Recombinant human granulocyte-macrophage colony-stimulating factor accelerates engraftment kinetics after allogeneic bone marrow transplantation for childhood acute lymphoblastic leukemia

Background and Objective. The use of recombinant human granulocyte-macropha ge stimulating factor (rhGM-CSF) has been shown to be well-tolerated and to reduce post-transplantation morbidity in adults undergoing HLA-identical a llogeneic bone marrow transplantation (BMT). There is however, limited expe rience in children. Design and Methods. We performed a prospective, comparative multicenter tri al using rhGM-CSF after allogeneic BMT in children with acute lymphoblastic leukemia (ALL). The study comprised 24 patients with ALL who received rhGM -CSF and 22 patients with ALL who did not receive rhGM-CSF. There were no s tatistically significant differences in the demographic characteristics bet ween the rhGM-CSF-treated and untreated groups. rhGM-CSF was given at a dos e of 10 mu g/kg/day infusion over 4 hours from day +1 until +28 or until th e absolute neutrophil count (ANC) was greater than or equal to 1x10(9)/L. A ll patients received HLA-identical sibling marrow and cyclosporine alone fo r graft-versus-host disease (GVHD) prophylaxis. The number of cells infused was similar in both groups. A software program (Statview 4.0, Abacus Conce pt, Inc., Berkeley, CA, USA) was used for statistical analysis. Results. The median of days to achieve ANC greater than or equal to 0.5x10( 9)/L was shorter in the rhGM-CSF-treated patients (14 days vs 18.5 days; p< 0.0001). Patients who received rhGM-CSF had a lower incidence of grade Ill- IV mucositis. The duration of hospital stay was significantly shorter in pa tients who received rhGM-CSF (31 days vs 45 days; p < 0.005). No difference s in GvHD severity, relapse or survival were observed. At the dose and sche dule used in the present study, rhGM-CSF was well-tolerated and no side eff ects were observed. Interpretations and Conclusions. rhGM-CSF at a dose of 10 mu g/kg/day in ch ildren with ALL undergoing allogeneic BMT is well tolerated, accelerates ne utrophil and platelet engraftment, reduces the intensity and severity of mu cositis and permits a more rapid discharge from hospital. (C)1999, Ferrata Storti Foundation.