Venetoclax plus hypomethylating agents in <scp><i>DDX41</i></scp>‐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 M. Patnaik, Aref Al‐Kali, Hassan B. Alkhateeb
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.