Treatment Patterns and Real-World Outcomes of Molecular Subgroups in Patients With AML Receiving Frontline Venetoclax-Based Therapy.

Abstract

For patients with newly diagnosed AML not suitable for intensive induction chemotherapy, venetoclax (VEN) plus azacitidine (AZA) is a standard of care therapy. This study describes real-world (rw) treatment patterns and outcomes of patients with AML receiving initial VEN-based therapy. Patients age ≥18 years diagnosed with AML who received first-line (1L) VEN-based therapy and had available dosing information were included from the COTA rw, electronic health records-based database. Patients with missing/imprecise key study dates were excluded. The index date for the study was the date of 1L initiation, unless otherwise noted. Rw time to next treatment, rw event-free survival, and rw overall survival (rwOS) were analyzed using the Kaplan-Meier method. A total of 331 patients met the inclusion criteria, of which the majority were male, White, and treated in community practices. In patients with available molecular data for the given marker, 8.8%, 19.7%, 11.0%, and 19.7% had mutations in <i>IDH1</i>, <i>IDH2</i>, <i>FLT3</i>-ITD, and <i>NPM1</i>, respectively. Following 1L, 115 patients initiated second-line (2L) therapy, of which 26.1% received intensive chemotherapy, 60.9% received low-intensity regimens, and 8.7% received investigational therapy. The median rwOS overall was 13.9 months and differed by mutation status (13.1 months for <i>IDH1</i>-positive patients, 42.0 months for <i>IDH2</i>-positive patients, not reached for <i>FLT3</i>-ITD-positive patients, and 42.0 months for <i>NPM1</i>-positive patients). The median rwOS for this study was comparable with results in the VIALE-A trial, despite the community-based nature of these data. There was no clear standard of care for patients who received 2L+ therapy. These data highlight the need for novel treatment for patients with AML following 1L VEN-based therapy.