Final safety and efficacy results from the CPX-351 early access program for older patients with high-risk or secondary acute myeloid leukemia

Leuk Lymphoma. 2020 May;61(5):1188-1194. doi: 10.1080/10428194.2020.1725503. Epub 2020 Feb 26.


CPX-351, a dual-drug liposomal encapsulation of cytarabine and daunorubicin at a synergistic 5:1 molar drug ratio, achieved superior efficacy compared with conventional chemotherapy in older adults with newly diagnosed, high-risk/secondary acute myeloid leukemia (AML) in phase 2 and 3 studies. Prior to CPX-351 commercialization, an expanded access program (EAP) provided CPX-351 access for this population in the United States. In this phase 4, single-arm, open-label study (NCT02533115), 52 patients were treated with CPX-351 for 1-2 induction cycles and ≤4 consolidation cycles. The primary endpoint was safety. The most common serious adverse events were febrile neutropenia (19%), pneumonia (10%), and infection (8%). The 30- and 60-d mortality rates were 0% and 6%, respectively. Remission was achieved by 44% of patients; 90% of patients were alive at study completion. Overall, these results support outcomes from prior studies and the use of CPX-351 in older adults with newly diagnosed, high-risk/secondary AML.

Keywords: Acute myeloid leukemia; CPX-351; expanded access program.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Aged
  • Antineoplastic Combined Chemotherapy Protocols* / adverse effects
  • Cytarabine / adverse effects
  • Daunorubicin / adverse effects
  • Humans
  • Leukemia, Myeloid, Acute* / drug therapy


  • CPX-351
  • Cytarabine
  • Daunorubicin

Associated data