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Evaluating the clinical effectiveness of autologous haematopoietic stem cell transplantation versus disease-modifying therapy in multiple sclerosis using a matching-adjusted indirect comparison: an exploratory study from the Autoimmune Diseases Working Party (ADWP) of the European Society of Bone and Marrow Transplantation (EBMT)

Bone Marrow Transplantation volume 55, pages 1473–1475 (2020)Cite this article

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Across many health services, there is a need to demonstrate that health care technologies are clinically effective and cost-effective in order for them to be recommended for routine use. Whilst a number of disease-modifying therapies (DMTs) are available for the treatment of relapsing/remitting multiple sclerosis (RRMS), the effectiveness of most of these therapies in delaying or preventing long-term disease progression is limited. The need for ongoing treatment using DMTs leads to a significant treatment burden for patients as well as considerable costs for the health service.

Autologous haematopoietic stem cell transplantation (aHSCT) is an alternative effective treatment approach for well selected patients with RRMS [1, 2]. We previously undertook an economic analysis that compared aHSCT versus mitoxantrone in patients with secondary progressive multiple sclerosis. In this setting, aHSCT would at best be expected to reduce, but not reverse, the progression of disability (as reflected by the Expanded Disability Status Scale [EDSS]) [3]. Whilst subject to uncertainty, the results of this analysis suggested that aHSCT has the potential to generate additional health gains at an acceptable cost to the UK National Health Service. However, there is now increasing recognition that aHSCT is best applied at an earlier stage in patients with highly active RRMS, where there is the potential for significant reversal of disability (i.e., improvement of EDSS scores) and this has been reflected by trends in aHSCT practice [4].

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Acknowledgements

This study was undertaken without research funding. JAS declares speaker fees from Sanofi, Jazz, Janssen, Mallinckrodt and Gilead and IDMC membership for Kiadis Pharma. This research was supported by the NIHR Sheffield Biomedical Research Centre/NIHR Sheffield Clinical Research Facility.

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Authors and Affiliations

  1. School of Health and Related Research, University of Sheffield, Sheffield, UK

    P. Tappenden, Y. Wang & I. Bermejo

  2. Academic Department of Neuroscience, Sheffield INHR BRC, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK

    B. Sharrack

  3. Department of Neuroscience, Uppsala University, Uppsala, Sweden

    J. Burman

  4. Kings Health Partners, Department of Haematology, Guys Hospital, London, UK

    M. Kazmi

  5. Haematology Department, Careggi University Hospital, Florence, Italy

    R. Saccardi

  6. Cabourn Statistics Ltd, Manchester, UK

    R. Harvey

  7. EBMT Paris study office / CEREST-TC—Department of Haematology, Saint Antoine Hospital—INSERM UMR 938—Université Pierre et Marie Curie, Paris, France

    M. Badoglio

  8. Assistance Publique-Hôpitaux de Paris, Saint-Louis Hospital, Autoimmune and Vascular Disease Unit, Internal Medicine (UF04), Center of Reference for Rare Systemic Autoimmune Diseases (FAI2R), Université de Paris, EA 3518, Paris, France

    D. Farge

  9. Department of Medicine, McGill University, Montreal, QC, Canada

    D. Farge

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

    J. A. Snowden

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  1. P. Tappenden
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Correspondence to P. Tappenden.

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Tappenden, P., Wang, Y., Sharrack, B. et al. Evaluating the clinical effectiveness of autologous haematopoietic stem cell transplantation versus disease-modifying therapy in multiple sclerosis using a matching-adjusted indirect comparison: an exploratory study from the Autoimmune Diseases Working Party (ADWP) of the European Society of Bone and Marrow Transplantation (EBMT). Bone Marrow Transplant 55, 1473–1475 (2020). https://doi.org/10.1038/s41409-019-0747-2

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