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Autologous haematopoietic stem cell transplantation (HSCT) for anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis: a retrospective survey of patients reported to European Society for Blood and Marrow Transplantation (EBMT) registry

Bone Marrow Transplantation volume 55, pages 1512–1515 (2020)Cite this article

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Anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis (AAV) represents a heterogeneous group of rare and potentially life-threatening multisystem autoimmune diseases (ADs), characterised by the development of necrotising inflammation of small blood vessels and the presence of ANCA with specificity for proteinase-3 or myeloperoxidase that share clinical manifestations, such as renal, respiratory, ocular and neurologic involvement [1]. The recent introduction of targeted biologic disease-modifying drugs (DMARDs), such as rituximab, provides significant clinical benefit [2], but relapses are common [3, 4] and AAV may adversely affect the quality of life even in patients in remission [5]. In this group of patients, autologous haematopoietic stem cell transplantation (HSCT) has been an occasional treatment modality and regarded as a ‘clinical option’ in EBMT recommendations in patients after failure of at least two lines of conventional treatment [6]. However, published clinical evidence is restricted to case reports and retrospective registry-based analyses, previously indicating that HSCT may be associated with beneficial clinical responses [7, 8].

In order to assess the current use and long-term outcome of HSCT in AAV, we retrospectively surveyed adult cases routinely reported to the EBMT registry between 1998 and 2018. All patients had provided written consent for transmission of their anonymised data in accordance with local practices and EBMT registry function. A specifically designed questionnaire was completed by each referring transplant centre to obtain relevant retrospective clinical and laboratory data. Primary endpoint was treatment response according to criteria provided in Supplementary Table 1. Other endpoints included overall survival (OS), progression-free survival (PFS) and relapse incidence (RI).

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Acknowledgements

We thank centres and their data managers who contributed to this study. We are indebted to the patients in this study and the multidisciplinary teams coordinating their care.

Author information

Author notes

  1. These authors contributed equally: Tobias Alexander, Clare Samuelson

Authors and Affiliations

  1. Department of Rheumatology and Clinical Immunology, University Medicine Berlin, Berlin, Germany

    Tobias Alexander

  2. Deutsches RheumaForschungszentrum Berlin - a Leibniz Institute, Berlin, Germany

    Tobias Alexander

  3. Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    Clare Samuelson & John A. Snowden

  4. Department of Rheumatology, University Hospital Basel, Basel, Switzerland

    Thomas Daikeler

  5. Department for Internal Medicine II (Oncology, Hematology, Rheumatology and Immunology), University Hospital Tuebingen, Tübingen, Germany

    Jörg Henes

  6. Department of Rheumatology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    Mohammed Akil

  7. Department of Hematology, University Hospital Umea, Umea, Sweden

    Lars Skagerlind

  8. Medizinische Klinik und Poliklinik I, Technische Universität Dresden, Dresden, Germany

    Gerhard Ehninger

  9. Department of Internal Medicine, University Hospital Kuopio, Kuopio, Finland

    Esa Jantunen

  10. University of Eastern Finland, Joensuu, Finland

    Esa Jantunen

  11. Institute of Clinical Medicine, Department of Medicine, Kuopio University Hospital, Joensuu, Finland

    Esa Jantunen

  12. Department of Medicine - North Carelia Hospital District, Joensuu, Finland

    Esa Jantunen

  13. EBMT Paris study office; Department of Haematology, Saint Antoine Hospital; INSERM UMR 938, Sorbonne University, Paris, France, Paris, France

    Manuela Badoglio

  14. Unité de Médecine Interne, Maladies Auto-immunes et Pathologie Vasculaire (UF 04), Hôpital St-Louis, AP-HP, Paris 6, 1 avenue Claude Vellefaux, Paris, France

    Dominique Farge

  15. Centre de Référence des Maladies auto-immunes systémiques Rares, Filière FAI2R, d’Ile-de-France, MATHEC St-Louis, Paris, France

    Dominique Farge

  16. EA 3518, Université de Paris, Paris, France

    Dominique Farge

  17. Department of Internal Medicine, McGill University, Montréal, QC, Canada

    Dominique Farge

Authors
  1. Tobias Alexander
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  2. Clare Samuelson
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  3. Thomas Daikeler
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  4. Jörg Henes
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  5. Mohammed Akil
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  6. Lars Skagerlind
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  7. Gerhard Ehninger
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  8. Esa Jantunen
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  9. Manuela Badoglio
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  10. Dominique Farge
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  11. John A. Snowden
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Consortia

on behalf of the EBMT Autoimmune Diseases Working Party

Contributions

TA, CS, TD, JH, DF and JAS designed and coordinated the study. MB was responsible for data input. MB, TA and CS collected data. TA, CS, TD and JAS co-wrote the manuscript. All authors reviewed and revised the manuscript and approved the final version.

Corresponding author

Correspondence to Tobias Alexander.

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Conflict of interest

JAS declares speaker fees from Sanofi, Jazz, Janssen, Mallinckrodt and Gilead. TA received travel grants from Neovii. The other authors declare that they have no conflict of interest.

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Alexander, T., Samuelson, C., Daikeler, T. et al. Autologous haematopoietic stem cell transplantation (HSCT) for anti-neutrophil cytoplasmic antibody (ANCA)-associated vasculitis: a retrospective survey of patients reported to European Society for Blood and Marrow Transplantation (EBMT) registry. Bone Marrow Transplant 55, 1512–1515 (2020). https://doi.org/10.1038/s41409-019-0763-2

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