References
- Meriggioli MN, Sanders DB. Autoimmune myasthenia gravis: emerging clinical and biological heterogeneity. Lancet Neurol. 2009;8(5):475–490. doi: 10.1016/S1474-4422(09)70063-8
- Berrih-Aknin S, Frenkian-Cuvelier M, Eymard B. Diagnostic and clinical classification of autoimmune myasthenia gravis. J Autoimmun. 2014;48-49:143–148. doi: 10.1016/j.jaut.2014.01.003
- Sudres M, Verdier J, Truffault F, et al. Pathophysiological mechanisms of autoimmunity. Ann N Y Acad Sci. 2018;1413(1):59–68. doi: 10.1111/nyas.13560
- Dalakas MC. Future perspectives in target-specific immunotherapies of myasthenia gravis. Ther Adv Neurol Disord. 2015;8(6):316–327. doi: 10.1177/1756285615605700
- Menon D, Urra Pincheira A, Bril V. Emerging drugs for the treatment of myasthenia gravis. Expert Opin Emerg Drugs. 2021;26(3):259–270. doi: 10.1080/14728214.2021.1952982
- Sánchez-Tejerina D, Sotoca J, Llaurado A, et al. New targeted agents in myasthenia gravis and future therapeutic strategies. J Clin Med. 2022;11(21):6394. doi: 10.3390/jcm11216394
- Berrih-Aknin S, Le Panse R. Myasthenia gravis: a comprehensive review of immune dysregulation and etiological mechanisms. J Autoimmun. 2014;52:90–100. doi: 10.1016/j.jaut.2013.12.011
- Thiruppathi M, Rowin J, Li Jiang Q, et al. Functional defect in regulatory T cells in myasthenia gravis. Ann N Y Acad Sci. 2012;1274(1):68–76. doi: 10.1111/j.1749-6632.2012.06840.x
- Rødgaard A, Nielsen FC, Djurup R, et al. Acetylcholine receptor antibody in myasthenia gravis: predominance of IgG subclasses 1 and 3. Clin Exp Immunol. 1987;67(1):82–8.
- Hoch W, McConville J, Helms S, et al. Auto-antibodies to the receptor tyrosine kinase MuSK in patients with myasthenia gravis without acetylcholine receptor antibodies. Nat Med. 2001;7(3):365–368. doi: 10.1038/85520
- Meriggioli MN, Sanders DB. Muscle autoantibodies in myasthenia gravis: Beyond diagnosis? Expert Rev Clin Immunol. 2012;8(5):427–438. doi: 10.1586/eci.12.34
- Lazaridis K, Tzartos SJ. Autoantibody specificities in myasthenia gravis; implications for improved diagnostics and therapeutics. Front Immunol. 2020;11. doi: 10.3389/fimmu.2020.00212
- Narayanaswami P, Sanders DB, Wolfe G, et al. International consensus guidance for management of myasthenia gravis: 2020 update. Neurology. 2021;96(3):114–122. doi: 10.1212/WNL.0000000000011124
- Dalakas MC. Advances in the therapeutic algorithm for myasthenia gravis. Nat Rev Neurol. 2023;19(7):393–394. doi: 10.1038/s41582-023-00825-y
- Ramaswamy SB, Singh S, Hooshmand S, et al. Current and upcoming treatment modalities in myasthenia gravis. J Clin Neuromuscul Dis. 2021;23(2):75–99. doi: 10.1097/CND.0000000000000377
- Sathasivam S. Steroids and immunosuppressant drugs in myasthenia gravis. Nat Clin Pract Neurol. 2008;4(6):317–327. doi: 10.1038/ncpneuro0810
- Hartung HP. Advances in the understanding of the mechanism of action of IVIg. J Neurol. 2008;255(S3):3–6. doi: 10.1007/s00415-008-3002-0
- Dalakas MC. Intravenous Immunoglobulin in Autoimmune Neuromuscular Diseases. JAMA. 2004;291(19):2367–2375. doi: 10.1001/jama.291.19.2367
- Ipe TS, Davis AR, Raval JS. Therapeutic plasma exchange in myasthenia gravis: a systematic literature review and meta-analysis of comparative evidence. Front Neurol. 2021;12. doi: 10.3389/fneur.2021.662856
- Imai T. Why is development of new treatments necessary for myasthenia gravis? Recent advances in clinical trials. Neurol Clin Neurosci. 2019;7(4):161–165. doi: 10.1111/ncn3.12301
