{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T22:04:28Z","timestamp":1790805868296,"version":"4.1.0"},"reference-count":17,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T00:00:00Z","timestamp":1772841600000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by\/4.0\/"},{"start":{"date-parts":[[2026,3,7]],"date-time":"2026-03-07T00:00:00Z","timestamp":1772841600000},"content-version":"tdm","delay-in-days":0,"URL":"http:\/\/doi.wiley.com\/10.1002\/tdm_license_1.1"}],"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":["American J Hematol"],"published-print":{"date-parts":[[2026,5]]},"abstract":"<jats:title>ABSTRACT<\/jats:title>\n                  <jats:p>Ravulizumab, a second\u2010generation complement\u00a0component 5 inhibitor (C5i) derived from eculizumab, with improved pharmacokinetics, is the current standard\u2010of\u2010care treatment for patients with paroxysmal nocturnal hemoglobinuria (PNH), where available. Pivotal trials have demonstrated durable long\u2010term efficacy, safety, and improved survival, and increasing real\u2010world evidence is required. This analysis utilized data from the International PNH Registry (NCT01374360) to evaluate the real\u2010world effectiveness and safety of ravulizumab, and to assess baseline characteristics at treatment initiation. Adults (aged 18\u201365\u2009years) enrolled in the registry as of July 1, 2024, who were treated with ravulizumab for \u2265\u20096\u2009months were included and stratified by prior eculizumab use (eculizumab\u2010experienced and C5i\u2010naive). Ravulizumab treatment outcomes were assessed for up to 24\u2009months and included change in lactate dehydrogenase (LDH) ratio from baseline, transfusion avoidance, and adverse event rates. Data for 203 eculizumab\u2010experienced and 23 C5i\u2010naive patients were analyzed. During treatment, LDH ratio was maintained near normal (&lt;\u20091.5\u2009\u00d7\u2009the upper limit of normal in both groups); 86.8% of eculizumab\u2010experienced and 76.5% of C5i\u2010naive patients were transfusion\u2010independent, and a low rate of major adverse vascular events was reported (eculizumab\u2010experienced, 0.4 per 100 person\u2010years; C5i\u2010naive, no events). These findings reinforce the pivotal trial outcomes and demonstrate the effectiveness and safety of ravulizumab in the real\u2010world setting, further supporting ravulizumab as the first\u2010line treatment of choice for patients with PNH, where available.<\/jats:p>\n                  <jats:p>\n                    <jats:bold>Trial Registration:<\/jats:bold>\n                    International PNH Registry: NCT01374360\n                  <\/jats:p>","DOI":"10.1002\/ajh.70268","type":"journal-article","created":{"date-parts":[[2026,3,8]],"date-time":"2026-03-08T08:40:58Z","timestamp":1772959258000},"page":"939-948","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":1,"title":["Real\u2010World Effectiveness and Safety of Ravulizumab in Patients With Paroxysmal Nocturnal Hemoglobinuria: Evidence From the International\n                    <scp>PNH<\/scp>\n                    Registry"],"prefix":"10.1002","volume":"101","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-4414-7699","authenticated-orcid":false,"given":"Alexander","family":"R\u00f6th","sequence":"first","affiliation":[{"name":"Department of Hematology and Stem Cell Transplantation, West German Cancer Center University Hospital Essen, University of Duisburg\u2010Essen  Essen Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Christopher J.","family":"Patriquin","sequence":"additional","affiliation":[{"name":"Division of Medical Oncology and Hematology University Health Network  Toronto Ontario Canada"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jeff","family":"Szer","sequence":"additional","affiliation":[{"name":"Department of Clinical Hematology Peter MacCallum Cancer Centre and the Royal Melbourne Hospital  Melbourne Victoria Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Louis","family":"Terriou","sequence":"additional","affiliation":[{"name":"Service de M\u00e9decine Interne et d'Immunologie Clinique, CHU Lille  Lille France"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ami S.","family":"Patel","sequence":"additional","affiliation":[{"name":"Alexion, AstraZeneca Rare Disease  Boston Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jesse","family":"Metzger","sequence":"additional","affiliation":[{"name":"Parexel  Newton Massachusetts USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Philippe","family":"Gustovic","sequence":"additional","affiliation":[{"name":"Alexion, AstraZeneca Rare Disease  Baar Switzerland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5026-693X","authenticated-orcid":false,"given":"Jun\u2010ichi","family":"Nishimura","sequence":"additional","affiliation":[{"name":"Department of Hematology and Oncology Osaka University Graduate School of Medicine  Osaka Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5741-1255","authenticated-orcid":false,"given":"Robert A.","family":"Brodsky","sequence":"additional","affiliation":[{"name":"Division of Hematology Johns Hopkins Medicine  Baltimore Maryland USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2026,3,7]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2014-02-522128"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.1053\/j.seminhematol.2008.11.004"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.1177\/20406207221091046"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.13783"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-09-876805"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2018-09-876136"},{"key":"e_1_2_11_8_1","first-page":"81","article-title":"Ravulizumab Demonstrates Long\u2010Term Efficacy, Safety and Favorable Patient Survival in Patients With Paroxysmal Nocturnal Hemoglobinuria","volume":"104","author":"Kulasekararaj A.","year":"2025","journal-title":"Annals of Hematology"},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.3324\/haematol.2013.093161"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1007\/s00277-020-04052-z"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/imj.13523"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.14080"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1182\/blood.2023021762"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.3346\/jkms.2016.31.2.214"},{"key":"e_1_2_11_15_1","first-page":"S1","article-title":"K\/DOQI Clinical Practice Guidelines for Chronic Kidney Disease: Evaluation, Classification, and Stratification","volume":"39","author":"National Kidney Foundation","year":"2002","journal-title":"American Journal of Kidney Diseases"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1111\/ejh.13564"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620720966137"},{"key":"e_1_2_11_18_1","article-title":"(DMT49) Meningococcal Infections in Eculizumab\u2010 or Ravulizumab\u2010Treated Patients With Neuromyelitis Optica Spectrum Disorder: Clinical and Real\u2010World Pharmacovigilance Update","volume":"26","author":"Fam S.","year":"2024","journal-title":"International Journal of MS Care"}],"container-title":["American Journal of 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