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Research paper
Autologous haematopoietic stem cell transplantation for aggressive multiple sclerosis: the Swedish experience
  1. Joachim Burman1,2,
  2. Ellen Iacobaeus3,
  3. Anders Svenningsson4,
  4. Jan Lycke5,
  5. Martin Gunnarsson6,7,
  6. Petra Nilsson8,
  7. Magnus Vrethem9,10,
  8. Sten Fredrikson11,
  9. Claes Martin12,
  10. Anna Sandstedt13,
  11. Bertil Uggla7,14,
  12. Stig Lenhoff15,
  13. Jan-Erik Johansson16,
  14. Cecilia Isaksson17,
  15. Hans Hägglund18,
  16. Kristina Carlson18,
  17. Jan Fagius1,2
  1. 1Department of Neuroscience, Uppsala University, Uppsala, Sweden
  2. 2Department of Neurology, Uppsala University Hospital, Uppsala, Sweden
  3. 3Neuroimmunology Unit, Department of Clinical Neuroscience, Karolinska Institute Solna, Center for Molecular Medicine, Stockholm, Sweden
  4. 4Department of Pharmacology and Clinical Neuroscience, Umeå University and University Hospital of Northern Sweden, Umeå, Sweden
  5. 5Department of Neurology, Institute of Neuroscience and Physiology, Sahlgrenska University Hospital, Gothenburg, Sweden
  6. 6Department of Neurology, Örebro University Hospital, Örebro, Sweden
  7. 7School of Health and Medical Sciences, Örebro University, Örebro, Sweden
  8. 8Department of Neurology, Skåne University Hospital Lund, Lund, Sweden
  9. 9Neurology and Clinical Neurophysiology, Faculty of Health Sciences, Department of Clinical and Experimental Medicine, Linköping University, Linköping, Sweden
  10. 10Department of Neurology and Neurophysiology, County Council of Östergötland, Linköping, Sweden
  11. 11Department of Clinical Neuroscience, Karolinska Institute Huddinge, Stockholm, Sweden
  12. 12Neurology Unit, Division of Internal Medicine, Danderyd Hospital, Karolinska Institute, Stockholm, Sweden
  13. 13Department of Hematology, Linköping University Hospital, Linköping, Sweden
  14. 14Division of Hematology, Department of Medicine, Örebro University Hospital, Örebro, Sweden
  15. 15Department of Hematology and Coagulation, Skåne University Hospital, Lund, Sweden
  16. 16Department of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden
  17. 17Department of Radiosciences, Umeå University, Umeå, Sweden
  18. 18Division of Hematology, Department of Medical Science, Uppsala University Hospital, Uppsala, Sweden
  1. Correspondence to Dr Joachim Burman, Department of Neurosciences, Uppsala University Hospital, Uppsala SE-751 85, Sweden; joachim.burman{at}neuro.uu.se

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.

  • Multiple Sclerosis

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