Traitements de fond de la sclérose en plaques. État des lieux et perspectives

Bulletin de l'Académie Nationale de Médecine - Tập 206 - Trang 741-751 - 2022
L. Magy1
1Service et laboratoire de neurologie, CRC sclérose en plaques, CHU Limoges, Limoges, France

Tài liệu tham khảo

Reich, 2018, N Engl J Med, 378, 169, 10.1056/NEJMra1401483 McGinley, 2021, Diagnosis and treatment of multiple sclerosis: a review, JAMA, 325, 765, 10.1001/jama.2020.26858 Yong, 2021, Mechanism-based criteria to improve therapeutic outcomes in progressive multiple sclerosis, Nat Rev Neurol [Internet] Trapp, 1998, Axonal transection in the lesions of multiple sclerosis, N Engl J Med, 338, 278, 10.1056/NEJM199801293380502 Barnett, 2004, Relapsing and remitting multiple sclerosis: Pathology of the newly forming lesion, Ann Neurol, 55, 458, 10.1002/ana.20016 Arneth, 2021, Contributions of T cells in multiple sclerosis: what do we currently know?, J Neurol, 268, 4587, 10.1007/s00415-020-10275-x Sanders, 2007, Janus faces of microglia in multiple sclerosis, Brain Res Rev, 54, 274, 10.1016/j.brainresrev.2007.03.001 Comi, 2020, Role of B cells in multiple sclerosis and related disorders, Ann Neurol [Internet] Kuhlmann, 2016, An updated histological classification system for multiple sclerosis lesions, Acta Neuropathol, 133, 13, 10.1007/s00401-016-1653-y Correale, 2016, Progressive multiple sclerosis: from pathogenic mechanisms to treatment, Brain, 140, 527 Fisher, 2008, Gray matter atrophy in multiple sclerosis: a longitudinal study, Ann Neurol, 64, 255, 10.1002/ana.21436 Magliozzi, 2006, Meningeal B-cell follicles in secondary progressive multiple sclerosis associate with early onset of disease and severe cortical pathology, Brain, 130, 1089, 10.1093/brain/awm038 Devonshire, 2012, Relapse and disability outcomes in patients with multiple sclerosis treated with fingolimod: subgroup analyses of the double-blind, randomised, placebo-controlled FREEDOMS study, Lancet neurology, 11, 420, 10.1016/S1474-4422(12)70056-X Confavreux, 2000, Relapses and progression of disability in multiple sclerosis, N Engl J Med, 343, 1430, 10.1056/NEJM200011163432001 Hauser, 2021, Curing multiple sclerosis: how to know when we’re there, Ann Neurol, 90, 539, 10.1002/ana.26155 University of California SFMT, 2019, Silent progression in disease activity–free relapsing multiple sclerosis, Ann Neurol, 85, 653, 10.1002/ana.25463 Kappos, 2020, Contribution of relapse-independent progression vs. relapse-associated worsening to overall confirmed disability accumulation in typical relapsing multiple sclerosis in a pooled analysis of 2 randomized clinical trials, JAMA Neurol, 77, 1132, 10.1001/jamaneurol.2020.1568 Kappos, 2016, Inclusion of brain volume loss in a revised measure of ‘no evidence of disease activity’ (NEDA-4) in relapsing–remitting multiple sclerosis, Mult Scler, 22, 1297, 10.1177/1352458515616701 Río, 2006, Defining the response to interferon-beta in relapsing-remitting multiple sclerosis patients, Ann Neurol, 59, 344, 10.1002/ana.20740 Rovaris, 2008, Cognitive impairment and structural brain damage in benign multiple sclerosis, Neurology, 71, 1521, 10.1212/01.wnl.0000319694.14251.95 Ferreira-Atuesta, 2021, The evolution of neurofilament light chain in multiple sclerosis, Front Neurosci, 15, 642384, 10.3389/fnins.2021.642384 1993, Interferon beta-1b is effective in relapsing-remitting multiple sclerosis. I. Clinical results of a multicenter, randomized, double-blind, placebo-controlled trial. The IFNB Multiple Sclerosis Study Group, Neurology, 43, 