- Howard JF. Myasthenia gravis: the role of complement at the neuromuscular junction. Ann N Y Acad Sci. 2018;1412(1):113–128. doi: 10.1111/nyas.13522
- Yanagidaira M, Nishida Y, Yokota T. Temporal correlation between serum CH50 level and symptom severity of myasthenia gravis during eculizumab therapy. Clin Neurol Neurosur. 2020;189:189. doi: 10.1016/j.clineuro.2019.105630
- Huda R, Tüzün E, Christadoss P. Targeting complement system to treat myasthenia gravis. Rev Neurosci. 2014;25(4):575–583. doi: 10.1515/revneuro-2014-0021
- Lewis LA, Ram S. Meningococcal disease and the complement system. Virulence. 2014;5(1):98–126. doi: 10.4161/viru.26515
- Study details | a study to examine the efficacy and safety of Pozelimab and cemdisiran combination therapy in adult patients with symptomatic generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT05070858
- Prevail study | see if you may qualify | generalized myasthenia gravis study [accessed 2023 Dec 20]. Available from: https://prevailmgstudy.alexionclinicaltrials.com/
- Study details | study of ALXN2050 in adult participants with generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT05218096
- Kim MS, Prasad V. The clinical trials portfolio for on-label and off-label studies of eculizumab. JAMA Intern Med. 2020;180(2):315–317. doi: 10.1001/jamainternmed.2019.4694
- Howard JF, Barohn RJ, Cutter GR, et al. A randomized, double-blind, placebo-controlled phase II study of eculizumab in patients with refractory generalized myasthenia gravis. Muscle Nerve. 2013;48(1):76–84. doi: 10.1002/mus.23839
- Howard JF, Utsugisawa K, Benatar M, et al. Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study. Lancet Neurol. 2017;16(12):976–986. doi: 10.1016/S1474-4422(17)30369-1
- DeHart-McCoyle M, Patel S, Du X. New and emerging treatments for myasthenia gravis. BMJ Med. 2023;2(1):e000241. doi: 10.1136/bmjmed-2022-000241
- Nowak RJ, Muppidi S, Beydoun SR, et al. Concomitant Immunosuppressive Therapy Use in Eculizumab-Treated Adults With Generalized Myasthenia Gravis During the REGAIN Open-Label Extension Study. Front Neurol. 2020;11:556104. doi: 10.3389/fneur.2020.556104
- Hallstensen RF, Bergseth G, Foss S, et al. Eculizumab treatment during pregnancy does not affect the complement system activity of the newborn. Immunobiology. 2015;220(4):452–459. doi: 10.1016/j.imbio.2014.11.003
- Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs Eculizumab in Adult Patients with PNH Naive to Complement Inhibitors: The 301 Study. Blood. 2019;133(6):530–539. doi: 10.1182/blood-2018-09-876136
- Vu T, Meisel A, Mantegazza R, et al. Terminal Complement Inhibitor Ravulizumab in Generalized Myasthenia Gravis. NEJM Evidence. 2022;1(5). doi: 10.1056/evidoa2100066
- Ultomiris approved in the US for adults with generalised myasthenia gravis [accessed 2023 Dec 20]. Available from: https://www.astrazeneca.com/media-centre/press-releases/2022/ultomiris-approved-in-the-us-for-adults-with-generalised-myasthenia-gravis.html
- Zilucoplan | C172H278N24O55 | CID 133083018 - PubChem [accessed 2023 Dec 20]. Available from: https://pubchem.ncbi.nlm.nih.gov/compound/133083018
- Howard JF, Vissing J, Gilhus NE, et al. Zilucoplan: an investigational complement C5 inhibitor for the treatment of acetylcholine receptor autoantibody–positive generalized myasthenia gravis. Expert Opin Investig Drugs. 2021;30(5):483–493. doi: 10.1080/13543784.2021.1897567
