Review of approved NMO therapies based on mechanism of action, efficacy and long-term effects
Tài liệu tham khảo
Jiao, 2013, Updated estimate of AQP4-IgG serostatus and disability outcome in neuromyelitis optica, Neurology, 81, 1197, 10.1212/WNL.0b013e3182a6cb5c
Jarius, 2012, Contrasting disease patterns in seropositive and seronegative neuromyelitis optica: a multicentre study of 175 patients, J Neuroinflammation, 9, 14, 10.1186/1742-2094-9-14
Ajmera, 2018, Evaluation of comorbidities and health care resource use among patients with highly active neuromyelitis optica, J Neurol Sci, 384, 96, 10.1016/j.jns.2017.11.022
Kitley, 2012, Prognostic factors and disease course in aquaporin-4 antibody-positive patients with neuromyelitis optica spectrum disorder from the United Kingdom and Japan, Brain, 135, 1834, 10.1093/brain/aws109
Mealy, 2018, Mortality in neuromyelitis optica is strongly associated with African ancestry, Neurol Neuroimmunol Neuroinflamm, 5, e468, 10.1212/NXI.0000000000000468
Beekman, 2019, Neuromyelitis optica spectrum disorder: patient experience and quality of life, Neurol Neuroimmunol Neuroinflamm, 6, e580, 10.1212/NXI.0000000000000580
Papp, 2018, Nationwide prevalence and incidence study of neuromyelitis optica spectrum disorder in Denmark, Neurology, 91, e2265, 10.1212/WNL.0000000000006645
Mealy, 2019, Long-term disability in neuromyelitis optica spectrum disorder with a history of myelitis is associated with age at onset, delay in diagnosis/preventive treatment, MRI lesion length and presence of symptomatic brain lesions, Mult Scler Relat Disord, 28, 64, 10.1016/j.msard.2018.12.011
Wingerchuk, 1999, The clinical course of neuromyelitis optica (Devic’s syndrome), Neurology, 53, 1107, 10.1212/WNL.53.5.1107
Wingerchuk, 2007, The spectrum of neuromyelitis optica, Lancet Neurol, 6, 805, 10.1016/S1474-4422(07)70216-8
Hinson, 2016, Autoimmune AQP4 channelopathies and neuromyelitis optica spectrum disorders, Handb Clin Neurol, 133, 377, 10.1016/B978-0-444-63432-0.00021-9
Hyun, 2015, Idiopathic aquaporin-4 antibody negative longitudinally extensive transverse myelitis, Mult Scler, 21, 710, 10.1177/1352458514551454
Bernard-Valnet, 2015, Neuromyelitis optica: a positive appraisal of seronegative cases, Eur J Neurol., 22, 1511, 10.1111/ene.12679
Melamed, 2015, Update on biomarkers in neuromyelitis optica, Neurol Neuroimmunol Neuroinflamm, 2, e134, 10.1212/NXI.0000000000000134
Narayan, 2018, MOG antibody disease: a review of MOG antibody seropositive neuromyelitis optica spectrum disorder, Mult Scler Relat Disord, 25, 66, 10.1016/j.msard.2018.07.025
Hamid, 2017, The impact of 2015 neuromyelitis optica spectrum disorders criteria on diagnostic rates, Mult Scler., 23, 228, 10.1177/1352458516663853
Flanagan, 2016, Epidemiology of aquaporin-4 autoimmunity and neuromyelitis optica spectrum, Ann Neurol., 79, 775, 10.1002/ana.24617
Badaut, 2000, Presence of aquaporin-4 and muscarinic receptors in astrocytes and ependymal cells in rat brain: a clue to a common function?, Neurosci Lett., 292, 75, 10.1016/S0304-3940(00)01364-1
Amiry-Moghaddam, 2000, Aquaporins and water homeostasis in the central nervous system, Comparative Biochemistry and Physiology, Part A124, 10.1016/S1095-6433(00)80225-1
Verkman, 2017, The aquaporin-4 water channel as a potential drug target in neurological disorders, Expert Opin. Ther. Targets, 21, 1161, 10.1080/14728222.2017.1398236
Pittock, 2006, Neuromyelitis optica brain lesions localized at sites of high aquaporin 4 expression, Arch Neurol., 63, 964, 10.1001/archneur.63.7.964
Filippini, 2000, Corticosteroids or ACTH for acute exacerbations in multiple sclerosis, Cochrane Database Syst Rev.
