Rationale for off-label treatments use in primary progressive multiple sclerosis: A review of the literature |
| |
| Affiliation: | 1. Hospital Center of Périgueux, 80, avenue Georges Pompidou, 24000 Périgueux, France;2. Université Clermont Auvergne, CHU de Clermont-Ferrand, Inserm, Neuro-Dol, 63000 Clermont-Ferrand, France;1. UNIROUEN, Inserm U1245, IRON group, Normandy Centre for Genomic and Personalized Medicine, Normandie university, Rouen University Hospital, 76031 Rouen, France;2. Department of Medical Oncology, Cancer Centre Henri Becquerel, 76000 Rouen, France;3. Normandie Univ, UNIROUEN, Inserm U1245, Normandy Centre for Genomic and Personalized Medicine, Rouen, France;4. Department of Pathology, Rouen University Hospital, 76031 Rouen, France;5. Department of Radiology, Rouen University Hospital, 76031 Rouen, France;6. Department of Neurosurgery, Rouen University Hospital, 76031 Rouen, France;7. Department of Hepatogastroenterology, Rouen University Hospital, 76031 Rouen, France;1. Inserm U1094, IRD U270, EpiMaCT – Epidemiology of chronic diseases in tropical zone, Institute of Epidemiology and Tropical Neurology, OmegaHealth, University Limoges, CHU de Limoges, Limoges, France;2. Department of Neurology, Centre de Référence SLA et autres maladies du neurone moteur, CHU de Limoges, Limoges, France;3. European Reference Network for Rare Neuromuscular Diseases (EURO-NMD) Health Care Providers (HCP) Limoges Tours, Limoges, France;4. Centre d’Épidémiologie de Biostatistique et de Méthodologie de la Recherche, CHU Limoges, Limoges, France;1. Neurology department, Grenoble Alpes university hospital, 38043 Grenoble, France;2. Neuroradiology department, Grenoble Alpes university hospital, 38043 Grenoble, France;3. Clinical pharmacology, Inserm CIC1406, Grenoble Alpes university hospital, 38043 Grenoble, France;4. Emergency department, Grenoble Alpes university hospital, 38043 Grenoble, France;5. Neurology department, Chambéry-Métropole-Savoie hospital, 73000 Chambéry, France |
| |
| Abstract: | BackgroundUntil recently, few therapeutic options, other than symptomatic treatment, were available for patients with primary progressive multiple sclerosis (PPMS). Ocrelizumab is the only approved treatment in this indication, and only since 2017. However, many patients in France are receiving off-label treatments for PPMS, mainly rituximab, mycophenolate mofetil, methotrexate, cyclophosphamide, and azathioprine.ObjectiveTo evaluate published data concerning the efficacy of these five treatments frequently used as off-label disease-modifying therapies.MethodsWe reviewed and summarized the studies published in Pubmed since the inception of the database.ResultsEvidence from randomized controlled trials is lacking to support the use of these treatments as disease-modifying therapies in PPMS.ConclusionThe literature lacks dedicated studies to support the off-label use of these disease-modifying therapies in PPMS. However, some limited data are available in the literature suggesting that the use of rituximab and cyclophosphamide could potentially be of some interest in specific subpopulations. |
| |
| Keywords: | Multiple sclerosis Primary progressive multiple sclerosis Treatment Off-label use |
| 本文献已被 ScienceDirect 等数据库收录！ |
|
