TY - JOUR T1 - Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience JF - Journal of Neurology, Neurosurgery & Psychiatry JO - J Neurol Neurosurg Psychiatry SP - 1116 LP - 1121 DO - 10.1136/jnnp-2013-307207 VL - 85 IS - 10 AU - Joachim Burman AU - Ellen Iacobaeus AU - Anders Svenningsson AU - Jan Lycke AU - Martin Gunnarsson AU - Petra Nilsson AU - Magnus Vrethem AU - Sten Fredrikson AU - Claes Martin AU - Anna Sandstedt AU - Bertil Uggla AU - Stig Lenhoff AU - Jan-Erik Johansson AU - Cecilia Isaksson AU - Hans Hägglund AU - Kristina Carlson AU - Jan Fagius Y1 - 2014/10/01 UR - http://jnnp.bmj.com/content/85/10/1116.abstract N2 - 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. ER -