A Phase III, Randomized Multicenter Study of Induction With or Without Granulocyte Colony-Stimulating Factor in AML

Background The effects of granulocyte colony-stimulating factors (G-CSF) on recovery following induction chemotherapy are widely accepted. However, their impact on response and survival has not been clarified yet. Male gender has been claimed to be a susceptibility factor for development of leukemia and shorten survival but effect of sex has not been analyzed in clinical trials utilizing G-CSF.

Design and Methods Efficacy and safety of G-CSF as an adjunct to de novo AML remission induction therapy was assessed in this prospective randomized Phase III multicenter trial. Patients were randomized to receive induction therapy consisting of either cytosine arabinoside 100mg/m2/d, days 1-10 and idarubicin 12 mg/m2/d, days 1-3 (control arm) or plus G-CSF (Filgrastim, 5 µg/kg/d starting from day 8 until absolute neutrophil count (ANC) over 0.5x109/L for two consecutive days) for a median duration of 14 days (G-CSF arm).

After achievement of CR, all patients received first consolidation course of Ara-C 1 gr/sq.m/d (d1-5) and Ida 12 mg/sq.m/d (d1-3). If patients did not have an HLA identical donor they were randomized to receive second course of consolidation either high dose Ara-C 3 gr/sq.m/d (d1, 3, 5) or G-CSF for collection of stem cells for the consecutive PBSCT.