ISSN:
1365-2516
Quelle:
Blackwell Publishing Journal Backfiles 1879-2005
Thema:
Medizin
Notizen:
Treatment of previously untreated patients (PUPs) and minimally treated patients (MTPs) with severe hemophilia A using FVIII concentrates is complicated by FVIII inhibitor formation in ∼30% of patients. The incidence of FVIII inhibitors was determined in a prospective clinical trial of sucrose-formulated full-length recombinant FVIII (rFVIII-FS, KOGENATE® Bayer; Kogenate® FS) in pediatric patients.Methods: PUPs and MTPs (=4 exposure days-EDs) with hemophilia A (〈2% FVIII) were enrolled from 19 EU and 13 US centers. Sixty patients were evaluable for inhibitor formation (EU, 31; US, 29). Patients were tested regularly with the Nijmegen-modified Bethesda assay (negative, ≤0.6 BU; Low Titer, 〉0.6-5 BU; High Titer, 〉5 BU).Results: In the EU cohort (31 Caucasian), 4 patients developed inhibitors (3 Low; 1 High). Five high titer inhibitors developed in the US cohort (17 Caucasian; 5 Black; 7 Other). Median EDs at inhibitor detection was 8 [range, 3-16], and at study end 1 EU and 4 US patients had 〈20 EDs. The incidence of inhibitors in patients achieving 20 ED was 16.4% (9/55).Conclusions: The rate of inhibitor formation in pediatric patients with severe hemophilia A treated with rFVIII-FS is consistent with that observed with plasma-derived and other recombinant FVIII products. Major gene disruptions were observed in all inhibitor patients.
Materialart:
Digitale Medien
URL:
http://dx.doi.org/10.1046/j.1365-2516.2002.d01-2_4.x