Watanabe, 2007, Therapeutic efficacy of plasma exchange in NMO-IgG-positive patients with neuromyelitis optica, Mult Scler., 13, 128, 10.1177/1352458506071174
Kleiter, 2018, Apheresis therapies for NMOSD attacks: a retrospective study of 207 therapeutic interventions, Neurol Neuroimmunol Neuroinflamm, 5, e504, 10.1212/NXI.0000000000000504
Elsone, 2014, Role of intravenous immunoglobulin in the treatment of acute relapses of neuromyelitis optica: experience in 10 patients, Mult Scler., 20, 501, 10.1177/1352458513495938
Greenberg, 2007, Idiopathic transverse myelitis: corticosteroids, plasma exchange, or cyclophosphamide, Neurology, 68, 1614, 10.1212/01.wnl.0000260970.63493.c8
Bichuetti, 2010, Neuromyelitis optica treatment: analysis of 36 patients, Arch Neurol., 67, 1131, 10.1001/archneurol.2010.203
Mandler, 1998, Devic’s neuromyelitis optica: a prospective study of seven patients treated with prednisone and azathioprine, Neurology., 51, 1219, 10.1212/WNL.51.4.1219
Jacob, 2009, Treatment of neuromyelitis optica with mycophenolate mofetil: retrospective analysis of 24 patients, Arch Neurol., 66, 1128, 10.1001/archneurol.2009.175
Cree, 2005, An open label study of the effects of rituximab in neuromyelitis optica, Neurology., 64, 1270, 10.1212/01.WNL.0000159399.81861.D5
Jacob, 2008, Treatment of neuromyelitis optica with rituximab: retrospective analysis of 25 patients, Arch Neurol., 65, 1443, 10.1001/archneur.65.11.noc80069
Clifford, 2011, Rituximab-associated progressive multifocal leukoencephalopathy in rheumatoid arthritis, Arch Neurol., 68, 1156, 10.1001/archneurol.2011.103
Zephir, 2015, Rituximab as first-line therapy in neuromyelitis optica: efficiency and tolerability, J Neurol., 262, 2329, 10.1007/s00415-015-7852-y
Kim, 2013, A 5-year follow-up of rituximab treatment in patients with neuromyelitis optica spectrum disorder, JAMA Neurol., 70, 1110, 10.1001/jamaneurol.2013.3071
Pellkofer, 2011, Long-term follow-up of patients with neuromyelitis optica after repeated therapy with rituximab, Neurology., 76, 1310, 10.1212/WNL.0b013e3182152881
Damato, 2016, Efficacy and Safety of Rituximab Therapy in Neuromyelitis Optica Spectrum Disorders: a Systematic Review and Meta-analysis, JAMA Neurol., 73, 1342, 10.1001/jamaneurol.2016.1637
Gao, 2019, Effectiveness of rituximab in neuromyelitis optica: a meta-analysis, BMC Neurol, 19, 36, 10.1186/s12883-019-1261-2
Lindsey, 2012, Variable results after rituximab in neuromyelitis optica, J Neurol Sci., 317, 103, 10.1016/j.jns.2012.02.017
Magraner, 2013, The effect of intravenous immunoglobulin on neuromyelitis optica, Neurologia., 28, 65, 10.1016/j.nrl.2012.03.014
Yaguchi, 2013, Efficacy of intravenous cyclophosphamide therapy for neuromyelitis optica spectrum disorder, Intern Med., 52, 969, 10.2169/internalmedicine.52.7885
Kim, 2011, Efficacy and safety of mitoxantrone in patients with highly relapsing neuromyelitis optica, Arch Neurol., 68, 473, 10.1001/archneurol.2010.322
Weinstock-Guttman, 2006, Study of mitoxantrone for the treatment of recurrent neuromyelitis optica (Devic disease), Arch Neurol., 63, 957, 10.1001/archneur.63.7.957
Gelfand, 2014, Massive CNS monocytic infiltration at autopsy in an alemtuzumab-treated patient with NMO, Neurol Neuroimmunol Neuroinflamm, 1, e34, 10.1212/NXI.0000000000000034
Kleiter, 2012, Failure of natalizumab to prevent relapses in neuromyelitis optica, Arch Neurol., 69, 239, 10.1001/archneurol.2011.216
Shimizu, 2010, IFNbeta-1b may severely exacerbate Japanese optic-spinal MS in neuromyelitis optica spectrum, Neurology., 75, 1423, 10.1212/WNL.0b013e3181f8832e
Papeix, 2007, Immunosuppressive therapy is more effective than interferon in neuromyelitis optica, Mult Scler., 13, 256, 10.1177/1352458506070732
Min, 2012, Development of extensive brain lesions following fingolimod (FTY720) treatment in a patient with neuromyelitis optica spectrum disorder, Mult Scler., 18, 113, 10.1177/1352458511431973
Ringelstein, 2015, Long-term Therapy With Interleukin 6 Receptor Blockade in Highly Active Neuromyelitis Optica Spectrum Disorder, JAMA Neurol., 72, 756, 10.1001/jamaneurol.2015.0533
Araki, 2014, Efficacy of the anti-IL-6 receptor antibody tocilizumab in neuromyelitis optica: a pilot study, Neurology., 82, 1302, 10.1212/WNL.0000000000000317
Dunkelberger, 2010, Complement and its role in innate and adaptive immune responses, Cell Res., 20, 34, 10.1038/cr.2009.139
Noris, 2012, STEC-HUS, atypical HUS and TTP are all diseases of complement activation, Nat Rev Nephrol., 8, 622, 10.1038/nrneph.2012.195
52 Hill A., Kelly R.J., Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. 1392-1397. 2013;121(25):4985–4996; quiz 5105.
