BACKGROUND AND PURPOSE: Treatment options in primary progressive multiple sclerosis (PPMS) are scarce and, with the exception of ocrelizumab, anti-inflammatory agents have failed to show efficacy in ameliorating disability progression. The aim of this study was to investigate a potential effect of anti-inflammatory disease-modifying treatment on disability outcomes in PPMS. METHODS: Using MSBase, a large, international, observational database, we identified patients with PPMS who were either never treated or treated with a disease-modifying agent. Propensity score matching was used to select subpopulations with similar baseline characteristics. Expanded Disability Status Scale (EDSS) outcomes were compared with an intention-to-treat and an as-treated approach in paired, pairwise-censored analyses. RESULTS: Of the 1284 included patients, 533 were matched (treated, n = 195; untreated n = 338). Median on-study pairwise-censored follow-up was 3.4 years (quartiles 1.2-5.5). No difference in the hazard of experiencing 3-month confirmed EDSS progression events was observed between the groups [hazard ratio (HR), 1.0; 95% confidence interval (CI), 0.6-1.7, P = 0.87]. We did not find significant differences in the hazards of confirmed EDSS improvement (HR, 1.0; 95% CI, 0.6-1.6, P = 0.91) or reaching a confirmed EDSS step ≥7 (HR, 1.1; 95% CI, 0.7-1.6, P = 0.69). CONCLUSION: Our pooled analysis of disease-modifying agents suggests that these therapies have no substantial effect on short- to medium-term disability outcomes in PPMS.

Cliniques Universitaires Saint Luc Brussels Belgium

Department of Basic Medical Sciences Neuroscience and Sense Organs University of Bari Bari Italy

Department of Medicine University of Melbourne Melbourne VIC Department of Neurology Royal Melbourne Hospital Melbourne VIC Department of Neuroscience Central Clinical School Alfred Hospital Monash University Melbourne VIC

Department of Medicine University of Melbourne Melbourne VIC Department of Neurology Royal Melbourne Hospital Melbourne VIC Department of Neuroscience Central Clinical School Alfred Hospital Monash University Melbourne VIC Department of Neurology Box Hill Hospital Monash University Melbourne VIC

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

Department of Neurology John Hunter Hospital Newcastle NSW School of Medicine and Public Health University of Newcastle Newcastle NSW Australia

Department of Neurology Royal Melbourne Hospital Melbourne VIC CORe Department of Medicine University of Melbourne Melbourne VIC Australia

Department of Neuroscience Imaging and Clinical Sciences University G d'Annunzio Chieti Italy

Departments of Medicine Clinical Research Biomedicine and Biomedical Engineering Neurologic Clinic and Policlinic University Hospital and University of Basel Basel Switzerland

Flinders Medical Centre Flinders University Adelaide SA Australia

Hôpital Notre Dame Montreal Quebec

Hospedale P A Micone Genova Italy

Hospital de Galdakao Usansolo Galdakao Spain

Hospital Germans Trias i Pujol Badalona Spain

Hospital Universitario Virgen Macarena Seville Spain

Hôtel Dieu de Lévis Lévis Quebec Canada

Neuro Rive Sud Hôpital Charles LeMoyne Greenfield Park Quebec

Neurology Unit ASUR Marche AV3 Macerata

Nuovo Ospedale Civile S Agostino Estense Modena

Orbis Medical Center Sittard The Netherlands

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$a Lorscheider, J $u Departments of Medicine, Clinical Research, Biomedicine and Biomedical Engineering, Neurologic Clinic and Policlinic, University Hospital and University of Basel, Basel, Switzerland.

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$a Anti-inflammatory disease-modifying treatment and disability progression in primary progressive multiple sclerosis: a cohort study / $c J. Lorscheider, J. Kuhle, G. Izquierdo, A. Lugaresi, E. Havrdova, D. Horakova, R. Hupperts, P. Duquette, M. Girard, A. Prat, F. Grand'Maison, P. Grammond, P. Sola, D. Ferraro, M. Trojano, C. Ramo-Tello, J. Lechner-Scott, E. Pucci, C. Solaro, M. Slee, V. Van Pesch, JL. Sanchez Menoyo, A. van der Walt, H. Butzkueven, L. Kappos, T. Kalincik, MSBase Study Group,

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$a BACKGROUND AND PURPOSE: Treatment options in primary progressive multiple sclerosis (PPMS) are scarce and, with the exception of ocrelizumab, anti-inflammatory agents have failed to show efficacy in ameliorating disability progression. The aim of this study was to investigate a potential effect of anti-inflammatory disease-modifying treatment on disability outcomes in PPMS. METHODS: Using MSBase, a large, international, observational database, we identified patients with PPMS who were either never treated or treated with a disease-modifying agent. Propensity score matching was used to select subpopulations with similar baseline characteristics. Expanded Disability Status Scale (EDSS) outcomes were compared with an intention-to-treat and an as-treated approach in paired, pairwise-censored analyses. RESULTS: Of the 1284 included patients, 533 were matched (treated, n = 195; untreated n = 338). Median on-study pairwise-censored follow-up was 3.4 years (quartiles 1.2-5.5). No difference in the hazard of experiencing 3-month confirmed EDSS progression events was observed between the groups [hazard ratio (HR), 1.0; 95% confidence interval (CI), 0.6-1.7, P = 0.87]. We did not find significant differences in the hazards of confirmed EDSS improvement (HR, 1.0; 95% CI, 0.6-1.6, P = 0.91) or reaching a confirmed EDSS step ≥7 (HR, 1.1; 95% CI, 0.7-1.6, P = 0.69). CONCLUSION: Our pooled analysis of disease-modifying agents suggests that these therapies have no substantial effect on short- to medium-term disability outcomes in PPMS.

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$a Kuhle, J $u Departments of Medicine, Clinical Research, Biomedicine and Biomedical Engineering, Neurologic Clinic and Policlinic, University Hospital and University of Basel, Basel, Switzerland.

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