Background: VEN + hypomethylating agents (HMAs)/low-dose cytarabine was approved for the treatment of pts with AML who are ≥75 y or ineligible for intensive chemotherapy in 11/2018.
Aims: To understand the real-world use, safety, and efficacy of VEN in the US clinical practice.
Methods: This is a retrospective, descriptive analysis of deidentified secondary data from the COTA AML database, which included pt-level curated electronic health record data from centers across the US. Data were abstracted for adults diagnosed with AML who initiated first-line (1L) treatment on or after 01/01/2018 and received ≥1 VEN treatment at any point. Pts with no diagnosis date or with concurrent primary malignancies were excluded. Follow-up was until the last recorded event (last visit or death). The study period for this analysis was 01/2018 to 09/2022.
Results: Of 3836 pts with AML in the database, 1163 pts initiated 1L treatment in or after 2018; of these, 636 received ≥1 VEN treatment at any point. See Table for baseline demographic data. Most VEN-treated pts were seen by community healthcare providers (407/636, 64%). Genetic/molecular data were limited. Of the most frequently tested mutations, the most common were TP53 (129/461, 28%), RUNX1 (109/444, 25%), TET2 (101/414, 24%), ASLX1 (92/417, 22%), and DNMT3A (91/424, 22%). Of pts who received VEN, 47% (299/636) received 1L VEN, and the majority (269/636, 42%) received VEN + HMAs. For pts with treatment prior to VEN (pretreated), 262/337 (78%) were previously treated with chemotherapy. 1L pts were more often treated in community centers than pretreated pts (73% vs 56%), had more comorbidities (56% vs 40%), and TP53 positivity (19% vs 10%) at baseline.
Of 284 VEN-treated pts with available response data, 180 (63%) had a complete response (CR). Median OS (mOS) of VEN-treated pts was 14.4 mo from 1L treatment initiation (de novo AML, 17 mo; secondary AML, 14 mo). mOS from diagnosis was 14 mo for newly diagnosed and 18 mo for pretreated pts. Toxicity was the main cause of dose reduction and discontinuation. Median duration of VEN treatment was 78 d. Dose reduction was reported in ~30% of VEN-treated pts. Of VEN-treated pts, 53% (338/636) discontinued at least 1 VEN regimen, with a median time to discontinuation of 49.5 d.
Summary/Conclusion: This study provides an overview of the real-world use of VEN in pts with AML in the US since its approval in 11/2018. Despite the study period starting earlier than VEN approval, almost half of pts with AML who initiated treatment after 2018 received 1L VEN. The real-world CR rate in VEN-treated pts with available data was 63%, but over half of VEN-treated pts had VEN treatment discontinuation.
Keywords: AML, Venetoclax, Real world data