{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,1]],"date-time":"2026-09-01T02:24:48Z","timestamp":1788229488201,"version":"build-2803163510"},"reference-count":42,"publisher":"Springer Science and Business Media LLC","issue":"5","license":[{"start":{"date-parts":[[2024,4,4]],"date-time":"2024-04-04T00:00:00Z","timestamp":1712188800000},"content-version":"tdm","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/4.0"},{"start":{"date-parts":[[2024,4,4]],"date-time":"2024-04-04T00:00:00Z","timestamp":1712188800000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by-nc\/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":["link.springer.com"],"crossmark-restriction":false},"short-container-title":["Adv Ther"],"published-print":{"date-parts":[[2024,5]]},"DOI":"10.1007\/s12325-024-02827-8","type":"journal-article","created":{"date-parts":[[2024,4,4]],"date-time":"2024-04-04T14:03:13Z","timestamp":1712239393000},"page":"2050-2069","update-policy":"https:\/\/doi.org\/10.1007\/springer_crossmark_policy","source":"Crossref","is-referenced-by-count":17,"title":["Safety and Efficacy of Pegcetacoplan in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria over 48\u00a0Weeks: 307 Open-Label Extension Study"],"prefix":"10.1007","volume":"41","author":[{"given":"Christopher J.","family":"Patriquin","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrija","family":"Bogdanovic","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Morag","family":"Griffin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Richard J.","family":"Kelly","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jaroslaw P.","family":"Maciejewski","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Brian","family":"Mulherin","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"R\u00e9gis","family":"Peffault de Latour","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alexander","family":"R\u00f6th","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Veena","family":"Selvaratnam","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeffrey","family":"Szer","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Mohammed","family":"Al-Adhami","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Regina","family":"Horneff","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lisa","family":"Tan","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Michael","family":"Yeh","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jens","family":"Panse","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"297","published-online":{"date-parts":[[2024,4,4]]},"reference":[{"issue":"Suppl 4","key":"2827_CR1","doi-asserted-by":"publisher","first-page":"S33","DOI":"10.1002\/ajh.26882","volume":"98","author":"AG Kulasekararaj","year":"2023","unstructured":"Kulasekararaj AG, Lazana I. Paroxysmal nocturnal hemoglobinuria: where are we going. Am J Hematol. 2023;98(Suppl 4):S33\u201343. https:\/\/doi.org\/10.1002\/ajh.26882.","journal-title":"Am J Hematol"},{"key":"2827_CR2","doi-asserted-by":"publisher","first-page":"1304","DOI":"10.1182\/blood.2019003812","volume":"137","author":"RA Brodsky","year":"2021","unstructured":"Brodsky RA. How I treat paroxysmal nocturnal hemoglobinuria. Blood. 2021;137:1304\u20139. https:\/\/doi.org\/10.1182\/blood.2019003812.","journal-title":"Blood"},{"key":"2827_CR3","doi-asserted-by":"publisher","first-page":"2804","DOI":"10.1182\/blood-2014-02-522128","volume":"124","author":"RA Brodsky","year":"2014","unstructured":"Brodsky RA. Paroxysmal nocturnal hemoglobinuria. Blood. 2014;124:2804\u201311. https:\/\/doi.org\/10.1182\/blood-2014-02-522128.","journal-title":"Blood"},{"key":"2827_CR4","doi-asserted-by":"publisher","first-page":"4985","DOI":"10.1182\/blood-2012-09-311381","volume":"121","author":"A Hill","year":"2013","unstructured":"Hill A, Kelly RJ, Hillmen P. Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood. 2013;121:4985\u20135105. https:\/\/doi.org\/10.1182\/blood-2012-09-311381.","journal-title":"Blood"},{"key":"2827_CR5","doi-asserted-by":"publisher","first-page":"552","DOI":"10.1056\/NEJMoa031688","volume":"350","author":"P Hillmen","year":"2004","unstructured":"Hillmen P, Hall C, Marsh JC, et al. Effect of eculizumab on hemolysis and transfusion requirements in patients with paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2004;350:552\u20139. https:\/\/doi.org\/10.1056\/NEJMoa031688.","journal-title":"N Engl J Med"},{"key":"2827_CR6","doi-asserted-by":"publisher","first-page":"1233","DOI":"10.1056\/NEJMoa061648","volume":"355","author":"P Hillmen","year":"2006","unstructured":"Hillmen P, Young NS, Schubert J, et al. The complement inhibitor eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2006;355:1233\u201343. https:\/\/doi.org\/10.1056\/NEJMoa061648.","journal-title":"N Engl J Med"},{"key":"2827_CR7","doi-asserted-by":"publisher","first-page":"1840","DOI":"10.1182\/blood-2007-06-094136","volume":"111","author":"RA Brodsky","year":"2008","unstructured":"Brodsky RA, Young NS, Antonioli E, et al. Multicenter phase\u00a03 study of the complement inhibitor eculizumab for the treatment of patients with paroxysmal nocturnal hemoglobinuria. Blood. 2008;111:1840\u20137. https:\/\/doi.org\/10.1182\/blood-2007-06-094136.","journal-title":"Blood"},{"key":"2827_CR8","doi-asserted-by":"publisher","first-page":"263","DOI":"10.1111\/j.1365-2141.2008.07183.x","volume":"142","author":"J Schubert","year":"2008","unstructured":"Schubert J, Hillmen P, R\u00f6th A, et al. Eculizumab, a terminal complement inhibitor, improves anaemia in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2008;142:263\u201372. https:\/\/doi.org\/10.1111\/j.1365-2141.2008.07183.x.","journal-title":"Br J Haematol"},{"key":"2827_CR9","doi-asserted-by":"publisher","unstructured":"Lee JW, Peffault de Latour R, Brodsky RA, et al. Effectiveness of eculizumab in patients with paroxysmal nocturnal hemoglobinuria (PNH) with or without aplastic anemia in the International PNH Registry. Am J Hematol. 2019;94:E37\u2013E41. https:\/\/doi.org\/10.1002\/ajh.25334.","DOI":"10.1002\/ajh.25334"},{"key":"2827_CR10","doi-asserted-by":"publisher","first-page":"540","DOI":"10.1182\/blood-2018-09-876805","volume":"133","author":"AG Kulasekararaj","year":"2019","unstructured":"Kulasekararaj AG, Hill A, Rottinghaus ST, et al. Ravulizumab (ALXN1210) vs eculizumab in C5-inhibitor-experienced adult patients with PNH: the 302 study. Blood. 2019;133:540\u20139. https:\/\/doi.org\/10.1182\/blood-2018-09-876805.","journal-title":"Blood"},{"key":"2827_CR11","doi-asserted-by":"publisher","first-page":"6786","DOI":"10.1182\/blood-2011-02-333997","volume":"117","author":"RJ Kelly","year":"2011","unstructured":"Kelly RJ, Hill A, Arnold LM, et al. Long-term treatment with eculizumab in paroxysmal nocturnal hemoglobinuria: sustained efficacy and improved survival. Blood. 2011;117:6786\u201392. https:\/\/doi.org\/10.1182\/blood-2011-02-333997.","journal-title":"Blood"},{"key":"2827_CR12","doi-asserted-by":"publisher","first-page":"84","DOI":"10.1111\/ejh.13970","volume":"111","author":"K Versmold","year":"2023","unstructured":"Versmold K, Alashkar F, Raiser C, et al. Long-term outcomes of patients with paroxysmal nocturnal hemoglobinuria treated with eculizumab in a real-world setting. Eur J Haematol. 2023;111:84\u201395. https:\/\/doi.org\/10.1111\/ejh.13970.","journal-title":"Eur J Haematol"},{"issue":"Suppl 1","key":"2827_CR13","first-page":"3471","volume":"130","author":"CE McKinley","year":"2017","unstructured":"McKinley CE, Richards SJ, Munir T, et al. Extravascular hemolysis due to C3-loading in patients with PNH treated with eculizumab: defining the clinical syndrome. Blood. 