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Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience
Department of Neuroscience, Uppsala University, Uppsala, Sweden; Department of Neurology, Uppsala University Hospital, Uppsala, Sweden .
Neuroimmunology Unit, Department of Clinical Neuroscience, Karolinska Institute Solna, Center for Molecular Medicine, Stockholm, Sweden.
Department of Pharmacology and Clinical Neuroscience, Umeå University and University Hospital of Northern Sweden, Umeå, Sweden .
Department of Neurology, Institute of Neuroscience and Physiology, Sahlgrenska University Hospital, Gothenburg, Sweden.
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2014 (English)In: Journal of Neurology, Neurosurgery and Psychiatry, ISSN 0022-3050, E-ISSN 1468-330X, Vol. 85, no 10, 1116-1121 p.Article in journal (Refereed) Published
Abstract [en]

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.

Place, publisher, year, edition, pages
London, United Kingdom: BMJ Publishing Group Ltd, 2014. Vol. 85, no 10, 1116-1121 p.
National Category
Neurology
Research subject
Neurology
Identifiers
URN: urn:nbn:se:oru:diva-37789DOI: 10.1136/jnnp-2013-307207ISI: 000344456000228PubMedID: 24554104Scopus ID: 2-s2.0-84893900422OAI: oai:DiVA.org:oru-37789DiVA: diva2:756386
Available from: 2014-10-17 Created: 2014-10-15 Last updated: 2017-03-15Bibliographically approved

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Gunnarsson, MartinUggla, BertilJohansson, Jan-Erik
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School of Medicine, Örebro University, SwedenOrebro University Hospital
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