A Phase 2 study of CPX-351 in combination with venetoclax in patients with newly diagnosed high-risk acute myeloid leukemia.

ABSTRACT: Venetoclax has been combined with intensive chemotherapy regimens in the treatment of acute myeloid leukemia (AML). We aimed to investigate the safety and efficacy of venetoclax combined with full-dose CPX-351 (CPX + VEN) in newly diagnosed (ND) AML. Seventeen patients with a median age of 59 years (range, 43-69) were treated; 71% had secondary AML, 47% had prior hypomethylating agent (HMA) exposure, 59% had myelodysplastic syndrome (MDS)-related (MR) mutations, 47% had complex karyotype, and 29% were TP53 mutated. The overall response rate (ORR) was 82% (95% CI, 57-96) with a composite complete remission rate (CRc) of 71% (95% CI, 50-93). Patients with MR mutations had an ORR of 100% (95% CI, 69-100), including a CRc of 90% (95% CI, 55-100). Patients with prior HMA exposure had a C

SUBMITTER: Jen WY 

PROVIDER: S-EPMC12542300 | biostudies-literature | 2025 Oct

REPOSITORIES: biostudies-literature