Venetoclax azacitidine achieves MRD clearance and enables transplant in NPM1 mutated AML with molecular failure

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Published: 20 Jun 2026
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Dr Chiara Sartor - University of Bologna, Bologna, Italy

Dr Chiara Sartor speaks to ecancer about results from the phase 2 GIMEMA AML2521 trial evaluating venetoclax plus azacitidine as a molecular relapse intervention and bridge to transplant in patients with NPM1 mutated acute myeloid leukaemia after intensive chemotherapy.

In this MRD driven study, the combination demonstrated high efficacy in maintaining remission and deepening molecular responses, with 97 percent of patients remaining in complete remission and 79 percent achieving MRD negativity.

Responses were rapid, with most patients achieving molecular reduction after the first cycle and MRD clearance within a median of two cycles.

Importantly, 85 percent of patients were successfully bridged to allogeneic transplant, the majority in MRD negative remission, supporting this strategy as an effective approach to prevent overt relapse.

Treatment was generally well tolerated, with manageable haematologic toxicity and minimal impact on transplant eligibility.

These findings highlight venetoclax plus azacitidine as a promising MRD guided strategy to improve outcomes in high risk NPM1 mutated AML by enabling timely and effective transition to curative transplant.