{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,5,30]],"date-time":"2026-05-30T20:10:29Z","timestamp":1780171829023,"version":"3.54.0"},"reference-count":52,"publisher":"Wiley","issue":"1","license":[{"start":{"date-parts":[[2023,4,19]],"date-time":"2023-04-19T00:00:00Z","timestamp":1681862400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"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":[[2023,7]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>Paroxysmal nocturnal haemoglobinuria (PNH) is a rare, non\u2010malignant haematological disorder associated with disabling fatigue and reduced health\u2010related quality of life. Post hoc analysis of PEGASUS phase 3 trial (NCT03500549) characterised improvements in patient\u2010reported fatigue measured by functional assessment of chronic illness therapy\u2010fatigue (FACIT\u2010fatigue) instrument item\u2010level ratings for pegcetacoplan and eculizumab for the treatment of PNH.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Item\u2010level responder analysis was conducted on a\u2009\u22652\u2010level change from baseline (CFB) clinically important response (CIR) for the FACIT\u2010fatigue 13 individual items rated on a 5\u2010level Likert scale. We evaluated \u22652\u2010level change against the minimal clinically important difference (MCID) of the FACIT\u2010fatigue total score (\u22655 points) and clinical parameters, haemoglobin (Hb; \u22651\u2009g\/dL) and normalised absolute reticulocyte count (ARC; 30\u2013100\u2009pg\/cells). Logistic regressions estimated baseline\u2010to\u2010Week\u201016 FACIT\u2010fatigue item\u2010level transitional probabilities; Kaplan\u2013Meier analysis estimated time to FACIT\u2010fatigue item CIR.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Pegcetacoplan versus eculizumab was associated with significantly greater odds of Week 16 CIR across 8\/13 items and on total score MCID (OR\u2009[CI] =\u200911.19 [3.73, 33.57]) and faster times to responses. The item\u2010level CIR threshold also showed clinical relevance on Hb level and ARC normalization.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Compared with eculizumab, pegcetacoplan was associated with clinically meaningful greater improvements on a majority of FACIT\u2010fatigue items.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.13969","type":"journal-article","created":{"date-parts":[[2023,3,25]],"date-time":"2023-03-25T20:02:00Z","timestamp":1679774520000},"page":"72-83","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":6,"title":["Fatigue and health\u2010related quality of life in paroxysmal nocturnal haemoglobinuria: A post hoc analysis of the pegcetacoplan <scp>PEGASUS<\/scp> trial data"],"prefix":"10.1111","volume":"111","author":[{"ORCID":"https:\/\/orcid.org\/0000-0001-6316-3112","authenticated-orcid":false,"given":"Jens","family":"Panse","sequence":"first","affiliation":[{"name":"Department of Oncology, Hematology, Hemostaseology and Stem Cell Transplantation University Hospital RWTH Aachen  Aachen Germany"},{"name":"Center for Integrated Oncology Aachen Bonn, Cologne, Duesseldorf (CIO ABCD)  Aachen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Koo","family":"Wilson","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Fishman","sequence":"additional","affiliation":[{"name":"Apellis Pharmaceuticals, Inc.  Waltham Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Piotr","family":"Wojciechowski","sequence":"additional","affiliation":[{"name":"Assignity Sp. z O.O.  Krakow Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Marlena","family":"Wdowiak","sequence":"additional","affiliation":[{"name":"Putnam Associates  Krakow Poland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Horneff","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher J.","family":"Patriquin","sequence":"additional","affiliation":[{"name":"Division of Hematology University Health Network  Toronto Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika","family":"Oliver","sequence":"additional","affiliation":[{"name":"Division of Hematology University of Alberta  Alberta Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Zalmai","family":"Hakimi","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,4,19]]},"reference":[{"key":"e_1_2_12_2_1","doi-asserted-by":"publisher","DOI":"10.2147\/btt.s1420"},{"key":"e_1_2_12_3_1","doi-asserted-by":"publisher","DOI":"10.1080\/10245332.2018.1444563"},{"issue":"2","key":"e_1_2_12_4_1","first-page":"19","article-title":"PESG PNH diagnosis, follow\u2010up and treatment guidelines","volume":"6","author":"Sahin F","year":"2016","journal-title":"Am J Blood Res"},{"key":"e_1_2_12_5_1","unstructured":"National Institute for Health and Care Excellence.Single technology appraisal: ravulizumab for treating paroxysmal nocturnal haemoglobinuria [ID1457].2021Accessed September 29 2021.https:\/\/www.nice.org.uk\/guidance\/ta698\/evidence\/committee-papers-pdf-9132078637"},{"issue":"20","key":"e_1_2_12_6_1","first-page":"S376","article-title":"Overview of venous thromboembolism","volume":"23","author":"Phillippe HM","year":"2017","journal-title":"Am J Manag Care"},{"key":"e_1_2_12_7_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277\u2010020\u201004052\u2010z"},{"key":"e_1_2_12_8_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.12543"},{"key":"e_1_2_12_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102141.2012.09166.x"},{"key":"e_1_2_12_10_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2018.04.001"},{"key":"e_1_2_12_11_1","doi-asserted-by":"publisher","DOI":"10.1007\/s12325\u2010021\u201001825\u20104"},{"key":"e_1_2_12_12_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277\u2010021\u201004715\u20105"},{"key":"e_1_2_12_13_1","doi-asserted-by":"publisher","DOI":"10.2147\/jbm.S339660"},{"key":"e_1_2_12_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102141.2008.07183.x"},{"key":"e_1_2_12_15_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.25960"},{"key":"e_1_2_12_16_1","doi-asserted-by":"publisher","DOI":"10.3389\/fimmu.2019.01157"},{"key":"e_1_2_12_17_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2017.28"},{"key":"e_1_2_12_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.blre.2007.10.002"},{"key":"e_1_2_12_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.lrr.2020.100224"},{"key":"e_1_2_12_20_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2019.236877"},{"key":"e_1_2_12_21_1","doi-asserted-by":"publisher","DOI":"10.2165\/11208300\u2010000000000\u201000000"},{"key":"e_1_2_12_22_1","doi-asserted-by":"publisher","DOI":"10.1002\/ajh.20144"},{"key":"e_1_2_12_23_1","unstructured":"Alexion.Solirus (eculizumab) [package insert]. 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