{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,9,30]],"date-time":"2026-09-30T00:54:20Z","timestamp":1790729660179,"version":"4.1.0"},"reference-count":29,"publisher":"Wiley","issue":"3","license":[{"start":{"date-parts":[[2020,3,30]],"date-time":"2020-03-30T00:00:00Z","timestamp":1585526400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc-nd\/4.0\/"}],"funder":[{"DOI":"10.13039\/100004339","name":"Sanofi","doi-asserted-by":"publisher","id":[{"id":"10.13039\/100004339","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":["onlinelibrary.wiley.com"],"crossmark-restriction":true},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2020,5]]},"abstract":"<jats:title>Abstract<\/jats:title><jats:sec><jats:title>Introduction<\/jats:title><jats:p>The efficacy and safety of recombinant factor VIII Fc fusion protein (rFVIIIFc) as an extended half\u2010life treatment for severe haemophilia A were demonstrated in the Phase 3 A\u2010LONG and Kids A\u2010LONG studies. Eligible subjects who completed A\u2010LONG and Kids A\u2010LONG could enrol in ASPIRE (NCT01454739), an open\u2010label extension study.<\/jats:p><\/jats:sec><jats:sec><jats:title>Aim<\/jats:title><jats:p>To report the long\u2010term safety and efficacy of rFVIIIFc in subjects with severe haemophilia A who enrolled in ASPIRE.<\/jats:p><\/jats:sec><jats:sec><jats:title>Methods<\/jats:title><jats:p>Previously treated subjects received one or more of the following regimens: individualized prophylaxis (IP), weekly prophylaxis, modified prophylaxis or episodic treatment. Subjects could switch treatment regimen at any time. The primary endpoint was inhibitor development.<\/jats:p><\/jats:sec><jats:sec><jats:title>Results<\/jats:title><jats:p>A total of 150 subjects from A\u2010LONG and 61 subjects from Kids A\u2010LONG enrolled in ASPIRE. Most subjects received the IP regimen (A\u2010LONG: n\u00a0=\u00a0110; Kids A\u2010LONG: n\u00a0=\u00a059). Median (range) treatment duration in ASPIRE for subjects from A\u2010LONG and Kids A\u2010LONG was 3.9 (0.1\u20105.3) years and 3.2 (0.3\u20103.9) years, respectively. No inhibitors were observed (0 per 1000 subject\u2010years; 95% confidence interval, 0\u20105.2) and the overall rFVIIIFc safety profile was consistent with prior studies. For subjects on the IP regimen, annualized bleed rates (ABR) remained low (median overall ABR for adults and adolescents was &lt;1.0) and extended\u2010dosing intervals were maintained (median of 3.5\u00a0days) for the majority of subjects in ASPIRE.<\/jats:p><\/jats:sec><jats:sec><jats:title>Conclusion<\/jats:title><jats:p>ASPIRE results, which include up to 5\u00a0years of follow\u2010up data, confirm earlier reports on the consistent and well\u2010characterized safety and efficacy of rFVIIIFc treatment for severe haemophilia A.<\/jats:p><\/jats:sec>","DOI":"10.1111\/hae.13953","type":"journal-article","created":{"date-parts":[[2020,3,30]],"date-time":"2020-03-30T09:37:08Z","timestamp":1585561028000},"page":"494-502","update-policy":"https:\/\/doi.org\/10.1002\/crossmark_policy","source":"Crossref","is-referenced-by-count":66,"title":["Recombinant factor VIII Fc fusion protein for the treatment of severe haemophilia A: Final results from the ASPIRE extension study"],"prefix":"10.1111","volume":"26","author":[{"ORCID":"https:\/\/orcid.org\/0000-0003-0145-4736","authenticated-orcid":false,"given":"Beatrice","family":"Nolan","sequence":"first","affiliation":[{"name":"Children's Health Ireland at Crumlin Dublin Ireland"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-5781-7669","authenticated-orcid":false,"given":"Johnny","family":"Mahlangu","sequence":"additional","affiliation":[{"name":"Haemophilia Comprehensive Care Centre Faculty of Health Sciences Charlotte Maxeke Johannesburg Academic Hospital and NHLS University of Witwatersrand Johannesburg South Africa"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ingrid","family":"Pabinger","sequence":"additional","affiliation":[{"name":"Medizinische Universit\u00e4t Wien Vienna Austria"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-6013-1254","authenticated-orcid":false,"given":"Guy","family":"Young","sequence":"additional","affiliation":[{"name":"Children\u2019s Hospital Los Angeles Los Angeles CA USA"},{"name":"University of Southern California Keck School of Medicine Los Angeles CA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-3959-8797","authenticated-orcid":false,"given":"Barbara A.","family":"Konkle","sequence":"additional","affiliation":[{"name":"Bloodworks Northwest Seattle WA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Chris","family":"Barnes","sequence":"additional","affiliation":[{"name":"The Royal Children\u2019s Hospital Parkville Vic. Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-2415-2194","authenticated-orcid":false,"given":"Keiji","family":"Nogami","sequence":"additional","affiliation":[{"name":"Nara Medical University Kashiwara Japan"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Elena","family":"Santagostino","sequence":"additional","affiliation":[{"name":"Angelo Bianchi Bonomi Hemophilia and Thrombosis Centre Fondazione IRCCS Ca\u2019 Granda, Ospedale Maggiore Policlinico Milan Italy"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3394-2099","authenticated-orcid":false,"given":"K. John","family":"Pasi","sequence":"additional","affiliation":[{"name":"Royal London Haemophilia Centre Barts and The London School of Medicine and Dentistry London UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Liane","family":"Khoo","sequence":"additional","affiliation":[{"name":"Royal Prince Alfred Hospital Camperdown NSW Australia"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Bent","family":"Winding","sequence":"additional","affiliation":[{"name":"Sobi Stockholm Sweden"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Huixing","family":"Yuan","sequence":"additional","affiliation":[{"name":"Sanofi Cambridge MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Joachim","family":"Fruebis","sequence":"additional","affiliation":[{"name":"Bioverativ, a Sanofi company Waltham MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Dan","family":"Rudin","sequence":"additional","affiliation":[{"name":"Bioverativ, a Sanofi company Waltham MA USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0003-3038-2145","authenticated-orcid":false,"given":"Johannes","family":"Oldenburg","sequence":"additional","affiliation":[{"name":"Institute of Experimental Haematology and Transfusion Medicine University Clinic Bonn Bonn Germany"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"311","published-online":{"date-parts":[[2020,3,30]]},"reference":[{"key":"e_1_2_9_2_1","unstructured":"National Hemophilia Foundation.MASAC document 241: MASAC recommendations concerning Prophylaxis.https:\/\/www.hemophilia.org\/Researchers\u2010Healthcare\u2010Providers\/Medical\u2010and\u2010Scientific\u2010Advisory\u2010Council\u2010MASAC\/MASAC\u2010Recommendations\/MASAC\u2010Recommendation\u2010Concerning\u2010Prophylaxis. 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