{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,7]],"date-time":"2026-10-07T16:19:22Z","timestamp":1791389962319,"version":"4.3.3"},"reference-count":22,"publisher":"Springer Science and Business Media LLC","issue":"9","license":[{"start":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T00:00:00Z","timestamp":1722297600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"},{"start":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T00:00:00Z","timestamp":1722297600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0"}],"funder":[{"DOI":"10.13039\/100007195","name":"Universit\u00e0 degli Studi di Napoli Federico II","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100007195","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["J Neurol"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:title>Abstract<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Introduction<\/jats:title>\n                    <jats:p>Eculizumab, a complement active antibody, and efgartigimod, an Fc fragment that blocks neonatal Fc receptor, are both approved to treat generalized myasthenia gravis (gMG) patients. The objective of this study is to describe the clinical response to both treatments in a real-life setting.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>We collected baseline and follow-up clinical data using the Myasthenia Gravis-Activities of Daily Living (MG-ADL), and Quantitative Myasthenia Gravis (QMG). We included 63 patients, 32 treated with eculizumab and 31 with efgartigimod. Of the efgartigimod-treated patients, 22 were anti-acetylcholine receptor antibody-positive (AChR-Ab\u2009+) and 9 were AChR-Ab- (3 MuSK-Ab\u2009+\u2009and 6 seronegative).<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Both treatments showed similar efficacy relative to the MG-ADL scale reduction (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.237). Efgartigimod had a similar effect on both AChR-Ab\u2009+\u2009and AChR-Ab- (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.280). Eculizumab was superior to efgartigimod relative to the QMG score reduction for the entire dataset (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.003) and was more likely to achieve a clinical response at the QMG compared to efgartigimod (OR 1.373;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.016). Steroid-sparing effect was higher for eculizumab than efgartigimod (\u2009\u2013\u200916.7 vs \u2009\u2013\u20095.2\u00a0mg of the baseline daily dose at follow-up;\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.001). Mean speed of prednisone reduction was \u2009\u2013\u200913.1\u00a0mg of the daily dose for each month of follow-up for eculizumab-treated patients and \u2009\u2013\u20093.2 for efgartigimod (\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.001). We found three serious events, all not related to treatment in the investigator\u2019s opinion. One eculizumab-treated patient experienced a severe pneumonia and died despite treatment.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>Our study provides evidence that eculizumab and efgartigimod can be used in clinical practice to reduce disability in gMG patients. Eculizumab-treated patients had a higher QMG response and steroid sparing effect. Efgartigimod may offer a more flexible schedule due to its cyclical use, no need for vaccination, and efficacy in AChR-Ab- patients.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1007\/s00415-024-12588-7","type":"journal-article","created":{"date-parts":[[2024,7,30]],"date-time":"2024-07-30T14:32:12Z","timestamp":1722349932000},"page":"6209-6219","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":56,"title":["A real-life experience with eculizumab and efgartigimod in generalized myasthenia gravis patients"],"prefix":"10.1007","volume":"271","author":[{"given":"Chiara","family":"Pane","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Vincenzo","family":"Di Stefano","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nunzia","family":"Cuomo","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alessio","family":"Sarnataro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Claudia","family":"Vinciguerra","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liliana","family":"Bevilacqua","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Filippo","family":"Brighina","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicasio","family":"Rini","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giorgia","family":"Puorro","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Angela","family":"Marsili","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Matteo","family":"Garibaldi","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Laura","family":"Fionda","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1323-6317","authenticated-orcid":false,"given":"Francesco","family":"Sacc\u00e0","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,7,30]]},"reference":[{"key":"12588_CR1","doi-asserted-by":"publisher","first-page":"2570","DOI":"10.1056\/NEJMra1602678","volume":"375","author":"NE Gilhus","year":"2016","unstructured":"Gilhus NE (2016) Myasthenia gravis. N Engl J Med 