Efficacy and Safety of Ziftomenib Combined with Venetoclax and Azacitidine in Relapsed/Refractory NPM1-Mutated Acute Myeloid Leukemia

Highlight

– Ziftomenib, an oral menin inhibitor, combined with venetoclax and azacitidine shows promising efficacy in relapsed/refractory NPM1-mutated AML patients.
– Composite complete remission (CRc) reached 46% overall, with higher rates in venetoclax-naïve patients (70%).
– The combination therapy was generally well tolerated with manageable adverse events including hematologic toxicities and rare differentiation syndrome.
– Median duration of response was 8.6 months, and median overall survival was not reached, suggesting durable responses.

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