Abstract
Venetoclax (VEN) in combination with hypomethylating agents (HMA) or low-dose cytarabine has become the standard of care for patients with acute myeloid leukemia (AML) who are ineligible to receive intensive induction chemotherapy. Clinical trials are performed in a controlled setting that can be difficult to emulate in the real world. We sought to investigate outcomes of patients treated with VEN-based therapy in the real world. Patients with an age of ≥65 years who received frontline VEN-based therapy were identified using the COTA database (n = 112). The majority of patients (91%) were treated in the community setting and had adverse-risk AML (63%). The real-world overall response rate (rwORR) was 55% with a median real-world overall survival (rwOS) of 13 months after VEN/HMA. The rwORR was lower and median rwOS was shorter than those reported in the VIALE-A trial, underscoring the importance of studying novel therapies using real-world data.
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Author contributions
P.A.P., C.K.W., and A.J.B. designed the research. E.H., H.F., M.S., and A.J.B. performed the research. F.W.H., P.A.P, E.H., and A.J.B. analyzed the data. F.W.H., P.A.P., Y.F.M., C.K.W., and A.J.B. wrote the paper.
Disclosure statement
P.A.P. has served on the advisory boards of Servier and BMS Celgene and is currently employed by Servier. Y.F.M. has received honoraria/consulting fees from BluePrint Medicines, GERON, OncLive. Y.F.M. participated in advisory boards and received honoraria from Sierra Oncology, Stemline Therapeutics, Blueprint Medicines, Morphosys, Taiho Oncology, and Novartis. Y.F.M. received travel reimbursement from Blueprint Medicines and Morphosys. None of these relationships were related to this work. A.J.B., E.H., H.F., M.S., and C.K.W. are all employees of COTA, Inc.