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Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience

Burman, Joachim ; Iacobaeus, Ellen ; Svenningsson, Anders ; Lycke, Jan ; Gunnarsson, Martin ; Nilsson, Petra LU ; Vrethem, Magnus ; Fredrikson, Sten ; Martin, Claes and Sandstedt, Anna , et al. (2014) In Journal of Neurology, Neurosurgery and Psychiatry 85(10). p.1116-1121
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... (More)
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. (Less)
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organization
publishing date
type
Contribution to journal
publication status
published
subject
in
Journal of Neurology, Neurosurgery and Psychiatry
volume
85
issue
10
pages
1116 - 1121
publisher
BMJ Publishing Group
external identifiers
  • wos:000344456000228
  • scopus:84908335130
  • pmid:24554104
ISSN
1468-330X
DOI
10.1136/jnnp-2013-307207
language
English
LU publication?
yes
id
f17f9aa2-e63d-47a6-ab85-521714f79b74 (old id 4871317)
date added to LUP
2016-04-01 13:35:08
date last changed
2024-10-25 05:00:23
@article{f17f9aa2-e63d-47a6-ab85-521714f79b74,
  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.}},
  author       = {{Burman, Joachim and Iacobaeus, Ellen and Svenningsson, Anders and Lycke, Jan and Gunnarsson, Martin and Nilsson, Petra and Vrethem, Magnus and Fredrikson, Sten and Martin, Claes and Sandstedt, Anna and Uggla, Bertil and Lenhoff, Stig and Johansson, Jan-Erik and Isaksson, Cecilia and Hagglund, Hans and Carlson, Kristina and Fagius, Jan}},
  issn         = {{1468-330X}},
  language     = {{eng}},
  number       = {{10}},
  pages        = {{1116--1121}},
  publisher    = {{BMJ Publishing Group}},
  series       = {{Journal of Neurology, Neurosurgery and Psychiatry}},
  title        = {{Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience}},
  url          = {{http://dx.doi.org/10.1136/jnnp-2013-307207}},
  doi          = {{10.1136/jnnp-2013-307207}},
  volume       = {{85}},
  year         = {{2014}},
}