Clinical benefits of granulocyte colony-stimulating factor therapy after hematopoietic stem cell transplant in children: results of a prospective randomized trial

Background and objectives: Hematopoietic stem cell transplantation (HSCT) is associated with profound neutropenia and significant morbidity and mortality. To evaluate the safety and efficacy of non-glycosylated recombinant human granulocyte colony-stimulating factor (rHuG-CSF) in accelerating myeloid recovery and its influence on infections, supportive therapy, and transplant-related mortality we carried out a randomized study in pediatric patients receiving HSCT. Design and methods: Two hundred and twenty-one consecutive children, recipients of an allogeneic or autologous bone marrow (BM) or peripheral blood progenitor cell (PBPC) transplant, were randomized to either receive rHuG-CSF 10 mg/kg (n=110) or not (n=111). Results: Myeloid engraftment was faster in the treated arm (14 vs 20 days, p=0.0001). Neutrophil recovery was accelerated both in the BM subgroups (allogeneic and autologous, p=0.002) and in the PBPC group (p=0.0005). All the other evaluated variables showed an advantage in favor of rHuG-CSF treated patients that was significant for platelet transfusion independence and time to discharge (p=0.02 and p=0.04, respectively) only in the BM subgroup. Interpretation and conclusions: We conclude that faster neutrophil recovery in BM recipients receiving rHuG-CSF led to clinical benefits, while, in the PBPC subgroup, it did not translate into clinical advantages.