655, 10.1212/WNL.43.4.655 Johnson, 1995, Copolymer 1 reduces relapse rate and improves disability in relapsing-remitting multiple sclerosis: results of a phase III multicenter, double-blind placebo-controlled trial. The Copolymer 1 Multiple Sclerosis Study Group, Neurology, 45, 1268, 10.1212/WNL.45.7.1268 Jacobs, 1996, Intramuscular interferon beta-1a for disease progression in relapsing multiple sclerosis, Ann Neurol, 39, 285, 10.1002/ana.410390304 1998, Randomised double-blind placebo-controlled study of interferon beta-1a in relapsing/remitting multiple sclerosis. PRISMS (Prevention of Relapses and Disability by Interferon beta-1a Subcutaneously in Multiple Sclerosis) Study Group, Lancet, 352, 1498, 10.1016/S0140-6736(98)03334-0 McDonald, 2001, Recommended diagnostic criteria for multiple sclerosis: guidelines from the International Panel on the diagnosis of multiple sclerosis, Ann Neurol, 50, 121, 10.1002/ana.1032 Polman, 2005, Diagnostic criteria for multiple sclerosis: 2005 revisions to the “McDonald Criteria”, Ann Neurol, 58, 840, 10.1002/ana.20703 Polman, 2011, Diagnostic criteria for multiple sclerosis: 2010 Revisions to the McDonald criteria, Ann Neurol, 69, 292, 10.1002/ana.22366 Thompson, 2018, Diagnosis of multiple sclerosis: 2017 revisions of the McDonald criteria, Lancet Neurol, 17, 162, 10.1016/S1474-4422(17)30470-2 Comi, 2001, Effect of early interferon treatment on conversion to definite multiple sclerosis: a randomised study, Lancet, 357, 1576, 10.1016/S0140-6736(00)04725-5 Jacobs, 2000, Intramuscular interferon beta-1A therapy initiated during a first demyelinating event in multiple sclerosis, N Engl J Med, 343, 898, 10.1056/NEJM200009283431301 Brex, 2002, A longitudinal study of abnormalities on MRI and disability from multiple sclerosis, N Engl J Med, 346, 158, 10.1056/NEJMoa011341 Fisniku, 2008, Disability and T2 MRI lesions: a 20-year follow-up of patients with relapse onset of multiple sclerosis, Brain, 131, 808, 10.1093/brain/awm329 Chung, 2019, A 30-year clinical and magnetic resonance imaging observational study of multiple sclerosis and clinically isolated syndromes, Ann Neurol, 0, 1 Sayao, 2007, Longitudinal follow-up of “benign” multiple sclerosis at 20 years, Neurology, 68, 496, 10.1212/01.wnl.0000253185.03943.66 Tallantyre, 2018, How common is truly benign MS in a UK population?, J Neurol Neurosurg Psychiatr, 90, 522, 10.1136/jnnp-2018-318802 Brown, 2019, Association of initial disease-modifying therapy with later conversion to secondary progressive multiple sclerosis, JAMA, 321, 175, 10.1001/jama.2018.20588 Lebrun, 2009, Association between clinical conversion to multiple sclerosis in radiologically isolated syndrome and magnetic resonance imaging, cerebrospinal fluid, and visual evoked potential: follow-up of 70 patients, Arch Neurol, 66, 841, 10.1001/archneurol.2009.119 Lebrun-Frenay, 2020, Radiologically isolated syndrome: 10-year risk estimate of a clinical event, Ann Neurol, 88, 407, 10.1002/ana.25799 Lebrun-Frénay, 2021, Risk factors and time to clinical symptoms of multiple sclerosis among patients with radiologically isolated syndrome, JAMA Netw Open, 4, e2128271, 10.1001/jamanetworkopen.2021.28271 Moisset, 2021, Untreated patients with multiple sclerosis: a study of French expert centers, Eur J Neurol, 28, 2026, 10.1111/ene.14790 Dhib-Jalbut, 2010, Interferon-β mechanisms of action in multiple sclerosis, Neurology, 74, S17, 10.1212/WNL.0b013e3181c97d99 Fox, 2014, BG-12 (dimethyl fumarate): a review of mechanism of action, efficacy, and safety, Curr Med Res