Venetoclax Induces Rapid Elimination Of Npm1 Mutant Measurable Residual Disease In Combination With Low-Intensity Chemotherapy In Acute Myeloid Leukaemia

Based on promising results in older adults with acute myeloid leukaemia (AML), we treated patients with NPM1(mut) measurable residual disease (MRD) using off-label venetoclax in combination with low-dose cytarabine or azacitidine. Twelve consecutive patients were retrospectively identified, including five with molecular persistence and seven with molecular relapse/progression. All patients with molecular persistence achieved durable molecular complete remission (CRMRD-) without transplantation. Six of seven patients with molecular relapse/progression achieved CRMRD- after 1-2 cycles of venetoclax. This paper highlights the promising efficacy of venetoclax-based therapy to reduce the relapse risk in patients with persistent or rising NPM1(mut) MRD.