RT Journal Article
SR Electronic
T1 Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience
JF Journal of Neurology, Neurosurgery & Psychiatry
JO J Neurol Neurosurg Psychiatry
FD BMJ Publishing Group Ltd
SP 1116
OP 1121
DO 10.1136/jnnp-2013-307207
VO 85
IS 10
A1 Joachim Burman
A1 Ellen Iacobaeus
A1 Anders Svenningsson
A1 Jan Lycke
A1 Martin Gunnarsson
A1 Petra Nilsson
A1 Magnus Vrethem
A1 Sten Fredrikson
A1 Claes Martin
A1 Anna Sandstedt
A1 Bertil Uggla
A1 Stig Lenhoff
A1 Jan-Erik Johansson
A1 Cecilia Isaksson
A1 Hans Hägglund
A1 Kristina Carlson
A1 Jan Fagius
YR 2014
UL http://jnnp.bmj.com/content/85/10/1116.abstract
AB 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.