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Autologous haematopoietic stem cell transplantation for immune-mediated neurological diseases: what, how, who and why?
  1. Gavin Brittain1,2,
  2. Alasdair J Coles3,
  3. G Giovannoni4,
  4. Paolo Antonio Muraro5,
  5. Jacqueline Palace6,
  6. Jennifer Petrie7,
  7. Elisa Roldan8,
  8. N J Scolding9,10,
  9. John A Snowden8,11,
  10. Basil Sharrack1,2
  1. 1 Department of Clinical Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
  2. 2 Sheffield Institute for Translational Neuroscience, The University of Sheffield, Sheffield, UK
  3. 3 Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK
  4. 4 Neuroscience and Trauma, Blizard Institute of Cell and Molecular Science, London, UK
  5. 5 Department of Brain Sciences, Imperial College London, London, UK
  6. 6 Department of Clinical Neurology, University of Oxford, Oxford, UK
  7. 7 Clinical Trials Research Unit, The University of Sheffield, Sheffield, UK
  8. 8 Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
  9. 9 Institute of Clinical Neurosciences, University of Bristol, Bristol, UK
  10. 10 Department of Neurology, Gloucestershire Royal Hospital, Gloucester, UK
  11. 11 Department of Oncology and Metabolism, The University of Sheffield, Sheffield, UK
  1. Correspondence to Dr Gavin Brittain, Department of Clinical Neurology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, S10 2JF, UK; gavin.brittain{at}sheffield.ac.uk

Abstract

In carefully selected patients, autologous haematopoietic stem cell transplantation (HSCT) is a safe, highly effective and cost-saving treatment modality for treatment-resistant, and potentially treatment-naïve, immune-mediated neurological disorders. Although the evidence base has been growing in the last decade, limited understanding has led to confusion, mistrust and increasing use of health tourism. In this article, we discuss what autologous HSCT is, which immune-mediated conditions can be treated with it, how to select patients, what are the expected outcomes and potential adverse effects, and how cost-effective this treatment is.

  • neuroimmunology
  • multiple sclerosis
  • myasthenia
  • stiff man syndrome
  • neuropathy

Data availability statement

Data sharing not applicable as no datasets generated and/or analysed for this study.

https://doi.org/10.1136/pn-2022-003531

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    Data availability statement

    Data sharing not applicable as no datasets generated and/or analysed for this study.

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    Footnotes

    • JAS and BS are joint senior authors.

    • Twitter @gavinbrittain, @gavingiovannoni, @elirolga89

    • Contributors The authors BS and JS contribute to this article equally as senior authors and on behalf of Autoimmune Diseases Working Party (ADWP) of the European Society for Blood and Marrow Transplantation (EBMT). The article idea was initially conceived by GG and written by GB with input from all authors. JAS and BS provided senior authorship jointly and all authors approved the final version.

    • Funding This research was supported/funded by the NIHR Sheffield Biomedical Research Centre (BRC)/NIHR Sheffield Clinical Research Facility (CRF). The views expressed are those of the author(s) and not necessarily those of the NHS, the NIHR or the Department of Health and Social Care (DHSC). StarMS: 16/126/26 is funded by the Efficacy and Mechanism Evaluation (EME) Programme, an MRC and NIHR partnership.

    • Competing interests None declared.

    • Provenance and peer review Commissioned; externally peer reviewed Ian Sutton, New South Wales, Australia.

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