- Howard JF, Nowak RJ, Wolfe GI, et al. Clinical effects of the self-administered subcutaneous complement inhibitor zilucoplan in patients with moderate to severe generalized myasthenia gravis: results of a phase 2 randomized, double-blind, placebo-controlled, multicenter clinical trial. JAMA Neurol. 2020;77(5):582–592. doi: 10.1001/jamaneurol.2019.5125
- Ra Pharmaceuticals Receives Orphan Drug Designation from the U.S. FDA for zilucoplan for the treatment of myasthenia gravis | business wire [accessed 2024 Jan 9]. Available from: https://www.businesswire.com/news/home/20190904005284/en/Ra-Pharmaceuticals-Receives-Orphan-Drug-Designation-from-the-U.S.-FDA-for-Zilucoplan-for-the-Treatment-of-Myasthenia-Gravis
- Juntas-Morales R, Masuda M, Howard JF, et al. Safety and efficacy of Zilucoplan in patients with generalised myasthenia gravis (RAISE): arandomised, double-blind, placebo-controlled, phase 3 study. Lancet Neurol. 2023;22(5):395–406. doi: 10.1016/S1474-4422(23)00080-7
- UCB announces U.S. FDA approval of ZILBRYSQ[®] (zilucoplan) for the treatment of adults with generalized myasthenia gravis | UCB [accessed 2023 Dec 20]. Available from: https://www.ucb.com/stories-media/Press-Releases/article/UCB-announces-US-FDA-approval-of-ZILBRYSQR-zilucoplan-for-the-treatment-of-adults-with-generalized-myasthenia-gravis
- Study details | open-label extension of Zilucoplan in subjects with generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT04225871
- Pyzik M, Kozicky LK, Gandhi AK, et al. The therapeutic age of the neonatal fc receptor. Nat Rev Immunol. 2023;23(7):415–432. doi: 10.1038/s41577-022-00821-1
- Gable KL, Guptill JT. Antagonism of the neonatal fc receptor as an emerging treatment for myasthenia gravis. Front Immunol. 2020;10:10. doi: 10.3389/fimmu.2019.03052
- Peter HH, Ochs HD, Cunningham-Rundles C, et al. Targeting FcRn for immunomodulation: benefits, risks, and practical considerations. J Allergy Clin Immunol. 2020;146(3):479–491.e5.
- A study of nipocalimab administered to adults with generalized myasthenia gravis - full text view - ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT04951622
- Study details | phase 3 study to assess the efficacy and safety of Batoclimab as induction and maintenance therapy in adult participants with generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT05403541
- Suzuki S, Uzawa A, Murai H. Efgartigimod for generalized myasthenia gravis with or without anti-acetylcholine receptor antibodies: a worldwide and Japanese perspective. Expert Rev Clin Immunol. 2022;18(12):1207–1215. doi: 10.1080/1744666X.2022.2136167
- Sivadasan A, Bril V. Clinical efficacy and safety of efgartigimod for treatment of myasthenia gravis. Immunotherapy. 2023;15(8):553–563. doi: 10.2217/imt-2022-0298
- Howard JF, Bril V, Burns TM, et al. Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis. Neurology. 2019;92(23):e2661–e2673. doi: 10.1212/WNL.0000000000007600
- Ulrichts P, Guglietta A, Dreier T, et al. Neonatal fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans. J Clin Investig. 2018;128(10):4372–4386. doi: 10.1172/JCI97911
- Howard JF Jr, Bril V, Vu T, et al. Safety, efficacy, and tolerability of Efgartigimod in patients with generalised myasthenia gravis (ADAPT): amulticentre, randomised, placebo-controlled, phase 3 trial. Lancet Neurol. 2021;20(7):526–536. doi: 10.1016/S1474-4422(21)00159-9.
