Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience

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Abstract

Background Autologous haematopoietic stem cell transplantation (HSCT) is a viable option for treatment of aggressive multiple sclerosis (MS). No randomised controlled trial has been performed, and thus, experiences from systematic and sustained follow-up of treated patients constitute important information about safety and efficacy. In this observational study, we describe the characteristics and outcome of the Swedish patients treated with HSCT for MS. Methods Neurologists from the major hospitals in Sweden filled out a follow-up form with prospectively collected data. Fifty-two patients were identified in total; 48 were included in the study and evaluated for safety and side effects; 41 patients had at least 1 year of follow-up and were further analysed for clinical and radiological outcome. In this cohort, 34 patients (83%) had relapsing-remitting MS, and mean follow-up time was 47 months. Results At 5 years, relapse-free survival was 87%; MRI event-free survival 85%; expanded disability status scale (EDSS) score progression-free survival 77%; and disease-free survival (no relapses, no new MRI lesions and no EDSS progression) 68%. Presence of gadolinium-enhancing lesions prior to HSCT was associated with a favourable outcome (disease-free survival 79% vs 46%, p=0.028). There was no mortality. The most common long-term side effects were herpes zoster reactivation (15%) and thyroid disease (8.4%). Conclusions HSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.

Detaljer

Författare
  • Joachim Burman
  • Ellen Iacobaeus
  • Anders Svenningsson
  • Jan Lycke
  • Martin Gunnarsson
  • Petra Nilsson
  • Magnus Vrethem
  • Sten Fredrikson
  • Claes Martin
  • Anna Sandstedt
  • Bertil Uggla
  • Stig Lenhoff
  • Jan-Erik Johansson
  • Cecilia Isaksson
  • Hans Hagglund
  • Kristina Carlson
  • Jan Fagius
Enheter & grupper
Forskningsområden

Ämnesklassifikation (UKÄ) – OBLIGATORISK

  • Neurologi
Originalspråkengelska
Sidor (från-till)1116-1121
TidskriftJournal of Neurology, Neurosurgery and Psychiatry
Volym85
Utgivningsnummer10
StatusPublished - 2014
PublikationskategoriForskning
Peer review utfördJa