2017;130(Suppl 1):3471.","journal-title":"Blood"},{"key":"2827_CR14","doi-asserted-by":"publisher","first-page":"251","DOI":"10.1007\/s00277-021-04715-5","volume":"101","author":"D Dingli","year":"2022","unstructured":"Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101:251\u201363. https:\/\/doi.org\/10.1007\/s00277-021-04715-5.","journal-title":"Ann Hematol"},{"key":"2827_CR15","doi-asserted-by":"publisher","first-page":"366","DOI":"10.1002\/ajh.24278","volume":"91","author":"M Loschi","year":"2016","unstructured":"Loschi M, Porcher R, Barraco F, et al. Impact of eculizumab treatment on paroxysmal nocturnal hemoglobinuria: a treatment versus no-treatment study. Am J Hematol. 2016;91:366\u201370. https:\/\/doi.org\/10.1002\/ajh.24278.","journal-title":"Am J Hematol"},{"key":"2827_CR16","doi-asserted-by":"publisher","first-page":"1157","DOI":"10.3389\/fimmu.2019.01157","volume":"10","author":"AM Risitano","year":"2019","unstructured":"Risitano AM, Marotta S, Ricci P, et al. Anti-complement treatment for paroxysmal nocturnal hemoglobinuria: time for proximal complement inhibition? A position paper from the SAAWP of the EBMT. Front Immunol. 2019;10:1157. https:\/\/doi.org\/10.3389\/fimmu.2019.01157.","journal-title":"Front Immunol"},{"key":"2827_CR17","doi-asserted-by":"publisher","first-page":"567","DOI":"10.3324\/haematol.2009.007229","volume":"95","author":"A Hill","year":"2010","unstructured":"Hill A, Rother RP, Arnold L, et al. Eculizumab prevents intravascular hemolysis in patients with paroxysmal nocturnal hemoglobinuria and unmasks low-level extravascular hemolysis occurring through C3 opsonization. Haematologica. 2010;95:567\u201373. https:\/\/doi.org\/10.3324\/haematol.2009.007229.","journal-title":"Haematologica"},{"key":"2827_CR18","unstructured":"Apellis. EMPAVELI (pegcetacoplan) Prescribing Information. 2023. https:\/\/pi.apellis.com\/files\/PI_Empaveli.pdf. Accessed Jan 24, 2024."},{"key":"2827_CR19","unstructured":"EMA. ASPAVELI (pegcetacoplan) Summary of Product Characteristics. Swedish Orphan Biovitrum AB; 2022. https:\/\/www.ema.europa.eu\/en\/documents\/product-information\/aspaveli-epar-product-information_en.pdf. Accessed Jan 24, 2024."},{"key":"2827_CR20","unstructured":"National Institute for Health and Care Excellence. Pegcetacoplan for treating paroxysmal nocturnal haemoglobinuria. March 9, 2022. http:\/\/www.nice.org.uk\/guidance\/ta778. Accessed Jan 24, 2024."},{"key":"2827_CR21","first-page":"1","volume":"3","author":"Canadian Agency for Drugs and Technologies in Health","year":"2023","unstructured":"Canadian Agency for Drugs and Technologies in Health. CADTH reimbursement recommendation: pegcetacoplan (Empaveli). Can J Med Technol. 2023;3:1\u201324.","journal-title":"Can J Med Technol"},{"key":"2827_CR22","unstructured":"Australian Government. Department of Health and Aged Care, Therapeutic Goods Administration. Australian public assessment report for pegcetacoplan. August 2022. https:\/\/www.tga.gov.au\/sites\/default\/files\/2022-09\/auspar-pegcetacoplan-220902.docx. Accessed Jan 24, 2024."},{"key":"2827_CR23","unstructured":"Apellis Pharmaceuticals, Inc. Apellis Pharmaceuticals reports first quarter 2023 financial results. Press release. May\u00a04, 2023. https:\/\/investors.apellis.com\/news-releases\/news-release-details\/apellis-pharmaceuticals-reports-first-quarter-2023-financial. Accessed Jan 24, 2024."},{"key":"2827_CR24","doi-asserted-by":"publisher","first-page":"1028","DOI":"10.1056\/NEJMoa2029073","volume":"384","author":"P Hillmen","year":"2021","unstructured":"Hillmen P, Szer J, Weitz I, et al. Pegcetacoplan versus eculizumab in paroxysmal nocturnal hemoglobinuria. N Engl J Med. 2021;384:1028\u201337. https:\/\/doi.org\/10.1056\/NEJMoa2029073.","journal-title":"N Engl J Med"},{"key":"2827_CR25","doi-asserted-by":"publisher","first-page":"e648","DOI":"10.1016\/S2352-3026(22)00210-1","volume":"9","author":"RP de Latour","year":"2022","unstructured":"de Latour RP, Szer J, Weitz IC, et al. Pegcetacoplan versus eculizumab in patients with paroxysmal nocturnal haemoglobinuria (PEGASUS): 48-week follow-up of a randomised, open-label, phase\u00a03, active-comparator, controlled trial. Lancet Haematol. 