{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,2,27]],"date-time":"2026-02-27T21:52:29Z","timestamp":1772229149550,"version":"3.50.1"},"reference-count":39,"publisher":"Wiley","issue":"5","license":[{"start":{"date-parts":[[2005,8,26]],"date-time":"2005-08-26T00:00:00Z","timestamp":1125014400000},"content-version":"vor","delay-in-days":0,"URL":"http:\/\/onlinelibrary.wiley.com\/termsAndConditions#vor"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["Haemophilia"],"published-print":{"date-parts":[[2005,9]]},"abstract":"<jats:p><jats:bold>Summary.\u2002<\/jats:bold> This clinical trial evaluated the safety and efficacy of ReFacto (St Louis\u2010derived active substance) in patients with severe or moderately severe haemophilia A over a period of 6\u2003months or 50\u2003exposure days (EDs), whichever occurred first. Sixty patients, 58 previously treated and two previously untreated, were enrolled into this study. This was an open\u2010label, multicentre, postmarketing surveillance study in which patients received prophylaxis or on\u2010demand treatment as determined by their doctor. Surgical prophylaxis was evaluated in seven patients requiring elective surgery. Thirty\u2010two patients aged &lt;1 to 66\u2003years (median 19.5) received prophylaxis and 28 patients, aged 1\u201371\u2003years (median 33.5), received on\u2010demand treatment. The majority of patients had severe haemophilia A (FVIII:C\u2003&lt;\u20032%): 84.4% in the prophylaxis group and 85.7% in the on\u2010demand group. Prophylaxis with ReFacto was associated with a median of 6.7 bleeds per year (range: 0\u201337). The investigator's assessment of final outcome for prophylactic treatment was excellent or effective for 93.1% of patients. ReFacto resolved 92.8% of bleeds with one or two infusions. The investigator's assessment was excellent or good for 98.2% of bleeds treated with ReFacto. Haemostasis was achieved for all seven surgical cases and ReFacto gave an excellent or good response for each. The nature and incidence of adverse events was as expected and no new safety concerns emerged. One previously treated patient (PTP) developed a high\u2010titre inhibitor (maximum 75\u2003BU) and one minimally treated patient (MTP) developed a low\u2010titre inhibitor while on the study but eventually achieved high titres (maximum 30\u2003BU) after immune tolerance therapy was initiated with a plasma\u2010derived FVIII product. One previously untreated patient (PUP) developed a transient low\u2010titre inhibitor (0.4\u2003BU). Other serious adverse events (SAEs) were unrelated to study treatment. There were no allergic events. The results of this study are consistent with the previously published ReFacto pivotal studies.<\/jats:p>","DOI":"10.1111\/j.1365-2516.2005.01131.x","type":"journal-article","created":{"date-parts":[[2005,8,26]],"date-time":"2005-08-26T09:32:38Z","timestamp":1125048758000},"page":"444-451","source":"Crossref","is-referenced-by-count":27,"title":["A postmarketing surveillance study of the safety and efficacy of ReFacto<sup>\u00ae<\/sup> (St Louis\u2010derived active substance) in patients with haemophilia A"],"prefix":"10.1111","volume":"11","author":[{"given":"M. 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