Wingerchuk, 2017, Neuromyelitis optica spectrum disorder diagnostic criteria: sensitivity and specificity are both important, Mult Scler., 23, 182, 10.1177/1352458516688352
Herwerth, 2016, In vivo imaging reveals rapid astrocyte depletion and axon damage in a model of neuromyelitis optica-related pathology, Ann Neurol., 79, 794, 10.1002/ana.24630
Jasiak-Zatonska, 2016, The Immunology of Neuromyelitis Optica-Current Knowledge, Clinical Implications, Controversies and Future Perspectives, Int J Mol Sci., 17, 273, 10.3390/ijms17030273
Bradl, 2018, Mechanisms for lesion localization in neuromyelitis optica spectrum disorders, Curr. Opin. Neurol., 31, 325, 10.1097/WCO.0000000000000551
Bennett, 2015, B lymphocytes in neuromyelitis optica, Neurol Neuroimmunol Neuroinflamm., 2, e104, 10.1212/NXI.0000000000000104
Petersone, 2018, T Cell/B Cell Collaboration and Autoimmunity: an Intimate Relationship, Front Immunol., 9, 1941, 10.3389/fimmu.2018.01941
Takeshita, 2017, Effects of neuromyelitis optica-IgG at the blood-brain barrier in vitro, Neurol Neuroimmunol Neuroinflamm., 4, e311, 10.1212/NXI.0000000000000311
Ratelade, 2012, Neuromyelitis optica: aquaporin-4 based pathogenesis mechanisms and new therapies, Int J Biochem Cell Biol., 44, 1519, 10.1016/j.biocel.2012.06.013
Agasing, 2020, Transcriptomics and proteomics reveal a cooperation between interferon and T-helper 17 cells in neuromyelitis optica, Nat Commun., 11, 2856, 10.1038/s41467-020-16625-7
Kaneko, 2018, CSF cytokine profile in MOG-IgG+ neurological disease is similar to AQP4-IgG+ NMOSD but distinct from MS: a cross-sectional study and potential therapeutic implications, J Neurol Neurosurg Psychiatry., 89, 927, 10.1136/jnnp-2018-317969
Pittock, 2019, Eculizumab in Aquaporin-4-Positive Neuromyelitis Optica Spectrum Disorder, N Engl J Med., 10.1056/NEJMoa1900866
Duchow, 2020, Emerging drugs for the treatment of neuromyelitis optica, Expert Opin Emerg Drugs., 10.1080/14728214.2020.1803828
65 Nishimoto N., Terao K., Mima T., Nakahara H., Takagi N., Kakehi T. Mechanisms and pathologic significances in increase in serum interleukin-6 (IL-6) and soluble IL-6 receptor after administration of an anti-IL-6 receptor antibody, tocilizumab, in patients with rheumatoid arthritis and Castleman disease. 1392-1397. 2008;112(10):3959–3964.
Yokota, 2005, Clinical study of tocilizumab in children with systemic-onset juvenile idiopathic arthritis, Clin Rev Allergy Immunol, 28, 231, 10.1385/CRIAI:28:3:231
Smolen, 2008, Effect of interleukin-6 receptor inhibition with tocilizumab in patients with rheumatoid arthritis (OPTION study): a double-blind, placebo-controlled, randomised trial, Lancet., 371, 987, 10.1016/S0140-6736(08)60453-5
Nishimoto, 2008, 151
Paul-Pletzer, 2006, Tocilizumab: blockade of interleukin-6 signaling pathway as a therapeutic strategy for inflammatory disorders, Drugs Today (Barc)., 42, 559, 10.1358/dot.2006.42.9.1025692
Scheinecker, 2009, Tocilizumab, Nature reviews Drug discovery., 8, 273, 10.1038/nrd2863
Araki, 2013, Clinical improvement in a patient with neuromyelitis optica following therapy with the anti-IL-6 receptor monoclonal antibody tocilizumab, Mod Rheumatol., 23, 827, 10.3109/s10165-012-0715-9
Yamamura, 2019, Trial of Satralizumab in Neuromyelitis Optica Spectrum Disorder, N Engl J Med., 381, 2114, 10.1056/NEJMoa1901747
Bennett, 2019, Efficacy of satralizumab as monotherapy in pre-specified subgroups of SAkuraStar, a double-blind placebo-controlled phase 3 clinical study in patients with neuromyelitis optica spectrum disorder (NMOSD), J Neuro Sci., 10.1016/j.jns.2019.10.1109
Cree, 2019, Inebilizumab for the treatment of neuromyelitis optica spectrum disorder (N-MOmentum): a double-blind, randomised placebo-controlled phase 2/3 trial, Lancet., 10.1016/S0140-6736(19)31817-3
Cree, 2020, The N-MOmentum study – a randomized, placebo-controlled, double-blind trial of inebilizumab for neuromyelitis optica spectrum disorder: randomized controlled period and open-label extension results
76 www.ocrelizumabinfo.com. Ocrelizumab and COVID-19 Pharmacovigilance Data. https://www.ocrelizumabinfo.com/content/dam/gene/ocrelizumabinfo/pdfs/covid-19-pharmacovigilance-data.pdf. Published 2020. Accessed.
