What is the effect of granulocyte macrophage colony-stimulating factor (GM-CSF) on induction therapy for the treatment of acute myeloid leukemia (AML)?

Updated: May 26, 2020
  • Author: Karen Seiter, MD; Chief Editor: Emmanuel C Besa, MD  more...
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Other groups have studied the effect of granulocyte macrophage colony-stimulating factor (GM-CSF) on induction therapy.

In an Eastern Cooperative Oncology Group (ECOG) study of yeast-derived GM-CSF in elderly patients with AML, no significant increase in induction chemotherapy response rate was observed; however, a significant decrease in the death rate from pneumonia and fungal infection was observed. [108] Neutrophil recovery rate was increased in the GM-CSF group (14 d vs 21 d, respectively), and overall survival was significantly improved (323 d vs 145 d, respectively). [108]

In a CALGB study of GM-CSF derived from E coli, no difference was observed in induction chemotherapy response rates between the GM-CSF group and the placebo group. [109] The risk of severe infection and resistant leukemia was similar in the 2 groups. However, in an EORTC study using GM-CSF derived from E coli, patients who randomly received GM-CSF after induction had a significantly lower complete rate (48%) than those who did not receive GM-CSF (77%). [110]

These data suggest that G-CSF and yeast-derived GM-CSF accelerate neutrophil recovery and decrease the risk of infection in patients who are undergoing induction therapy. [110] For this reason, most clinicians use either of these growth factors in patients who are at high risk for complications from infection.

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