Blood Cancer Journal (Oct 2021)
Real-world experience of CPX-351 as first-line treatment for patients with acute myeloid leukemia
- Christina Rautenberg,
- Friedrich Stölzel,
- Christoph Röllig,
- Matthias Stelljes,
- Verena Gaidzik,
- Michael Lauseker,
- Oliver Kriege,
- Mareike Verbeek,
- Julia Marie Unglaub,
- Felicitas Thol,
- Stefan W. Krause,
- Mathias Hänel,
- Charlotte Neuerburg,
- Vladan Vucinic,
- Christian-Friedrich Jehn,
- Julia Severmann,
- Maxi Wass,
- Lars Fransecky,
- Jens Chemnitz,
- Udo Holtick,
- Kerstin Schäfer-Eckart,
- Josephine Schröder,
- Sabrina Kraus,
- William Krüger,
- Ulrich Kaiser,
- Sebastian Scholl,
- Kathrin Koch,
- Lea Henning,
- Guido Kobbe,
- Rainer Haas,
- Nael Alakel,
- Maximilian-Alexander Röhnert,
- Katja Sockel,
- Maher Hanoun,
- Uwe Platzbecker,
- Tobias A. W. Holderried,
- Anke Morgner,
- Michael Heuser,
- Tim Sauer,
- Katharina S. Götze,
- Eva Wagner-Drouet,
- Konstanze Döhner,
- Hartmut Döhner,
- Christoph Schliemann,
- Johannes Schetelig,
- Martin Bornhäuser,
- Ulrich Germing,
- Thomas Schroeder,
- Jan Moritz Middeke
Affiliations
- Christina Rautenberg
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Friedrich Stölzel
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Christoph Röllig
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Matthias Stelljes
- Department of Medicine A, University Hospital Münster
- Verena Gaidzik
- Department of Internal Medicine III, University Hospital of Ulm
- Michael Lauseker
- Institute for Medical Information Processing, Biometry and Epidemiology, Ludwig-Maximilians-University
- Oliver Kriege
- Department of Medicine III, University Medical Center, Johannes Gutenberg University Mainz
- Mareike Verbeek
- Department of Medicine III, Technical University of Munich
- Julia Marie Unglaub
- Department of Internal Medicine V, University Hospital Heidelberg
- Felicitas Thol
- Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School
- Stefan W. Krause
- Department V for Internal Medicine, University Hospital Erlangen
- Mathias Hänel
- Department of Internal Medicine III, Klinikum Chemnitz
- Charlotte Neuerburg
- Department of Oncology, Hematology and Rheumatology, University Hospital Bonn
- Vladan Vucinic
- Leipzig: Department of Hematology and Cell Therapy, Medical Oncology, Hemostaseology
- Christian-Friedrich Jehn
- Department of Hematology, Oncology and Stem Cell Transplantation, Asklepios Clinic St. Georg
- Julia Severmann
- Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen
- Maxi Wass
- Clinic and Policlinic for Internal Medicine IV, University Hospital Halle (Saale)
- Lars Fransecky
- Departmenf for Internal Medicine II, University Schleswig-Holstein
- Jens Chemnitz
- Gemeinschaftsklinikum Mittelrhein GGmbH
- Udo Holtick
- Department I of Internal Medicine, University Hospital of Cologne
- Kerstin Schäfer-Eckart
- Department of Internal Medicine V, Oncology and Hematology, Klinikum Nürnberg
- Josephine Schröder
- Clinic for Heaematology and Stem Cell Transplantation HELIOS Clinic Berlin-Buch GmbH
- Sabrina Kraus
- Division of Hematology and Oncology, Department of Internal Medicine II, University of Würzburg, Medical Center
- William Krüger
- Clinic and Policlinic for Internal Medicine C, Hematology and Oncology, University of Greifswald
- Ulrich Kaiser
- Department of Hematology and Oncology, St. Bernward Krankenhaus
- Sebastian Scholl
- Department of Internal Medicine II, Hematology and Oncology, University Hospital Jena
- Kathrin Koch
- Department of Medicine III, Technical University of Munich
- Lea Henning
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Guido Kobbe
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Rainer Haas
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Nael Alakel
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Maximilian-Alexander Röhnert
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Katja Sockel
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Maher Hanoun
- Department of Hematology and Stem Cell Transplantation, West German Cancer Center Essen, University Hospital Essen
- Uwe Platzbecker
- Leipzig: Department of Hematology and Cell Therapy, Medical Oncology, Hemostaseology
- Tobias A. W. Holderried
- Department of Oncology, Hematology and Rheumatology, University Hospital Bonn
- Anke Morgner
- Department of Internal Medicine III, Klinikum Chemnitz
- Michael Heuser
- Department of Hematology, Hemostasis, Oncology and Stem Cell Transplantation, Hannover Medical School
- Tim Sauer
- Department of Internal Medicine V, University Hospital Heidelberg
- Katharina S. Götze
- Department of Medicine III, Technical University of Munich
- Eva Wagner-Drouet
- Department of Medicine III, University Medical Center, Johannes Gutenberg University Mainz
- Konstanze Döhner
- Department of Internal Medicine III, University Hospital of Ulm
- Hartmut Döhner
- Department of Internal Medicine III, University Hospital of Ulm
- Christoph Schliemann
- Department of Medicine A, University Hospital Münster
- Johannes Schetelig
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Martin Bornhäuser
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- Ulrich Germing
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Thomas Schroeder
- Department of Hematology, Oncology and Clinical Immunology, University Hospital Duesseldorf, Medical Faculty, Heinrich Heine—University
- Jan Moritz Middeke
- Medizinische Klinik und Poliklinik I, University Hospital Carl Gustav Carus Dresden, Technical University Dresden
- DOI
- https://doi.org/10.1038/s41408-021-00558-5
- Journal volume & issue
-
Vol. 11,
no. 10
pp. 1 – 8
Abstract
Abstract To investigate the efficacy and toxicities of CPX-351 outside a clinical trial, we analyzed 188 patients (median age 65 years, range 26–80) treated for therapy-related acute myeloid leukemia (t-AML, 29%) or AML with myelodysplasia-related changes (AML-MRC, 70%). Eighty-six percent received one, 14% two induction cycles, and 10% received consolidation (representing 22% of patients with CR/CRi) with CPX-351. Following induction, CR/CRi rate was 47% including 64% of patients with available information achieving measurable residual disease (MRD) negativity (<10−3) as measured by flow cytometry. After a median follow-up of 9.3 months, median overall survival (OS) was 21 months and 1-year OS rate 64%. In multivariate analysis, complex karyotype predicted lower response (p = 0.0001), while pretreatment with hypomethylating agents (p = 0.02) and adverse European LeukemiaNet 2017 genetic risk (p < 0.0001) were associated with lower OS. Allogeneic hematopoietic cell transplantation (allo-HCT) was performed in 116 patients (62%) resulting in promising outcome (median survival not reached, 1-year OS 73%), especially in MRD-negative patients (p = 0.048). With 69% of patients developing grade III/IV non-hematologic toxicity following induction and a day 30-mortality of 8% the safety profile was consistent with previous findings. These real-world data confirm CPX-351 as efficient treatment for these high-risk AML patients facilitating allo-HCT in many patients with promising outcome after transplantation.
