Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2021 Mar;192(6):1026-1030.
doi: 10.1111/bjh.16722. Epub 2020 May 26.

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

Ing S Tiong  1   2 , Richard Dillon  3   4 , Adam Ivey  1 , Tse-Chieh Teh  1 , Phillip Nguyen  1 , Nicholas Cummings  1 , David C Taussig  5 , Annie-Louise Latif  6 , Nicola E Potter  3 , Manohursingh Runglall  7 , Nigel H Russell  4 , Kavita Raj  4 , Anthony P Schwarer  8 , Chun Yew Fong  2 , Andrew P Grigg  2 , Andrew H Wei  1
Affiliations

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

Ing S Tiong et al. Br J Haematol. 2021 Mar.

Abstract

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 risk in patients with persistent or rising NPM1mut MRD.

Keywords: AML; MRD; NPM1; venetoclax.

PubMed Disclaimer

Figures

Fig 1
Fig 1
Swimmer plot of patients treated with venetoclax in combination with low‐intensity chemotherapy for molecular persistence (cohort 1: cases 1–5) and progression (cohort 2: cases 6–12).
See this image and copyright information in PMC

References

    1. Ivey A, Hills RK, Simpson MA, et al. Assessment of minimal residual disease in standard‐risk AML. N Engl J Med. 2016;374(5):422–33. - PubMed
    1. Balsat M, Renneville A, Thomas X, et al. Postinduction Minimal Residual Disease Predicts Outcome and Benefit From Allogeneic Stem Cell Transplantation in Acute Myeloid Leukemia With NPM1 Mutation: A Study by the Acute Leukemia French Association Group. J Clin Oncol. 2017;35(2):185–93. - PubMed
    1. Kronke J, Schlenk RF, Jensen KO, et al. Monitoring of minimal residual disease in NPM1‐mutated acute myeloid leukemia: a study from the German‐Austrian acute myeloid leukemia study group. J Clin Oncol. 2011;29(19):2709–16. - PubMed
    1. Shayegi N, Kramer M, Bornhauser M, et al. The level of residual disease based on mutant NPM1 is an independent prognostic factor for relapse and survival in AML. Blood. 2013;122(1):83–92. - PubMed
    1. Dillon R, Hills R, Freeman S, et al. Molecular MRD status and outcome after transplantation in NPM1‐mutated AML. Blood. 2020;135(9):680–8. - PMC - PubMed

Publication types