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Venetoclax induces rapid elimination of <i>NPM1</i> mutant measurable residual disease in combination with low‐intensity chemotherapy in acute myeloid leukaemia

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

2020British Journal of Haematology88 citationsDOIOpen Access PDF

Abstract

Summary 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 (CR MRD‐ ) without transplantation. Six of seven patients with molecular relapse/progression achieved CR MRD‐ 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.

Topics & Concepts

VenetoclaxNPM1MedicineCytarabineOncologyInternal medicineMinimal residual diseaseMyeloid leukemiaRegimenFludarabineChemotherapyMyeloidAzacitidineLeukemiaChronic lymphocytic leukemiaCyclophosphamideBiologyChromosomeKaryotypeGene expressionGeneDNA methylationBiochemistryAcute Myeloid Leukemia ResearchHistone Deacetylase Inhibitors ResearchMultiple Myeloma Research and Treatments
Venetoclax induces rapid elimination of <i>NPM1</i> mutant measurable residual disease in combination with low‐intensity chemotherapy in acute myeloid leukaemia | Litcius