Venetoclax plus hypomethylating agents in DDX41-mutated acute myeloid leukaemia and myelodysplastic syndrome: Mayo Clinic series on 12 patients.
Ahmad NanaaRong HeJames M ForanTalha BadarNaseema GangatAnimesh D PardananiWilliam J HoganMark R LitzowMrinal PatnaikAref A Al-KaliHassan B AlkhateebPublished in: British journal of haematology (2023)
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.
Keyphrases
- end stage renal disease
- acute myeloid leukemia
- ejection fraction
- chronic kidney disease
- newly diagnosed
- prognostic factors
- peritoneal dialysis
- squamous cell carcinoma
- immune response
- primary care
- acute lymphoblastic leukemia
- patient reported outcomes
- liver failure
- allogeneic hematopoietic stem cell transplantation
- patient reported
- smoking cessation
- respiratory failure
- mechanical ventilation