Real‐world analysis of <scp>CPX</scp> ‐351 in <scp>AML</scp> ‐ <scp>MR</scp> : A multicentre study from the <scp>MARROW</scp> consortium
CPX-351 is a standard front-line induction regimen for newly diagnosed acute myeloid leukaemia (AML) with myelodysplasia-related changes (AML-MRC). The 2022 International Consensus Classification (ICC) and World Health Organization (WHO) classifications redefine AML with myelodysplasia-related (AML-MR) to include myelodysplasia-related mutations as well as cytogenetic abnormalities. Clinical outcomes of patients treated with CPX-351 within these refined AML-MR classifications remain unclear. We conducted a retrospective, multicentre study of 235 adults with newly diagnosed AML-MR treated with CPX-351 across seven US academic centres. Patients were stratified by age (younger: <60 vs. older: ≥60 years) and AML-MR subgroup: cytogenetics (AML-MRc), molecular (AML-MRm) and antecedent haematological disorder (AML-AHD). Outcomes included complete remission (CR) and CR with incomplete recovery (CR/CRi), rates of allogeneic haematopoietic stem cell transplant (alloHSCT) and overall survival (OS). The overall CR/CRi rate of CPX-351 was 52%, with no difference by age. AML-MRm had the highest CR/CRi rate (57%). Among CR/CRi responders, 55% underwent alloHSCT (<60 years: 53% vs. ≥60 years: 57%). Median OS was 13.8 months with no significant difference by age. Younger AML-MRm patients had longer median OS compared with older AML-MRm patients (38.0 vs. 19.5 months; p = 0.05). Favourable outcomes in AML-MRm, particularly in younger patients, support molecular classification in guiding therapy and selectively extending CPX-351 use beyond older adults.
Authors
- Charles Foucar (ORCID: https://orcid.org/0000-0001-7972-6979)
- Kieran Sahasrabudhe (ORCID: https://orcid.org/0000-0001-8919-1189)
- Jesus D. Gonzalez‐Lugo (ORCID: https://orcid.org/0000-0001-5870-4594)
- Lauren Gabriele Banaszak (ORCID: https://orcid.org/0000-0002-7682-0350)
- Alice S. Mims (ORCID: https://orcid.org/0000-0002-8971-2199)
- Daniel T. Peters (ORCID: https://orcid.org/0000-0002-4655-0994)
- Alexander Joseph Ambinder (ORCID: https://orcid.org/0000-0003-0855-9653)
- Tara L. Lin (ORCID: https://orcid.org/0000-0002-0242-6449)
- Joshua F. Zeidner (ORCID: https://orcid.org/0000-0002-9014-1514)
- Brittany Knick Ragon (ORCID: https://orcid.org/0000-0002-2460-0698)
- Christine M. McMahon (ORCID: https://orcid.org/0000-0001-9745-9595)
- Onyee Chan (ORCID: https://orcid.org/0000-0003-2872-1961)
- Michael J. Hochman (ORCID: https://orcid.org/0000-0003-0594-154X)
- Krzysztof Mrózek (ORCID: https://orcid.org/0000-0002-1408-5063)
- Deedra Nicolet (ORCID: https://orcid.org/0000-0001-7796-0251)
- Ann‐Kathrin Eisfeld (ORCID: https://orcid.org/0000-0001-6442-1419)
- Yazan F. Madanat (ORCID: https://orcid.org/0000-0001-9573-2690)
- Jeffery Lancet
- Najla Al‐Ali
- Tamanna Haque (ORCID: https://orcid.org/0000-0003-0589-9038)
- Bianca Barredo
- Justice Ameyi
Institutions
- University of North Carolina at Chapel Hill (US)
- Memorial Sloan Kettering Cancer Center (US)
- Emory University (US)
- University of New Mexico (US)
- Moffitt Cancer Center (US)
- Piedmont Cancer Institute (US)
- Levine Cancer Institute (US)
- University of Wisconsin Carbone Cancer Center (US)
- University of Kansas Medical Center (US)
- Sidney Kimmel Comprehensive Cancer Center (US)
- Winship Cancer Institute
- UNC Lineberger Comprehensive Cancer Center
- Wake Forest University (US)
- The Ohio State University (US)
- The University of Texas Southwestern Medical Center (US)
- University of Colorado Denver (US)
Publication Details
- Journal
- British Journal of Haematology
- Published
- 2025-12-03
- DOI
- https://doi.org/10.1111/bjh.70274
- Primary Topic
- Acute Myeloid Leukemia Research
- Type
- article
- Field-Weighted Citation Impact
- 0.00
Funders
- Medical Research Council
- National Cancer Institute
- National Institute of General Medical Sciences
- U.S. National Library of Medicine