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Safety and efficacy of KOGENATE ® Bayer in previously untreated patients (PUPs) and minimally treated patients (MTPs)
Author(s) -
Giangrande P. L. F.
Publication year - 2002
Publication title -
haemophilia
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.213
H-Index - 92
eISSN - 1365-2516
pISSN - 1351-8216
DOI - 10.1046/j.1351-8216.2001.00133.x
Subject(s) - medicine , pediatrics
Summary. Safety and efficacy of KOGENATE ® Bayer (Kogenate ® FS), a second‐generation full‐length recombinant factor VIII formulated with sucrose as stabilizer and produced without the addition of human albumin during purification and the final formulation, was assessed in a prospective, international clinical trial including 31 previously untreated patients (PUPs) and minimally treated patients (MTPs) with severe haemophilia A in home therapy and surgery. Factor VIII inhibitor development was monitored and mutation type profiles were analysed. As of 30 June 2000, the patients received a total of 2.729 infusions (mean 88; range 6–274) for bleeding episodes, surgery or prophylactic treatment. No unexpected drug‐related adverse events were observed. Four patients developed an inhibitor after 3–12 exposure days (EDs). One patient successfully underwent immune tolerance treatment; inhibitors in two patients disappeared spontaneously with on‐demand treatment, while the inhibitor titre remains low in one patient. Twenty‐nine patients (93%) with more than 20 EDs can be regarded as at low risk for inhibitor development. In conclusion, KOGENATE ® Bayer is efficacious and well‐tolerated for treatment of children with severe haemophilia A. The incidence of inhibitor formation is not different to that observed with other recombinant or plasma‐derived products.