{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T12:43:35Z","timestamp":1790772215970,"version":"4.1.0"},"reference-count":40,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2023,8,16]],"date-time":"2023-08-16T00:00:00Z","timestamp":1692144000000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"funder":[{"DOI":"10.13039\/501100016300","name":"Octapharma","doi-asserted-by":"publisher","id":[{"id":"10.13039\/501100016300","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":[[2023,11]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Objectives<\/jats:title><jats:p>We aimed to indirectly compare the efficacy of personalized prophylaxis with simoctocog alfa (Nuwiq\u00ae) versus three extended half\u2010life (EHL) recombinant FVIII (rFVIII) concentrates.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Treatment effects were compared using matching\u2010adjusted indirect comparisons after matching individual patient\u2010level baseline characteristics for simoctocog alfa (pharmacokinetic [PK]\u2010guided personalized prophylaxis) against published aggregate personalized prophylaxis data for efmoroctocog alfa, damoctocog alfa pegol, and rurioctocog alfa pegol.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A higher percentage (<jats:italic>p<\/jats:italic>\u2009&lt;\u2009.001) of patients with zero bleeds was found with simoctocog alfa compared with efmoroctocog alfa (75% vs. 45%), damoctocog alfa pegol (77% vs. 38%), and rurioctocog alfa pegol (target trough level 1%\u20133%; 78% vs. 42%). Similar efficacy was found comparing simoctocog alfa against rurioctocog alfa pegol 8%\u201312% (77% vs. 62%). The mean total annualized bleeding rate was lower (<jats:italic>p<\/jats:italic>\u2009&lt;\u2009.001) with simoctocog alfa than damoctocog alfa pegol (1.5 vs. 4.9). Consistent with approved dosing, the mean FVIII weekly dose was higher (<jats:italic>p<\/jats:italic>\u2009&lt;\u2009.001) for simoctocog alfa than efmoroctocog alfa, damoctocog alfa pegol, or rurioctocog alfa pegol 1%\u20133%, but lower (<jats:italic>p<\/jats:italic>\u2009&lt;\u2009.001) than rurioctocog alfa pegol 8%\u201312%.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusions<\/jats:title><jats:p>Indirect comparisons demonstrated that PK\u2010guided, personalized prophylaxis with simoctocog alfa can lead to higher zero bleed rates compared with personalized EHL rFVIII concentrate regimens, albeit with higher weekly doses, and a lower percentage of patients treated twice weekly or less.<\/jats:p><\/jats:sec>","DOI":"10.1111\/ejh.14073","type":"journal-article","created":{"date-parts":[[2023,8,20]],"date-time":"2023-08-20T21:39:27Z","timestamp":1692567567000},"page":"757-767","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":4,"title":["Clinical efficacy of simoctocog alfa versus extended half\u2010life recombinant <scp>FVIII<\/scp> concentrates in hemophilia A patients undergoing personalized prophylaxis using a matching\u2010adjusted indirect comparison method"],"prefix":"10.1111","volume":"111","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0599-5724","authenticated-orcid":false,"given":"Craig M.","family":"Kessler","sequence":"first","affiliation":[{"name":"Lombardi Comprehensive Cancer Center Georgetown University Medical Center  Washington DC USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6185-4809","authenticated-orcid":false,"given":"Fernando F.","family":"Corrales\u2010Medina","sequence":"additional","affiliation":[{"name":"Division of Pediatric Hematology\u2013Oncology, Department of Pediatrics Miller School of Medicine, University of Miami  Miami Florida USA"},{"name":"Hemophilia Treatment Center University of Miami  Miami Florida USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-1915-3897","authenticated-orcid":false,"given":"Pier Mannuccio","family":"Mannucci","sequence":"additional","affiliation":[{"name":"Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico Angelo Bianchi Bonomi Hemophilia and Thrombosis Center  Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3937-3499","authenticated-orcid":false,"given":"V\u00edctor","family":"Jim\u00e9nez\u2010Yuste","sequence":"additional","affiliation":[{"name":"Hospital Universitario La Paz\u2014Hematology Department Aut\u00f3noma University  Madrid Spain"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-0069-8176","authenticated-orcid":false,"given":"Michael D.","family":"Tarantino","sequence":"additional","affiliation":[{"name":"Bleeding and Clotting Disorders Institute  Peoria Illinois USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2023,8,16]]},"reference":[{"key":"e_1_2_11_2_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14046"},{"key":"e_1_2_11_3_1","doi-asserted-by":"publisher","DOI":"10.2450\/2019.0211\u201019"},{"key":"e_1_2_11_4_1","doi-asserted-by":"publisher","DOI":"10.2450\/2012.0166\u201011"},{"key":"e_1_2_11_5_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12438"},{"key":"e_1_2_11_6_1","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365\u20102516.2010.02370.x"},{"key":"e_1_2_11_7_1","doi-asserted-by":"publisher","DOI":"10.1177\/2040620719858471"},{"key":"e_1_2_11_8_1","unstructured":"Nuwiq\u00ae.Prescribing information.2021."},{"key":"e_1_2_11_9_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12793"},{"key":"e_1_2_11_10_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12797"},{"key":"e_1_2_11_11_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12859"},{"key":"e_1_2_11_12_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.12322"},{"key":"e_1_2_11_13_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13251"},{"key":"e_1_2_11_14_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14313"},{"key":"e_1_2_11_15_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.13438"},{"key":"e_1_2_11_16_1","doi-asserted-by":"publisher","DOI":"10.1136\/bmj.d4849"},{"key":"e_1_2_11_17_1","doi-asserted-by":"publisher","DOI":"10.1111\/hae.14511"},{"key":"e_1_2_11_18_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.critrevonc.2022.103678"},{"key":"e_1_2_11_19_1","doi-asserted-by":"publisher","DOI":"10.1016\/j.jval.2012.05.004"},{"key":"e_1_2_11_20_1","unstructured":"PhillippoD AdesT DiasS PalmerS AbramsKR WeltonN.NICE DSU Technical Support Document 18: methods for population\u2010adjusted indirect comparisons in submissions to NICE (technical support documents). 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