Opin, 30, 251, 10.1185/03007995.2013.849236 Kalincik, 2019, Comparison of fingolimod, dimethyl fumarate and teriflunomide for multiple sclerosis, J Neurol Neurosurg Psychiatr, 90, 458, 10.1136/jnnp-2018-319831 Lorscheider, 2021, Comparative analysis of dimethyl fumarate and fingolimod in relapsing–remitting multiple sclerosis, J Neurol, 268, 941, 10.1007/s00415-020-10226-6 Briner, 2019, Time course of lymphocyte repopulation after dimethyl fumarate-induced grade 3 lymphopenia: contribution of patient age, Ther Adv Neurol Disord, 12, 10.1177/1756286419843450 Miller, 2021, An updated review of teriflunomide's use in multiple sclerosis, Neurodegener Dis Manag, 11, 387, 10.2217/nmt-2021-0014 Hauser, 2021, Safety of ocrelizumab in patients with relapsing and primary progressive multiple sclerosis, Neurology, 97, e1546, 10.1212/WNL.0000000000012700 Tallantyre, 2022, COVID-19 Vaccine Response in People with Multiple Sclerosis, Annals Neurol, 91, 89, 10.1002/ana.26251 Hauser, 2020, Ofatumumab versus teriflunomide in multiple sclerosis, N Engl J Med, 383, 546, 10.1056/NEJMoa1917246 Edan, 2011, Mitoxantrone prior to interferon beta-1b in aggressive relapsing multiple sclerosis: a 3-year randomised trial, J Neurol Neurosurg Psychiatry, 82, 1344, 10.1136/jnnp.2010.229724 Miller, 2003, A controlled trial of natalizumab for relapsing multiple sclerosis, N Engl J Med, 348, 15, 10.1056/NEJMoa020696 Polman, 2006, A randomized, placebo-controlled trial of natalizumab for relapsing multiple sclerosis, N Engl J Med, 354, 899, 10.1056/NEJMoa044397 McGuigan, 2015, Stratification and monitoring of natalizumab-associated progressive multifocal leukoencephalopathy risk: recommendations from an expert group, J Neurol Neurosurg Psychiatr, 87, 117 Vukusic, 2019, Progressive multifocal leukoencephalopathy incidence and risk stratification among natalizumab users in France, JAMA Neurol, 77, 1 Jeffery, 2016, Fingolimod: a review of its mode of action in the context of its efficacy and safety profile in relapsing forms of multiple sclerosis, Expert Rev Neurother, 16, 31, 10.1586/14737175.2016.1123094 Cohen, 2010, Oral fingolimod or intramuscular interferon for relapsing multiple sclerosis, N Engl J Med, 362, 402, 10.1056/NEJMoa0907839 Rammohan, 2020, The development of cladribine tablets for the treatment of multiple sclerosis: a comprehensive review, Drugs, 80, 1901, 10.1007/s40265-020-01422-9 Giovannoni, 2010, A placebo-controlled trial of oral cladribine for relapsing multiple sclerosis, N Engl J Med, 362, 416, 10.1056/NEJMoa0902533 Giovannoni, 2018, Safety and efficacy of cladribine tablets in patients with relapsing–remitting multiple sclerosis: results from the randomized extension trial of the CLARITY study, Mult Scler, 24, 1594, 10.1177/1352458517727603 Giovannoni, 2019, Efficacy of cladribine tablets in high disease activity subgroups of patients with relapsing multiple sclerosis: a post hoc analysis of the CLARITY study, Mult Scler, 25, 819, 10.1177/1352458518771875 Bayry, 2015, IVIg for relapsing–remitting multiple sclerosis: promises and uncertainties, Trends Pharmacol Sci, 36, 419, 10.1016/j.tips.2015.04.012 Brochet, 2020, Double-blind, randomized controlled trial of therapeutic plasma exchanges vs. sham exchanges in moderate-to-severe relapses of multiple sclerosis, J Clin Apher, 35, 281, 10.1002/jca.21788 Hauser, 2008, B-cell depletion with rituximab in relapsing-remitting multiple sclerosis, N Engl J Med, 358, 676, 10.1056/NEJMoa0706383 Alping, 2016, Rituximab versus fingolimod after natalizumab in multiple sclerosis