{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,5]],"date-time":"2026-09-05T01:52:26Z","timestamp":1788573146783,"version":"build-2803163510"},"reference-count":18,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2022,6,21]],"date-time":"2022-06-21T00:00:00Z","timestamp":1655769600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/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"}]},{"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":[[2022,9]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Background<\/jats:title><jats:p>The effects of eculizumab treatment in paroxysmal nocturnal hemoglobinuria (PNH) patients with or without high\u2010disease activity (HDA), defined by LDH\u2009\u2265\u20091.5\u2009\u00d7\u2009ULN and history of major adverse vascular events (MAVEs; including thrombotic events [TEs]); anemia; and\/or physician\u2010reported abdominal pain, dyspnea, dysphagia, erectile dysfunction, fatigue, and\/or hemoglobinuria, in the International PNH Registry were evaluated.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Registry patients were stratified by baseline HDA and eculizumab\u2010treatment status. Longitudinal changes in laboratory and clinical PNH\u2010related endpoints were evaluated using linear mixed models (continuous variables) or Poisson regression (incidence rates).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>As of May 1, 2017, 3009 patients (HDA\/eculizumab\u2010treated, <jats:italic>n<\/jats:italic>\u00a0=\u00a0913; HDA\/never\u2010treated, <jats:italic>n<\/jats:italic>\u00a0=\u00a0651; no\u2010HDA\/eculizumab\u2010treated, <jats:italic>n<\/jats:italic>\u00a0=\u00a0173; no\u2010HDA\/never\u2010treated, <jats:italic>n<\/jats:italic>\u00a0=\u00a01272) were analyzed. Higher proportions of eculizumab\u2010treated patients had HDA and history of MAVEs. In patients with and without HDA, respectively, eculizumab treatment resulted in reductions from baseline for (1) LDH ratio (mean [SD]: \u22125.3 [4.0] and \u22122.3 [3.8]); (2) incidence rate ratio (IRR) for MAVEs (\u221280% and \u221270%); (3) IRR for TEs (\u221280% for both); and (4) units of red blood cell transfusions per year (from 6.8 to 2.8 and 3.6 to 2.5\u00a0units).<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Eculizumab treatment in a real\u2010world setting improved outcomes, including substantial decreases in hemolysis, MAVE rates, TEs, and transfusions in PNH patients regardless of HDA.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.13773","type":"journal-article","created":{"date-parts":[[2022,4,7]],"date-time":"2022-04-07T16:14:46Z","timestamp":1649348086000},"page":"197-204","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":14,"title":["Effect of eculizumab treatment in patients with paroxysmal nocturnal hemoglobinuria with or without high disease activity: <scp>Real\u2010world<\/scp> findings from the International Paroxysmal Nocturnal Hemoglobinuria Registry"],"prefix":"10.1111","volume":"109","author":[{"given":"Britta","family":"H\u00f6chsmann","sequence":"first","affiliation":[{"name":"Institute of Transfusion Medicine, University of Ulm, and Institute for Clinical Transfusion Medicine and Immunogenetics German Red Cross Blood Transfusion Service Baden\u2010W\u00fcrttemberg\u2010Hessen and University Hospital Ulm  Ulm Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flore Sicre","family":"de Fontbrune","sequence":"additional","affiliation":[{"name":"H\u00f4pital Saint Louis  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jong Wook","family":"Lee","sequence":"additional","affiliation":[{"name":"Department of Hematology, Seoul St. Mary's Hospital, College of Medicine The Catholic University of Korea  Seoul Republic of Korea"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9589-4136","authenticated-orcid":false,"given":"Alexander D.","family":"Kulagin","sequence":"additional","affiliation":[{"name":"Pavlov First Saint Petersburg State Medical University  St. Petersburg Russia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Peter","family":"Hillmen","sequence":"additional","affiliation":[{"name":"St. James's University Hospital  Leeds UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amanda","family":"Wilson","sequence":"additional","affiliation":[{"name":"Alexion, AstraZeneca Rare Disease  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jing L.","family":"Marantz","sequence":"additional","affiliation":[{"name":"Alexion, AstraZeneca Rare Disease  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hubert","family":"Schrezenmeier","sequence":"additional","affiliation":[{"name":"Institute of Transfusion Medicine, University of Ulm, and Institute for Clinical Transfusion Medicine and Immunogenetics German Red Cross Blood Transfusion Service Baden\u2010W\u00fcrttemberg\u2010Hessen and University Hospital Ulm  Ulm 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