- ADAPT/ADAPT+ study show improvements in myasthenia gravis efficacy scales with efgartigimod [accessed 2023 Dec 20]. Available from: https://www.neurologylive.com/view/adapt-study-show-improvements-myasthenia-gravis-efficacy-scales-efgartigimod
- Evaluating the Pharmacodynamic Noninferiority of Efgartigimod PH20 SC Administered Subcutaneously as Compared to Efgartigimod Administered Intravenously in Patients With Generalized Myasthenia Gravis - Full Text View - ClinicalTrials.gov [accessed 2023 Dec 20]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT04735432
- Subcutaneous Efgartigimod PH20 Demonstrates Efficacy for Generalized Myasthenia Gravis in Open Label ADAPT-SC+ Trial [accessed 2023 Dec 20]. Available from: https://www.neurologylive.com/view/subcutaneous-efgartigimod-ph20-demonstrates-efficacy-generalized-mg-open-label-adapt-sc-trial
- argenx Announces Positive Topline Phase 3 Data From ADAPT-SC Study Evaluating Subcutaneous Efgartigimod for Generalized Myasthenia Gravis | Business Wire [accessed 2023 Dec 20]. Available from: https://www.businesswire.com/news/home/20220321005941/en/argenx-Announces-Positive-Topline-Phase-3-Data-From-ADAPT-SC-Study-Evaluating-Subcutaneous-Efgartigimod-for-Generalized-Myasthenia-Gravis
- Efgartigimod Approved for Generalized Myasthenia Gravis [accessed 2023 Dec 20]. Available from: https://www.neurologylive.com/view/efgartigimod-approved-for-generalized-myasthenia-gravis
- FDA approves subcutaneous efgartigimod as treatment for generalized myasthenia gravis [accessed 2023 Dec 20]. Available from: https://www.neurologylive.com/view/fda-approves-subcutaneous-efgartigimod-vyvgart-hytrulo-generalized-myasthenia-gravis
- Bril V, Benatar M, Andersen H, et al. Efficacy and safety of Rozanolixizumab in moderate to severe generalized myasthenia gravis: a phase 2 randomized control trial. Neurology. 2021;96(6):E853–E865. doi: 10.1212/WNL.0000000000011108
- Bril V, Drużdż A, Grosskreutz J, et al. Articles safety and efficacy of Rozanolixizumab in patients with generalised myasthenia gravis (MycarinG): a randomised, double-blind, placebo-controlled, adaptive phase 3 study. Lancet Neurol. 2023;22(5):383–394. doi: 10.1016/S1474-4422(23)00077-7
- Janssen to highlight latest research from Nipocalimab Clinical Development Program to address unmet need in myasthenia gravis at AANEM 2023 meeting [accessed 2023 Dec 20]. Available from: https://www.jnj.com/janssen-to-highlight-latest-research-from-nipocalimab-clinical-development-program-to-address-unmet-need-in-myasthenia-gravis-at-aanem-2023-meeting
- Johnson & Johnson reports positive topline results for nipocalimab from a phase 3 pivotal study in generalized myasthenia gravis (gMG) and a phase 2 study in Sjögren’s disease (SjD) [accessed 2024 Mar 1]. Available from: https://www.jnj.com/media-center/press-releases/johnson-johnson-reports-positive-topline-results-for-nipocalimab-from-a-phase-3-pivotal-study-in-generalized-myasthenia-gravis-gmg-and-a-phase-2-study-in-sjogrens-disease-sjd
- Yan C, Duan RS, Yang H, et al. Therapeutic effects of Batoclimab in Chinese patients with generalized myasthenia gravis: a double-blinded, randomized, placebo-controlled phase II study. Neurol Ther. 2022;11(2):815–834. doi: 10.1007/s40120-022-00345-9
- Yi JS, Guptill JT, Stathopoulos P, et al. B cells in the pathophysiology of myasthenia gravis. Muscle Nerve. 2018;57(2):172–184. doi: 10.1002/mus.25973
- Musette P, Bouaziz JD. B cell modulation strategies in autoimmune diseases: New concepts. Front Immunol. 2018;9(APR). doi: 10.3389/fimmu.2018.00622
