{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,3]],"date-time":"2026-09-03T18:50:16Z","timestamp":1788461416279,"version":"build-2803163510"},"reference-count":28,"publisher":"Frontiers Media SA","license":[{"start":{"date-parts":[[2024,10,14]],"date-time":"2024-10-14T00:00:00Z","timestamp":1728864000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"content-domain":{"domain":["frontiersin.org"],"crossmark-restriction":true},"short-container-title":["Front. Neurol."],"abstract":"<jats:sec><jats:title>Background<\/jats:title><jats:p>Efgartigimod was approved in Japan in January 2022 for the treatment of generalized myasthenia gravis (gMG), regardless of antibody status. This case series describes a real-world experience in Japan of efgartigimod treatment for gMG patients with diverse backgrounds.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>We retrospectively analyzed the medical records of 16 Japanese patients (11 females and five males, mean age 40.4\u2009years) with gMG who received efgartigimod at the Kumamoto University Hospital between August 2022 and September 2023. The outcomes were Quantitative Myasthenia Gravis (QMG) responders (\u2265 3 point reduction), IgG levels, and change in prednisolone dose, in the first cycle of efgartigimod.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Fifteen patients completed one cycle of efgartigimod. Of the 14 patients for whom QMG scores were obtained, 10 patients were QMG responders. Four of the five patients with Myasthenia Gravis Foundation of America class V were QMG responders. Improvement in QMG after efgartigimod treatment was observed in one patient with myasthenic crisis and in one refractory patient who had unsuccessful eculizumab treatment. The mean reductions from baseline in IgG levels at weeks 1, 2, 3, and follow-up were 38.3, 56.1, 63.1, and 43.9%, respectively. A decrease in prednisolone dose was observed in seven patients. The most common adverse events were headache (three patients) and diarrhea (two patients). One patient discontinued efgartigimod treatment due to a treatment-related adverse event of rash.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Improvements in the outcomes of patients with gMG, including patients with severe gMG, myasthenic crisis, and refractory to anti-complementary therapy, were observed after the first cycle of efgartigimod treatment. Our real-world experience in Japan suggests the future possibilities for the treatment with efgartigimod for gMG with diverse backgrounds.<\/jats:p><\/jats:sec>","DOI":"10.3389\/fneur.2024.1472845","type":"journal-article","created":{"date-parts":[[2024,10,14]],"date-time":"2024-10-14T13:33:31Z","timestamp":1728912811000},"update-policy":"https:\/\/doi.org\/10.3389\/crossmark-policy","source":"Crossref","is-referenced-by-count":17,"title":["Efgartigimod treatment for generalized myasthenia gravis: a single-center case series of 16 patients"],"prefix":"10.3389","volume":"15","author":[{"given":"Toshiya","family":"Nomura","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michie","family":"Imamura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Masao","family":"Imura","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hironori","family":"Mizutani","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mitsuharu","family":"Ueda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1965","published-online":{"date-parts":[[2024,10,14]]},"reference":[{"key":"ref1","doi-asserted-by":"publisher","first-page":"111793","DOI":"10.1016\/j.mad.2023.111793","article-title":"Sex differences in neuromuscular disorders","volume":"211","author":"Vinciguerra","year":"2023","journal-title":"Mech Ageing Dev"},{"key":"ref2","doi-asserted-by":"publisher","first-page":"419","DOI":"10.1212\/WNL.0000000000002790","article-title":"International consensus guidance for management of myasthenia gravis: executive summary","volume":"87","author":"Sanders","year":"2016","journal-title":"Neurology"},{"key":"ref3","doi-asserted-by":"publisher","first-page":"114","DOI":"10.1212\/WNL.0000000000011124","article-title":"International consensus guidance for management of myasthenia gravis: 2020 update","volume":"96","author":"Narayanaswami","year":"2021","journal-title":"Neurology"},{"key":"ref4","doi-asserted-by":"publisher","first-page":"976","DOI":"10.1016\/S1474-4422(17)30369-1","article-title":"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","volume":"16","author":"Howard","year":"2017","journal-title":"Lancet Neurol"},{"key":"ref5","doi-asserted-by":"publisher","first-page":"e610","DOI":"10.1212\/WNL.0000000000011207","article-title":"Post-intervention status in patients with refractory myasthenia gravis treated with eculizumab during REGAIN and its open-label extension","volume":"96","author":"Mantegazza","year":"2021","journal-title":"Neurology"},{"key":"ref6","doi-asserted-by":"publisher","first-page":"526","DOI":"10.1016\/S1474-4422(21)00159-9","article-title":"Safety, efficacy, and tolerability of efgartigimod in patients with generalised myasthenia gravis (ADAPT): a multicentre, randomised, placebo-controlled, phase 3 trial","volume":"20","author":"Howard","year":"2021","journal-title":"Lancet Neurol"},{"key":"ref7","doi-asserted-by":"publisher","first-page":"4372","DOI":"10.1172\/JCI97911","article-title":"Neonatal fc receptor antagonist efgartigimod safely and sustainably reduces IgGs in humans","volume":"128","author":"Ulrichts","year":"2018","journal-title":"J Clin