{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,6,10]],"date-time":"2026-06-10T15:07:00Z","timestamp":1781104020435,"version":"3.54.1"},"reference-count":31,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2026,1,12]],"date-time":"2026-01-12T00:00:00Z","timestamp":1768176000000},"content-version":"vor","delay-in-days":11,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,1,1]],"date-time":"2026-01-01T00:00:00Z","timestamp":1767225600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"funder":[{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82360268"],"award-info":[{"award-number":["82360268"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100001809","name":"National Natural Science Foundation of China","doi-asserted-by":"publisher","award":["82471487"],"award-info":[{"award-number":["82471487"]}],"id":[{"id":"10.13039\/501100001809","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["CNS Neurosci Ther"],"published-print":{"date-parts":[[2026,1]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:sec>\n                    <jats:title>Background<\/jats:title>\n                    <jats:p>Efgartigimod, a neonatal Fc receptor (FcRn) blocker, is approved for generalized Myasthenia Gravis (gMG), but predictors of early response are unclear. Using minimal symptom expression (MSE) as the therapeutic target, we developed and externally validated a clinical model to predict early MSE response\u00a0after starting efgartigimod. This efgartigimod\u2010tailored model estimates the baseline probability of achieving early MSE\u00a0and may assist in individualized treatment selection at therapy initiation.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods<\/jats:title>\n                    <jats:p>\n                      We retrospectively analyzed 118 adults with gMG treated at five tertiary centers in China (efgartigimod 10\u2009mg\/kg IV weekly \u00d74). MSE was defined as Myasthenia Gravis\u2013Activities of Daily Living (MG\u2010ADL) \u2264\u20091 sustained \u2265\u20094\u2009weeks; early MSE response was achievement within 4\u2009weeks of initiation. Patients were split into a derivation cohort (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200964; three centers) and an external validation cohort (\n                      <jats:italic>n<\/jats:italic>\n                      \u2009=\u200954; two centers). Candidate predictors included demographics, baseline severity, and laboratory indices. Variables associated with early MSE in univariable analyses entered multivariable logistic regression to construct a nomogram. Discrimination (AUC\u2010ROC), calibration (curves and Spiegelhalter\n                      <jats:italic>Z<\/jats:italic>\n                      \u2010test), and clinical utility (decision curve analysis, DCA) were assessed, with bootstrap internal validation.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results<\/jats:title>\n                    <jats:p>\n                      Early MSE response occurred in 26\/64 derivation and 22\/54 validation patients. Lower bulbar MG\u2010ADL (OR 0.633,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.040), higher FVC% (OR 1.042,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.048), and lower IgG (OR 0.795,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.036) independently predicted early MSE response. The nomogram showed strong discrimination\u2014derivation AUC 0.869 (95% CI 0.797\u20130.941), bootstrap AUC 0.880 (0.806\u20130.954), and external AUC 0.839 (0.760\u20130.919)\u2014and good calibration (Spiegelhalter\n                      <jats:italic>Z<\/jats:italic>\n                      : 1.03,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.303; 0.94,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.347; 1.17,\n                      <jats:italic>p<\/jats:italic>\n                      \u2009=\u20090.242). DCA indicated net benefit across thresholds 0.05\u20130.82, with validation curves mirroring derivation.\n                    <\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusions<\/jats:title>\n                    <jats:p>A three\u2010factor nomogram (bulbar MG\u2010ADL, FVC%, and serum IgG) provides an efgartigimod\u2010specific baseline estimate of early sustained\u2010MSE response probability, which may help neurologists select appropriate candidates, counsel expected benefit, and tailor follow\u2010up intensity or alternative escalation strategies.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Trail Registration<\/jats:title>\n                    <jats:p>Chinese Clinical Trial Registry (ChiCTR2500101971)<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1002\/cns.70746","type":"journal-article","created":{"date-parts":[[2026,1,12]],"date-time":"2026-01-12T12:44:09Z","timestamp":1768221849000},"update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Development and External Validation of a Multivariable Model to Predict Early Minimal Symptom Expression Response in Adult Generalized Myasthenia Gravis Patients Treated With Efgartigimod"],"prefix":"10.1002","volume":"32","author":[{"given":"Yufang","family":"Yang","sequence":"first","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6828-1839","authenticated-orcid":false,"given":"Tao","family":"Liang","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0472-4298","authenticated-orcid":false,"given":"Mingming","family":"Zhao","sequence":"additional","affiliation":[{"name":"Department of Neurology Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hongxia","family":"Yang","sequence":"additional","affiliation":[{"name":"Center for the Diagnosis and Treatment of Myasthenia Gravis Shijiazhuang People's Hospital  Shijiazhuang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zhilan","family":"Zhao","sequence":"additional","affiliation":[{"name":"Department of