2022;9:e648\u201359. https:\/\/doi.org\/10.1016\/S2352-3026(22)00210-1.","journal-title":"Lancet Haematol"},{"key":"2827_CR26","doi-asserted-by":"publisher","first-page":"2468","DOI":"10.1182\/bloodadvances.2022009129","volume":"7","author":"RSM Wong","year":"2023","unstructured":"Wong RSM, Navarro-Cabrera JR, Comia NS, et al. Pegcetacoplan controls hemolysis in complement inhibitor-naive patients with paroxysmal nocturnal hemoglobinuria. Blood Adv. 2023;7:2468\u201378. https:\/\/doi.org\/10.1182\/bloodadvances.2022009129.","journal-title":"Blood Adv"},{"key":"2827_CR27","doi-asserted-by":"publisher","first-page":"1334","DOI":"10.1002\/ajh.25960","volume":"95","author":"C de Castro","year":"2020","unstructured":"de Castro C, Grossi F, Weitz IC, et al. C3 inhibition with pegcetacoplan in subjects with paroxysmal nocturnal hemoglobinuria treated with eculizumab. Am J Hematol. 2020;95:1334\u201343. https:\/\/doi.org\/10.1002\/ajh.25960.","journal-title":"Am J Hematol"},{"key":"2827_CR28","doi-asserted-by":"publisher","first-page":"1971","DOI":"10.1007\/s00277-022-04903-x","volume":"101","author":"RSM Wong","year":"2022","unstructured":"Wong RSM, Pullon HWH, Amine I, et al. Inhibition of C3 with pegcetacoplan results in normalization of hemolysis markers in paroxysmal nocturnal hemoglobinuria. Ann Hematol. 2022;101:1971\u201386. https:\/\/doi.org\/10.1007\/s00277-022-04903-x.","journal-title":"Ann Hematol"},{"key":"2827_CR29","doi-asserted-by":"publisher","first-page":"528","DOI":"10.1002\/cncr.10245","volume":"94","author":"D Cella","year":"2002","unstructured":"Cella D, Lai JS, Chang CH, Peterman A, Slavin M. Fatigue in cancer patients compared with fatigue in the general United States population. Cancer. 2002;94:528\u201338. https:\/\/doi.org\/10.1002\/cncr.10245.","journal-title":"Cancer"},{"key":"2827_CR30","doi-asserted-by":"publisher","first-page":"E28","DOI":"10.1002\/ajh.26841","volume":"98","author":"D Dingli","year":"2023","unstructured":"Dingli D, Yeh M, Al-Adhami M, Min J, Savage J, Weitz I. Abstracts from the THSNA Thrombosis & Hemostasis Summit of North America: Increased D-dimer normalization and reduced incidence of thrombosis in pegcetacoplan-treated patients with paroxysmal nocturnal hemoglobinuria: week\u00a048 interim results from the 307 long-term extension study. Am J Hematol. 2023;98:E28\u20139. https:\/\/doi.org\/10.1002\/ajh.26841.","journal-title":"Am J Hematol"},{"key":"2827_CR31","doi-asserted-by":"publisher","first-page":"63","DOI":"10.1186\/s41687-023-00609-4","volume":"7","author":"D Cella","year":"2023","unstructured":"Cella D, Johansson P, Ueda Y, et al. Clinically important change for the FACIT-Fatigue scale in paroxysmal nocturnal hemoglobinuria: a derivation from International PNH Registry patient data. J Patient Rep Outcomes. 2023;7:63. https:\/\/doi.org\/10.1186\/s41687-023-00609-4.","journal-title":"J Patient Rep Outcomes"},{"key":"2827_CR32","doi-asserted-by":"publisher","first-page":"72","DOI":"10.1111\/ejh.13969","volume":"111","author":"J Panse","year":"2023","unstructured":"Panse J, Wilson K, Fishman J, et al. Fatigue and health-related quality of life in paroxysmal nocturnal haemoglobinuria: a post hoc analysis of the pegcetacoplan PEGASUS trial data. Eur J Haematol. 