{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,12]],"date-time":"2026-09-12T05:57:59Z","timestamp":1789192679027,"version":"build-2803163510"},"reference-count":23,"publisher":"Wiley","issue":"9","license":[{"start":{"date-parts":[[2017,9,10]],"date-time":"2017-09-10T00:00:00Z","timestamp":1505001600000},"content-version":"vor","delay-in-days":9,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"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":["Internal Medicine Journal"],"published-print":{"date-parts":[[2017,9]]},"abstract":"Abstract<\/jats:title>Background<\/jats:title>Eculizumab reduces intravascular haemolysis and improves disease symptoms in patients with paroxysmal nocturnal haemoglobinuria (PNH<\/jats:styled-content>).<\/jats:p><\/jats:sec>Aims<\/jats:title>To characterise, in a real\u2010world setting, the effect of eculizumab in patients with haemolytic PNH<\/jats:styled-content> (lactase dehydrogenase (LDH<\/jats:styled-content>)\u2009\u2265\u20091.5 upper limit of normal) and no history of red blood cell transfusion, including those with high disease activity (HDA<\/jats:styled-content>).<\/jats:p><\/jats:sec>Methods<\/jats:title>Three populations from the International PNH<\/jats:styled-content> Registry were studied: (i) non\u2010transfused, untreated; (ii) non\u2010transfused, eculizumab\u2010treated and (iii) transfused, eculizumab\u2010treated (\u22651 transfusions in 6\u2009months prior to eculizumab initiation). Using multivariate linear regression, the primary outcome was mean absolute change from baseline to 6\u2009months in LDH<\/jats:styled-content> (U\/L) in non\u2010transfused patients who were treated with eculizumab versus those who remained untreated. Secondary outcomes were mean changes in functional assessment of chronic illness therapy (FACIT<\/jats:styled-content>)\u2010Fatigue and European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC\u2010QLQ<\/jats:styled-content>)\u2010C30<\/jats:styled-content> Fatigue scores from baseline to last available assessment.<\/jats:p><\/jats:sec>Results<\/jats:title>The study population included (i) 144 non\u2010transfused, untreated patients; (ii) 45 non\u2010transfused, eculizumab\u2010treated patients and (iii) 105 transfused, eculizumab\u2010treated patients. Of these, 136\/144, 43\/45 and 99\/105 had HDA<\/jats:styled-content> respectively. Compared with untreated patients, non\u2010transfused, treated patients had greater absolute reduction in LDH<\/jats:styled-content> (\u22121318.8 vs \u221239.4; P<\/jats:italic>\u2009<\u20090.001) and greater percentage reduction in LDH<\/jats:styled-content> (\u221269.9 vs \u22121.6%; P<\/jats:italic>\u2009<\u20090.001). Clinically meaningful improvements in FACIT<\/jats:styled-content>\u2010Fatigue (73.7 vs 24.6%, respectively) and in EORTC\u2010QLQ\u2010C30<\/jats:styled-content> (84.2 vs 33.3%, respectively) were observed. Non\u2010transfused, treated patients with HDA<\/jats:styled-content> had significantly reduced LDH<\/jats:styled-content> levels (P<\/jats:italic>\u2009<\u20090.001) and clinically meaningful improvements in FACIT<\/jats:styled-content>\u2010Fatigue (P<\/jats:italic>\u2009=\u20090.003) and EORTC\u2010QLQ\u2010C30<\/jats:styled-content> (P<\/jats:italic>\u2009=\u20090.020) versus untreated patients.<\/jats:p><\/jats:sec>Conclusion<\/jats:title>Significant LDH<\/jats:styled-content> reduction and clinically meaningful improvement in fatigue were observed in patients with PNH<\/jats:styled-content> and HDA<\/jats:styled-content> treated with eculizumab versus untreated patients, irrespective of transfusion history.<\/jats:p><\/jats:sec>","DOI":"10.1111\/imj.13523","type":"journal-article","created":{"date-parts":[[2017,6,13]],"date-time":"2017-06-13T07:36:47Z","timestamp":1497339407000},"page":"1026-1034","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":23,"title":["Clinical benefit of eculizumab in patients with no transfusion history in the International Paroxysmal Nocturnal Haemoglobinuria Registry"],"prefix":"10.1111","volume":"47","author":[{"given":"Antonio M.","family":"Almeida","sequence":"first","affiliation":[{"name":"Servi\u00e7o de Hematologia Instituto Portugu\u00eas de Oncologia de Lisboa, Francisco Gentil Lisbon Portugal"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Camille","family":"Bedrosian","sequence":"additional","affiliation":[{"name":"Alexion Pharma International New Haven Connecticut USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"Cole","sequence":"additional","affiliation":[{"name":"Alexion Pharma International Lexington Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Petra","family":"Muus","sequence":"additional","affiliation":[{"name":"Radboudumc Nijmegen The Netherlands"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Hubert","family":"Schrezenmeier","sequence":"additional","affiliation":[{"name":"Institute of Clinical Transfusion Medicine and Immunogenetics German Red Cross Blood Transfusion Service Baden\u2010W\u00fcrttemberg\u2013Hessen Hessen Germany"},{"name":"University Hospital Ulm Ulm Germany"},{"name":"Institute of Transfusion Medicine University of Ulm Ulm Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6783-2301","authenticated-orcid":false,"given":"Jeff","family":"Szer","sequence":"additional","affiliation":[{"name":"Royal Melbourne Hospital Melbourne Victoria Australia"},{"name":"University of Melbourne Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Wendell F.","family":"Rosse","sequence":"additional","affiliation":[{"name":"Duke University Medical Center Durham North Carolina USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2017,9,10]]},"reference":[{"key":"e_1_2_7_2_1","first-page":"419","volume-title":"Hematology Basic Principles and Practices","author":"Brodsky R","year":"2005"},{"key":"e_1_2_7_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2005-04-1717"},{"key":"e_1_2_7_4_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12185-013-1346-4"},{"key":"e_1_2_7_5_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.21757"},{"key":"e_1_2_7_6_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJM199511093331904"},{"key":"e_1_2_7_7_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1445-5994.2012.02924.x"},{"key":"e_1_2_7_8_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2013.093161"},{"key":"e_1_2_7_9_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa061648"},{"key":"e_1_2_7_10_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-06-094136"},{"key":"e_1_2_7_11_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2007-06-095646"},{"key":"e_1_2_7_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/bjh.12347"},{"key":"e_1_2_7_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.V120.21.3472.3472"},{"key":"e_1_2_7_14_1","doi-asserted-by":"publisher","DOI":"10.1056\/NEJMoa031688"},{"key":"e_1_2_7_15_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2011-02-333997"},{"key":"e_1_2_7_16_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-012-1433-2"},{"key":"e_1_2_7_17_1","unstructured":"CellaD. 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