Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience.
J Neurol Neurosurg Psychiatry
; 85(10): 1116-21, 2014 Oct.
Article
in En
| MEDLINE
| ID: mdl-24554104
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.Key words
Full text:
1
Collection:
01-internacional
Database:
MEDLINE
Main subject:
Transplantation, Autologous
/
Brain
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Hematopoietic Stem Cell Transplantation
/
Multiple Sclerosis
Type of study:
Clinical_trials
/
Observational_studies
/
Prognostic_studies
/
Risk_factors_studies
Limits:
Adolescent
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Adult
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Child
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Female
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Humans
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Male
/
Middle aged
Country/Region as subject:
Europa
Language:
En
Journal:
J Neurol Neurosurg Psychiatry
Year:
2014
Document type:
Article
Affiliation country:
Sweden