Granulocyte–Macrophage Colony-Stimulating Factor in the Therapy of Adults with De Novo Acute Myeloblastic Leukemia: An Update of a Double-Blind Randomized, Placebo-Controlled Trial

We investigated whether granulocytemacrophage colony-stimulating factor (GMCSF) given concomitantly with chemotherapy (CT) improves the outcome of adults with de novo acute myeloblastic leukemia (AML) by increasing the efficacy of CT and reducing infections. CT included cytarabine (ara-C) daunorubicin, and etoposide (DAV) for induction and early consolidation therapy and one cycle with high-dose (patients aged ≤50 years) or intermediate-dose ara-C (patients aged >50 years) /daunorubicin for late consolidation therapy. Eighty patients were randomized after DAV 1 to receive either GM-CSF (Escherichia coli, 250 µg/m2 per day, s.c.) or placebo starting 48 h prior to DAV II and the subsequent courses and given throughout CT until the absolute neutrophil count had recovered to >500/µl. The CR was 81% in the GM-CSF and 79% in the placebo group (p=0.57; Fisher’s exact test). After a median follow up of 35 months the probability of relapsefree survival (RFS) at 41 months was 42% in the GM-CSF and 41% in the placebo group (p=0.89; log-rank test). The probability of RFS for patients aged ≤50 years was 65% in the GM-CSF versus 58% in the placebo group (p=0.31; log-rank test). In patients aged >50 years the probability of RFS under GM-CSF was 20% versus 31% in the placebo group (p=0.28; log-rank test).