Joachim Burman1, Ellen Iacobaeus2, Anders Svenningsson3, Jan Lycke4, Martin Gunnarsson5, Petra Nilsson6, Magnus Vrethem7, Sten Fredrikson8, Claes Martin9, Anna Sandstedt10, Bertil Uggla11, Stig Lenhoff12, Jan-Erik Johansson13, Cecilia Isaksson14, Hans Hägglund15, Kristina Carlson15, Jan Fagius1. 1. Department of Neuroscience, Uppsala University, Uppsala, Sweden Department of Neurology, Uppsala University Hospital, Uppsala, Sweden. 2. Neuroimmunology Unit, Department of Clinical Neuroscience, Karolinska Institute Solna, Center for Molecular Medicine, Stockholm, Sweden. 3. Department of Pharmacology and Clinical Neuroscience, Umeå University and University Hospital of Northern Sweden, Umeå, Sweden. 4. Department of Neurology, Institute of Neuroscience and Physiology, Sahlgrenska University Hospital, Gothenburg, Sweden. 5. Department of Neurology, Örebro University Hospital, Örebro, Sweden School of Health and Medical Sciences, Örebro University, Örebro, Sweden. 6. Department of Neurology, Skåne University Hospital Lund, Lund, Sweden. 7. 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. 8. Department of Clinical Neuroscience, Karolinska Institute Huddinge, Stockholm, Sweden. 9. Neurology Unit, Division of Internal Medicine, Danderyd Hospital, Karolinska Institute, Stockholm, Sweden. 10. Department of Hematology, Linköping University Hospital, Linköping, Sweden. 11. School of Health and Medical Sciences, Örebro University, Örebro, Sweden Division of Hematology, Department of Medicine, Örebro University Hospital, Örebro, Sweden. 12. Department of Hematology and Coagulation, Skåne University Hospital, Lund, Sweden. 13. Department of Hematology and Coagulation, Sahlgrenska University Hospital, Gothenburg, Sweden. 14. Department of Radiosciences, Umeå University, Umeå, Sweden. 15. Division of Hematology, Department of Medical Science, Uppsala University Hospital, Uppsala, Sweden.
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. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.
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. Published by the BMJ Publishing Group Limited. For permission to use (where not already granted under a licence) please go to http://group.bmj.com/group/rights-licensing/permissions.
Authors: J-P Stellmann; K H Stürner; F Ufer; S Havemeister; J Pöttgen; F Ayuk Ayuketang; N Kröger; M A Friese; C Heesen Journal: Nervenarzt Date: 2015-08 Impact factor: 1.214
Authors: J Burman; K Kirgizov; K Carlson; M Badoglio; G L Mancardi; G De Luca; B Casanova; J Ouyang; R Bembeeva; J Haas; P Bader; J Snowden; D Farge Journal: Bone Marrow Transplant Date: 2017-03-20 Impact factor: 5.483