Intended for healthcare professionals

Practice Uncertainties

Is stem cell transplantation safe and effective in multiple sclerosis?

BMJ 2022; 377 doi: (Published 09 June 2022) Cite this as: BMJ 2022;377:e061514
  1. Basil Sharrack, consultant neurologist and professor of clinical neurology,
  2. Jennifer Petrie, trial manager,
  3. Alasdair Coles, professor of neuroimmunology and consultant neurologist,
  4. John A Snowden, consultant haematologist, director of blood and marrow transplantation programme, professor of haematology
  1. 1Department of Neurology and Sheffield NIHR Neuroscience BRC, Sheffield Teaching Hospitals NHS Foundation Trust and University of Sheffield, Sheffield, UK
  2. 2Clinical Trials Research Unit, University of Sheffield, Sheffield, UK
  3. 3Department of Clinical Neurosciences, University of Cambridge, Cambridge, UK
  4. 4Department of Haematology, Sheffield Teaching Hospitals NHS Foundation Trust, Sheffield, UK
  5. 5Department of Oncology and Metabolism, Medical School, University of Sheffield, Sheffield, UK
  1. Correspondence to B Sharrack basil.sharrack{at}

What you need to know

  • Autologous haematopoietic stem cell transplantation (aHSCT) is a relatively new treatment for patients with highly active relapsing-remitting multiple sclerosis (MS) that has not responded to treatment with disease modifying therapies

  • Very limited evidence from two small randomised controlled trials shows that aHSCT may slow disease activity and disability progression in some patients, but it is not known how this compares with newer, highly effective disease modifying therapies

  • Patients with highly active relapsing-remitting MS can be considered for aHSCT in specialist centres, and should be treated as part of a clinical trial or included in registry studies

Multiple sclerosis (MS) is an immune mediated disease of the central nervous system. It can lead to visual, sensory, and motor impairment, and difficulties with balance and sphincter control. More than 130 000 people in the UK, and more than 2.5 million people worldwide, are affected. MS is an important cause of disability in young adults, with most being diagnosed between the ages of 20 and 40.12

Most patients (85%) have a relapsing-remitting (RR) clinical course (RRMS), with episodes of new or worsening symptoms, known as relapses, which improve or resolve over weeks or months.3 This is followed by periods of remission, with or without persistent disability. Disease modifying therapies (DMTs) are offered as standard treatment to patients with active relapsing-remitting disease.4 Highly effective DMTs result in NEDA (no evidence of disease activity), defined as the absence of clinical relapses, disability progression, or disease activity visible on magnetic resonance imaging (MRI), in 34-48% of patients at 2 years.5 Over time, most patients with RRMS (50% at 10 years) transition to a secondary progressive course, where disability increases gradually. A smaller proportion of patients with MS (10-15%) have a primary progressive course from onset, with worsening disability independent of relapses.6 …

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