Granulocyte-macrophage colony-stimulating factor support in therapy of high-risk acute lymphoblastic leukemia in children

Background. Our purpose was to increase the dose intensity of chemotherapy and reduce the days with neutropenic fever in childhood high-risk (HR) acut e lymphoblastic leukemia (ALL) by systematic use of granulocyte-macrophage colony-stimulating factor (GMCSF). Procedure. All children with HR-ALL in F inland during 1990-1996 were included. Two open-label study groups were for med: 1) 34 children diagnosed between January, 1992, and December, 1996, re ceived seven or nine courses (depending on cranial RT or no cranial RT) of GM-CSF at 5 mu g/kg s.c. daily until an absolute neutrophil count (ANC) of 1,000 x 10(6)/liter at scheduled places in the protocol and 2) 80 control c hildren, those diagnosed between January, 1990, and December, 1991, plus al l with significant coexpression of myeloid markers, did not receive CM-CSF. Results. Dose intensity increased in patients who received regular GM-CSF support. The intensive phase of therapy, including induction, consolidation courses, and delayed intensification, was 33 days shorter(P < 0.001) in ch ildren with seven courses and 26 days shorter (P < 0.01) in those with nine courses of CM-CSF compared to controls. The number of infections during th e whole ALL therapy was reduced by use of GM-CSF in children aged >5 years (Pi 0.001), but not in those aged <5 years. The mean total duration of intr avenous antibiotics per child was 39 days in the GM-CSF group and 48 days i n the control group (P < 0.001). Systematic use of GM-CSF was cost-effectiv e. Conclusions. Systematic use of CM-CSF improved dose intensity by shorten ing the intensive treatment period by about 4 weeks. Use of GM-CSF reduced the days for inpatient antibiotics by about 1 week per child, which transla tes into reduced costs. (C) 2000 Wiley-Liss, Inc.