Neurology Second Affiliated Hospital of Guizhou University of Traditional Chinese Medicine  Guiyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lu","family":"Yu","sequence":"additional","affiliation":[{"name":"Department of Neurology Guizhou Provincial People's Hospital  Guiyang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Linlin","family":"Yan","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Siyuan","family":"Li","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peng","family":"Zhang","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-9218-3219","authenticated-orcid":false,"given":"Guoyan","family":"Qi","sequence":"additional","affiliation":[{"name":"Center for the Diagnosis and Treatment of Myasthenia Gravis Shijiazhuang People's Hospital  Shijiazhuang China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0003-2054-7281","authenticated-orcid":false,"given":"Jian","family":"Yin","sequence":"additional","affiliation":[{"name":"Department of Neurology Beijing Hospital, National Center of Gerontology, Institute of Geriatric Medicine, Chinese Academy of Medical Sciences  Beijing China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6849-7928","authenticated-orcid":false,"given":"Zucai","family":"Xu","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7574-3293","authenticated-orcid":false,"given":"Zhong","family":"Luo","sequence":"additional","affiliation":[{"name":"Department of Neurology Affiliated Hospital of Zunyi Medical University  Zunyi China"},{"name":"Key Laboratory of Brain Function and Brain Disease Prevention and Treatment of Guizhou Province (Qiankehe Foundation\u2010ZSYS(2025)030)  Zunyi China"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,1,12]]},"reference":[{"key":"e_1_2_14_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/s41582-021-00547-z"},{"key":"e_1_2_14_3_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2020.596981"},{"key":"e_1_2_14_4_1","doi-asserted-by":"publisher","DOI":"10.3810\/hp.2013.10.1079"},{"key":"e_1_2_14_5_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474-4422(21)00159-9"},{"key":"e_1_2_14_6_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2024-334086"},{"key":"e_1_2_14_7_1","doi-asserted-by":"publisher","DOI":"10.1212\/CPJ.0000000000200276"},{"key":"e_1_2_14_8_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.27974"},{"key":"e_1_2_14_9_1","doi-asserted-by":"publisher","DOI":"10.1177\/17562864241311127"},{"key":"e_1_2_14_10_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28334"},{"key":"e_1_2_14_11_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28003"},{"key":"e_1_2_14_12_1","doi-asserted-by":"publisher","DOI":"10.1136\/jnnp-2024-334404"},{"key":"e_1_2_14_13_1","doi-asserted-by":"publisher","DOI":"10.18553\/jmcp.2024.30.6.517"},{"key":"e_1_2_14_14_1","doi-asserted-by":"publisher","DOI":"10.1002\/mus.28417"},{"key":"e_1_2_14_15_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa2501561"},{"key":"e_1_2_14_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/cns.70391"},{"key":"e_1_2_14_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16098"},{"key":"e_1_2_14_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.intimp.2025.114603"},{"key":"e_1_2_14_19_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-023-00464-x"},{"key":"e_1_2_14_20_1","doi-asserted-by":"publisher","DOI":"10.1007\/s10072-022-06493-y"},{"key":"e_1_2_14_21_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00415-023-11733-y"},{"key":"e_1_2_14_22_1","doi-asserted-by":"publisher","DOI":"10.1007\/s40120-024-00682-x"},{"key":"e_1_2_14_23_1","article-title":"Peak Expiratory Flow and the Single\u2010Breath Counting Test as Markers of Respiratory Function in Patients With Myasthenia Gravis","volume":"12","author":"Aguirre F.","year":"2020","journal-title":"Neurolog\u00eda"},{"key":"e_1_2_14_24_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.25087"},{"key":"e_1_2_14_25_1","doi-asserted-by":"publisher","DOI":"10.1111\/cns.14761"},{"key":"e_1_2_14_26_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0000000000210299"},{"key":"e_1_2_14_27_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1472845"},{"key":"e_1_2_14_28_1","doi-asserted-by":"publisher","DOI":"10.3389\/fneur.2024.1407418"},{"key":"e_1_2_14_29_1","doi-asserted-by":"publisher","DOI":"10.1111\/1744-9987.14065"},{"key":"e_1_2_14_30_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2024.1325171"},{"key":"e_1_2_14_31_1","doi-asserted-by":"publisher","DOI":"10.3109\/00207454.2011.596293"},{"key":"e_1_2_14_32_1","volume-title":"Hypergammaglobulinemia (Polyclonal Gammopathy)","author":"Rasel M.","year":"2025"}],"container-title":["CNS Neuroscience &amp; Therapeutics"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cns.70746","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/full-xml\/10.1002\/cns.70746","content-type":"application\/xml","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1002\/cns.70746","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2026,2,15]],"date-time":"2026-02-15T10:59:53Z","timestamp":1771153193000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1002\/cns.70746"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2026,1]]},"references-count":31,"journal-issue":{"issue":"1","published-print":{"date-parts":[[2026,1]]}},"alternative-id":["10.1002\/cns.70746"],"URL":"https:\/\/doi.org\/10.1002\/cns.70746","archive":["Portico"],"relation":{},"ISSN":["1755-5930","1755-5949"],"issn-type":[{"value":"1755-5930","type":"print"},{"value":"1755-5949","type":"electronic"}],"subject":[],"published":{"date-parts":[[2026,1]]},"assertion":[{"value":"2025-09-29","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2025-12-31","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2026-01-12","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}],"article-number":"e70746"}}