{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,7]],"date-time":"2026-09-07T21:47:50Z","timestamp":1788817670615,"version":"build-2803163510"},"reference-count":31,"publisher":"SAGE Publications","license":[{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"unspecified","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2024,8,1]],"date-time":"2024-08-01T00:00:00Z","timestamp":1722470400000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/journals.sagepub.com\/page\/policies\/text-and-data-mining-license"}],"funder":[{"DOI":"10.13039\/100015661","name":"UCB Pharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100015661","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["journals.sagepub.com"],"crossmark-restriction":true},"short-container-title":["Ther Adv Neurol Disord"],"published-print":{"date-parts":[[2024,8]]},"abstract":"<jats:sec>\n                    <jats:title>Background:<\/jats:title>\n                    <jats:p>Clinical efficacy of zilucoplan has been demonstrated in a 12-week, placebo-controlled, phase III study in patients with acetylcholine receptor autoantibody-positive generalised myasthenia gravis (gMG). However, placebo-controlled zilucoplan data past 12\u2009weeks are not available.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Objectives:<\/jats:title>\n                    <jats:p>Predict the treatment effect of zilucoplan versus control (placebo or standard of care) in patients with gMG up to 24\u2009weeks.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Design:<\/jats:title>\n                    <jats:p>A model-informed analysis (MIA) within a Bayesian framework.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Methods:<\/jats:title>\n                    <jats:p>Part 1 of the MIA comprised a control meta-regression using aggregate data on control response over time from randomised studies and a national myasthenia gravis (MG) registry. In Part 2, a combined Bayesian analysis of individual patient-level data from the phase II (NCT03315130), RAISE (NCT04115293) and RAISE-XT (NCT04225871) studies of zilucoplan was conducted using posterior distributions from Part 1 as informative priors. Population mean treatment effect in the change from baseline (CFB) at week 24 in MG-Activities of Daily Living (MG-ADL) and quantitative MG (QMG) scores for zilucoplan versus control were assessed.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Results:<\/jats:title>\n                    <jats:p>At week 24, the predicted mean CFB in MG-ADL score was \u22124.55 (95% credible interval: \u22126.04, \u22123.13) with zilucoplan versus \u22122.00 (\u22123.35, \u22120.64) with control (difference: \u22122.55 [\u22123.76, \u22121.40]). The probability of a favourable treatment effect as measured by MG-ADL score at week 24 with zilucoplan versus control was &gt;99.9%. There was an 82.8% probability that the difference in the predicted mean CFB in MG-ADL score at week 24 was greater than the clinically meaningful threshold (\u2a7e2.0-point improvement). Comparable results were observed with QMG.<\/jats:p>\n                  <\/jats:sec>\n                  <jats:sec>\n                    <jats:title>Conclusion:<\/jats:title>\n                    <jats:p>This MIA demonstrates the maintenance of efficacy with zilucoplan versus control up to 24\u2009weeks. Through combining real-world evidence with data from randomised studies, this novel method to estimate long-term treatment efficacy facilitated reduced exposure to placebo in the phase III RAISE study. This methodology could be used to reduce the length of future placebo-controlled studies.<\/jats:p>\n                  <\/jats:sec>","DOI":"10.1177\/17562864241279125","type":"journal-article","created":{"date-parts":[[2026,1,6]],"date-time":"2026-01-06T07:17:07Z","timestamp":1767683827000},"update-policy":"https:\/\/doi.org\/10.1177\/sage-journals-update-policy","source":"Crossref","is-referenced-by-count":2,"title":["Maintenance of zilucoplan efficacy in patients with generalised myasthenia gravis up to 24\u2009weeks: a model-informed analysis"],"prefix":"10.1177","volume":"17","author":[{"ORCID":"https:\/\/orcid.org\/0009-0000-6483-0925","authenticated-orcid":false,"given":"Guillemette","family":"de la Borderie","sequence":"first","affiliation":[{"name":"UCB, 420 rue d\u2019Estienne d\u2019Orves, Colombes 92700, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Damien","family":"Chimits","sequence":"additional","affiliation":[{"name":"UCB, Colombes, France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Babak","family":"Boroojerdi","sequence":"additional","affiliation":[{"name":"UCB, Monheim, Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Melissa","family":"Brock","sequence":"additional","affiliation":[{"name":"UCB, Morrisville, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Petra W.","family":"Duda","sequence":"additional","affiliation":[{"name":"UCB, Cambridge, MA, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Fiona","family":"Grimson","sequence":"additional","affiliation":[{"name":"UCB, Slough, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Paul","family":"Mahoney","sequence":"additional","affiliation":[{"name":"UCB, Slough, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Foteini","family":"Strimenopoulou","sequence":"additional","affiliation":[{"name":"UCB, Slough, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8455-980X","authenticated-orcid":false,"given":"Gary","family":"Cutter","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, The University of Alabama at Birmingham School of Public Health, Birmingham, AL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0563-1691","authenticated-orcid":false,"given":"Inmaculada","family":"Aban","sequence":"additional","affiliation":[{"name":"Department of Biostatistics, The University of Alabama at Birmingham School of Public Health, Birmingham, AL, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9120-9488","authenticated-orcid":false,"given":"Susanna","family":"Brauner","sequence":"additional","affiliation":[{"name":"Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden"},{"name":"Department of Neurology, Karolinska University Hospital, Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Malin","family":"Petersson","sequence":"additional","affiliation":[{"name":"Department of Clinical Neuroscience, Karolinska Institutet, Stockholm, Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-7136-8617","authenticated-orcid":false,"suffix":"Jr","given":"James F.","family":"Howard","sequence":"additional","affiliation":[{"name":"Department of Neurology, The University of North Carolina at Chapel Hill, Chapel Hill, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nathan","family":"Bennett","sequence":"additional","affiliation":[{"name":"UCB, Morrisville, NC, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"179","published-online":{"date-parts":[[2024,9,21]]},"reference":[{"key":"e_1_3_4_2_2","doi-asserted-by":"publisher","DOI":"10.1038\/s41572-019-0079-y"},{"key":"e_1_3_4_3_2","doi-asserted-by":"publisher","DOI":"10.1111\/nyas.13522"},{"key":"e_1_3_4_4_2","doi-asserted-by":"publisher","DOI":"10.1002\/mus.26695"},{"key":"e_1_3_4_5_2","first-page":"255","article-title":"Clinical characteristics of refractory myasthenia gravis patients","volume":"86","author":"Suh J","year":"2013","unstructured":"Suh J, Goldstein JM, Nowak RJ. 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