Real-world experience with first-line CPX-351 treatment in patients with acute myeloid leukemia - long-term follow-up with focus on younger patients

Christina Rautenberg¹, Jan Moritz Middeke², Christoph Röllig², Matthias Stelljes³, Verena Gaidzik⁴, Oliver Kriege⁵, Mareike Verbeek⁶, Katrin Koch⁶, Julia Marie Unglaub⁷, Felicitas Thol⁸, Stefan W Krause⁹, Mathias Hänel¹⁰, Charlotte Neuerburg¹¹, Vladan Vucinic¹², Christian-Friedrich Jehn¹³, Maxi Wass¹³, Michael Heuser^{8

14}, Lars Fransecky¹⁵, Jens Chemnitz¹⁶, Udo Holtick¹⁷, Kerstin Schäfer-Eckart¹⁸, Josephine Schröder¹⁹, Sabrina Kraus²⁰, William Krüger²¹, Ulrich Kaiser²², Sebastian Scholl²³, Guido Kobbe²⁴, Paul Sebastian Jäger²⁴, Nael Alakel², Malte von Bonin², Katja Sockel², Maher Hanoun¹, Uwe Platzbecker¹², Tobias A W Holderried¹¹, Anke Morgner¹⁰, Tim Sauer⁷, Katharina S Götze⁶, Eva Wagner-Drouet⁵, Konstanze Döhner⁴, Hartmut Döhner⁴, Christoph Schliemann³, Johannes Schetelig², Martin Bornhäuser², Ulrich Germing²⁴, Sara Flossdorf²⁵, Thomas Schroeder^#²⁶, Friedrich Stölzel^#¹⁵

Affiliations

¹ Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen, Essen, Germany.
² Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden, Dresden, Germany.
³ Department of Medicine A, University Hospital Münster, Münster, Germany.
⁴ Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany.
⁵ Department of Medicine III, University Medical Center, Johannes Gutenberg University Mainz, Mainz, Germany.
⁶ Department of Medicine III, Technical University of Munich, Munich, Germany.
⁷ Department of Internal Medicine V, University Hospital Heidelberg, Heidelberg, Germany.
⁸ Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
⁹ Department 5 for Internal Medicine, University Hospital Erlangen, Erlangen, Germany.
¹⁰ Department of Internal Medicine III, Chemnitz Hospital, Chemnitz, Germany.
¹¹ Department of Hematology, Oncology, Stem Cell Transplantation, Immune and Cell Therapy, Clinical Immunology and Rheumatology, University Hospital Bonn, Bonn, Germany.
¹² Department of Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases, Leipzig, Germany.
¹³ Department of Hematology, Oncology and Stem Cell Transplantation, Asklepios Clinic St. Georg, Hamburg, Germany.
¹⁴ Clinic and Policlinic for Internal Medicine IV, University Hospital Halle (Saale), Halle (Saale), Germany.
¹⁵ Division of Stem Cell Transplantation and Cellular Imunotherapies, Department of Hematology and Oncology, University Hospital Schleswig-Holstein, Kiel, Germany.
¹⁶ Gemeinschaftsklinikum Mittelrhein GGmbH, Koblenz, Germany.
¹⁷ Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
¹⁸ Department of Internal Medicine V, Oncology and Hematology, Klinikum Nürnberg, Nürnberg, Germany.
¹⁹ Clinic for Hematology and Stem Cell Transplantation HELIOS Clinic Berlin-Buch GmbH, Berlin, Germany.
²⁰ Division of Hematology and Oncology, Department of Internal Medicine II, University of Würzburg, Medical Center, Würzburg, Germany.
²¹ Clinic and Policlinic for Internal Medicine C, Hematology and Oncology, University of Greifswald, Greifswald, Germany.
²² Department of Hematology and Oncology, St. Bernward Krankenhaus, Hildesheim, Germany.
²³ Department of Internal Medicine II, Hematology and Oncology, University Hospital Jena, Jena, Germany.
²⁴ Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine - University, Duesseldorf, Germany.
²⁵ Department for Medical Informatics, Biometry and Epidemiology, University Hospital Essen, Essen, Germany.
²⁶ Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen, Essen, Germany. Thomas.schroeder@uk-essen.de.

^# Contributed equally.

PMID: 40199848
PMCID: PMC11978746
DOI: 10.1038/s41408-025-01274-0

Real-world experience with first-line CPX-351 treatment in patients with acute myeloid leukemia - long-term follow-up with focus on younger patients

Christina Rautenberg et al. Blood Cancer J. 2025.

. 2025 Apr 8;15(1):60.

doi: 10.1038/s41408-025-01274-0.

