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Treosulfan compared to busulfan in allogeneic haematopoietic stem cell transplantation for myelofibrosis: a registry-based study from the Chronic Malignancies Working Party of the EBMT

Bone Marrow Transplantation (2024)Cite this article

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Abstract

We aimed to compare outcomes following treosulfan (TREO) or busulfan (BU) conditioning in a large cohort of myelofibrosis (MF) patients from the EBMT registry. A total of 530 patients were included; 73 received TREO and 457 BU (BU ≤ 6.4 mg/kg in 134, considered RIC, BU > 6.4 mg/kg in 323 considered higher dose (HD)). Groups were compared using adjusted Cox models. Cumulative incidences of engraftment and acute GVHD were similar across the 3 groups. The TREO group had significantly better OS than BU-HD (HR:0.61, 95% CI: 0.39–0.93) and a trend towards better OS over BU-RIC (HR: 0.66, 95% CI: 0.41–1.05). Moreover, the TREO cohort had a significantly better Progression-Free-Survival (PFS) than both the BU-HD (HR: 0.57, 95% CI: 0.38–0.84) and BU-RIC (HR: 0.60, 95% CI: 0.39–0.91) cohorts, which had similar PFS estimates. Non-relapse mortality (NRM) was reduced in the TREO and BU-RIC cohorts (HR: 0.44, 95% CI: 0.24–0.80 TREO vs BU-HD; HR: 0.54, 95% CI: 0.28–1.04 TREO vs BU-RIC). Of note, relapse risk did not significantly differ across the three groups. In summary, within the limits of a registry-based study, TREO conditioning may improve PFS in MF HSCT and have lower NRM than BU-HD with a similar relapse risk to BU-RIC. Prospective studies are needed to confirm these findings.

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Fig. 1: Outcome according to conditioning regimen.

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Data are available on request to the corresponding author.

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Acknowledgements

All participating centers and patients who gave their consent for the registry are thanked.

Author information

Authors and Affiliations

  1. Hôpital Saint-Louis, APHP, Université de Paris Cité, Paris, France

    Marie Robin

  2. Deptartment of Biology, University of Rome Tor Vergata, Rome, Italy

    Simona Iacobelli

  3. EBMT Leiden Study Unit, Leiden, The Netherlands

    Linda Koster

  4. University Hospital | Basel, Basel, Switzerland

    Jakob Passweg

  5. King’s College Hospital, London, UK

    Daniele Avenoso

  6. University Hospital of Wales, Cardiff, UK

    Keith M. O. Wilson

  7. HUCH Comprehensive Cancer Center, Helsinki, Finland

    Urpu Salmenniemi

  8. University of Heidelberg, Heidelberg, Germany

    Peter Dreger

  9. Leiden University Hospital, Leiden, The Netherlands

    Peter von dem Borne

  10. Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    John A. Snowden

  11. Bristol Royal Hospital for Children, Bristol, UK

    Stephen Robinson

  12. ASST Papa Giovanni XXIII, Bergamo, Italy

    Maria Chiara Finazzi

  13. University Hospital | Essen, Duesseldorf, Germany

    Thomas Schroeder

  14. Northern Centre for Bone Marrow Transplantation, Newcastle, England

    Matthew Collin

  15. Hannover Medical School, Hannover, Germany

    Matthias Eder

  16. Service d’Hématologie Clinique et Thérapie Cellulaire, CHU Bordeaux, F-33000, Bordeaux, France

    Edouard Forcade

  17. CHU Nice—Hôpital de l’ARCHET, Nice, France

    Michael Loschi

  18. Department of Oncology/Hematology, IRCCS Humanitas Research Hospital, via Manzoni 56, Rozzano, 20089, Milan, Italy

    Stefania Bramanti

  19. Instituto de Biomedicina de Sevilla IBIS, CSIC, Hospital Universitario Virgen del Rocío Universidad de Sevilla, Sevilla, Spain

    Jose Antonio Pérez-Simón

  20. Maria Skłodowska-Curie National Research Institute of Oncology, Gliwice, Poland

    Tomasz Czerw

  21. Unit of Blood Diseases and Stem Cell Transplantation, University of Brescia—ASST Spedali Civili, Brescia, Italy

    Nicola Polverelli

  22. Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Nicola Polverelli

  23. University Clinical Centre Medical University of Warsaw, Warsaw, Poland

    Joanna Drozd-Sokolowska

  24. University College London Hospitals NHS Trust, London, UK

    Kavita Raj

  25. Department of Hematology, Hospital Clínico Universitario, Valencia, Spain

    Juan Carlos Hernández-Boluda

  26. University College Hospital London, London, UK

    Donal P. McLornan

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  1. Marie Robin
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Contributions

MR initiated and proposed the study to the CMWP, MR, TC, NP, JDS, KR, JCHD, and DML were involved in the study design, SI made the statistics, LK made data management, MR, JP, AD, KW, US, PD, PVDB, SR, TCl, SB, JAPS recruited patients and checked the data, all co-authors approved the paper and contributed to the paper.

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Correspondence to Marie Robin.

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Robin, M., Iacobelli, S., Koster, L. et al. Treosulfan compared to busulfan in allogeneic haematopoietic stem cell transplantation for myelofibrosis: a registry-based study from the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02269-4

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