Forsthuber, 2018, B cell-based therapies in CNS autoimmunity: differentiating CD19 and CD20 as therapeutic targets, Ther Adv Neurol Disord., 11, 10.1177/1756286418761697
Alexopoulos, 2016, Anti-B-Cell Therapies in Autoimmune Neurological Diseases: rationale and Efficacy Trials, Neurotherapeutics., 13, 20, 10.1007/s13311-015-0402-6
Wilson, 2018, Condition-dependent generation of aquaporin-4 antibodies from circulating B cells in neuromyelitis optica, Brain., 141, 1063, 10.1093/brain/awy010
Atkins, 2013, Hematopoietic stem cell therapy for multiple sclerosis: top 10 lessons learned, Neurotherapeutics., 10, 68, 10.1007/s13311-012-0162-5
Mancardi, 2008, Autologous haematopoietic stem-cell transplantation in multiple sclerosis, Lancet Neurol, 7, 626, 10.1016/S1474-4422(08)70138-8
Sormani, 2017, Autologous hematopoietic stem cell transplantation in multiple sclerosis: a meta-analysis, Neurology., 88, 2115, 10.1212/WNL.0000000000003987
Aouad, 2015, Resolution of aquaporin-4 antibodies in a woman with neuromyelitis optica treated with human autologous stem cell transplant, J Clin Neurosci., 22, 1215, 10.1016/j.jocn.2015.02.007
Peng, 2010, A preliminary result of treatment of neuromyelitis optica with autologous peripheral hematopoietic stem cell transplantation, Neurologist., 16, 375, 10.1097/NRL.0b013e3181b126e3
Greco, 2015, Autologous hematopoietic stem cell transplantation in neuromyelitis optica: a registry study of the EBMT Autoimmune Diseases Working Party, Mult Scler., 21, 189, 10.1177/1352458514541978
Steinman, 2016, Restoring immune tolerance in neuromyelitis optica: part I, Neurol Neuroimmunol Neuroinflamm., 3, e276, 10.1212/NXI.0000000000000276
Bar-Or, 2016, Restoring immune tolerance in neuromyelitis optica: part II, Neurol Neuroimmunol Neuroinflamm., 3, e277, 10.1212/NXI.0000000000000277
Tappenden, 2010, Autologous haematopoietic stem cell transplantation for secondary progressive multiple sclerosis: an exploratory cost-effectiveness analysis, Bone Marrow Transplant., 45, 1014, 10.1038/bmt.2009.305
Massey, 2018, Regenerating Immunotolerance in Multiple Sclerosis with Autologous Hematopoietic Stem Cell Transplant, Front Immunol., 9, 410, 10.3389/fimmu.2018.00410
Brod, 2020, In MS: immunosuppression is passe, Mult Scler Relat Disord., 40, 10.1016/j.msard.2020.101967
Winthrop, 2015, Opportunistic infections and biologic therapies in immune-mediated inflammatory diseases: consensus recommendations for infection reporting during clinical trials and postmarketing surveillance, Ann Rheum Dis., 74, 2107, 10.1136/annrheumdis-2015-207841
Montalban, 2017, Ocrelizumab versus Placebo in Primary Progressive Multiple Sclerosis, N Engl J Med., 376, 209, 10.1056/NEJMoa1606468
93 OCREVUS PI-. 2020.
Mikulska, 2018, ESCMID Study Group for Infections in Compromised Hosts (ESGICH) Consensus Document on the safety of targeted and biological therapies: an infectious diseases perspective (Agents targeting lymphoid cells surface antigens [I]: CD19, CD20 and CD52), Clin Microbiol Infect., 24, S71, 10.1016/j.cmi.2018.02.003