BACKGROUND: Natalizumab was not shown to modify disability in progressive multiple sclerosis (MS). This matched observational study compared the effectiveness of autologous haematopoietic stem cell transplantation (AHSCT) with natalizumab in progressive MS. METHODS: Patients with primary/secondary progressive MS from seven AHSCT MS centres and the MSBase registry, treated with AHSCT or natalizumab, were matched on a propensity score derived from sex, age, Expanded Disability Status Scale (EDSS), number of relapses 12/24 months before baseline, time from MS onset, the most effective prior therapy and country. The pairwise-censored groups were compared on hazards of 6-month confirmed EDSS worsening and improvement, relapses and annualised relapse rates (ARRs), using Andersen-Gill proportional hazards models and conditional negative binomial model. RESULTS: 39 patients treated with AHSCT (37 with secondary progressive MS, mean age 37 years, EDSS 5.7, 28% with recent disability progression, ARR 0.54 during the preceding year) were matched with 65 patients treated with natalizumab. The study found no evidence for difference in hazards of confirmed EDSS worsening (HR 1.49, 95% CI 0.70 to 3.14) and improvement (HR 1.50, 95% CI 0.22 to 10.29) between AHSCT and natalizumab over up to 4 years. The relapse activity was also similar while treated with AHSCT and natalizumab (ARR: mean±SD 0.08±0.28 vs 0.08±0.25; HR 1.05, 95% CI 0.39 to 2.82). In the AHSCT group, 3 patients experienced febrile neutropenia during mobilisation, 9 patients experienced serum sickness, 6 patients required intensive care unit admission and 36 patients experienced complications after discharge. No treatment-related deaths were reported. CONCLUSION: This study does not support the use of AHSCT to control disability in progressive MS with advanced disability and low relapse activity.

CORe Department of Medicine University of Melbourne Parkville Victoria Australia

Department of Haematology 1st Faculty of Medicine Charles University Prague Czech Republic

Department of Haematology 3rd Faculty of Medicine Charles University Prague Czech Republic

Department of Haematology Haukeland University Hospital Bergen Norway

Department of Haematology Sheffield Teaching Hospitals NHS Foundation Trust Sheffield UK

Department of Haematology St Vincent's Hospital Sydney Darlinghurst New South Wales Australia

Department of Medical Sciences Neurology Uppsala University Uppsala Sweden

Department of Medicine Ottawa Hospital Research Institute Ottawa Ontario Canada

Department of Neurology and Center of Clinical Neuroscience 1st Faculty of Medicine Charles University Prague Czech Republic

Department of Neurology Austin Health Melbourne Victoria Australia

Department of Neurology Haukeland University Hospital Bergen Norway

Department of Neurology St Vincent's Hospital Sydney Darlinghurst New South Wales Australia

Department of Neuroscience and Sheffield NIHR Translational Neuroscience Biomedical Research Centre Sheffield Teaching Hospitals NHS Foundation Trust Sheffield UK

Department of Oncology and Metabolism Medical School The University of Sheffield Sheffield UK

General University Hospital Prague Prague Czech Republic

Neuroimmunology Centre Department of Neurology Royal Melbourne Hospital Parkville Victoria Australia

St Vincent's Clinical School University of New South Wales Sydney New South Wales Australia

University Hospital Kralovske Vinohrady Prague Czech Republic

University of Melbourne Melbourne Victoria Australia

University of New South Wales Sydney New South Wales Australia

University of Syndey Sydney New South Wales Australia

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