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

Br J Haematol. 2021 Mar;192(6):1026-1030. doi: 10.1111/bjh.16722. Epub 2020 May 26.

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.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Adult
  • Aged
  • Aged, 80 and over
  • Bridged Bicyclo Compounds, Heterocyclic / administration & dosage*
  • Female
  • Humans
  • Leukemia, Myeloid, Acute* / drug therapy
  • Leukemia, Myeloid, Acute* / genetics
  • Male
  • Middle Aged
  • Mutation*
  • Neoplasm Proteins / genetics*
  • Neoplasm, Residual
  • Nuclear Proteins / genetics*
  • Nucleophosmin
  • Retrospective Studies
  • Sulfonamides / administration & dosage*

Substances

  • Bridged Bicyclo Compounds, Heterocyclic
  • NPM1 protein, human
  • Neoplasm Proteins
  • Nuclear Proteins
  • Sulfonamides
  • Nucleophosmin
  • venetoclax