Re-treatment of relapse in elderly AML with time-limited venetoclax-based regimen: a case report and literature review

Venetoclax and hypomethylating agent combination therapy is now the standard of care in acute myeloid leukemia (AML) for patients ineligible for intensive therapy, including the elderly; however, venetoclax and low-dose cytarabine remain a viable option for select patients. Relapse in this cohort remains a significant challenge, with a poor prognosis and an unmet need for further treatment options. This case discusses an elderly patient with NPM1-mutated AML successfully retreated with time-limited venetoclax and low-dose cytarabine at relapse. The sustained response observed contributes to the limited literature on the efficacy of re-treatment with venetoclax-based therapy in this setting. However, prospective data are required to assess the efficacy and safety of this strategy as well as to establish the role of molecular monitoring. The role of time-limited venetoclax therapy for preventing treatment resistance and limiting treatment-related adverse effects remains an important question in the cohort ineligible for intensive therapies, given the poor prognosis and limited options of relapsed/refractory AML post-venetoclax and hypomethylating agent therapy.