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Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group

Leukemia volume 29, pages 2126–2133 (2015)Cite this article

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Abstract

The aim of this work is to produce recommendations on the management of allogeneic stem cell transplantation (allo-SCT) in primary myelofibrosis (PMF). A comprehensive systematic review of articles released from 1999 to 2015 (January) was used as a source of scientific evidence. Recommendations were produced using a Delphi process involving a panel of 23 experts appointed by the European LeukemiaNet and European Blood and Marrow Transplantation Group. Key questions included patient selection, donor selection, pre-transplant management, conditioning regimen, post-transplant management, prevention and management of relapse after transplant. Patients with intermediate-2- or high-risk disease and age <70 years should be considered as candidates for allo-SCT. Patients with intermediate-1-risk disease and age <65 years should be considered as candidates if they present with either refractory, transfusion-dependent anemia, or a percentage of blasts in peripheral blood (PB) >2%, or adverse cytogenetics. Pre-transplant splenectomy should be decided on a case by case basis. Patients with intermediate-2- or high-risk disease lacking an human leukocyte antigen (HLA)-matched sibling or unrelated donor, should be enrolled in a protocol using HLA non-identical donors. PB was considered the most appropriate source of hematopoietic stem cells for HLA-matched sibling and unrelated donor transplants. The optimal intensity of the conditioning regimen still needs to be defined. Strategies such as discontinuation of immune-suppressive drugs, donor lymphocyte infusion or both were deemed appropriate to avoid clinical relapse. In conclusion, we provided consensus-based recommendations aimed to optimize allo-SCT in PMF. Unmet clinical needs were highlighted.

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Authors and Affiliations

  1. Department of Stem Cell Transplantation, University Hospital, Hamburg, Germany

    N M Kröger & H Alchalby

  2. Clinical Research Division, Fred Hutchinson Cancer Research Center, University of Washington School of Medicine, Seattle, WA, USA

    J H Deeg

  3. Department of Hematology, Hospital of Navarra, Navarra Health Service, Pamplona, Spain

    E Olavarria

  4. Department of Haematology, Clinical Oncology and Hemostasiology, University of Leipzig, Leipzig, Germany

    D Niederwieser

  5. Ospedale San Martino, Genoa, Italy

    A Bacigalupo

  6. Research Foundation, Ospedale Papa Giovanni XXIII, Bergamo, Italy

    T Barbui

  7. Hematology and Bone Marrow Transplant Unit, Ospedale Papa Giovanni XXIII, Bergamo, Italy

    A Rambaldi

  8. Division of Hematology and Oncology, Mayo Clinic, Scottsdale, AZ, USA

    R Mesa

  9. Division of Hematology, Department of Medicine, Mayo Clinic, Rochester, MN, USA

    A Tefferi

  10. Academic Department of Hematology and Oncology, Johannes Wesling Teaching Hospital, Hans-Nolte-Strasse 1, Minden, Germany

    M Griesshammer

  11. Princess Margaret Cancer Center, University of Toronto, Toronto, ON, Canada

    V Gupta

  12. Department of Haematology Guy's and St Thomas' NHS Foundation Trust, London, UK

    C Harrison & D McLornan

  13. Department of Experimental and Clinical Medicine, University of Florence, Florence, Italy

    A M Vannucchi

  14. Hospital Clínic, IDIBAPS, University of Barcelona, Barcelona, Spain

    F Cervantes

  15. Department of Hematology and Bone Marrow Transplantation, Saint Louis Hospital, Paris, France

    M Robin

  16. Department of Bone Marrow Transplantation, WTZ, Comprehensive Cancer Centre, University Hospital Essen, Essen, Germany

    M Ditschkowski

  17. Mayo Clinic Cancer Center, Scottsdale, AZ, USA

    V Fauble

  18. Department of Haematological Medicine, King’s College Hospital, London, UK

    D McLornan

  19. Division of Hematology/Oncology, Massachusetts General Hospital, Boston, MA, USA

    K Ballen

  20. Department of Stem Cell Transplantation and Cellular Therapy, The University of Texas MD Anderson Cancer Center, Houston, TX, USA

    U R Popat

  21. Division of Hematology, Department of Medicine, University Hospital Ospedale di Circolo e Fondazione Macchi, Varese, Italy

    F Passamonti

  22. University of Illinois Hospital & Health Sciences System and University of Illinois Cancer Center, Chicago, IL, USA

    D Rondelli

  23. Center for the Study of Myelofibrosis, IRCCS Policlinico S. Matteo Foundation,, Pavia, Italy

    G Barosi

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  1. N M Kröger
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Correspondence to G Barosi.

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NMK declares speakers’ bureau and research funding from Sanofi and Novartis; FC declares consulting and speakers’ bureau from Novartis, AOP and Baxter; DM declares honoraria, speakers’ bureau and research funding from Novartis; AR declares consulting from Piere Fabre Pharma, and Travels from Piere Fabre Pharma and Novartis; EO declares travel expenses for Novartis, Bristol Meyers Squibb and Sanofi; CH declares honoraria and speakers’ bureau from Novartis, Sanofi and Shire. GB declares consulting from Celgene; DN declares speakers’ bureau and travels from Novartis; TB declares consulting, speakers’ bureau from Novartis and Shire; RM declares honoraria and consulting from Novartis, and funding from Incyte, Gilead, Genentech; VG declares honoraria, consulting, and research funding from Novartis and Incyte; AMV declares consulting and speakers’ bureau from Novartis; MD declares consulting from MSD Sharp and Dohme, and research funding from Biosciences; URP declares research funding from CTI Pharma and Otsuka; DR declares consulting from Incyte, and speakers’ bureau from Sanofi. The remaining authors declare no conflict of interest.

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NMK and GB designed the research; GB did the systematic review of literature; NMK, JHD, EO, AB and DN wrote the first draft of the recommendations; TB, AR, RM, AT, MG, VG, CH, HA, AMV, FC, MR, MD, VF, DM, KB, URP, FP and DR criticized the first draft of the recommendations and provided important input in the text of the recommendations; GB wrote the first draft of the manuscript; and all the authors approved the final version of the manuscript.

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Kröger, N., Deeg, J., Olavarria, E. et al. Indication and management of allogeneic stem cell transplantation in primary myelofibrosis: a consensus process by an EBMT/ELN international working group. Leukemia 29, 2126–2133 (2015). https://doi.org/10.1038/leu.2015.233

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