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

TL;DR

An elderly AML patient with NPM1 mutation responded well to re-treatment with venetoclax and low-dose cytarabine after relapse, suggesting potential for this approach in similar cases.

Contribution

This case report adds to the limited evidence on re-treating relapsed AML with a time-limited venetoclax-based regimen.

Findings

The patient achieved a sustained response after re-treatment with venetoclax and low-dose cytarabine.

The case highlights the potential of re-treatment with venetoclax-based therapy in relapsed AML.

Prospective studies are needed to confirm the efficacy and safety of this strategy.

Abstract

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.

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