2023;111:72\u201383. https:\/\/doi.org\/10.1111\/ejh.13969.","journal-title":"Eur J Haematol"},{"key":"2827_CR33","doi-asserted-by":"publisher","unstructured":"Hillmen P, Risitano AM, Peffault de Latour R. Pegcetacoplan versus eculizumab in PNH. Reply. N Engl J Med. 2021;385:1725\u20136. https:\/\/doi.org\/10.1056\/NEJMc2106424.","DOI":"10.1056\/NEJMc2106424"},{"issue":"Suppl 1","key":"2827_CR34","doi-asserted-by":"publisher","first-page":"2937","DOI":"10.1182\/blood-2022-163117","volume":"140","author":"M Griffin","year":"2022","unstructured":"Griffin M, Kelly R, Deeren D, et al. Intensive pegcetacoplan dosing in the management of acute hemolysis as part of the 307 open-label extension study. Blood. 2022;140(Suppl 1):2937\u20139. https:\/\/doi.org\/10.1182\/blood-2022-163117.","journal-title":"Blood"},{"key":"2827_CR35","doi-asserted-by":"publisher","first-page":"406","DOI":"10.1007\/s12185-013-1404-y","volume":"98","author":"Y Kanakura","year":"2013","unstructured":"Kanakura Y, Ohyashiki K, Shichishima T, et al. Long-term efficacy and safety of eculizumab in Japanese patients with PNH: AEGIS trial. Int J Hematol. 2013;98:406\u201316. https:\/\/doi.org\/10.1007\/s12185-013-1404-y.","journal-title":"Int J Hematol"},{"key":"2827_CR36","doi-asserted-by":"publisher","first-page":"62","DOI":"10.1111\/bjh.12347","volume":"162","author":"P Hillmen","year":"2013","unstructured":"Hillmen P, Muus P, R\u00f6th A, et al. Long-term safety and efficacy of sustained eculizumab treatment in patients with paroxysmal nocturnal haemoglobinuria. Br J Haematol. 2013;162:62\u201373. https:\/\/doi.org\/10.1111\/bjh.12347.","journal-title":"Br J Haematol"},{"key":"2827_CR37","doi-asserted-by":"publisher","first-page":"205","DOI":"10.1111\/ejh.13783","volume":"109","author":"AG Kulasekararaj","year":"2022","unstructured":"Kulasekararaj AG, Griffin M, Langemeijer S, et al. Long-term safety and efficacy of ravulizumab in patients with paroxysmal nocturnal hemoglobinuria: 2-year results from two pivotal phase\u00a03 studies. Eur J Haematol. 2022;109:205\u201314. https:\/\/doi.org\/10.1111\/ejh.13783.","journal-title":"Eur J Haematol"},{"key":"2827_CR38","doi-asserted-by":"publisher","unstructured":"Lee JW, Sicre de Fontbrune F, Wong Lee Lee L, et al. Ravulizumab (ALXN1210) vs eculizumab in adult patients with PNH naive to complement inhibitors: the 301 study. Blood. 2019;133:530\u20139. https:\/\/doi.org\/10.1182\/blood-2018-09-876136.","DOI":"10.1182\/blood-2018-09-876136"},{"key":"2827_CR39","unstructured":"FDA. FABHALTA (iptacopan) Prescribing Information. 2023. https:\/\/www.accessdata.fda.gov\/drugsatfda_docs\/label\/2023\/218276s000lbl.pdf. Accessed Jan 24 2024."},{"key":"2827_CR40","unstructured":"ULTOMIRIS (ravulizumab-cwvz) Prescribing Information. 2022. https:\/\/alexion.com\/Documents\/Ultomiris_USPI.pdf. Accessed Jan 24 2024."},{"key":"2827_CR41","unstructured":"EMA. ULTOMIRIS (ravulizumab-cwvz) Summary of Product Characteristics. Swedish Orphan Biovitrum AB; 2023. https:\/\/www.ema.europa.eu\/en\/medicines\/human\/EPAR\/ultomiris#ema-inpage-item-product-info. Accessed Jan 24 2024."},{"key":"2827_CR42","doi-asserted-by":"crossref","unstructured":"de Latour RP, Szer J, Kulasekararaj A, et al. The COMPLETE study: a single arm, multicenter observational study to evaluate effectiveness of pegcetacoplan under real world conditions in patients with paroxysmal nocturnal hemoglobinuria (PNH). HemaSphere. 2023;7(S3):e36410cc. https:\/\/journals.lww.com\/hemasphere\/fulltext\/2023\/08003\/pb2064__the_complete_study__a_single_arm,.1938.aspx.","DOI":"10.1097\/01.HS9.0000975056.36410.cc"}],"container-title":["Advances in