Safety considerations for drugs newly approved for treating acute myeloid leukemia.

Introduction: Acute myeloid leukemia (AML) is typically characterized by a poor prognosis, mainly due to the median age at diagnosis. Until recently, treatment options were limited to intensive chemotherapy (IC) for young patients or hypomethylating agents for those ineligible for IC. Since 2017, nine molecules were registered for newly-diagnosed AML: midostaurin, gilteritinib, quizartinib, enasidenib, ivosidenib, gemtuzumab ozogamicin, CPX-351, glasdegib, and venetoclax.

Areas covered: The review examines the safety profile of these drugs and their interactions with other agents used in supportive care. The PubMed and Google Scholar databases were searched for articles in English concerning new agents in AML from 2017 until 2023. Further relevant publications were obtained by reviewing the prescribing information and Food and Drug Administration (FDA) data.

Expert opinion: The therapeutic spectrum in AML has broadened over several years and can also improve outcomes in older patients. However, in addition to their well-known cytotoxic activity, new molecules cause several unique, off-target toxicities. Also, potential drug-drug interactions (DDI) should be taken into consideration when choosing optimal first-line therapy; this remains a challenge in clinical practice.