A RANDOMIZED PLACEBO-CONTROLLED PHASE-III STUDY OF GRANULOCYTE-MACROPHAGE COLONY-STIMULATING FACTOR IN ADULT PATIENTS (GREATER-THAN-55 TO 70 YEARS OF AGE) WITH ACUTE MYELOGENOUS LEUKEMIA - A STUDY OF THE EASTERN-COOPERATIVE-ONCOLOGY-GROUP (E1490)

The treatment of adult patients greater than 55 to 70 years of age wit h acute myelogenous leukemia (AML) is associated with a treatment-rela ted mortality of approximately 25%. This prospective, double-blind ran domized study was designed to see if the use of granulocyte-macrophage colony stimulating factor (GM-CSF; yeast-derived) could shorten the p eriod of neutropenia and to determine any effect this would have on th erapy-related morbidity and mortality. A total of 124 patients entered this study. Induction consisted of standard daunorubicin and cytarabi ne. A day-10 bone marrow was examined; if this was aplastic without le ukemia, patients received blinded placebo or GM-CSF from day 11 until neutrophil recovery, Patients who entered complete remission received the identical study medication (blinded GM-CSF or placebo) in consolid ation that they had received during induction. The overall complete re mission rate was 52%; 60% for the GM-CSF arm and 44% for the placebo a rm (P = .08). Median times to neutrophil recovery were significantly s hortened on the GM-CSF arm. The overall treatment-related toxicity fro m start of GM-CSF/placebo was reduced on the GM-CSF arm (P = .049). Si milarly, the infectious toxicity was significantly reduced on the GM-C SF arm (P = .015). The median survival for all patients was 10.6 month s in the GM-CSF group and 4.8 months in the placebo arm (P = .048). It appears that GM-CSF is safe and efficacious for adult patients greate r than 55 to 70 years of age with AML; its major impact is in reducing the duration of neutropenia and therapy-related mortality and morbidi ty. This may result in a better response rate. (C) 1995 by The America n Society of Hematology.