Authors

Affiliations

¹ Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen, Essen, Germany.
² Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden, Dresden, Germany.
³ Department of Medicine A, University Hospital Münster, Münster, Germany.
⁴ Department of Internal Medicine III, University Hospital of Ulm, Ulm, Germany.
⁵ Department of Medicine III, University Medical Center, Johannes Gutenberg University Mainz, Mainz, Germany.
⁶ Department of Medicine III, Technical University of Munich, Munich, Germany.
⁷ Department of Internal Medicine V, University Hospital Heidelberg, Heidelberg, Germany.
⁸ Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School, Hannover, Germany.
⁹ Department 5 for Internal Medicine, University Hospital Erlangen, Erlangen, Germany.
¹⁰ Department of Internal Medicine III, Chemnitz Hospital, Chemnitz, Germany.
¹¹ Department of Hematology, Oncology, Stem Cell Transplantation, Immune and Cell Therapy, Clinical Immunology and Rheumatology, University Hospital Bonn, Bonn, Germany.
¹² Department of Hematology, Cellular Therapy, Hemostaseology and Infectious Diseases, Leipzig, Germany.
¹³ Department of Hematology, Oncology and Stem Cell Transplantation, Asklepios Clinic St. Georg, Hamburg, Germany.
¹⁴ Clinic and Policlinic for Internal Medicine IV, University Hospital Halle (Saale), Halle (Saale), Germany.
¹⁵ Division of Stem Cell Transplantation and Cellular Imunotherapies, Department of Hematology and Oncology, University Hospital Schleswig-Holstein, Kiel, Germany.
¹⁶ Gemeinschaftsklinikum Mittelrhein GGmbH, Koblenz, Germany.
¹⁷ Department I of Internal Medicine, University Hospital of Cologne, Cologne, Germany.
¹⁸ Department of Internal Medicine V, Oncology and Hematology, Klinikum Nürnberg, Nürnberg, Germany.
¹⁹ Clinic for Hematology and Stem Cell Transplantation HELIOS Clinic Berlin-Buch GmbH, Berlin, Germany.
²⁰ Division of Hematology and Oncology, Department of Internal Medicine II, University of Würzburg, Medical Center, Würzburg, Germany.
²¹ Clinic and Policlinic for Internal Medicine C, Hematology and Oncology, University of Greifswald, Greifswald, Germany.
²² Department of Hematology and Oncology, St. Bernward Krankenhaus, Hildesheim, Germany.
²³ Department of Internal Medicine II, Hematology and Oncology, University Hospital Jena, Jena, Germany.
²⁴ Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine - University, Duesseldorf, Germany.
²⁵ Department for Medical Informatics, Biometry and Epidemiology, University Hospital Essen, Essen, Germany.
²⁶ Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen, Essen, Germany. Thomas.schroeder@uk-essen.de.

^# Contributed equally.

PMID: 40199848
PMCID: PMC11978746
DOI: 10.1038/s41408-025-01274-0

No abstract available

PubMed Disclaimer

Conflict of interest statement

Competing interests: Thomas Schroeder: Advisory Board with honoraria: JAZZ Pharmaceuticals; lecture fees: JAZZ Pharmaceuticals; Reasearch Funding: JAZZ Pharmaceuticals. Jan Moritz Middeke: Consultancy work: Janssen, Roche, Gilead, Abbvie, JAZZ Pharmaceuticals, Pfizer, Astellas, Novartis, AstraZeneca, Glycostem; Shareholdings: Cancilico; Synagen; Honoraria: Novartis, Roche, Janssen, Abbvie, Pfizer, Sanofi, Astellas, Beigene: Reasearch Funding: JAZZ Pharmaceuticals, Janssen, Novartis; Financial travel support: Beigene. Friedrich Stölzel: Advisory Board with honoraria: JAZZ Pharmaceuticals, medac, Pierre Fabre, Servier, Astellas, Travel Support: Johnson & Johnson and Servier; Reasearch Funding: JAZZ Pharmaceuticals. Christina Rautenberg: Advisory Board with honoraria: BMS/Celgene Deutschland GmbH, Servier; Lecture fees: JAZZ Pharmaceuticals, BMS/Celgene Deutschland GmbH, Pfizer; Financial travel support: JAZZ Pharmeceuticals, BMS/Celgene Deutschland GmbH, medac; Reasearch Funding: JAZZ Pharmaceuticals. Hartmut Döhner: Advisory Board with honoraria: AbbVie, AstraZeneca, Gilead, Janssen, Jazz, Pfizer, Servier, Stemline, Syndax; Clinical Research Funding to Institution: AbbVie, Astellas, Bristol Myers Squibb, Celgene, Jazz Pharmaceuticals, Kronos Bio, Servier; financial travel support: Abbvie, Servier. Katharina Götze: Advisory Board with honoraria: AbbVie, JAZZ, BMS/Celgene Deutschland GmbH, Alexion; research funding: BMS/Celgene. Tim Sauer: Advisory Board with honoraria: AbbVie, Takeda, Astellas. Guido Kobbe: received lecture fees from BMS/Celgene Deutschland GmbH, Novartis, Jazz Pharmaceuticals and Janssen-Cilag GmbH. Ulrich Germing: research funding: BMS/Celgene Deutschland GmbH, Novartis. Christoph Schlimann: Advisory Board with honoraria: AbbVie, Astellas, AstraZeneca, BMS/Celgene Deutschland GmbH, Laboratories Delbert, JAZZ Pharmaceuticals, Novartis, Otsuka, Pfizer, Roche; Research support (institutional): JAZZ Pharmaceuticals; financial travel support: AbbVie, BMS/Celgene Deutschland GmbH, JAZZ Pharmaceuticals, Pfizer. Verena I. Gaidzik: Advisory Board with honoraria: Jazz Pharmaceuticals, Abbvie, Boehringer-Ingelheim; Speakers Bureau: Pfizer, Janssen, Abbvie, Otsuka; Travel Support: Abbvie, JAZZ Pharmaceuticals. Stefan W. Krause: Lecture fees: Eickeler, expenses for meetings: Abbvie, JAZZ Pharmaceuticals, Alexion. Vladan Vucinic: Advisory Board with honoraria from Novartis, BMS/Celgene Germany GmbH, Takeda, Abbvie, Gilead/Kite, J&J. Felicitas Thol: Advisory Board with honoraria: BMS/Celgene Germany GmbH, Novartis, AbbVie, Menarini, Rigel; Lecture fees: AbbVie, Astellas. Julia Marie Unglaub: Financial travel support: JAZZ Pharmaceuticals and AbbVie. Ethical approval and consent to participate: Treatment, monitoring and response assessment of the patients followed local guidelines of the respective centers which themselves are adapted from international guidelines, e.g. European Leukemia Net Recommendations. The study was approved by the ethics committee of the Heinrich-Heine-University, Duesseldorf (approval number: 2020–877) and all patients gave written informed consent for scientific use of their data.

