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Navigating ‘grey areas’ and challenges during evaluation of transplant eligibility in specific myelofibrosis populations: a perspective on behalf of the Chronic Malignancies Working Party of the EBMT

Bone Marrow Transplantation volume 60, pages 10–18 (2025)Cite this article

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Abstract

Significant efforts have been made to effectively select myelofibrosis (MF) patients who can benefit from allogeneic hematopoietic cell transplantation (allo-HCT), the only current cure for MF. The recent EBMT/ELN 2024 recommendations offer valuable guidance for hematologists and transplant physicians. However, several grey areas remain in day-to-day clinical practice regarding the feasibility and optimal preparation for transplantation in patients with this disease. Effective spleen size reduction, often achieved with JAK inhibitors, appears crucial for transplant success. For resistant cases, switching JAK inhibitors, splenectomy, or spleen irradiation may be considered, taking into account patient profiles, treatment availability and center preferences. Managing splanchnic vein thromboses, portal, and pulmonary hypertension is critical as these conditions may affect transplant outcomes. Cytopenias, particularly transfusion-dependent anemia and thrombocytopenia, complicate treatment and impact on outcomes, though new drugs show promise. Comorbidities play a significant role and tools like the Hematopoietic Cell Transplantation-Comorbidity Index (HCT-CI) and frailty assessments are useful for evaluating transplant risks while allowing the implementation of corrective measures. Especially in low- and medium-income countries where access to novel therapies may be challenging, allo-HCT still represents an attractive therapeutic option for MF. Future directions include integrating new therapeutics into the transplant algorithm and leveraging artificial intelligence for more informed risk assessment, highlighting the need for tailored approaches to improve allo-HCT outcomes in such a setting.

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Fig. 1: Transplant outcomes for myelofibrosis patients in Brazil.
Fig. 2: Comprehensive assessment of myelofibrosis patients for transplant.

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Acknowledgements

We would like to thank Juan Carlos García Pagán (Hepatology Unit, Hospital Clínic, Barcelona) and Enrique Santas (Cardiology Department, Hospital Clínico, Valencia) for kindly reviewing the present manuscript.

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Author notes

  1. These authors contributed equally: Nicola Polverelli, Juan Carlos Hernández-Boluda, Nico Gagelmann, Carmelo Gurnari, Michele Malagola, Fernando Barroso Duarte, Vaneuza A. M. Funke, Caterina Zerbi, Donal P. McLornan.

Authors and Affiliations

  1. Unit of Bone Marrow Transplantation and Cellular Therapies - Division of Hematology, Fondazione IRCCS Policlinico San Matteo, Pavia, Italy

    Nicola Polverelli & Caterina Zerbi

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

    Juan Carlos Hernández-Boluda

  3. Department of Stem Cell Transplantation, University Medical Center Hamburg-Eppendorf, Hamburg, Germany

    Nico Gagelmann

  4. Department of Biomedicine and Prevention, University of Rome Tor Vergata, Rome, Italy

    Carmelo Gurnari

  5. Translational Hematology and Oncology Research Department, Taussig Cancer Center, Cleveland Clinic, Cleveland, OH, USA

    Carmelo Gurnari

  6. Chair of Hematology, Department of Clinical and Experimental Sciences, University of Brescia, Unit of Blood Disease and Stem Cell Transplantation, ASST Spedali Civili, Brescia, Italy

    Michele Malagola

  7. Hospital Universitario Walter Cantídio, Universidade Federal do Ceara, Fortaleza, Brazil

    Fernando Barroso Duarte

  8. Blood and Marrow Transplantation Programme, Hospital de Clínicas, Federal University of Parana, Curitiba, Paraná, Brazil

    Vaneuza A. M. Funke

  9. Hospital Nossa Senhora das Graças, Curitiba, Brazil

    Vaneuza A. M. Funke

  10. Department of Haematology and Stem Cell transplantation, University College London Hospitals NHS Foundation Trust, London, UK

    Donal P. McLornan

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  1. Nicola Polverelli
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NP, JCHB, NC, CG, MM, FBD, VAM, CZ, DPM wrote and approved the final version of the manuscript.

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Correspondence to Nicola Polverelli or Donal P. McLornan.

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Polverelli, N., Hernández-Boluda, J.C., Gagelmann, N. et al. Navigating ‘grey areas’ and challenges during evaluation of transplant eligibility in specific myelofibrosis populations: a perspective on behalf of the Chronic Malignancies Working Party of the EBMT. Bone Marrow Transplant 60, 10–18 (2025). https://doi.org/10.1038/s41409-024-02437-6

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