A Pilot Study of Priming With Granulocyte Macrophage Colony-Stimulating Factor Plus All-trans Retinoic Acid Combined With Remission Induction Chemotherapy in Patients With Acute Myeloid Leukemia

Abstract

Priming with granulocyte macrophage colony-stimulating factor (GM-CSF) plus all-trans retinoic acid (ATRA) during induction chemotherapy may enhance response rates and survival in patients with acute myeloid leukemia (AML) due to the differentiation of human myeloblastic leukemia cells into granulocytes. GM-CSF was administered to patients during induction chemotherapy and ATRA was ingested orally on days 1 to 14. Patients undergoing a regimen with GM-CSF and ATRA were evaluated as compared with a historical control group of subjects. For patients enrolled in this study, the complete remission plus complete remission with incomplete platelet recovery rate was 61.5% as compared with 41.4% for the historical control group of subjects. The relapse rate was 38.5% and 44.8% for the study and control group of subjects, respectively. Two-year probabilities were 45.5% (study group) and 47.4% (control group) for disease-free survival and 38.5% (study group) and 36.2% (control group) for overall survival. The most frequent side effects included fever, headache, and skin lesions with pruritus and dyspnea with pulmonary congestion, similar to ATRA syndrome. Priming with GM-CSF plus ATRA during anthracycline-based chemotherapy is feasible in terms of response rate, but the toxicity of the regimen is significant.