title: European data of a clinical trial with a sucrose formulated recombinant factor VIII in previously treated haemophilia A patients.
abstract: To increase the safety of antihaemophilic treatment, the production process of full-length recombinant factor VIII (FVIII) KOGENATE Bayer (Kogenate FS) has been modified. Human albumin is no longer added as stabilizer during purification and in final formulation. Instead, the new KOGENATE Bayer production process uses sucrose as a stabilizer in the formulation and adds solvent/detergent virus inactivation step. An European clinical trial was carried out in Germany and France in previously treated patients with severe haemophilia A who had more than 100 exposure days to exogenous FVIII. Pharmacokinetic data was analysed according to one-stage and chromogenic assays. Efficacy and safety during home therapy and in surgical procedures were evaluated; inhibitor formation was carefully monitored. Safety and efficacy were evaluated in 33 European patients for 24 months. Patients received more than 13 million IU KOGENATE Bayer. Over 75% of patients accrued more than 100 exposure days with the new product. Of 875 bleeding episodes, 90.7% were treated with 1 or 2 infusions and 75.8% of responses to treatment were rated as 'excellent' or 'good'. Prophylactic treatment was the most common mode of therapy (60.7% of infusions). The product was well-tolerated and FVIII recovery studies were consistent throughout the study period. Only 0.26% of adverse events were reported to be drug related. No evidence of de novo inhibitor formation was observed. Overall, KOGENATE Bayer was efficacious, safe and well-tolerated for the treatment of haemophilia A in multitransfused patients.
authors.affiliation: Hospital Necker Enfants Malades, Paris, France. c-rot@psinet.fr
authors.forename: C
authors.initials: C
authors.lastname: Rothschild
authors.forename: I
authors.initials: I
authors.lastname: Scharrer
authors.forename: H-H
authors.initials: HH
authors.lastname: Brackmann
authors.forename: N
authors.initials: N
authors.lastname: Stieltjes
authors.forename: M
authors.initials: M
authors.lastname: Vicariot
authors.forename: M-F
authors.initials: MF
authors.lastname: Torchet
authors.forename: W
authors.initials: W
authors.lastname: Effenberger
mesh_terms: D000293:Adolescent; D000328:Adult; D016913:Blood Component Transfusion / Q000706:statistics & numerical data; D002648:Child; D003257:Consumer Product Safety; D005169:Factor VIII / Q000493:pharmacokinetics / Q000627:therapeutic use*; D006467:Hemophilia A / Q000188:drug therapy*; D006801:Humans; D008875:Middle Aged; D013395:Sucrose; D016896:Treatment Outcome
chemical_list: D013395:Sucrose; C078147:F8 protein, human; D005169:Factor VIII