Phase II trial of CPX-351 in patients with acute myeloid leukemia at high risk for induction mortality

Ghayas C. Issa, Hagop M. Kantarjian, Lianchun Xiao, Jing Ning, Yesid Alvarado, Gautam Borthakur, Naval Daver, Courtney D. DiNardo, Elias Jabbour, Prithviraj Bose, Nitin Jain, Tapan M. Kadia, Kiran Naqvi, Naveen Pemmaraju, Koichi Takahashi, Srdan Verstovsek, Micheal Andreeff, Steven M. Kornblau, Zeev Estrov, Alessandra FerrajoliGuillermo Garcia-Manero, Maro Ohanian, William G. Wierda, Farhad Ravandi, Jorge E. Cortes

Research output: Contribution to journalArticlepeer-review

1 Scopus citations

Abstract

CPX-351 is a liposomal formulation of cytarabine/daunorubicin with a 5:1 fixed molar ratio. We investigated the safety and efficacy of escalating doses of CPX-351 in patients with acute myeloid leukemia (AML) at high risk of induction mortality with standard chemotherapy determined through assessment of leukemia and patient-related risk factors for intensive chemotherapy in an open-label, phase II trial. Patients were randomized to receive 50 or 75 units/m2 on days 1, 3, and 5. Once safety was established, a 100 units/m2 arm was opened. Fifty-six patients were enrolled, 16, 24, and 16 in the 50, 75, and 100 units/m2 arms, respectively. The composite complete remission rate (complete remission + complete remission with incomplete blood count recovery) was lowest with 50 units/m2 (19%) compared with 75 units/m2 (38%) and 100 units/m2 (44%) (P = 0.35). The 50 units/m2 arm had a median OS of 4.3 months, compared with 8.6 and 6.2 months for the 75 and 100 units/m2 respectively (P = 0.04). Nonhematologic grade 3/4 treatment-emergent adverse events included febrile neutropenia (34%), pneumonia (23%), and sepsis (16%). CPX-351 at 75 units/m2 has favorable safety and efficacy for AML patients at high risk of induction mortality with some tolerating the standard dose of 100 units/m2.

Original languageEnglish (US)
Pages (from-to)2914-2924
Number of pages11
JournalLeukemia
Volume34
Issue number11
DOIs
StatePublished - Nov 1 2020

ASJC Scopus subject areas

  • Hematology
  • Oncology
  • Cancer Research

Fingerprint

Dive into the research topics of 'Phase II trial of CPX-351 in patients with acute myeloid leukemia at high risk for induction mortality'. Together they form a unique fingerprint.

Cite this