Therapy"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-024-02827-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/article\/10.1007\/s12325-024-02827-8\/fulltext.html","content-type":"text\/html","content-version":"vor","intended-application":"text-mining"},{"URL":"https:\/\/link.springer.com\/content\/pdf\/10.1007\/s12325-024-02827-8.pdf","content-type":"application\/pdf","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2024,4,26]],"date-time":"2024-04-26T17:14:05Z","timestamp":1714151645000},"score":1,"resource":{"primary":{"URL":"https:\/\/link.springer.com\/10.1007\/s12325-024-02827-8"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2024,4,4]]},"references-count":42,"journal-issue":{"issue":"5","published-print":{"date-parts":[[2024,5]]}},"alternative-id":["2827"],"URL":"https:\/\/doi.org\/10.1007\/s12325-024-02827-8","relation":{},"ISSN":["0741-238X","1865-8652"],"issn-type":[{"value":"0741-238X","type":"print"},{"value":"1865-8652","type":"electronic"}],"subject":[],"published":{"date-parts":[[2024,4,4]]},"assertion":[{"value":"7 December 2023","order":1,"name":"received","label":"Received","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"19 February 2024","order":2,"name":"accepted","label":"Accepted","group":{"name":"ArticleHistory","label":"Article History"}},{"value":"4 April 2024","order":3,"name":"first_online","label":"First Online","group":{"name":"ArticleHistory","label":"Article History"}},{"order":1,"name":"Ethics","group":{"name":"EthicsHeading","label":"Declarations"}},{"value":"Michael Yeh is an employee of Apellis and holds stock options. Mohammed Al-Adhami was an employee of Apellis at the time of study completion. Christopher J. Patriquin reports relationships with Alexion, Apellis, Regeneron, Takeda, BioCryst, Swedish Orphan Biovitrum AB, Amgen, and Novartis. Andrija Bogdanovic reports relationships with Novartis, Takeda, Pfizer, and Apellis. Morag Griffin reports consultancy at Biocryst and Regeneron; honoraria from Alexion and Swedish Orphan Biovitrum AB; advisory board member at Novartis, Biocryst, Alexion, Amgen, and Swedish Orphan Biovitrum AB; and educational work sponsored by Apellis with unrestricted grant paid to Medscape. Richard J. Kelly has received research funding from Novartis, reports consultancy for Swedish Orphan Biovitrum AB, sat on advisory boards for Alexion, AbbVie, Amgen, Jazz, Novartis, and Swedish Orphan Biovitrum AB, and received lecture fees from Alexion, Astellas, Amgen, Biologix, Jazz, and Swedish Orphan Biovitrum AB. Jaroslaw P. Maciejewski has nothing to disclose. Brian Mulherin has nothing to disclose. R\u00e9gis Peffault de Latour has nothing to disclose. Alexander R\u00f6th reports personal fees from Alexion, Apellis, Kira, Novartis, Roche, Swedish Orphan Biovitrum AB, Sanofi, Bioverativ, and Grifols; and grants from Roche. Veena Selvaratnam has received consultant fees from Apellis. Jeffrey Szer reports consultancy at Novartis, Alexion, and Apellis; honoraria from Takeda, Pfizer, Novartis, Prevail Therapeutics, and Alexion; speakers\u2019 bureau at Takeda, Pfizer, Novartis, and Alexion; and membership on an entity\u2019s board of directors or advisory committees at Prevail Therapeutics and Alexion. Regina Horneff is an employee of Swedish Orphan Biovitrum AB. Lisa Tan is a consultant for Swedish Orphan Biovitrum AB. Jens Panse reports consultancy and honoraria at Blueprint Medicines, Amgen, F. Hoffmann-La Roche, MSD, Bristol Myers Squibb, Alexion, AstraZeneca, Novartis, Pfizer, Gilead, Boehringer Ingelheim, Swedish Orphan Biovitrum AB, and Apellis; and honoraria from Swiss Biopharma.","order":2,"name":"Ethics","group":{"name":"EthicsHeading","label":"Conflict of Interest"}},{"value":"Parent study and 307-OLE protocols were designed and monitored in accordance with the ethical principles of Good Clinical Practice and the Declaration of Helsinki [\u2013]. These protocols were approved by an institutional review board or independent ethics committee at each center. Each patient provided written informed consent before undergoing study-related procedures.","order":3,"name":"Ethics","group":{"name":"EthicsHeading","label":"Ethical Approval"}}]}}