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Transplantation for myelofibrosis patients in the ruxolitinib era: a registry study from the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire

Bone Marrow Transplantation (2024)Cite this article

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Abstract

In this SFGM-TC registry study, we report the results after stem cell transplantation (HSCT) in 305 myelofibrosis patients, in order to determine potential risk factors associated with outcomes, especially regarding previous treatment with ruxolitinib. A total of 102 patients were transplanted from an HLA-matched-sibling donor (MSD), and 143 patients received ruxolitinib. In contrast with previous studies, our results showed significantly worse outcomes for ruxolitinib patients regarding overall survival (OS) and non-relapse mortality (NRM), especially in the context of unrelated donors (URD). When exploring reasons for potential confounders regarding the ruxolitinib effect, an interaction between the type of donor and the use of ATG was found, therefore subsequent analyses were performed separately for each type of donor. Multivariable analyses did not confirm a significant negative impact of ruxolitinib in transplantation outcomes. In the setting of URD, only age and Fludarabine-Melphalan (FM) conditioning were associated with increased NRM. For MSD, only Karnoksfy <70% was associated with reduced OS. However, a propensity score analysis showed that ruxolitinib had a negative impact on OS but only in non-responding patients, consistent with previous data. To conclude, with all the precautions due to confounders and bias, ruxolitinib itself does not appear to increase mortality after HSCT.

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Fig. 1: Incidence of acute GVHD grade 3–4.
Fig. 2: Outcomes according to the type of donor and ruxolitinib before transplantation.

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Data availability

The data that support the findings of this study are available via SFGM-TC. These data are available upon reasonable request and with the permission of SFGM-TC.

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Acknowledgements

The authors acknowledge all centers, investigators, data managers, patients, and their families for their participation in this study, and all the SFGM-TC members.

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Authors and Affiliations

  1. Service d’hématologie - greffe, Hôpital Saint Louis, APHP, Université de Paris, Paris, France

    Sara Villar & Marie Robin

  2. Hematology Department, Clínica Universidad de Navarra, Pamplona, Spain

    Sara Villar

  3. APHP, Saint-Louis University Hospital, Department of Biostatistics, Paris, France

    Sylvie Chevret

  4. Cliniques Universitaires St-Luc, Brussels, Brussels, Belgium

    Xavier Poire

  5. Hematology Department, CHU Amiens, Amiens, France

    Magalie Joris

  6. Hematology Department, Nantes University Hospital, Nantes, France

    Patrice Chevallier

  7. Hematology Department, Institut Gustave Roussy, Villejuif, France

    Jean-Henri Bourhis

  8. Service d’hématologie et thérapie Cellulaire, CHU Bordeaux, Hôpital Haut-Leveque, Pessac, France

    Edouard Forcade

  9. Hematology Department, Caen University Hospital, Caen, France

    Sylvain Chantepie

  10. Hematology Department, CHU de Lille, Lille, France

    David Beauvais

  11. SFGM-TC, Lyon, France

    Nicole Raus

  12. Department of Clinical Hematology and Cellular Therapy, CHU de Clermont-Ferrand, Site Estaing, Clermont-Ferrand, France

    Jacques-Olivier Bay

  13. Hematology Department, Cote D’Azur University, CHU of Nice, Nice, France

    Michael Loschi

  14. Hematology Department, Institut Paoli Calmettes, Marseille, France

    Raynier Devillier

  15. Department of Clinical Hematology and Cellular Therapy, Centre de Recherche Saint-Antoine (CRSA), Saint-Antoine Hospital, Assistance Publique - Hôpitaux de Paris, INSERM UMRs 938 Sorbonne University, Paris, France

    Remy Duléry

  16. Hematology Department, Montpellier University Hospital, Montpellier, France, Montpellier, France

    Patrice Ceballos

  17. Hematology Department, CHRU Brabois, Nancy, France

    Marie Thérèse Rubio

  18. CNRS UMR 7365, Équipe 6, Biopôle de L’Université de Lorraine, Vandoeuvre Les Nancy, France

    Marie Thérèse Rubio

  19. Hematology Department, CHU Liege, University of Liege, Liege, Belgium

    Sophie Servais

  20. Hematology Department, Pitié Salpêtrière Hospital, AP-HP, Paris, France

    Stephanie Nguyen

Authors
  1. Sara Villar
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  2. Sylvie Chevret
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  3. Xavier Poire
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  4. Magalie Joris
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  6. Jean-Henri Bourhis
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  8. Sylvain Chantepie
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  9. David Beauvais
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  11. Jacques-Olivier Bay
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  12. Michael Loschi
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  15. Patrice Ceballos
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  16. Marie Thérèse Rubio
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  17. Sophie Servais
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  18. Stephanie Nguyen
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  19. Marie Robin
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Contributions

MR and SV wrote the manuscript. SC performed the statistical analysis. XP, AC, PC, JHB, EF, SC, DB, JOB, ML, RD, RDu, PCe, MTR, YB, and SN recruited the patients. NR has done data management. All authors reviewed and approved the final manuscript.

Corresponding author

Correspondence to Marie Robin.

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Competing interests

The authors declare no competing interests in relationship with the current study. MR: research support by Novartis, MEDAC, NEOVII, ASTEX, ABBVIE. RD: research funding from Ligue contre le Cancer, Arthur Sachs, Monahan Foundation, Servier Foundation, Philippe Foundation, DCP AP-HP, honoraria from Novartis and Takeda, and non-financial support from Kite Pharma/Gilead, all outside the submitted work.

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Villar, S., Chevret, S., Poire, X. et al. Transplantation for myelofibrosis patients in the ruxolitinib era: a registry study from the Société Francophone de Greffe de Moelle et de Thérapie Cellulaire. Bone Marrow Transplant (2024). https://doi.org/10.1038/s41409-024-02268-5

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