CPX-351 vs daunorubicin, cytarabine, and gemtuzumab ozogamicin in older adults with non–adverse-risk AML: the NCRI AML18 trial

S Steven Knapper (School of Medicine, Cardiff University, Cardiff, United Kingdom) L Laura W. Dillon (1Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute Cancer Research Center, Washington, DC) M Malavika Babu A Abin Thomas I Ian Thomas (Cardiff University, Cardiff, United Kingdom) C Christopher S. Hourigan (1Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute Cancer Research Center, Washington, DC) G Georgia Andrew (4Laboratory of Myeloid Malignancies, National Heart, Lung, and Blood Institute, Bethesda, MD) R Richard Dillon (5King's College London, London, United Kingdom) A Amanda Gilkes (42Department of Haematology, University of Cardiff, Cardiff, United Kingdom) N Nuria Marquez Almuina (3Centre for Trials Research, Cardiff University, Cardiff, United Kingdom) S Sophie King (Centre for Trials Research,Cardiff University, Cardiff, United Kingdom) N Nicholas McCarthy (6Department of Clinical Immunology Service, School of Infection, Inflammation and Immunology, University of Birmingham College of Medicine and Health, Birmingham, United Kingdom) R Reem Bahr (2Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute, Cancer Research Center, Washington, DC) R Rasha W. Al-Ali (2Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute, Cancer Research Center, Washington, DC) L Louisa Stone (7Clinical Haematology, Auckland City Hospital, Auckland, New Zealand) T Tom Coats (8Department of Clinical Haematology, Royal Devon and Exeter NHS Foundation Trust, Exeter, United Kingdom) J Jennifer Byrne S Simone Green (Castle Hill Hospital, Hull, United Kingdom) U Ulrik Malthe Overgaard (Copenhagen University Hospital, Copenhagen, Denmark) R Rob S. Sellar (University College London, London, United Kingdom) M Mike Dennis (The Christie NHS, Manchester, United Kingdom) P Priyanka Mehta (University Hospitals Bristol and Weston NHS Trust, Bristol, United Kingdom) R Robert Hills (15Nuffield Department of Population Health, University of Oxford, Oxford, United Kingdom) S Sylvie D. Freeman (47School of Infection, Inflammation and Immunology, University of Birmingham College of Medicine and Health, Birmingham, United Kingdom) N Nigel H. Russell (Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom)

Abstract

Abstract We compared daunorubicin/cytarabine plus fractionated gemtuzumab ozogamicin (DAGO2) with CPX-351 (CPX; 1:2 randomization) in 439 patients with acute myeloid leukemia (AML) aged ≥60 years (median age, 68 years) without known adverse-risk cytogenetics. Median follow-up was 35 months. Patients not in measurable residual disease (MRD)-negative remission after course 1 could enter a second randomization between standard and intensified chemotherapy. Post–course 1, the overall response rate (complete remission [CR] + CR with incomplete hematological recovery) was greater after DAGO2 (60% vs 47.5%; odds ratio [OR], 0.61; P = .016). Following course 2, the overall response was not significantly different (85% for DAGO2 vs 78% for CPX; P = .095). More patients attained CR with MRD negativity after course 1 in the DAGO2 arm (47% vs 29% for CPX; OR, 0.46; P = .004). We observed better 3-year event-free survival (34% vs 27%; hazard ratio [HR], 0.73; P = .012) and overall survival (52% vs 35%; HR, 0.62; P = .001) with DAGO2. CPX did not provide a survival benefit in patients with myelodysplasia (MDS)–related mutations and was associated with poorer survival in patients with NPM1 (HR, 2.83) and FLT3 mutations (HR, 2.14). Overall, 37% of patients underwent transplantation in first remission, with no difference in transplantation frequency or survival after transplant between randomization groups. Among patients entering the course 2 randomization (n = 107), survival was equivalent between standard and intensified CPX doses (P = .565). In conclusion, in this population of older patients with AML without known adverse-risk cytogenetics, DAGO2 resulted in superior survival compared with CPX. CPX did not benefit patients with MDS-related mutations over DAGO2. This trial was registered at www.ClinicalTrials.gov as #NCT02272478.

Article Details

Journal Blood
Volume / Issue Vol. 147, Issue 10
Published March 05, 2026
Pages 1048-1057
ISSN 0006-4971
Publisher Elsevier BV

Journal Info

Blood

Elsevier BV

ISSN: 0006-4971 Health Sciences

Authors (25)

S

Steven Knapper

School of Medicine, Cardiff University, Cardiff, United Kingdom

L

Laura W. Dillon

1Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute Cancer Research Center, Washington, DC

M

Malavika Babu

A

Abin Thomas

I

Ian Thomas

Cardiff University, Cardiff, United Kingdom

C

Christopher S. Hourigan

1Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute Cancer Research Center, Washington, DC

G

Georgia Andrew

4Laboratory of Myeloid Malignancies, National Heart, Lung, and Blood Institute, Bethesda, MD

R

Richard Dillon

5King's College London, London, United Kingdom

A

Amanda Gilkes

42Department of Haematology, University of Cardiff, Cardiff, United Kingdom

N

Nuria Marquez Almuina

3Centre for Trials Research, Cardiff University, Cardiff, United Kingdom

S

Sophie King

Centre for Trials Research,Cardiff University, Cardiff, United Kingdom

N

Nicholas McCarthy

6Department of Clinical Immunology Service, School of Infection, Inflammation and Immunology, University of Birmingham College of Medicine and Health, Birmingham, United Kingdom

R

Reem Bahr

2Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute, Cancer Research Center, Washington, DC

R

Rasha W. Al-Ali

2Fralin Biomedical Research Institute, Virginia Tech Fralin Biomedical Research Institute, Cancer Research Center, Washington, DC

L

Louisa Stone

7Clinical Haematology, Auckland City Hospital, Auckland, New Zealand

T

Tom Coats

8Department of Clinical Haematology, Royal Devon and Exeter NHS Foundation Trust, Exeter, United Kingdom

J

Jennifer Byrne

S

Simone Green

Castle Hill Hospital, Hull, United Kingdom

U

Ulrik Malthe Overgaard

Copenhagen University Hospital, Copenhagen, Denmark

R

Rob S. Sellar

University College London, London, United Kingdom

M

Mike Dennis

The Christie NHS, Manchester, United Kingdom

P

Priyanka Mehta

University Hospitals Bristol and Weston NHS Trust, Bristol, United Kingdom

R

Robert Hills

15Nuffield Department of Population Health, University of Oxford, Oxford, United Kingdom

S

Sylvie D. Freeman

47School of Infection, Inflammation and Immunology, University of Birmingham College of Medicine and Health, Birmingham, United Kingdom

N

Nigel H. Russell

Guy's and St Thomas' NHS Foundation Trust, London, United Kingdom