanical ventilation after pediatric allogeneic hematopoietic SCT: results from a prospective, multicenter registry

“…Advancement over the years in the HSCT process has resulted in higher OS rates 1 and in a growing number of indications for transplantation. [2][3][4] However, HSCT still is a highrisk procedure, and a considerable proportion of children require transfer to the pediatric intensive care unit (PICU) because of complications associated with it. 5 Acute respiratory failure is the leading cause for PICU admission in children after HSCT.…”