Figures

**Fig. 1. Outcome after allo-HCT.**
A Posttransplant RFS, OS, NRM and CIR for the entire cohort. B Posttransplant RFS, OS, NRM and CIR depending on disease status prior allo-HCT: patients in CR (n = 94) are depicted in orange (solid line), MRD-negative patients (n = 23) are depicted green (dotted line) and MRD-positive patients (n = 14) in red (dotted line), while R/R patients (n = 27) are depicted in black (solid line).

**Fig. 2. Outcome after allo-HCT depending on age (<60 vs. ≥ 60 yrs).**
RFS, OS, NRM and CIR from transplant for patients <60 years depicted in purple (n = 39) compared to patients ≥60 years depicted in blue (n = 83).

See this image and copyright information in PMC

References

1. Lancet JE, Uy GL, Newell LF, Lin TL, Ritchie EK, Stuart RK, et al. CPX-351 versus 7+3 cytarabine and daunorubicin chemotherapy in older adults with newly diagnosed high-risk or secondary acute myeloid leukaemia: 5-year results of a randomised, open-label, multicentre, phase 3 trial. Lancet Haematol. 2021;8:e481–91. - PubMed
1. Lancet JE, Uy GL, Cortes JE, Newell LF, Lin TL, Ritchie EK, et al. CPX-351 (cytarabine and daunorubicin) liposome for injection versus conventional cytarabine plus daunorubicin in older patients with newly diagnosed secondary acute myeloid leukemia. JCO. 2018;36:2684–92. - PMC - PubMed
1. Heuser M, Ofran Y, Boissel N, Brunet Mauri S, Craddock C, Janssen J, et al. Acute myeloid leukaemia in adult patients: ESMO Clinical Practice Guidelines for diagnosis, treatment and follow-up. Ann Oncol. 2020;31:697–712. - PubMed
1. Rautenberg C, Stölzel F, Röllig C, Stelljes M, Gaidzik V, Lauseker M, et al. Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia. Blood Cancer J. 2021;11:164. - PMC - PubMed
1. Chiche E, Rahmé R, Bertoli S, Dumas PY, Micol JB, Hicheri Y, et al. Real-life experience with CPX-351 and impact on the outcome of high-risk AML patients: a multicentric French cohort. Blood Adv. 2021;5:176–84. - PMC - PubMed

Publication types

Actions

LinkOut - more resources

Full Text Sources
- Nature Publishing Group
- PubMed Central

Save citation to file

Email citation

Add to Collections

Add to My Bibliography

Your saved search

Create a file for external citation management software

Your RSS Feed

Real-world experience with first-line CPX-351 treatment in patients with acute myeloid leukemia - long-term follow-up with focus on younger patients

Affiliations

Real-world experience with first-line CPX-351 treatment in patients with acute myeloid leukemia - long-term follow-up with focus on younger patients

Authors

Affiliations

Conflict of interest statement

Figures

References

Publication types

LinkOut - more resources

Full Text Sources