Long-term efficacy and safety of a pasteurized, plasma-derived factor VIII concentrate (Beriate~R P) in patients with haemophilia A

摘要

Introduction: Beriate~R P was first introduced in Germany in 1990 as factor VIII (FVIII):C~R HS Behring and subsequent product improvements yielded an albumin-free formulation with a specific activity of approximately 170 ILJ/mg protein. In 1992, the concentration was raised to 100IU FVIII/mL in the reconstituted product, with a mean specific activity of 270 IU/mg protein. Pathogen safety is achieved by careful donor selection and a combination of pasteurization and chromatographic purification steps.Materials and methods: We analysed the efficacy and safety of Beriate~R P in the clinical setting from 1996 to 2005 with a focus on surgical patients. Of the 36 patients (mean age: 38 years; range 1-72 years), 29 had severe haemophilia A, two had moderate haemophilia, two had mild haemophilia, and three had sub-clinical haemophilia. Most patients (n = 28) had more than 100 exposure days, representing a total of 202 patient-years with a consumption of 27,811,500 IU of Beriate~R P.Results: There was no evidence of seroconversion towards relevant viruses, no inhibitor development (35 previously treated patients, one previously untreated patient), no abnormal immunological findings or allergic reactions. In all 36 patients treated for acute bleeding and prophylaxis, and 24 surgeries (15 total joint replacements, eight orthopaedic procedures, one cholecystectomy) in 16 patients with severe haemophilia A, efficacy of Beriate~R P was always rated as "excellent" or "good", and no thrombosis was reported. Conclusion: Beriate~R P has an excellent efficacy and safety profile. Many patients who were initiated on Beriate~R P at our centre remain on the treatment today.