patients, Ann Neurol, 79, 950, 10.1002/ana.24651 Boremalm, 2019, Natalizumab, rituximab and fingolimod as escalation therapy in multiple sclerosis, Eur J Neurol, 275, 364 Salzer, 2016, Rituximab in multiple sclerosis: a retrospective observational study on safety and efficacy, Neurology, 87, 2074, 10.1212/WNL.0000000000003331 Fassas, 1997, Peripheral blood stem cell transplantation in the treatment of progressive multiple sclerosis: first results of a pilot study, Bone Marrow Transplant, 20, 631, 10.1038/sj.bmt.1700944 Mariottini, 2018, Safety and efficacy of autologous hematopoietic stem-cell transplantation following natalizumab discontinuation in aggressive multiple sclerosis, Eur J Neurol, 4, 329 Moore, 2019, Prospective phase II clinical trial of autologous haematopoietic stem cell transplant for treatment refractory multiple sclerosis, J Neurol Neurosurg Psychiatry, 90, 514, 10.1136/jnnp-2018-319446 Nash, 2014, High-dose Immunosuppressive therapy and autologous hematopoietic cell transplantation for relapsing-remitting multiple sclerosis (HALT-MS), JAMA Neurol [Internet] Zhukovsky, 2021, Autologous haematopoietic stem cell transplantation compared with alemtuzumab for relapsing–remitting multiple sclerosis: an observational study, J Neurol Neurosurg Psychiatry, 92, 189, 10.1136/jnnp-2020-323992 Harding, 2019, Clinical outcomes of escalation vs. early intensive disease-modifying therapy in patients with multiple sclerosis, JAMA Neurol, 76, 536, 10.1001/jamaneurol.2018.4905 Iaffaldano, 2021, Long-term disability trajectories in relapsing multiple sclerosis patients treated with early intensive or escalation treatment strategies, Ther Adv Neurol Disord, 14, 10.1177/17562864211019574 Spelman, 2021, Treatment escalation vs. immediate initiation of highly effective treatment for patients with relapsing-remitting multiple sclerosis: data from 2 different national strategies, JAMA Neurol, 78, 1197, 10.1001/jamaneurol.2021.2738 He, 2020, Timing of high-efficacy therapy for multiple sclerosis: a retrospective observational cohort study, Lancet Neurol, 19, 307, 10.1016/S1474-4422(20)30067-3 Stankiewicz, 2019, An argument for broad use of high efficacy treatments in early multiple sclerosis, Neurol Neuroimmunol Neuroinflammation, 7, e636, 10.1212/NXI.0000000000000636 Wiendl, 2021, Multiple Sclerosis Therapy Consensus Group (MSTCG): position statement on disease-modifying therapies for multiple sclerosis (white paper), Ther Adv Neurol Disord, 14, 10.1177/17562864211039648 Ontaneda, 2020, Determining the effectiveness of early intensive versus escalation approaches for the treatment of relapsing-remitting multiple sclerosis: the DELIVER-MS study protocol, Contemp Clin Trials, 95, 106009, 10.1016/j.cct.2020.106009 Yano, 2019, Discontinuation of disease-modifying therapy for patients with relapsing-remitting multiple sclerosis: effect on clinical and MRI outcomes, Mult Scler Relat Disord, 35, 119, 10.1016/j.msard.2019.07.021 Kaminsky, 2020, Discontinuation of disease-modifying treatments for multiple sclerosis in patients aged over 50 with disease inactivity, J Neurol, 267, 3518, 10.1007/s00415-020-10029-9 McFaul, 2021, Defining benign/burnt-out MS and discontinuing disease-modifying therapies, Neurol Neuroimmunol Neuroinflamm, 8, e960, 10.1212/NXI.0000000000000960 Hartung, 2021, Stopping disease-modifying therapy in relapsing and progressive multiple sclerosis, Curr Opin Neurol [Internet], 10.1097/WCO.0000000000000960 Boremalm, 2021, Discontinuation and dose reduction of rituximab in relapsing–remitting