J Lorscheider1, J Kuhle1, G Izquierdo2, A Lugaresi3, E Havrdova4, D Horakova4, R Hupperts5, P Duquette6, M Girard6, A Prat6, F Grand'Maison7, P Grammond8, P Sola9, D Ferraro9, M Trojano10, C Ramo-Tello11, J Lechner-Scott12,13, E Pucci14, C Solaro15, M Slee16, V Van Pesch17, J L Sanchez Menoyo18, A van der Walt19,20,21, H Butzkueven19,20,21,22, L Kappos1, T Kalincik20,23. 1. Departments of Medicine, Clinical Research, Biomedicine and Biomedical Engineering, Neurologic Clinic and Policlinic, University Hospital and University of Basel, Basel, Switzerland. 2. Hospital Universitario Virgen Macarena, Seville, Spain. 3. Department of Neuroscience, Imaging and Clinical Sciences, University G. d'Annunzio, Chieti, Italy. 4. Department of Neurology and Center of Clinical Neuroscience, First Faculty of Medicine, Charles University and General University Hospital, Prague, Czech Republic. 5. Orbis Medical Center, Sittard, The Netherlands. 6. Hôpital Notre Dame, Montreal, Quebec. 7. Neuro Rive-Sud, Hôpital Charles LeMoyne, Greenfield Park, Quebec. 8. Hôtel-Dieu de Lévis, Lévis, Quebec, Canada. 9. Nuovo Ospedale Civile S.Agostino/Estense, Modena. 10. Department of Basic Medical Sciences, Neuroscience and Sense Organs, University of Bari, Bari, Italy. 11. Hospital Germans Trias i Pujol, Badalona, Spain. 12. Department of Neurology, John Hunter Hospital, Newcastle, NSW. 13. School of Medicine and Public Health, University of Newcastle, Newcastle, NSW, Australia. 14. Neurology Unit, ASUR Marche, AV3, Macerata. 15. Hospedale P. A. Micone, Genova, Italy. 16. Flinders Medical Centre, Flinders University, Adelaide, SA, Australia. 17. Cliniques Universitaires Saint-Luc, Brussels, Belgium. 18. Hospital de Galdakao-Usansolo, Galdakao, Spain. 19. Department of Medicine, University of Melbourne, Melbourne, VIC. 20. Department of Neurology, Royal Melbourne Hospital, Melbourne, VIC. 21. Department of Neuroscience, Central Clinical School Alfred Hospital, Monash University, Melbourne, VIC. 22. Department of Neurology, Box Hill Hospital, Monash University, Melbourne, VIC. 23. CORe, Department of Medicine, University of Melbourne, Melbourne, VIC, Australia.
Abstract
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.
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.
Authors: Tianci Chu; Lisa B E Shields; Wenxin Zeng; Yi Ping Zhang; Yuanyi Wang; Gregory N Barnes; Christopher B Shields; Jun Cai Journal: Neural Regen Res Date: 2021-07 Impact factor: 5.135
Authors: Vanessa F Moreira Ferreira; Danielle Caefer; Natalie Erlich-Malona; Brian C Healy; Tanuja Chitnis; James M Stankiewicz Journal: Mult Scler Int Date: 2020-12-17
Authors: Helmut Butzkueven; Tim Spelman; Dana Horakova; Stella Hughes; Claudio Solaro; Guillermo Izquierdo; Eva Kubala Havrdová; Francois Grand'Maison; Alexandre Prat; Marc Girard; Raymond Hupperts; Marco Onofrj; Alessandra Lugaresi; Bruce Taylor; Gavin Giovannoni; Ludwig Kappos; Stephen L Hauser; Xavier Montalban; Licinio Craveiro; Rita Freitas; Fabian Model; James Overell; Erwan Muros-Le Rouzic; Annette Sauter; Qing Wang; David Wormser; Jerry S Wolinsky Journal: Eur J Neurol Date: 2021-05-06 Impact factor: 6.288