Journal article

Venetoclax induces rapid elimination of NPM1 mutant measurable residual disease in combination with low-intensity chemotherapy in acute myeloid leukaemia

Ing S Tiong, Richard Dillon, Adam Ivey, Tse-Chieh Teh, Phillip Nguyen, Nicholas Cummings, David C Taussig, Annie-Louise Latif, Nicola E Potter, Manohursingh Runglall, Nigel H Russell, Kavita Raj, Anthony P Schwarer, Chun Yew Fong, Andrew P Grigg, Andrew H Wei

British Journal of Haematology | WILEY | Published : 2020


Based on promising results in older adults with acute myeloid leukaemia (AML), we treated patients with NPM1mut 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 r..

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Funding Acknowledgements

AHW is funded by the Leukemia & Lymphoma Society (LLS) Specialized Center of Research (SCOR) (Strasser) and by the Medical Research Future Fund (MRFF). DCT is funded by the NIHR RM/ICR Biomedical Research Centre.