{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,10]],"date-time":"2026-10-10T15:35:59Z","timestamp":1791646559528,"version":"4.3.4"},"reference-count":21,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2024,7,10]],"date-time":"2024-07-10T00:00:00Z","timestamp":1720569600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"DOI":"10.13039\/100006396","name":"Alexion Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100006396","id-type":"DOI","asserted-by":"publisher"}]}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["J Peripheral Nervous Sys"],"published-print":{"date-parts":[[2024,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background and Aims<\/jats:title><jats:p>Guillain\u2010Barr\u00e9 syndrome (GBS) is an acute, self\u2010limited, immune\u2010mediated peripheral neuropathy. Current treatments for GBS include intravenous immunoglobulin (IVIg) and plasma exchange, which may not sufficiently benefit severely affected patients. This study evaluated the efficacy and safety of eculizumab add\u2010on therapy to IVIg (standard\u2010of\u2010care treatment) in patients with severe GBS.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This phase 3, multicenter, double\u2010blind, randomized, placebo\u2010controlled clinical trial (NCT04752566), enrolled Japanese adults (age\u2009\u2265\u200918\u2009years) with severe GBS (Hughes functional grade [FG] score FG3 or FG4\/FG5 within 2\u2009weeks of onset of GBS). Participants were randomized 2:1 to receive intravenous infusion of eculizumab or placebo (once weekly for 4\u2009weeks) with IVIg treatment with 20\u2009weeks of follow\u2010up. Primary efficacy endpoint was the time to first reach FG score \u22641 (able to run). Key secondary endpoints were proportion of participants achieving FG \u22641 at weeks 8 and 24 and FG improvement \u22653 at week 24. Pharmacodynamic analysis of serum free C5 concentration over time was performed. Safety was evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The analysis included 57 participants (eculizumab, <jats:italic>n<\/jats:italic>\u2009=\u200937; placebo, <jats:italic>n<\/jats:italic>\u2009=\u200920). Primary endpoint was not achieved (hazard ratio, 0.9; 95% CI, 0.45\u20131.97; <jats:italic>p<\/jats:italic>\u2009=\u2009.89). Key secondary endpoints did not reach statistical significance. Serum C5 concentration was reduced by 99.99% at 1\u2009h postdose and sustained to week 5 but returned to baseline at the end of follow\u2010up period. No new safety signals for eculizumab were identified.<\/jats:p><\/jats:sec><jats:sec><jats:title>Interpretation<\/jats:title><jats:p>Although well tolerated, eculizumab treatment did not show significant effects on motor function recovery compared to placebo in patients with GBS.<\/jats:p><\/jats:sec>","DOI":"10.1111\/jns.12646","type":"journal-article","created":{"date-parts":[[2024,7,11]],"date-time":"2024-07-11T02:29:02Z","timestamp":1720664942000},"page":"339-349","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":27,"title":["Efficacy and safety of eculizumab in Guillain\u2010Barr\u00e9 syndrome: A phase 3, multicenter, double\u2010blind, randomized, placebo\u2010controlled clinical trial"],"prefix":"10.1111","volume":"29","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-4716-8578","authenticated-orcid":false,"given":"Satoshi","family":"Kuwabara","sequence":"first","affiliation":[{"name":"Department of Neurology, Graduate School of Medicine Chiba University  Chiba Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Susumu","family":"Kusunoki","sequence":"additional","affiliation":[{"name":"Department of Neurology Kindai University Faculty of Medicine  Osaka\u2010Sayama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Motoi","family":"Kuwahara","sequence":"additional","affiliation":[{"name":"Department of Neurology Kindai University Faculty of Medicine  Osaka\u2010Sayama Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoshihisa","family":"Yamano","sequence":"additional","affiliation":[{"name":"Department of Neurology St. Marianna University School of Medicine Kawasaki  Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yoichiro","family":"Nishida","sequence":"additional","affiliation":[{"name":"Department of Neurology and Neurological Science, Graduate School of Medical and Dental Sciences Tokyo Medical and Dental University  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hirokazu","family":"Ishida","sequence":"additional","affiliation":[{"name":"Alexion Pharma GK  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Tomoyuki","family":"Kasuya","sequence":"additional","affiliation":[{"name":"Alexion Pharma GK  Tokyo Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Erik","family":"Kupperman","sequence":"additional","affiliation":[{"name":"AstraZeneca Rare Disease Alexion  Boston USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Qun","family":"Lin","sequence":"additional","affiliation":[{"name":"AstraZeneca Rare Disease Alexion  Boston USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Glen","family":"Frick","sequence":"additional","affiliation":[{"name":"AstraZeneca Rare Disease Alexion  Boston USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Sonoko","family":"Misawa","sequence":"additional","affiliation":[{"name":"Department of Neurology, Graduate School of Medicine Chiba University  Chiba Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2024,7,10]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awt285"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.vaccine.2010.06.003"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140\u20106736(21)00517\u20101"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/118.3.597"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410440512"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/14651858.CD001798.pub3"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199204233261705"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410410304"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(96)09095-2"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1093\/brain\/awm004"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1523\/JNEUROSCI.4401\u201006.2007"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410390512"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1016\/s0161\u20105890(96)00078\u20108"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(18)30114\u20105"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1212\/WNL.0b013e3182104407"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1016\/S1474\u20104422(07)70130\u20108"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1084\/jem.178.5.1771"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1002\/ana.410400209"},{"key":"e_1_2_11_20_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.14860"},{"key":"e_1_2_11_21_1","doi-asserted-by":"publisher","DOI":"10.1111\/ene.16094"},{"key":"e_1_2_11_22_1","doi-asserted-by":"publisher","DOI":"10.1159\/000324710"}],"container-title":["Journal of the Peripheral Nervous System"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/pdf\/10.1111\/jns.12646","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,9,20]],"date-time":"2024-09-20T11:13:30Z","timestamp":1726830810000},"score":1,"resource":{"primary":{"URL":"https:\/\/onlinelibrary.wiley.com\/doi\/10.1111\/jns.12646"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,7,10]]},"references-count":21,"journal-issue":{"issue":"3","published-print":{"date-parts":[[2024,9]]}},"alternative-id":["10.1111\/jns.12646"],"URL":"https:\/\/doi.org\/10.1111\/jns.12646","archive":["Portico"],"relation":{},"ISSN":["1085-9489","1529-8027"],"issn-type":[{"value":"1085-9489","type":"print"},{"value":"1529-8027","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,7,10]]},"assertion":[{"value":"2023-12-22","order":0,"name":"received","label":"Received","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-07-02","order":2,"name":"accepted","label":"Accepted","group":{"name":"publication_history","label":"Publication History"}},{"value":"2024-07-10","order":3,"name":"published","label":"Published","group":{"name":"publication_history","label":"Publication History"}}]}}