European data of a clinical trial with a sucrose formulated recombinant factor VIII in previously treated haemophilia A patients |
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Authors: | C. Rothschild,I. Scharrer,H.-H. Brackmann,N. Stieltjes,M. Vicariot,¶ ,M.-F. Torchet,W. Effenberger |
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Affiliation: | Hospital Necker Enfants Malades, Paris, France,;University Hospital, Frankfurt, Germany,;University of Bonn, Germany,;Hospital Cochin, Paris, France,;CHU Morvan, Brest, France |
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Abstract: | Summary. 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. |
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Keywords: | Haemophilia A recombinant FVIII full-length FVIII sucrose formulation |
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