multiple sclerosis, J Neurol, 268, 2161, 10.1007/s00415-021-10399-8 Vukusic, 2004, Pregnancy and multiple sclerosis (the PRIMS study): clinical predictors of post-partum relapse, Brain, 127, 1353, 10.1093/brain/awh152 Vukusic, 2015, Multiple sclerosis and pregnancy in the “treatment era”, Nat Rev Neurol, 11, 280, 10.1038/nrneurol.2015.53 Vukusic, 2021, Pregnancy with multiple sclerosis, Rev Neurol, 177, 180, 10.1016/j.neurol.2020.05.005 Mahad, 2015, Progressive multiple sclerosis 1. Pathological mechanisms in progressive multiple sclerosis, Lancet Neurol, 14, 183, 10.1016/S1474-4422(14)70256-X Feinstein, 2015, Progressive multiple sclerosis 2. Treatment of progressive multiple sclerosis: what works, what does not, and what is needed, Lancet Neurol, 14, 194, 10.1016/S1474-4422(14)70231-5 Ontaneda, 2015, Progressive multiple sclerosis 3. Clinical trials in progressive multiple sclerosis: lessons learned and future perspectives, Lancet Neurol, 14, 208, 10.1016/S1474-4422(14)70264-9 Lublin, 2014, Defining the clinical course of multiple sclerosis: the 2013 revisions, Neurology, 83, 278, 10.1212/WNL.0000000000000560 Montalban, 2016, Ocrelizumab versus placebo in primary progressive multiple sclerosis, N Engl J Med, 376, 209, 10.1056/NEJMoa1606468 Wolinsky, 2020, Long-term follow-up from the ORATORIO trial of ocrelizumab for primary progressive multiple sclerosis: a post-hoc analysis from the ongoing open-label extension of the randomised, placebo-controlled, phase 3 trial, Lancet Neurol, 19, 998, 10.1016/S1474-4422(20)30342-2 Kappos, 2018, Articles Siponimod versus placebo in secondary progressive multiple sclerosis (EXPAND): a double-blind, randomised, phase 3 study, Lancet, 391, 1263, 10.1016/S0140-6736(18)30475-6 Koch, 2021, Hydroxychloroquine for primary progressive multiple sclerosis, Ann Neurol [Internet], 10.1002/ana.26239 Cree, 2020, Safety and efficacy of MD1003 (high-dose biotin) in patients with progressive multiple sclerosis (SPI2): a randomised, double-blind, placebo-controlled, phase 3 trial, Lancet Neurol, 19, 988, 10.1016/S1474-4422(20)30347-1 De Angelis, 2019, Novel multiple sclerosis drugs in the pipeline, Clin Pharmacol Ther, 105, 1082, 10.1002/cpt.1412 Montalban, 2019, Placebo-controlled trial of an oral BTK inhibitor in multiple sclerosis, N Engl J Med, 380, 2406, 10.1056/NEJMoa1901981 Reich, 2021, Safety and efficacy of tolebrutinib, an oral brain-penetrant BTK inhibitor, in relapsing multiple sclerosis: a phase 2b, randomised, double-blind, placebo-controlled trial, Lancet Neurol, 20, 10, 10.1016/S1474-4422(21)00237-4 Uccelli, 2021, Safety, tolerability, and activity of mesenchymal stem cells versus placebo in multiple sclerosis (MESEMS): a phase 2, randomised, double-blind crossover trial, Lancet Neurol, 20, 13, 10.1016/S1474-4422(21)00301-X Geladaris, 2021, Microglia: the missing link to decipher and therapeutically control MS progression?, IJMS, 22, 3461, 10.3390/ijms22073461 Bodini, 2016, Dynamic imaging of individual remyelination profiles in multiple sclerosis, Ann Neurol, 79, 726, 10.1002/ana.24620 Kolb, 2021, 7T MRI differentiates remyelinated from demyelinated multiple sclerosis lesions, Ann Neurol, 90, 612, 10.1002/ana.26194 Green, 2017, Clemastine fumarate as a remyelinating therapy for multiple sclerosis (ReBUILD): a randomised, controlled, double-blind, crossover trial, Lancet, 390, 2481, 10.1016/S0140-6736(17)32346-2 Lubetzki, 2020, Remyelination in multiple sclerosis: from basic science to clinical translation, Lancet Neurol, 19, 678, 10.1016/S1474-4422(20)30140-X