- Lee DSW, Rojas OL, Gommerman JL. B cell depletion therapies in autoimmune disease: advances and mechanistic insights. Nat Rev Drug Discov. 2021;20(3):179–199. doi: 10.1038/s41573-020-00092-2
- Weiner GJ. Rituximab: mechanism of action. Semin Hematol. 2010;47(2):115–123. doi: 10.1053/J.SEMINHEMATOL.2010.01.011
- Tandan R, Hehir MK, Waheed W, et al. Rituximab treatment of myasthenia gravis: a systematic review. Muscle Nerve. 2017;56(2):185–196. doi: 10.1002/mus.25597
- Nowak RJ, Coffey CS, Goldstein JM, et al. Phase 2 trial of Rituximab in acetylcholine receptor antibody-positive generalized myasthenia gravis: the BeatMG study. Neurology. 2022;98(4):E376–E389. doi: 10.1212/WNL.0000000000013121
- Piehl F, Eriksson-Dufva A, Budzianowska A, et al. Efficacy and safety of Rituximab for new-onset generalized myasthenia gravis: the RINOMAX Randomized Clinical Trial. JAMA Neurol. 2022;79(11):1105–1112. doi: 10.1001/jamaneurol.2022.2887
- Stathopoulos P, Kumar A, Heiden JAV, et al. Mechanisms underlying B cell immune dysregulation and autoantibody production in MuSK myasthenia gravis. Ann N Y Acad Sci. 2018;1412(1):154–165. doi: 10.1111/nyas.13535
- Hehir MK, Hobson-Webb LD, Benatar M, et al. Rituximab as treatment for anti-MuSK myasthenia gravis multicenter blinded prospective review. Neurology. 2017;89(10):1069–1077. doi: 10.1212/WNL.0000000000004341
- Das G, Damotte V, Gelfand JM, et al. Rituximab before and during pregnancy. Neurol Neuroimmunol Neuroinflamm. 2018;5(3). doi: 10.1212/nxi.0000000000000453
- Smith JB, Hellwig K, Fink K, et al. Rituximab, MS, and pregnancy. Neurol Neuroimmunol Neuroinflamm. 2020;7(4):E734. doi: 10.1212/NXI.0000000000000734
- Bennett CL, Focosi D, Socal MP, et al. Progressive multifocal leukoencephalopathy in patients treated with rituximab: a 20-year review from the Southern Network on adverse reactions. Lancet Haematol. 2021;8(8):e593–e604. doi: 10.1016/S2352-3026(21)00167-8
- Du FH, Mills EA, Mao-Draayer Y. Next-generation anti-CD20 monoclonal antibodies in autoimmune disease treatment. Autoimmun Highlights. 2017;8(1). doi: 10.1007/s13317-017-0100-y
- Alabbad S, AlGaeed M, Sikorski P, et al. Monoclonal antibody-based therapies for myasthenia gravis. BioDrugs. 2020;34(5):557–566. doi: 10.1007/s40259-020-00443-w
- Waters MJ, Field D, Ravindran J. Refractory myasthenia gravis successfully treated with ofatumumab. Muscle Nerve. 2019;60(6):E45–E47. doi: 10.1002/MUS.26707
- Study Details | Myasthenia Gravis Inebilizumab Trial | ClinicalTrials.gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT04524273
- Kister I, Gulati S, Boz C, et al. Neuromyelitis optica in patients with myasthenia gravis who underwent thymectomy. Arch Neurol. 2006;63(6):851–856. doi: 10.1001/archneur.63.6.851
- Scheibe F, Ostendorf L, Prüss H, et al. Daratumumab for treatment-refractory antibody-mediated diseases in neurology. Eur J Neurol. 2022;29(6):1847–1854. doi: 10.1111/ene.15266
- A study of TAK-079 in people with generalized myasthenia gravis - full text view - ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://classic.clinicaltrials.gov/ct2/show/NCT04159805
- Gomez Mancilla B, Meriggioli MN, Genge A, et al. Efficacy and safety of iscalimab, a novel anti-CD40 monoclonal antibody, in moderate-to-severe myasthenia gravis: a phase 2 randomized study. J Clin Neurosci. 2024;119:76–84. doi: 10.1016/j.jocn.2023.11.013
- Qin Y, Gao C, Luo J. Metabolism characteristics of Th17 and regulatory T cells in autoimmune diseases. Front Immunol. 2022;13:13. doi: 10.3389/fimmu.2022.828191
- Ishihara K, Hirano T. IL-6 in autoimmune disease and chronic inflammatory proliferative disease. Cytokine Growth Factor Rev. 2002;13(4–5):357–368. doi: 10.1016/S1359-6101(02)00027-8
