{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T22:55:42Z","timestamp":1785884142920,"version":"3.56.0"},"reference-count":17,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T00:00:00Z","timestamp":1785801600000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"},{"start":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T00:00:00Z","timestamp":1785801600000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/100018748","name":"Argenx","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100018748","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Journal of Neuromuscular Diseases"],"abstract":"<jats:sec>\n                    <jats:title>Background and objectives<\/jats:title>\n                    <jats:p>To evaluate the safety and clinical efficacy of efgartigimod in adults with severe generalized myasthenia gravis (MG) exacerbations requiring ventilatory or enteral support.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>Prospective, single-arm, open-label study conducted between November 2024 and April 2025. Sixteen consecutive adults with AChR- or MuSK-antibody-positive generalized MG requiring invasive or non-invasive ventilatory support or enteral feeding were enrolled; all met 2016 MG Guidelines criteria for impending myasthenic crisis. MGFA classification included Class IIIb (n=14), IIIa (n=1), and IVb (n=1). The primary endpoint was clinically meaningful improvement (CMI) in MG Activities of Daily Living (MG-ADL), defined as \u22652-point reduction from baseline at weeks 4 and 8.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>All 16 participants (mean age 58.5\u00b117.4 years; 8 women) completed eight weeks of follow-up. At week 8, 12 patients (75%; 95% CI 47\u201391%) achieved the primary endpoint. Median MG-ADL decreased from 11 (IQR 9\u201313) to 7 (IQR 6\u20139) (median change \u22125 points; p=0.003). Median QMG decreased from 21 (IQR 19\u201323) to 13 (IQR 11\u201316) (median change \u22127.5 points; p&lt;0.001), with 56% meeting QMG-CMI by week 1. Among 12 participants receiving corticosteroids (mean starting dose 21.2 mg\/day), 50% reduced or discontinued by week 8. No treatment-related serious adverse events occurred.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Discussion<\/jats:title>\n                    <jats:p>Efgartigimod produced rapid, clinically meaningful improvement in severe MG exacerbations without safety concerns. FcRn blockade may represent a practical alternative to plasma exchange or IVIG in acute care settings. Findings are limited by the absence of a control arm, which precludes definitive attribution of clinical improvement to efgartigimod alone.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Clinical trials registration<\/jats:title>\n                    <jats:p>NCT06688253.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/22143602261450402","type":"journal-article","created":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T22:35:53Z","timestamp":1785882953000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":0,"title":["Prospective study of efgartigimod in severe myasthenia gravis exacerbations requiring ventilatory or enteral support"],"prefix":"10.1177","author":[{"given":"Shahar","family":"Shelly","sequence":"first","affiliation":[{"name":"Technion-Israel Institute of Technology"},{"name":"Rambam Medical Center"},{"name":"Department of Neurology, Mayo Clinic, Rochester, MN, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ayal","family":"Rosenberg","sequence":"additional","affiliation":[{"name":"Technion-Israel Institute of Technology"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohamad","family":"Zoabi","sequence":"additional","affiliation":[{"name":"Rambam Medical Center"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Saliba","family":"Makhoul","sequence":"additional","affiliation":[{"name":"Rambam Medical Center"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Roni","family":"Ramon-Gonen","sequence":"additional","affiliation":[{"name":"Bar-Ilan University"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gil I.","family":"Wolfe","sequence":"additional","affiliation":[{"name":"University at Buffalo\/SUNY"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2026,8,4]]},"reference":[{"key":"e_1_3_5_2_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000002790"},{"key":"e_1_3_5_3_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.ensci.2024.100541"},{"key":"e_1_3_5_4_2","doi-asserted-by":"publisher","DOI":"10.1080\/14656566.2022.2122710"},{"key":"e_1_3_5_5_2","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00159-9"},{"key":"e_1_3_5_6_2","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000201501"},{"key":"e_1_3_5_7_2","doi-asserted-by":"publisher","DOI":"10.1001\/archneur.56.1.25"},{"key":"e_1_3_5_8_2","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.j396"},{"key":"e_1_3_5_9_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2025.1524200"},{"key":"e_1_3_5_10_2","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2024.1418503"},{"key":"e_1_3_5_11_2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jneuroim.2024.578431"},{"key":"e_1_3_5_12_2","first-page":"9455237","article-title":"Efgartigimod as Rescue Medication in a Patient with Therapy-Refractory Myasthenic Crisis","volume":"2024","author":"Alhaj Omar O","year":"2024","unstructured":"Alhaj Omar O, Diel NJ, Gerner ST, et al. Efgartigimod as Rescue Medication in a Patient with Therapy-Refractory Myasthenic Crisis. Case Rep Neurol Med 2024; 2024: 9455237. https:\/\/doi.org\/10.1155\/2024\/9455237","journal-title":"Case Rep Neurol Med"},{"key":"e_1_3_5_13_2","doi-asserted-by":"publisher","DOI":"10.1177\/17562864241254895"},{"key":"e_1_3_5_14_2","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1321058"},{"key":"e_1_3_5_15_2","doi-asserted-by":"publisher","DOI":"10.1002\/acn3.52142"},{"key":"e_1_3_5_16_2","doi-asserted-by":"publisher","DOI":"10.1186\/s12883-024-03804-y"},{"key":"e_1_3_5_17_2","doi-asserted-by":"publisher","DOI":"10.1177\/17562864241307687"},{"key":"e_1_3_5_18_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41598-024-79918-7"}],"container-title":["Journal of Neuromuscular Diseases"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602261450402","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/full-xml\/10.1177\/22143602261450402","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/journals.sagepub.com\/doi\/pdf\/10.1177\/22143602261450402","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,8,4]],"date-time":"2026-08-04T22:35:54Z","timestamp":1785882954000},"score":1,"resource":{"primary":{"URL":"https:\/\/journals.sagepub.com\/doi\/10.1177\/22143602261450402"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,8,4]]},"references-count":17,"alternative-id":["10.1177\/22143602261450402"],"URL":"https:\/\/doi.org\/10.1177\/22143602261450402","relation":{},"ISSN":["2214-3599","2214-3602"],"issn-type":[{"value":"2214-3599","type":"print"},{"value":"2214-3602","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,8,4]]},"article-number":"22143602261450402"}}