375:2570\u20132581","journal-title":"N Engl J Med"},{"key":"12588_CR2","doi-asserted-by":"publisher","first-page":"1023","DOI":"10.1016\/S1474-4422(15)00145-3","volume":"14","author":"NE Gilhus","year":"2015","unstructured":"Gilhus NE, Verschuuren JJ (2015) Myasthenia gravis: subgroup classification and therapeutic strategies. Lancet Neurol 14:1023\u20131036","journal-title":"Lancet Neurol"},{"key":"12588_CR3","first-page":"255","volume":"86","author":"J Suh","year":"2013","unstructured":"Suh J, Goldstein JM, Nowak RJ (2013) Clinical characteristics of refractory myasthenia gravis patients. Yale J Biol Med 86:255\u2013260","journal-title":"Yale J Biol Med"},{"key":"12588_CR4","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16180","volume":"20","author":"F Sacc\u00e0","year":"2023","unstructured":"Sacc\u00e0 F, Salort-Campana E, Jacob S, Cort\u00e9s-Vicente E, Schneider-Gold C (2023) Refocusing generalized myasthenia gravis: Patient burden, disease profiles, and the role of evolving therapy. Eur J Neurol 20:e16180. https:\/\/doi.org\/10.1111\/ene.16180","journal-title":"Eur J Neurol"},{"key":"12588_CR5","doi-asserted-by":"publisher","first-page":"976","DOI":"10.1016\/S1474-4422(17)30369-1","volume":"16","author":"JF Howard Jr","year":"2017","unstructured":"Howard JF Jr, Utsugisawa K, Benatar M et al (2017) 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 16:976\u2013986","journal-title":"Lancet Neurol"},{"key":"12588_CR6","doi-asserted-by":"publisher","first-page":"526","DOI":"10.1016\/S1474-4422(21)00159-9","volume":"20","author":"JF Howard Jr","year":"2021","unstructured":"Howard JF Jr, Bril V, Vu T et al (2021) Safety, efficacy, and tolerability of efgartigimod in patients with generalized myasthenia gravis (ADAPT): a multicentre, randomized, placebo-controlled, phase 3 trial. Lancet Neurol 20:526\u2013536","journal-title":"Lancet Neurol"},{"key":"12588_CR7","doi-asserted-by":"publisher","first-page":"16","DOI":"10.1177\/1941875210382918","volume":"1","author":"LC Wendell","year":"2011","unstructured":"Wendell LC, Levine JM (2011) Myasthenic crisis. Neurohospitalist 1:16\u201322","journal-title":"Neurohospitalist"},{"key":"12588_CR8","doi-asserted-by":"publisher","first-page":"141","DOI":"10.1002\/mus.20950","volume":"37","author":"D Grob","year":"2008","unstructured":"Grob D, Brunner N, Namba T, Pagala M (2008) Lifetime course of myasthenia gravis. Muscle Nerve 37:141\u2013149","journal-title":"Muscle Nerve"},{"key":"12588_CR9","doi-asserted-by":"publisher","first-page":"3854","DOI":"10.1111\/ene.15872","volume":"30","author":"F Sacc\u00e0","year":"2023","unstructured":"Sacc\u00e0 F, Pane C, Espinosa PE, Sormani MP, Signori A (2023) Efficacy of innovative therapies in myasthenia gravis: a systematic review, meta-analysis and network meta-analysis. Eur J Neurol 30:3854\u20133867","journal-title":"Eur J Neurol"},{"key":"12588_CR10","doi-asserted-by":"publisher","first-page":"287","DOI":"10.3233\/JND-200584","volume":"8","author":"N Katyal","year":"2021","unstructured":"Katyal N, Narula N, Govindarajan R (2021) Clinical experience with eculizumab in treatment-refractory acetylcholine receptor antibody-positive generalized myasthenia gravis. J Neuromuscul Dis 8:287\u2013294","journal-title":"J Neuromuscul Dis"},{"key":"12588_CR11","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.96.15_supplement.1917","author":"S Muppidi","year":"2021","unstructured":"Muppidi S, Klink AJ, Parthan A, Sader SC, Balanean A, Gajra A et al (2021) Real-world use of eculizumab in generalized myasthenia gravis in the united states: results from a pilot retrospective chart-review study. Neurology. https:\/\/doi.org\/10.1212\/WNL.96.15_supplement.1917","journal-title":"Neurology"},{"key":"12588_CR12","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.98.18_supplement.1391","author":"H Murai","year":"2022","unstructured":"Murai H, Suzuki S, Fukamizu Y, Osawa T, Kikui H, Utsugisawa K (2022) Real-world effectiveness of eculizumab in generalized myasthenia gravis in Japan: 1-year post-marketing surveillance data. Neurology. https:\/\/doi.org\/10.1212\/WNL.98.18_supplement.1391","journal-title":"Neurology"},{"issue":"4","key":"12588_CR13","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16189","volume":"31","author":"R Frangiamore","year":"2024","unstructured":"Frangiamore R, Rinaldi E, Vanoli F, Andreetta F, Ciusani E, Bonanno S et al (2024) Efgartigimod in generalized myasthenia gravis: A real-life experience at a national reference center. Eur J Neurol 31(4):e16189","journal-title":"Eur J Neurol"},{"key":"12588_CR14","doi-asserted-by":"publisher","DOI":"10.1101\/2024.01.31.24302082","author":"JM Dion\u00edsio","year":"2024","unstructured":"Dion\u00edsio JM, Ambrose P, Burke G, Farrugia M, Garcia-Reitboeck P, Hewamadduma C et al (2024) Efgartigimod efficacy and safety in refractory Myasthenia Gravis - UK\u2019s first real-world experience. Medrxiv. https:\/\/doi.org\/10.1101\/2024.01.31.24302082","journal-title":"Medrxiv"},{"key":"12588_CR15","doi-asserted-by":"publisher","DOI":"10.1212\/CPJ.0000000000200276","volume":"14","author":"S Suzuki","year":"2024","unstructured":"Suzuki S, Uzawa A, Nagane Y et al (2024) Therapeutic responses to efgartigimod for generalized myasthenia gravis in Japan. Neurol Clin Pract 14:e200276","journal-title":"Neurol Clin Pract"},{"key":"12588_CR16","doi-asserted-by":"crossref","unstructured":"Wolfe GI, Herbelin L, Nations SP, Foster B, Bryan WW, Barohn RJ (1999) Myasthenia gravis activities of daily living profile. Neurology 52:1487\u20131489","DOI":"10.1212\/WNL.52.7.1487"},{"key":"12588_CR17","doi-asserted-by":"publisher","first-page":"1968","DOI":"10.1212\/01.WNL.0000163988.28892.79","volume":"64","author":"RS Bedlack","year":"2005","unstructured":"Bedlack RS, Simel DL, Bosworth H, Samsa G, Tucker-Lipscomb B, Sanders DB (2005) Quantitative myasthenia gravis score: assessment of responsiveness and longitudinal validity. Neurology 64:1968\u20131970","journal-title":"Neurology"},{"key":"12588_CR18","doi-asserted-by":"publisher","first-page":"1284444","DOI":"10.3389\/fneur.2023.1284444","volume":"14","author":"JF Howard Jr","year":"2024","unstructured":"Howard JF Jr, Bril V, Vu T et al (2024) Long-term safety, tolerability, and efficacy of efgartigimod (ADAPT+): interim results from a phase 3 open- label extension study in participants with generalized myasthenia gravis. Front Neurol 14:1284444","journal-title":"Front Neurol"},{"key":"12588_CR19","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16280","author":"A Meisel","year":"2024","unstructured":"Meisel A, Sacc\u00e0 F, Spillane J, Vissing J (2024) Expert consensus recommendations for improving and standardising the assessment of patients with generalised myasthenia gravis. Eur J Neurol. https:\/\/doi.org\/10.1111\/ene.16280","journal-title":"Eur J Neurol"},{"key":"12588_CR20","doi-asserted-by":"publisher","first-page":"87","DOI":"10.1002\/mus.28003","volume":"69","author":"M Singer","year":"2024","unstructured":"Singer M, Khella S, Brid S et al (2024) Single institution experience with efgartigimod in patients with myasthenia gravis: patient selection, dosing schedules, treatment response, and adverse events. Muscle Nerve 69:87\u201392","journal-title":"Muscle Nerve"},{"key":"12588_CR21","doi-asserted-by":"publisher","first-page":"762","DOI":"10.1002\/mus.27974","volume":"68","author":"N Katyal","year":"2023","unstructured":"Katyal N, Halldorsdottir K, Govindarajan R et al (2023) Safety and outcomes with efgartigimod use for acetylcholine receptor-positive generalized myasthenia gravis in clinical practice. Muscle Nerve 68:762\u2013766","journal-title":"Muscle Nerve"},{"key":"12588_CR22","doi-asserted-by":"publisher","first-page":"3707","DOI":"10.1007\/s10072-023-06900-y","volume":"44","author":"C Vinciguerra","year":"2023","unstructured":"Vinciguerra C, Bevilacqua L, Toriello A et al (2023) Starting eculizumab as rescue therapy in refractory myasthenic crisis. Neurol Sci 44:3707\u20133709","journal-title":"Neurol Sci"}],"updated-by":[{"DOI":"10.1007\/s00415-025-13061-9","type":"correction","label":"Correction","source":"publisher","updated":{"date-parts":[[2025,4,29]],"date-time":"2025-04-29T00:00:00Z","timestamp":1745884800000}}],"container-title":["Journal of Neurology"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-024-12588-7.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s00415-024-12588-7\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s00415-024-12588-7.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,4,29]],"date-time":"2025-04-29T09:31:51Z","timestamp":1745919111000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s00415-024-12588-7"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,30]]},"references-count":22,"journal-issue":{"issue":"9","published-print":{"date-parts":[[2024,9]]}},"alternative-id":["12588"],"URL":"https:\/\/doi.org\/10.1007\/s00415-024-12588-7","relation":{},"ISSN":["0340-5354","1432-1459"],"issn-type":[{"value":"0340-5354","type":"print"},{"value":"1432-1459","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,30]]},"assertion":[{"value":"26 May 2024","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"15 July 2024","order":2,"name":"revised","label":"Revised","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"16 July 2024","order":3,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"30 July 2024","order":4,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"25 April 2025","order":5,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Update","order":6,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Missing tables 1 and 2 foot note have been updated","order":7,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"29 April 2025","order":8,"name":"change_date","label":"Change Date","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"Correction","order":9,"name":"change_type","label":"Change Type","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"A Correction to this paper has been published:","order":10,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"https:\/\/doi.org\/10.1007\/s00415-025-13061-9","URL":"https:\/\/doi.org\/10.1007\/s00415-025-13061-9","order":11,"name":"change_details","label":"Change Details","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"None.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of interest"}}]}}