- Study details | efficacy and safety of Tocilizumab in the treatment of generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT05067348
- Study details | a study to evaluate efficacy, safety, pharmacokinetics, and pharmacodynamics of satralizumab in patients with generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT04963270
- Papp KA, Leonardi C, Menter A, et al. Brodalumab, an anti-interleukin-17-receptor antibody for psoriasis. N Engl J Med. 2012;366(13):1181–1189. doi: 10.1056/NEJMoa1109017
- Langley RG, Elewski BE, Lebwohl M, et al. Secukinumab in plaque psoriasis — results of two phase 3 trials. N Engl J Med. 2014;371(4):326–338. doi: 10.1056/nejmoa1314258
- Study details | therapy of antibody-mediated autoimmune diseases by Bortezomib (TAVAB) | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT02102594
- Kohler S, Märschenz S, Grittner U, et al. Bortezomib in antibody-mediated autoimmune diseases (TAVAB): Study protocol for a unicentric, non-randomised, non-placebo controlled trial. BMJ Open. 2019;9(1):e024523. doi: 10.1136/BMJOPEN-2018-024523
- Field-Smith A, Morgan GJ, Davies FE. Bortezomib (VelcadeTM) in the Treatment of Multiple Myeloma. Ther Clin Risk Manag. 2006;2(3):271. doi: 10.2147/TCRM.2006.2.3.271
- Mackay F, Woodcock SA, Lawton P, et al. Mice transgenic for BAFF develop lymphocytic disorders along with autoimmune manifestations. J Exp Med. 1999;190(11):1697–1710. doi: 10.1084/JEM.190.11.1697
- Hewett K, Sanders DB, Grove RA, et al. Randomized study of adjunctive belimumab in participants with generalized myasthenia gravis. Neurology. 2018;90(16):E1425–E1434. doi: 10.1212/WNL.0000000000005323
- Study details | a study of TACI(Transmembrane activator and Calcium-modulator and Cyclophilin Ligand (CAML) Interactor)-antibody fusion protein injection (RC18) in subjects with systemic myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT04302103
- Mohamed AJ, Yu L, Bäckesjö CM, et al. Bruton’s tyrosine kinase (Btk): function, regulation, and transformation with special emphasis on the PH domain. Immunol Rev. 2009;228(1):58–73. doi: 10.1111/J.1600-065X.2008.00741.X
- Study details | efficacy and safety of tolebrutinib (SAR442168) tablets in adult participants with generalized myasthenia gravis | ClinicalTrials.Gov [accessed 2024 Jan 9]. Available from: https://clinicaltrials.gov/study/NCT05132569
- Miliotou AN, Papadopoulou LC. CAR T-cell therapy: a new era in cancer immunotherapy. Curr Pharm Biotechnol. 2018;19(1):5–18. doi: 10.2174/1389201019666180418095526
- Cappell KM, Kochenderfer JN. Long-term outcomes following CAR T cell therapy: what we know so far. Nat Rev Clin Oncol. 2023;20(6):359–371. doi: 10.1038/s41571-023-00754-1
- Chen Y, Sun J, Liu H, et al. Immunotherapy deriving from CAR-T cell treatment in autoimmune diseases. J Immunol Res. 2019;2019:1–9. doi: 10.1155/2019/5727516
- Bonifant CL, Jackson HJ, Brentjens RJ, et al. Toxicity and management in CAR T-cell therapy. Mol Ther Oncolytics. 2016;3:16011. doi: 10.1038/MTO.2016.11
- Zhang C, Liu J, Zhong JF, et al. Engineering CAR-T cells. Biomark Res. 2017;5(1). doi: 10.1186/S40364-017-0102-Y
- Oh S, Mao X, Manfredo-Vieira S, et al. Precision targeting of autoantigen-specific B cells in muscle-specific tyrosine kinase myasthenia gravis with chimeric autoantibody receptor T cells. Nat Biotechnol. 2023;41(9):1229–1238. doi: 10.1038/S41587-022-01637-Z
- Granit V, Benatar M, Kurtoglu M, et al. Safety and clinical activity of autologous RNA chimeric antigen receptor T-Cell therapy in myasthenia gravis (MG-001): a prospective, multicentre, open-label, non-randomised phase 1b/2a study. Lancet Neurol. 2023;227:578–590. doi: 10.1016/S1474-4422(23)00194-1. Available from: www.thelancet.com/neurology
