Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
. 2025 Sep;39(9):2266-2269.
doi: 10.1038/s41375-025-02675-7. Epub 2025 Jul 3.

Induction chemotherapy with CPX-351 in acute myeloid leukemia: revisiting the role of early bone marrow assessment

Julian Ronnacker  1 , Leo Ruhnke  2 , Christoph Röllig  2 , Jan Moritz Middeke  2 , Regina Herbst  3 , Anke Morgner  3 , Julia M Unglaub  4 , Tim Sauer  4 , Karin Huber  5 , David Baden  5 , Lars Fransecky  5 , Melanie Nogueira Gezer  6 , Martina Crysandt  6 , Edgar Jost  6 , Madlen Jentzsch  7 , Klaus H Metzeler  7 , Andrew F Berdel  1 , Marc-André Urbahn  1 , Lina Kolloch  1 , Matthias Stelljes  1 , Uwe Platzbecker  7 , Tim H Brümmendorf  6 , Claudia Baldus  5 , Carsten Müller-Tidow  4 , Mathias Hänel  3 , Martin Bornhäuser  2 , Klaus Wethmar  1 , Utz Krug  8 , Georg Lenz  1 , Jan-Henrik Mikesch  1 , Christoph Schliemann  9
Affiliations

Induction chemotherapy with CPX-351 in acute myeloid leukemia: revisiting the role of early bone marrow assessment

Julian Ronnacker et al. Leukemia. 2025 Sep.
No abstract available

PubMed Disclaimer

Conflict of interest statement

Competing interests: CR received honoraria from AbbVie, Astellas, Bristol-Meyer-Squibb, Daiichi Sankyo, Jazz, Janssen, Novartis, Otsuka, Pfizer, Servier, and institutional research funding from AbbVie, Astellas, Novartis, Pfizer. JMM has served as a consultant for Janssen, Roche, Gilead, Abbvie, JAZZ Pharmaceuticals, Pfizer, Astellas, Novartis, AstraZeneca, Beigene, Glycostem; is a shareholder of Cancilico and Synagen; has received research funding from Servier, Janssen, and Novartis; has received travel support from Beigene. JMU received travel grants from Jazz Pharmaceuticals and AbbVie. TS received honoraria and travel support from Jazz Pharmaceuticals and is part of the advisory board of Jazz Pharmaceuticals. MJ received honoraria from Jazz Pharmaceuticals. AFB received speaker honoraria and travel grant from AbbVie. MH received honoraria from Novartis, Gilead, the Falk Foundation, SOBI, and BMS. MH is consultant for Janssen, Kite, Amgen, Sanofi, BMS, and BeiGene. MH received travel support from Abbvie. MS received grants not related to this manuscript from Pfizer. MS received honoraria not related to this manuscript from Pfizer, Jazz, Amgen, Novartis, Gilead, Celgene, medac, BMS, MSD, Incyte. MB received lecture fees and honoraria for advisory board participation from Jazz Pharmaceuticals and served on advisory boards for Acitrexx and MSD. GL received research grants not related to this manuscript from AGIOS, AQUINOX, AstraZeneca, Bayer, Celgene, Gilead, Janssen, MorphoSys, Novartis, F. Hoffmann-La Roche Ltd, and Verastem. GL received honoraria from ADC Therapeutics, AbbVie, Amgen, AstraZeneca, Bayer, BeiGene, BMS, Celgene, Constellation, Genase, Genmab, Gilead, Hexal/Sandoz, Immagene, Incyte, Janssen, Karyopharm, Lilly, Miltenyi, MorphoSys, MSD, NanoString, Novartis, PentixaPharm, Pierre Fabre, F. Hoffmann-La Roche Ltd, and Sobi. CS received institutional research support not related to this manuscript from Jazz Pharmaceuticals. CS received honoraria not related to this manuscript from AbbVie, Astellas, AstraZeneca, BMS, Laboratories Delbert, Jazz Pharmaceuticals, Novartis, Otsuka, Pfizer, Roche, and travel grants from AbbVie, BMS, Jazz Pharmaceuticals, Pfizer. Ethical approval and consent to participate: All described methods were performed in accordance with the relevant guidelines and regulations. Written informed consent was obtained from participants for inclusion in the clinical AML registry of the SAL (NCT03188874). The study was approved by the local Ethics Committee of Westphalia-Lippe (AZ 2023-032-f-S) as well as by the institutional review board of the SAL.

Figures

Fig. 1
Fig. 1. Cut-off definition and outcome by early bone marrow response in patients receiving CPX-351 induction.
The predictive value of early response assessment was evaluated by receiver operating characteristics, calculating the AUC and the Youden index. A cut-off of 5% BM blasts was identified as the optimal threshold for predicting induction response (A). Patients who failed to achieve early blast clearance (BM blast count <5%) were at higher risk of IF (B). Sankey diagram visualizing patient trajectories between early and definitive response assessment (C). OS did not differ between patients with and without early blast clearance (D) but differed significantly when stratified by definitive response after one course of CPX-351 (E). AUC area under the curve, CI confidence interval, CR complete remission, CRi CR with incomplete hematologic recovery, HR hazard ratio, MLFS morphologic leukemia-free state, ROC receiver operating characteristics.
See this image and copyright information in PMC

References

    1. Khoury JD, Solary E, Abla O, Akkari Y, Alaggio R, Apperley JF, et al. The 5th edition of the World Health Organization Classification of Haematolymphoid Tumours: Myeloid and Histiocytic/Dendritic Neoplasms. Leukemia. 2022;36:1703–19. - PMC - 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. J Clin Oncol. 2018;36:2684–92. - PMC - PubMed
    1. Guolo F, Fianchi L, Martelli MP, Chiusolo P, Lussana F, Grimaldi F, et al. Optimal duration of CPX-351 treatment and best timing for consolidation with allogeneic stem cell transplantation: evidence from a large real-world Italian study. Blood. 2023;142:731.
    1. Mehta P, Campbell V, Maddox J, Floisand Y, Kalakonda AJM, O’Nions J, et al. CREST-UK: Real-world effectiveness, safety and outpatient delivery of CPX-351 for first-line treatment of newly diagnosed therapy-related AML and AML with myelodysplasia-related changes in the UK. Br J Haematol. 2024;205:1326–36. - PubMed
    1. Benitez LL, Perissinotti AJ, Rausch CR, Klaus J, Clark SM, Filtz M, et al. Multicenter comparison of high-dose cytarabine-based regimens versus liposomal daunorubicin and cytarabine (CPX-351) in patients with secondary acute myeloid leukemia. Leuk Lymphoma. 2021;62:2184–92. - PubMed