Granulocyte‐macrophage‐colony stimulating factor for prevention of neutropenia and infections in children and adolescents with solid tumors. Results of a prospective randomized study

Background. Chemotherapy is an essential modality of curative strategies in pediatric oncology. Dose and dose intensity are, above all, restricted by the myelosuppressive effects of cytotoxic drugs. Neutropenia constitutes an important risk of morbidity and mortality. Granulocytemacrophage‐colony stimulating factor (GM‐CSF) is a hematopoietic growth factor that increases the number of circulating neutrophils as demonstrated in adults. Methods. A prospective randomized study of the effects of GM‐CSF was performed with 11 patients who were treated for solid tumors and received GM‐CSF for 2 weeks starting 48 hours after completion of chemotherapy. Forty‐two intraindividual identical chemotherapy‐courses with and 42 without GM‐CSF were compared. The monitoring program included the surveillance of the hematological reconstitution and the number and duration of infectious episodes. Results. The average nadir of the absolute neutrophil count (ANC) with GM‐CSF was higher than without GM‐CSF. The average number of days with an ANC below 500/μl was significantly reduced by GM‐CSF. Fewer infectious episodes were observed among those who received GM‐CSF therapy. Erythropoiesis was not significantly influenced by GM‐CSF, whereas patients with GM‐CSF therapy showed a longer thrombocytopenia without requiring more platelet transfusions. Rashes developed in two patients. Conclusions. In children and adolescents undergoing intensive chemotherapy for solid tumors, GM‐CSF reduces neutropenia and infectious episodes at the cost of mild thrombocytopenia Cancer 1995; 76:510–6.