A review of FDA-approved acute myeloid leukemia therapies beyond '7+3'

Introduction: The standard anthracycline and cytarabine-based chemotherapy for acute myeloid leukemia (AML) has changed relatively little since the 1970s and produces unsatisfactory outcomes in many patients. In the past two decades, a better understanding of the pathophysiology and heterogeneity of this disease has led to promising new therapies, resulting in a flurry of new drug approvals. Areas covered: The MEDLINE database, ClinicalTrials.gov and conference proceedings were reviewed for the most salient literature concerning FDA-approved drugs for AML beyond standard chemotherapy: gemtuzumab ozogamicin, hypomethylating agents, Fms-like tyrosine kinase 3 (FLT3) inhibitors, isocitrate dehydrogenase (IDH) inhibitors, venetoclax, liposomal cytarabine and daunorubicin (CPX-351), and hedgehog pathway inhibitors. Key evidence for their efficacy is discussed. For each drug category, indications, typical usage and responses, major toxicities, and future directions for research are highlighted. Expert opinion: The treatment paradigm for AML is rapidly evolving. Promising new drugs targeting driver mutations have improved outcomes in specific AML subgroups. In parallel, advances in low-intensity therapies have allowed patients unfit for standard induction chemotherapy to achieve meaningful disease control. Further work is ongoing to identify synergistic drug combinations as well as optimal treatment selection guided by individual patient and disease features.

Automated summary

The treatment paradigm for AML is rapidly evolving, with promising new drugs targeting driver mutations improving outcomes in specific AML subgroups, and advances in low-intensity therapies providing meaningful disease control for patients unfit for standard induction chemotherapy. Ongoing work focuses on identifying synergistic drug combinations and optimal treatment selection guided by individual patient and disease features.