Early Real-World First-Line Treatment With Venetoclax Plus HMAs Versus HMA Monotherapy Among Patients With AML in a Predominately US Community Setting

This retrospective cohort study used an electronic health record-derived, de-identified, US patient-level database to evaluate early real-world treatment experience among patients with newly diagnosed acute myeloid leukemia (AML) receiving venetoclax + hypomethylating agents (HMAs) or HMA monotherapy. In a predominantly community-based database of 619 patients treated with venetoclax + HMAs and 480 treated with HMA monotherapy, benefits in real-world outcomes were observed with venetoclax + HMAs over HMA monotherapy. Background: Venetoclax in combination with hypomethylating agents (HMAs) is standard-of-care in patients with newly diagnosed acute myeloid leukemia (AML) who are > 75 years old or unfit for intensive chemotherapy. We examined early real-world treatment experience among patients with AML receiving venetoclax + HMAs or HMA monotherapy. Patients and methods: This retrospective cohort study used an electronic health record-derived, deidentified, United States nationwide database comprised of patient-level structured and unstructured data, curated via technology-enabled abstraction. Patients with an AML diagnosis on or after January 1, 2014, who had > 2 clinic visits, and initiated treatment with venetoclax + HMAs from June 1, 2018 to March 31, 2021, or HMA monotherapy from January 1, 2016 to May 31, 2018, were included. Kaplan-Meier analysis was used to estimate time to last administration (TTLA) and overall survival (OS). Results: Overall, 619 patients treated with venetoclax + HMAs and 480 treated with HMA monotherapy were selected from the database. Median age at diagnosis was 76 and 78 years, respectively, most patients were treated in community practice (83.4% and 89.4%, respectively), and almost half had secondary AML (47.2% and 47.3%, respectively). Adjusted analyses showed both significantly longer TTLA (3.6 months vs. 2.3 months; hazard ratio [HR] = 0.69 [95% confidence interval (CI), 0.60-0.80], P < .0001) and OS (9.3 months vs. 5.9 months; HR = 0.71 [95% CI, 0.610.82], P < .0001) in patients treated with venetoclax + HMAs versus HMA monotherapy, respectively. Conclusion: This study shows benefit in real-world outcomes of venetoclax + HMAs relative to HMA monotherapy in patients with newly diagnosed AML, using a predominantly community-based database.