title: Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience.
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.
authors.affiliation: Department of Neuroscience, Uppsala University, Uppsala, Sweden Department of Neurology, Uppsala University Hospital, Uppsala, Sweden.
authors.forename: Joachim
authors.initials: J
authors.lastname: Burman
authors.affiliation: Neuroimmunology Unit, Department of Clinical Neuroscience, Karolinska Institute Solna, Center for Molecular Medicine, Stockholm, Sweden.
authors.forename: Ellen
authors.initials: E
authors.lastname: Iacobaeus
authors.affiliation: Department of Pharmacology and Clinical Neuroscience, Umeå University and University Hospital of Northern Sweden, Umeå, Sweden.
authors.forename: Anders
authors.initials: A
authors.lastname: Svenningsson
authors.affiliation: Department of Neurology, Institute of Neuroscience and Physiology, Sahlgrenska University Hospital, Gothenburg, Sweden.
authors.forename: Jan
authors.initials: J
authors.lastname: Lycke
authors.affiliation: Department of Neurology, Örebro University Hospital, Örebro, Sweden School of Health and Medical Sciences, Örebro University, Örebro, Sweden.
authors.forename: Martin
authors.initials: M
authors.lastname: Gunnarsson
authors.affiliation: Department of Neurology, Skåne University Hospital Lund, Lund, Sweden.
authors.forename: Petra
authors.initials: P
authors.lastname: Nilsson
authors.affiliation: Neurology and Clinical Neurophysiology, Faculty of Health Sciences, Department of Clinical and Experimental Medicine, Linköping University, Linköping, Sweden Department of Neurology and Neurophysiology, County Council of Östergötland, Linköping, Sweden.
authors.forename: Magnus
authors.initials: M
authors.lastname: Vrethem
authors.affiliation: Department of Clinical Neuroscience, Karolinska Institute Huddinge, Stockholm, Sweden.
authors.forename: Sten
authors.initials: S
authors.lastname: Fredrikson
authors.affiliation: Neurology Unit, Division of Internal Medicine, Danderyd Hospital, Karolinska Institute, Stockholm, Sweden.
authors.forename: Claes
authors.initials: C
authors.lastname: Martin
authors.affiliation: Department of Hematology, Linköping University Hospital, Linköping, Sweden.
authors.forename: Anna
authors.initials: A
authors.lastname: Sandstedt
authors.affiliation: School of Health and Medical Sciences, Örebro University, Örebro, Sweden Division of Hematology, Department of Medicine, Örebro University Hospital, Örebro, Sweden.
authors.forename: Bertil
authors.initials: B
authors.lastname: Uggla
authors.affiliation: Department of Hematology and Coagulation, Skåne University Hospital, Lund, Sweden.
authors.forename: Stig
authors.initials: S
authors.lastname: Lenhoff
authors.affiliation: Department of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden.
authors.forename: Jan-Erik
authors.initials: JE
authors.lastname: Johansson
authors.affiliation: Department of Radiosciences, Umeå University, Umeå, Sweden.
authors.forename: Cecilia
authors.initials: C
authors.lastname: Isaksson
authors.affiliation: Division of Hematology, Department of Medical Science, Uppsala University Hospital, Uppsala, Sweden.
authors.forename: Hans
authors.initials: H
authors.lastname: Hägglund
authors.affiliation: Division of Hematology, Department of Medical Science, Uppsala University Hospital, Uppsala, Sweden.
authors.forename: Kristina
authors.initials: K
authors.lastname: Carlson
authors.affiliation: Department of Neuroscience, Uppsala University, Uppsala, Sweden Department of Neurology, Uppsala University Hospital, Uppsala, Sweden.
authors.forename: Jan
authors.initials: J
authors.lastname: Fagius
mesh_terms: D000293:Adolescent; D000328:Adult; D001921:Brain / Q000473:pathology / Q000601:surgery*; D002648:Child; D004185:Disability Evaluation; D018572:Disease-Free Survival; D005260:Female; D005500:Follow-Up Studies; D018380:Hematopoietic Stem Cell Transplantation / Q000009:adverse effects* / Q000401:mortality*; D006801:Humans; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D009103:Multiple Sclerosis / Q000401:mortality / Q000473:pathology / Q000601:surgery*; D059906:Neuroimaging; D012008:Recurrence; D013548:Sweden; D014182:Transplantation, Autologous / Q000009:adverse effects* / Q000401:mortality*; D016896:Treatment Outcome; D055815:Young Adult
keywords: Multiple Sclerosis