Invest"},{"key":"ref8","doi-asserted-by":"publisher","first-page":"e2661","DOI":"10.1212\/WNL.0000000000007600","article-title":"Randomized phase 2 study of FcRn antagonist efgartigimod in generalized myasthenia gravis","volume":"92","author":"Howard","year":"2019","journal-title":"Neurology"},{"key":"ref9","year":"2024"},{"key":"ref10","year":"2024"},{"key":"ref11","year":"2024"},{"key":"ref12","doi-asserted-by":"publisher","first-page":"e16189","DOI":"10.1111\/ene.16189","article-title":"Efgartigimod in generalized myasthenia gravis: a real-life experience at a national reference center","volume":"31","author":"Frangiamore","year":"2024","journal-title":"Eur J Neurol"},{"key":"ref13","doi-asserted-by":"publisher","first-page":"762","DOI":"10.1002\/mus.27974","article-title":"Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice","volume":"68","author":"Katyal","year":"2023","journal-title":"Muscle Nerve"},{"key":"ref14","doi-asserted-by":"publisher","first-page":"87","DOI":"10.1002\/mus.28003","article-title":"Single institution experience with efgartigimod in patients with myasthenia gravis: patient selection, dosing schedules, treatment response, and adverse events","volume":"69","author":"Singer","year":"2024","journal-title":"Muscle Nerve"},{"key":"ref15","doi-asserted-by":"publisher","first-page":"467","DOI":"10.1002\/mus.28042","article-title":"Retrospective review of patients with myasthenia gravis switched from plasma exchange therapy to efgartigimod treatment","volume":"69","author":"Mehrabyan","year":"2024","journal-title":"Muscle Nerve"},{"key":"ref16","doi-asserted-by":"publisher","first-page":"3462","DOI":"10.1007\/s00415-024-12293-5","article-title":"Real-world experience with efgartigimod in patients with myasthenia gravis","volume":"271","author":"Fuchs","year":"2024","journal-title":"J Neurol"},{"key":"ref17","doi-asserted-by":"publisher","first-page":"e200276","DOI":"10.1212\/CPJ.0000000000200276","article-title":"Therapeutic responses to efgartigimod for generalized myasthenia gravis in Japan","volume":"14","author":"Suzuki","year":"2024","journal-title":"Neurol Clin Pract"},{"key":"ref18","doi-asserted-by":"publisher","first-page":"254","DOI":"10.1007\/s00415-023-11970-1","article-title":"Efgartigimod beyond myasthenia gravis: the role of FcRn-targeting therapies in stiff-person syndrome","volume":"271","author":"Stefano","year":"2024","journal-title":"J Neurol"},{"key":"ref19","doi-asserted-by":"publisher","first-page":"19","DOI":"10.1111\/cen3.12739","article-title":"The Japanese clinical guidelines 2022 for myasthenia gravis and Lambert\u2013Eaton myasthenic syndrome","volume":"14","author":"Murai","year":"2023","journal-title":"Clin Exp Neuro"},{"key":"ref20","year":"2024"},{"key":"ref21","doi-asserted-by":"publisher","first-page":"1487","DOI":"10.1212\/wnl.52.7.1487","article-title":"Myasthenia gravis activities of daily living profile","volume":"52","author":"Wolfe","year":"1999","journal-title":"Neurology"},{"key":"ref22","doi-asserted-by":"publisher","first-page":"769","DOI":"10.1111\/j.1749-6632.1998.tb11015.x","article-title":"Reliability testing of the quantitative myasthenia gravis score","volume":"841","author":"Barohn","year":"1998","journal-title":"Ann N Y Acad Sci"},{"key":"ref23","doi-asserted-by":"publisher","first-page":"341","DOI":"10.1007\/s40265-022-01678-3","article-title":"Efgartigimod: first approval","volume":"82","author":"Heo","year":"2022","journal-title":"Drugs"},{"key":"ref24","doi-asserted-by":"publisher","first-page":"1321058","DOI":"10.3389\/fneur.2024.1321058","article-title":"Case report: recovery from refractory myasthenic crisis to minimal symptom expression after add-on treatment with efgartigimod","volume":"15","author":"Watanabe","year":"2024","journal-title":"Front Neurol"},{"key":"ref25","doi-asserted-by":"publisher","first-page":"3707","DOI":"10.1007\/s10072-023-06900-y","article-title":"Starting eculizumab as rescue therapy in refractory myasthenic crisis","volume":"44","author":"Vinciguerra","year":"2023","journal-title":"Neurol Sci"},{"key":"ref26","doi-asserted-by":"publisher","first-page":"212","DOI":"10.1002\/mus.27326","article-title":"The use of eculizumab in ventilator-dependent myasthenia gravis patients","volume":"64","author":"Usman","year":"2021","journal-title":"Muscle Nerve"},{"key":"ref27","doi-asserted-by":"publisher","DOI":"10.1177\/17562864231213240","article-title":"Guideline for the management of myasthenic syndromes","volume":"16","author":"Wiendl","year":"2023","journal-title":"Ther Adv Neurol Disord"},{"key":"ref28","doi-asserted-by":"publisher","first-page":"1069","DOI":"10.1212\/WNL.0000000000004341","article-title":"Rituximab as treatment for anti-MuSK myasthenia gravis","volume":"89","author":"Hehir","year":"2017","journal-title":"Neurology"}],"container-title":["Frontiers in Neurology"],"original-title":[],"link":[{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2024.1472845\/full","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,10,14]],"date-time":"2024-10-14T13:33:32Z","timestamp":1728912812000},"score":1,"resource":{"primary":{"URL":"https:\/\/www.frontiersin.org\/articles\/10.3389\/fneur.2024.1472845\/full"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,10,14]]},"references-count":28,"alternative-id":["10.3389\/fneur.2024.1472845"],"URL":"https:\/\/doi.org\/10.3389\/fneur.2024.1472845","relation":{},"ISSN":["1664-2295"],"issn-type":[{"value":"1664-2295","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,10,14]]},"article-number":"1472845"}}