{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,25]],"date-time":"2026-08-25T03:52:47Z","timestamp":1787629967670,"version":"build-2736575974"},"reference-count":38,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2023,11,23]],"date-time":"2023-11-23T00:00:00Z","timestamp":1700697600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100019531","name":"Apellis Pharmaceuticals","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100019531","id-type":"DOI","asserted-by":"publisher"}]},{"DOI":"10.13039\/501100012112","name":"Swedish Orphan Biovitrum","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100012112","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":[[2024,4]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To describe real\u2010world use\/effectiveness of pegcetacoplan (PEG) in paroxysmal nocturnal haemoglobinuria (PNH).<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Data were drawn from the Adelphi PNH Disease Specific Programme\u2122, a cross\u2010sectional survey conducted in France, Italy, Germany, Spain and the United States from January to November 2022. Patients had a confirmed PNH diagnosis and received PEG for \u22651\u2009month. Physicians reported patient characteristics, treatment use\/satisfaction and their perception of patients' fatigue and health\u2010related quality of life (HRQoL). Patients reported treatment satisfaction and completed questionnaires assessing fatigue, HRQoL and productivity. Descriptive statistics were reported.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Overall, 14 physicians provided data for 61 patients who had received 1080\u2009mg\/dose PEG for 1.3\u201314.8\u2009months. At data collection compared to PEG initiation: haemoglobin was 2.5\u2009g\/dL higher on average; proportion of patients with lactate dehydrogenase (LDH) \u22651.5\u2009\u00d7\u2009upper limit of normal was reduced by 27.4%; physician\u2010perceived fatigue was lower and HRQoL better. Physician\u2010 and patient\u2010reported treatment satisfaction was high for &gt;90% of patients. Physicians and patients were more satisfied with PEG than previously prescribed C5 complement inhibitors. Mean work impairment and activity impairment in the 7\u2009days prior to data collection were 32.9% and 22.4%, respectively.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>These real\u2010world data support the effectiveness of PEG through positive effects on haemoglobin, LDH, fatigue and HRQoL.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14139","type":"journal-article","created":{"date-parts":[[2023,11,25]],"date-time":"2023-11-25T00:32:30Z","timestamp":1700872350000},"page":"516-529","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":10,"title":["Pegcetacoplan in paroxysmal nocturnal haemoglobinuria: Its use, its clinical effectiveness, and its influence on health\u2010related quality of life and productivity"],"prefix":"10.1111","volume":"112","author":[{"given":"Koo","family":"Wilson","sequence":"first","affiliation":[{"name":"Sobi  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Carly","family":"Rich","sequence":"additional","affiliation":[{"name":"Sobi  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zalmai","family":"Hakimi","sequence":"additional","affiliation":[{"name":"Sobi  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Horneff","sequence":"additional","affiliation":[{"name":"Sobi  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Fishman","sequence":"additional","affiliation":[{"name":"Apellis Pharmaceuticals  Waltham Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jennifer","family":"Mellor","sequence":"additional","affiliation":[{"name":"Adelphi Real World  Cheshire UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lucy","family":"Earl","sequence":"additional","affiliation":[{"name":"Adelphi Real World  Cheshire UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Yasmin","family":"Taylor","sequence":"additional","affiliation":[{"name":"Adelphi Real World  Cheshire UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alice","family":"Simons","sequence":"additional","affiliation":[{"name":"Adelphi Real World  Cheshire UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joe","family":"Conyers","sequence":"additional","affiliation":[{"name":"Adelphi Real World  Cheshire UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Mulherin","sequence":"additional","affiliation":[{"name":"Hematology Oncology of Indiana  Indianapolis Indiana USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elaine","family":"Majerus","sequence":"additional","affiliation":[{"name":"Department of Medicine Washington University  St. Louis Missouri USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"R\u00f6th","sequence":"additional","affiliation":[{"name":"Department of Hematology and Stem Cell Transplantation University Hospital Essen, West German Cancer Center, University of Duisburg\u2010Essen  Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,11,23]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2017.28"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2019003812"},{"key":"e_1_2_12_4_1","unstructured":"Global PNH Patient Registry.About the Global PNH Patient Registry. 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