• 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, and Jan Fagius.
• Department of Neuroscience, Uppsala University, Uppsala, Sweden Department of Neurology, Uppsala University Hospital, Uppsala, Sweden.
• J. Neurol. Neurosurg. Psychiatr.. 2014 Oct 1;85(10):1116-21.

BackgroundAutologous 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.MethodsNeurologists 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.ResultsAt 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%).ConclusionsHSCT is a very effective treatment of inflammatory active MS and can be performed with a high degree of safety at experienced centres.Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.

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