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Wiley, British Journal of Haematology, 2023

DOI: 10.1111/bjh.19105

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

Journal article published in 2023 by Ahmad Nanaa ORCID, Rong He ORCID, James M. Foran ORCID, Talha Badar ORCID, Naseema Gangat ORCID, Animesh Pardanani ORCID, William J. Hogan, Mark R. Litzow, Mrinal Patnaik, Aref Al‐Kali ORCID, Hassan B. Alkhateeb ORCID
This paper was not found in any repository, but could be made available legally by the author.
This paper was not found in any repository, but could be made available legally by the author.

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Abstract

SummaryVenetoclax (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.