Key clinical point: Treatment with a combination of hypomethylating agents (HMA) and venetoclax (VEN) was similarly effective in patients with acute myeloid leukemia (AML) with or without spliceosome mutations with specific mutational pairs demonstrating favorable outcomes.
Major finding: Overall response rate (89% vs. 79%; P = .32), composite complete response (79% vs. 75%; P = .65), and median overall survival (OS; 35 vs. 14 months; P = .58) were not significantly different in patients with vs. without spliceosome mutations. Co-occurrence of IDH2 and SRSF2 mutations was associated with favorable outcomes (1- and 2-year OS, 100% and 88%, respectively).
Study details: Findings are from a retrospective study of 119 adult patients with AML treated with frontline HMA+VEN-based therapy. Mutation in the spliceosome gene was observed in 39 patients.
Disclosures: No specific funding source was identified. Some investigators reported research funding, support, honoraria, consultancy, and advisory roles for various pharmaceutical companies.
Source: Lachowiez CA et al. Blood Adv. 2021 Apr 22. doi: 10.1182/bloodadvances.2020004173 .