Novel therapies for acute myeloid leukemia. Does age still matter?

Abstract

The prognosis of patients with AML varies significantly with age, driven by biological heterogeneity and age-associated factors such as comorbidities, functional status, and hospitalization burden. Many novel therapies have been approved in recent years; however, pivotal trials often include patients within a restricted age range, limiting the extrapolation of their findings across the broader AML population. For example, the FLT3 inhibitor midostaurin was added to chemotherapy for patients aged 18-60 years, while the BCL-2 inhibitor venetoclax was combined with azacitidine in patients aged 75 years and older, leaving important knowledge gaps regarding their efficacy and safety in other age groups. Moreover, for several novel therapies, particularly in populations outside the original trial age range, supporting evidence is derived primarily from single-arm studies or real-world experience rather than randomized controlled trials, further complicating clinical decision-making. This review explores the efficacy and safety of widely used traditional and novel therapies for AML, with particular focus on the impact of age on these different therapeutic regimens.