Venetoclax plus hypomethylating agents in DDX41-mutated acute myeloid leukaemia and myelodysplastic syndrome: Mayo Clinic series on 12 patients

Ahmad Nanaa, Rong He, James M. Foran, Talha Badar, Naseema Gangat, Animesh Pardanani, William J. Hogan, Mark R. Litzow, Mrinal Patnaik, Aref Al-Kali, Hassan B. Alkhateeb

Research output: Contribution to journalArticlepeer-review

Abstract

Venetoclax (VEN) is an FDA-approved selective inhibitor of B-cell leukaemia/lymphoma-2 (BCL-2), used for treating elderly or unfit acute myeloid leukaemia (AML) patients unable to undergo intensive chemotherapy. Combining VEN with hypomethylating agents (HMAs) has shown impressive response rates in high-risk myelodysplastic syndromes (MDS) and relapsed/refractory AML. However, the efficacy of VEN and HMAs in treating DDX41-mutated (mDDX41) MDS/AML patients remains uncertain. Despite the favourable prognostic nature of mDDX41 MDS/AML patients, there is a lack of clinical experience regarding their response to different treatment regimens, leading to an unknown optimal therapeutic approach.

Original languageEnglish (US)
Pages (from-to)171-176
Number of pages6
JournalBritish journal of haematology
Volume204
Issue number1
DOIs
StatePublished - Jan 2024

Keywords

  • DDX41
  • hypomethylating agents
  • response
  • treatment
  • venetoclax

ASJC Scopus subject areas

  • Hematology

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