A randomized, double-blind, placebo-controlled study with pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF) as anadjunct to chemotherapy for adults with de novo acute myeloid leukemia

To determine the safety, biologic, and clinical benefits of pegylated recom binant human megakaryocyte growth and development factor (PEG-rHuMGDF; Amge n, Thousand Oaks, CA) after myelosuppressive chemotherapy in acute myeloid leukemia (AML),108 adult patients with de novo AML were randomized to recei ve either PEG-rHuMGDF (2.5 mu g/kg/d or 5 mu g/kg/d) for up to 21 doses (gr oup A), a single dose of 2.5 mu g/kg PEG-rHuMGDF, 7 daily doses of 2.5 mu g /kg PEG-rHuMGDF (group B), or placebo. The greatest biologic activity was s een in group A with a median peak platelet count of 1,084 x 10(9)/L, occurr ing at a median 9 days after the last dose of study drug, compared with 517 x 10(9)/L and 396 x 10(9)/L in group B and placebo group, respectively. Th rombocytosis (platelets >1,000 x 10(9)/L) was seen at rates of 52%, 8%, and 9% in groups A, B, and placebo, respectively, but were not associated with any adverse event. There was no effect on median time to transfusion indep endent platelet recovery (greater than or equal to 20 x 10(9)/L). The media n time to neutrophil recovery (greater than or equal to 500/mu L) and red b lood cell transfusion requirements were similar in all groups, and there wa s no apparent stimulation of leukemia. PEG-rHuMGDF was biologically active and well tolerated. Further investigation of dose and scheduling is require d, specifically earlier dosing before and during chemotherapy. (C) 1999 by The American Society of Hematology.