LETTER TO THE EDITOR SUMMARY:

Current strategies for the treatment of acute myeloid leukemia (AML) focus on the induction of cell death by chemotherapy or targeted therapy. In elderly patients with chemorefractory disease the prognosis is still poor. We herein present a novel biomodulatory therapy consisting of low-dose 5-azacytidine (AZA) in combination with pioglitazone (PGZ) and all-trans retinoic acid (ATRA). Five elderly patients with AML primary refractory to standard induction therapies received AZA, PGZ, and ATRA (APA) on a compassionate-use basis outside a clinical trial. APA treatment induced ongoing morphologic complete remission (CR) in three of five, including two molecular CRs. Early increases of granulocytes during biomodulatory treatment indicated a differentiation inducing effect of APA. In conclusion, this study suggests that low-toxic salvage therapy with APA is clinically active in elderly AML patients who are refractory to standard induction chemotherapy.

Copyright © 2014, Ferrata Storti Foundation.