Autologous haematopoietic stem cell transplantation for immune-mediated neurological diseases: what, how, who and why?

Pract Neurol. 2023 Apr;23(2):139-145. doi: 10.1136/pn-2022-003531. Epub 2022 Sep 26.

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.

Keywords: multiple sclerosis; myasthenia; neuroimmunology; neuropathy; stiff man syndrome.

MeSH terms

  • Hematopoietic Stem Cell Transplantation*
  • Humans
  • Multiple Sclerosis*
  • Nervous System Diseases* / etiology
  • Nervous System Diseases* / therapy