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

Journal article
Authors 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 Hägglund
Kristina Carlson
Jan Fagius
Published in Journal of Neurology, Neurosurgery and Psychiatry
Volume 85
Pages 1116-1121
ISSN 00223050
Publication year 2014
Published at
Pages 1116-1121
Language en
Subject categories Neurosciences

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 diseasefree 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.

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