Efficacy and Safety of CPX-351 Versus 7+3 in a Phase 3 Exploratory Analysis in Patients with High-Risk/Secondary Acute Myeloid Leukemia (AML) with Prior Hypomethylating Agent Exposure Who Achieved Remission

Introduction: Patients (pts) with newly diagnosed, secondary AML (sAML) may have previously received hypomethylating agent (HMA) therapy for an antecedent hematologic malignancy (eg, myelodysplastic syndrome [MDS]). Outcomes for pts with MDS who progress following HMA therapy are typically poor, with remission rates <30% and median overall survival (OS) of ~6 months (Prébet T, et al. J Clin Oncol. 2011; Jabbour E, et al. Cancer 2010). CPX-351 (Vyxeos®; daunorubicin and cytarabine liposome for injection), a dual-drug liposomal encapsulation of cytarabine [C] and daunorubicin [D] at a synergistic 5:1 molar ratio, is approved by the US FDA and the EMA for the treatment of adults with newly diagnosed therapy-related AML or AML with myelodysplasia-related changes. In a large randomized, open-label, multicenter, phase 3 study (NCT01696084) in older pts with newly diagnosed, high-risk/sAML, induction followed by consolidation with CPX-351 significantly improved OS (9.56 vs 5.95 months; hazard ratio [HR] = 0.69; 1-sided P = 0.003) versus conventional 7+3, with a safety profile comparable to that of 7+3. An exploratory subgroup analysis of the phase 3 study was performed to compare outcomes in pts with any prior HMA exposure who achieved complete remission (CR) or CR with incomplete neutrophil or platelet recovery (CRi) with CPX-351 versus 7+3.