{"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":1789192679092,"version":"build-2803163510"},"reference-count":50,"publisher":"Wiley","issue":"4","license":[{"start":{"date-parts":[[2022,7,7]],"date-time":"2022-07-07T00:00:00Z","timestamp":1657152000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["European J of Haematology"],"published-print":{"date-parts":[[2022,10]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>To assess the clinical, humanistic and economic burden of paroxysmal nocturnal haemoglobinuria (PNH) among C5 inhibitor (C5i)\u2010treated patients with PNH.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>This was a web\u2010based, cross\u2010sectional survey (01FEB2021\u201031MAR2021) of adults with PNH treated with eculizumab (France, Germany, United Kingdom) or ravulizumab (Germany). Self\u2010reported outcomes included: patient characteristics; patient\u2010reported symptoms; and standardised patient\u2010reported outcomes (e.g. Functional Assessment of Chronic Illness Therapy [FACIT]\u2010Fatigue, European Organization for Research and Treatment of Cancer Quality of Life Questionnaire Core 30 [EORTC QLQ\u2010C30]).<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>Among 71 included patients, 98.6% were C5i\u2010treated for \u22653\u00a0months (88.7% \u226512\u2009months); among those with self\u2010reported haemoglobin (Hb) levels (<jats:italic>n<\/jats:italic>\u00a0=\u200963), most (85.7%) were anaemic (defined as \u226412.0\u00a0g\/dL). Fatigue was the most common symptom at both diagnosis (73.2%) and survey time (63.4%); there were no statistically significant differences in symptom prevalence between treatment subgroups (eculizumab vs. ravulizumab). Total FACIT\u2010Fatigue and EORTC QLQ\u2010C30 scores were substantially lower than European general population references, but there were no statistically significant differences between treatment subgroups. Hb\u2010level subgroups (&lt;10.5\u00a0g\/dL vs. \u226510.5\u00a0d\/dL) followed similar trends for all measures, with few significant subgroup differences.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Results suggest that there remains a considerable burden and unmet need among C5i\u2010treated patients with PNH that requires improved therapies.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.13816","type":"journal-article","created":{"date-parts":[[2022,6,24]],"date-time":"2022-06-24T05:04:02Z","timestamp":1656047042000},"page":"351-363","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":35,"title":["The burden of illness of patients with paroxysmal nocturnal haemoglobinuria receiving <scp>C5<\/scp> inhibitors in France, Germany and the United Kingdom: Patient\u2010reported insights on symptoms and quality of life"],"prefix":"10.1111","volume":"109","author":[{"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 (CIO) Aachen, Bonn, Cologne, Duesseldorf  Aachen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Flore","family":"Sicre de Fontbrune","sequence":"additional","affiliation":[{"name":"Hematology transplant Unit\u2014French Reference Center for Aplastic Anemia H\u00f4pital Saint\u2010Louis  Paris France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Pascale","family":"Burmester","sequence":"additional","affiliation":[{"name":"Stiftung Lichterzellen  Cologne Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Maria","family":"Piggin","sequence":"additional","affiliation":[{"name":"PNH Support  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joana E.","family":"Matos","sequence":"additional","affiliation":[{"name":"Kantar Health  New York New York USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Halley","family":"Costantino","sequence":"additional","affiliation":[{"name":"Cerner Enviza (previously Kantar Health)  Malvern Pennsylvania USA"}],"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":"Zalmai","family":"Hakimi","sequence":"additional","affiliation":[{"name":"Apellis Pharmaceuticals  Zug Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jameel","family":"Nazir","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Renaud","family":"Desgraz","sequence":"additional","affiliation":[{"name":"Apellis Pharmaceuticals  Zug Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Fishman","sequence":"additional","affiliation":[{"name":"Department of Haematology King's College Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Emmelie","family":"Persson","sequence":"additional","affiliation":[{"name":"Swedish Orphan Biovitrum AB  Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3180-3570","authenticated-orcid":false,"given":"Austin","family":"Kulasekararaj","sequence":"additional","affiliation":[{"name":"Department of Haematology King's College Hospital  London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2022,7,7]]},"reference":[{"key":"e_1_2_8_2_1","doi-asserted-by":"publisher","DOI":"10.1038\/nrdp.2017.28"},{"key":"e_1_2_8_3_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277\u2010020\u201004052\u2010z"},{"key":"e_1_2_8_4_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.12543"},{"key":"e_1_2_8_5_1","unstructured":"Orphanet.Paroxysmal nocturnal hemoglobinuria.Orphanet J Rare Dis. 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