{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,11,2]],"date-time":"2026-11-02T08:58:09Z","timestamp":1793609889164,"version":"4.5.0"},"reference-count":24,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T00:00:00Z","timestamp":1694736000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/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":["European J of Haematology"],"published-print":{"date-parts":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Data from the International PNH Registry (NCT01374360) were used to estimate the overall survival and first occurrence of thromboembolic events\/major adverse vascular events (TEs\/MAVEs) for eculizumab\u2010treated patients with paroxysmal nocturnal hemoglobinuria (PNH) compared with a contemporaneous untreated cohort.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Patients enrolled in the Registry from March 16, 2007, to February 14, 2022, were included. Treated patients received eculizumab for &gt;35\u2009days; untreated patients did not receive eculizumab at any time. Univariable and multivariable analyses were performed using a Cox proportional hazards regression model comparing eculizumab treatment periods to untreated periods and were adjusted for baseline covariates (e.g., high disease activity [HDA], transfusion dependency, and eculizumab treatment status).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>The analysis included 4118 patients. The univariable hazard ratio (HR) (95% CI) for mortality in eculizumab\u2010treated time versus untreated time was 0.51 (0.41\u20130.64; <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001). Significant baseline covariates included age, sex, history of bone marrow failure, \u22654 erythrocyte transfusions within 12\u2009months before baseline, and an estimated glomerular filtration rate\u2009\u2264\u200960\u2009mL\/min\/1.73\u2009m<jats:sup>2<\/jats:sup> (all <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001). In the adjusted analysis, patients with baseline HDA had the greatest reduction in mortality risk (HR [95% CI], 0.51 [0.36\u20130.72]). Treated patients had approximately 60% reduction in TE\/MAVE risk during treated versus untreated time (HR [95% CI]: TE: 0.40 [0.26\u20130.62], MAVE: 0.37 [0.26\u20130.54]; <jats:italic>p<\/jats:italic>\u2009&lt;\u20090.0001).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>Using data from the largest Registry of patients with PNH, with \u226514\u2009years of overall follow\u2010up, we demonstrate that treatment with eculizumab conferred a 49% relative benefit in survival and an approximately 60% reduction in TE\/MAVE risk.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14080","type":"journal-article","created":{"date-parts":[[2023,9,15]],"date-time":"2023-09-15T14:30:11Z","timestamp":1694788211000},"page":"796-804","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":22,"title":["Long\u2010term effectiveness of eculizumab: Data from the International <scp>PNH<\/scp> Registry"],"prefix":"10.1111","volume":"111","author":[{"given":"Louis","family":"Terriou","sequence":"first","affiliation":[{"name":"Universit\u00e9 de Lille, Inserm, CHU Lille, Service de M\u00e9decine Interne et Immunologie Clinique, Centre de R\u00e9f\u00e9rence des Maladies Autoimmunes Syst\u00e9miques Rares du Nord et Nord\u2010Ouest de France (CeRAINO) Institut de Recherche Translationnelle sur l'Inflammation (INFINITE) \u2013 U1286  Lille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jong Wook","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Hematology, Seoul St. Mary's Hospital The Catholic University of Korea  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Cecily","family":"Forsyth","sequence":"additional","affiliation":[{"name":"Central Coast Haematology  North Gosford New South Wales Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Morag","family":"Griffin","sequence":"additional","affiliation":[{"name":"Department of Haematology Leeds Teaching Hospitals NHS Trust  Leeds UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6783-2301","authenticated-orcid":false,"given":"Jeff","family":"Szer","sequence":"additional","affiliation":[{"name":"Department of Clinical Haematology Peter MacCallum Cancer Centre and the Royal Melbourne Hospital  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"R\u00f6th","sequence":"additional","affiliation":[{"name":"Department of Hematology and Stem Cell Transplantation University Hospital Essen and University of Duisburg\u2010Essen  Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Gustovic","sequence":"additional","affiliation":[{"name":"Alexion AstraZeneca Rare Disease  Z\u00fcrich Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Metzger","sequence":"additional","affiliation":[{"name":"Parexel  Newton Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ami S.","family":"Patel","sequence":"additional","affiliation":[{"name":"Alexion AstraZeneca Rare Disease  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2089-8650","authenticated-orcid":false,"given":"Christopher J.","family":"Patriquin","sequence":"additional","affiliation":[{"name":"Division of Hematology University of Toronto  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,9,15]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-02-522128"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2017.28"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-04-1717"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2013.093161"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.3346\/jkms.2016.31.2.214"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.24278"},{"key":"e_1_2_11_8_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2008-01-133918"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199511093331904"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2012-09-311381"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2004.04992.x"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-02-333997"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1097\/01.md.0000126763.68170.46"},{"key":"e_1_2_11_14_1","unstructured":"Alexion Europe SAS.Soliris. 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