- Study details | open-label study to evaluate the safety of various dosing regimens of MuSK-CAART for MuSK myasthenia gravis | ClinicalTrials.Gov [accessed 2023 Dec 20]. Available from: https://www.clinicaltrials.gov/study/NCT05451212
- Brudno JN, Lam N, Vanasse D, et al. Safety and feasibility of anti-CD19 CAR T cells with fully human binding domains in patients with B-cell lymphoma. Nat Med. 2020;26(2):270–280. doi: 10.1038/S41591-019-0737-3
- Ng SA, Sullivan KM. Application of stem cell transplantation to autoimmune diseases. Curr Opin Hematol. 2019;26(6):392. doi: 10.1097/MOH.0000000000000531
- Rush CA, Atkins HL, Freedman MS. Autologous hematopoietic stem cell transplantation in the treatment of multiple sclerosis. Cold Spring Harb Perspect Med. 2019;9(3):a029082. doi: 10.1101/CSHPERSPECT.A029082
- Bryant A, Atkins H, Pringle CE, et al. Myasthenia gravis treated with autologous hematopoietic stem cell transplantation. JAMA Neurol. 2016;73(6):652–658. doi: 10.1001/JAMANEUROL.2016.0113
- Study Details | Autologous Stem Cell Transplant for Neurologic Autoimmune Diseases | ClinicalTrials.gov [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT00716066
- Rodríguez-Fuentes DE, Fernández-Garza LE, Samia-Meza JA, et al. Mesenchymal stem cells Current clinical applications: a systematic review. Arch Med Res. 2021;52(1):93–101. doi: 10.1016/J.ARCMED.2020.08.006
- Angelini C, Martignago M, Bisciglia B. New treatments for myasthenia: a focus on antisense oligonucleotides. Drug Des Devel Ther. 2013;7:13–17. doi: 10.2147/DDDT.S25716
- Lev-Lehman E, Evron T, Broide RS, et al. Synaptogenesis and myopathy under acetylcholinesterase overexpression. J Mol Neurosci. 2000;14(1–2):93–105. doi: 10.1385/JMN:14:1-2:093
- Sussman JD, Argov Z, McKee D, et al. Antisense treatment for myasthenia gravis: experience with monarsen. Ann N Y Acad Sci. 2008;1132(1):283–290. doi: 10.1196/ANNALS.1405.022
- Menon D, Barnett C, Bril V. Novel treatments in myasthenia gravis. Front Neurol. 2020;11:11. doi: 10.3389/fneur.2020.00538
- Bourque PR, Pringle CE, Cameron W, et al. Subcutaneous immunoglobulin therapy in the chronic management of myasthenia gravis: a retrospective cohort study. PLoS One. 2016;11(8):e0159993. doi: 10.1371/JOURNAL.PONE.0159993
- Beecher G, Anderson D, Siddiqi ZA. Subcutaneous immunoglobulin in myasthenia gravis exacerbation: A prospective, open-label trial. Neurology. 2017;89(11):1135–1141. doi: 10.1212/WNL.0000000000004365
- Yang H, Tüzün E, Alagappan D, et al. IL-1 receptor antagonist-mediated therapeutic effect in murine myasthenia gravis is associated with suppressed serum proinflammatory cytokines, C3, and anti-acetylcholine receptor IgG1. J Immunol. 2005;175(3):2018–2025. doi: 10.4049/JIMMUNOL.175.3.2018
- Tüzün E, Meriggioli MN, Rowin J, et al. Myasthenia gravis patients with low plasma IL-6 and IFN-gamma benefit from etanercept treatment. J Autoimmun. 2005;24(3):261–268. doi: 10.1016/J.JAUT.2005.01.013
- Taylor TRP, Galloway J, Davies R, et al. Demyelinating events following initiation of anti-TNFα therapy in the British Society for Rheumatology Biologics Registry in rheumatoid arthritis. Neurol Neuroimmunol Neuroinflamm. 2021;8(3). doi: 10.1212/NXI.0000000000000992
- Mantegazza R. Current and emerging therapies for the treatment of myasthenia gravis. Neuropsychiatr Dis Treat. 2011;7(1):151–160. doi: 10.2147/NDT.S8915
- Study details | safety and efficacy of tofacitinib in patients with refractory myasthenia gravis: a pilot study | ClinicalTrials.Gov. [accessed 2023 Dec 20]. Available from: https://clinicaltrials.gov/study/NCT04431895