{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,10,1]],"date-time":"2026-10-01T21:26:04Z","timestamp":1790889964847,"version":"4.1.0"},"reference-count":12,"publisher":"Georg Thieme Verlag KG","issue":"11","funder":[{"DOI":"10.13039\/501100000801","name":"Bayer HealthCare","doi-asserted-by":"crossref","id":[{"id":"10.13039\/501100000801","id-type":"DOI","asserted-by":"crossref"}]}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Thromb Haemost"],"published-print":{"date-parts":[[2012]]},"abstract":"<jats:title>Summary<\/jats:title><jats:p>The benefits of prophylaxis of haemophilia A patients regarding joint health and quality-of-life are well established. However, adherence to an up to every-other-day infusion regimen is a barrier to widespread adoption of prophylaxis. BAY 79\u20134980 is an investigational drug consisting of rFVIII-FS (sucrose-formulated recombinant FVIII) reconstituted with liposome solvent. Previous clinical studies showed extended protection from bleeding after a single injection of BAY 79\u20134980 (13.3 \u00b1 6.2 days) compared with rFVIII-FS (7.2 \u00b1 1.7 days). The effect of once-a-week prophylaxis with BAY 79\u20134980 (35 IU\/kg) compared with three times-per-week rFVIII-FS (25 IU\/kg) in previously treated, severe haemophilia A patients was evaluated in a 52-week, double-blind, two-arm, randomised, controlled study. The primary and secondary endpoints were protection from total bleeds and joint bleeds, respectively. Short- and long-term safety and tolerability of BAY 79\u20134980 including effects on lipid levels were assessed. A total of 139 and 131 subjects were evaluable for safety and efficacy analyses, respectively. A large difference in efficacy between treatment groups was observed with 72.1% (49\/68) in the rFVIII-FS control group demonstrating &lt;9 bleeds\/year compared with 38.1% (24\/63) of BAY 79\u20134980-treated subjects. A similar difference was seen in annualised joint bleeds, with 43 subjects (63.2%) in the control group demonstrating &lt;5 joint bleeds\/year compared with 24 subjects (38.1%) treated with BAY 79\u20134980. The distribution of bleeds seven days post-prophylactic treatment with BAY 79\u20134980 showed that 61% of bleeds occurred after day 4 post dosing. There were no safety concerns identified. The investigational treatment arm was prematurely discontinued due to failure to achieve the primary endpoint.<\/jats:p>","DOI":"10.1160\/th12-03-0188","type":"journal-article","created":{"date-parts":[[2012,9,28]],"date-time":"2012-09-28T00:52:01Z","timestamp":1348793521000},"page":"913-922","source":"Crossref","is-referenced-by-count":23,"title":["Efficacy and safety of prophylaxis with once-weekly BAY 79\u20134980 compared with thrice-weekly rFVIII-FS in haemophilia A patients"],"prefix":"10.1160","volume":"108","author":[{"given":"Uri","family":"Martinowitz","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerzy","family":"Windyga","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Giovanni","family":"Di Minno","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Andrzej","family":"Hellmann","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ingrid","family":"Pabinger","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Monika","family":"Enriquez","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Lawrence","family":"Schwartz","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"J\u00f8rgen","family":"Ingerslev","sequence":"first","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jerry","family":"Powell","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]},{"name":"the LipLong Study Investigators","sequence":"additional","affiliation":[],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"194","published-online":{"date-parts":[[2017,11,29]]},"reference":[{"key":"10.1160\/TH12-03-0188-1","first-page":"691","volume":"73","author":"Berntorp","year":"1995","journal-title":"Bull World Health Organ"},{"key":"10.1160\/TH12-03-0188-2","doi-asserted-by":"publisher","DOI":"10.1046\/j.1365-2516.2001.00534.x"},{"key":"10.1160\/TH12-03-0188-3","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2141.2007.06703.x"},{"key":"10.1160\/TH12-03-0188-4","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02155.x"},{"key":"10.1160\/TH12-03-0188-5","doi-asserted-by":"crossref","first-page":"1061","DOI":"10.1160\/TH04-08-0485","volume":"93","author":"Baru","year":"2005","journal-title":"Thromb Haemost"},{"key":"10.1160\/TH12-03-0188-6","doi-asserted-by":"crossref","first-page":"277","DOI":"10.1111\/j.1538-7836.2007.02856.x","volume":"6","author":"Powell","year":"2008","journal-title":"J Thromb Haemost"},{"key":"10.1160\/TH12-03-0188-7","doi-asserted-by":"publisher","DOI":"10.1182\/blood-2006-03-008276"},{"key":"10.1160\/TH12-03-0188-8","doi-asserted-by":"crossref","first-page":"429","DOI":"10.1160\/TH08-03-0133","volume":"100","author":"Spira","year":"2008","journal-title":"Thromb Haemost"},{"key":"10.1160\/TH12-03-0188-9","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2008.01699.x"},{"key":"10.1160\/TH12-03-0188-10","doi-asserted-by":"crossref","first-page":"52","DOI":"10.1160\/TH07-06-0409","volume":"99","author":"Musso","year":"2008","journal-title":"Thromb Haemost"},{"key":"10.1160\/TH12-03-0188-11","doi-asserted-by":"publisher","DOI":"10.1111\/j.1365-2516.2009.02079.x"},{"key":"10.1160\/TH12-03-0188-12","doi-asserted-by":"publisher","DOI":"10.1111\/j.1538-7836.2008.03032.x"}],"container-title":["Thrombosis and Haemostasis"],"original-title":[],"language":"en","link":[{"URL":"http:\/\/www.thieme-connect.de\/products\/ejournals\/pdf\/10.1160\/TH12-03-0188.pdf","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2019,7,4]],"date-time":"2019-07-04T04:12:57Z","timestamp":1562213577000},"score":1,"resource":{"primary":{"URL":"http:\/\/www.thieme-connect.de\/DOI\/DOI?10.1160\/TH12-03-0188"}},"subtitle":["A randomised, active-controlled, double-blind study"],"short-title":[],"issued":{"date-parts":[[2012]]},"references-count":12,"journal-issue":{"issue":"11","published-online":{"date-parts":[[2017,11,29]]},"published-print":{"date-parts":[[2012]]}},"URL":"https:\/\/doi.org\/10.1160\/th12-03-0188","relation":{},"ISSN":["0340-6245","2567-689X"],"issn-type":[{"value":"0340-6245","type":"print"},{"value":"2567-689X","type":"electronic"}],"subject":[],"published":{"date-parts":[[2012]]}}}