Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience. Issue 10 (19th February 2014)

• Record Type: Journal Article
• Authors: Burman, Joachim; Iacobaeus, Ellen; Svenningsson, Anders; Lycke, Jan; Gunnarsson, Martin; Nilsson, Petra; Vrethem, Magnus; Fredrikson, Sten; Martin, Claes; Sandstedt, Anna; Uggla, Bertil; Lenhoff, Stig; Johansson, Jan-Erik; Isaksson, Cecilia; Hägglund, Hans; Carlson, Kristina; Fagius, Jan
• Is Part Of: Journal of neurology, neurosurgery and psychiatry. Volume 85:Issue 10(2014)
• Page Start: 1116
• Page End: 1121
• Publication Date: 2014-02-19
• Subjects: Multiple Sclerosis; Neurology -- Periodicals; Nervous system -- Surgery -- Periodicals; Psychiatry -- Periodicals; 616.8
• DOI: 10.1136/jnnp-2013-307207
• Languages: English
• ISSNs: 0022-3050

Abstract

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.