Venetoclax induces rapid elimination of NPM1 mutant measurable residual disease in combination with low-intensity chemotherapy in acute myeloid leukaemia.
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Based on promising results in older adults with acute myeloid leukaemia (AML), we treated patients with NPM1<sup>mut</sup> 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 (CR<sub>MRD-</sub> ) without transplantation. Six of seven patients with molecular relapse/progression achieved CR<sub>MRD-</sub> 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<sup>mut</sup> MRD.
Aged, 80 and over
Leukemia, Myeloid, Acute
Bridged Bicyclo Compounds, Heterocyclic
British journal of haematology, 2021, 192 (6), pp. 1026 - 1030