title: Updates from guardian™: a comprehensive registration programme.
abstract: Turoctocog alfa is an approved B-domain truncated recombinant factor VIII concentrate for adults and children with haemophilia A. Clinical data for turoctocog alfa have been reported from the guardian(™) 1, guardian(™) 2 and guardian(™) 3 phase III trials. guardian(™) 1 and guardian(™) 3 phase III trials enrolled 150 adolescents/adults (≥ 12 yr), and 63 children (<12 yr), respectively, with previously treated severe haemophilia A and no history of inhibitors; 188 of these patients continued into the ongoing guardian(™) 2 extension trial. In the three trials, patients have received prophylaxis with turoctocog alfa three times weekly or every second day, with breakthrough bleeds resolved to an expected postinjection level of ≥ 0.50 IU/mL. No safety concerns have arisen, and no patients have developed confirmed inhibitors (primary endpoint). Indeed, no confirmed inhibitors have been detected in >200 patients treated for a cumulative total of >54,000 exposure days in the phase III trials. Pooled efficacy data show a favourable long-term effect of turoctocog alfa on annualised bleeding rate and a success rate for haemostatic response of 86%; 90% of bleeds were resolved with 1-2 doses. This article reviews the clinical development of turoctocog alfa with reference to the guardian(™) clinical programme, describing results obtained to date and ongoing trials.
authors.affiliation: Unidade de Hemofilia IHTC 'Claudio L. P. Correa', Instituto Nacional de Ciência e Tecnologia do Sangue, Hemocentro UNICAMP, University of Campinas, Campinas, Brazil.
authors.forename: Margareth C
authors.initials: MC
authors.lastname: Ozelo
mesh_terms: D000293:Adolescent; D000328:Adult; D002648:Child; D017326:Clinical Trials, Phase III as Topic; D005169:Factor VIII / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D055815:Young Adult
chemical_list: C577506:recombinant factor VIII N8; D005169:Factor VIII
keywords: clinical trial; efficacy; haemophilia A; recombinant factor VIII; safety; turoctocog alfa