Blood Journal
Leading the way in experimental and clinical research in hematology

A randomized placebo-controlled phase III study of granulocyte- macrophage colony-stimulating factor in adult patients (> 55 to 70 years of age) with acute myelogenous leukemia: a study of the Eastern Cooperative Oncology Group (E1490)

  1. JM Rowe,
  2. JW Andersen,
  3. JJ Mazza,
  4. JM Bennett,
  5. E Paietta,
  6. FA Hayes,
  7. D Oette,
  8. PA Cassileth,
  9. EA Stadtmauer, and
  10. PH Wiernik
  1. Hematology Unit, University of Rochester Medical Center, NY 14642, USA.

Abstract

The treatment of adult patients greater than 55 to 70 years of age with acute myelogenous leukemia (AML) is associated with a treatment-related mortality of approximately 25%. This prospective, double-blind randomized study was designed to see if the use of granulocyte- macrophage colony stimulating factor (GM-CSF; yeast-derived) could shorten the period of neutropenia and to determine any effect this would have on therapy-related morbidity and mortality. A total of 124 patients entered this study. Induction consisted of standard daunorubicin and cytarabine. A day-10 bone marrow was examined; if this was aplastic without leukemia, 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 consolidation that they had received during induction. The overall complete remission rate was 52%; 60% for the GM- CSF arm and 44% for the placebo arm (P = .08). Median times to neutrophil recovery were significantly shortened on the GM-CSF arm. The overall treatment-related toxicity from start of GM-CSF/placebo was reduced on the GM-CSF arm (P = .049). Similarly, the infectious toxicity was significantly reduced on the GM-CSF arm (P = .015). The median survival for all patients was 10.6 months 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 greater than 55 to 70 years of age with AML; its major impact is in reducing the duration of neutropenia and therapy-related mortality and morbidity. This may result in a better response rate.