Successful immune tolerance treatment with monoclonal or recombinant factor VIII concentrates in high responding inhibitor patients.
Very high purity factor VIII (FVIII) concentrates (plasma-derived or produced by recombinant-DNA technology) were used to achieve immune tolerance in five patients with high-responding inhibitors to FVIII. The mean time required for inhibitor disappearance was 5 months. Four out of five patients showed normalisation of the half-life of infused FVIII after 8-18 months of treatment with 100 IU FVIII/kg body weight administered once daily. Highly purified FVIII products thus appear to be suitable for achieving immune tolerance without negative effects on endogenous von Willebrand factor levels and activity.
Rocino
A
A
Ospedale Nuovo Pellegrini, Divisione de Ematologiaosi, Centro Emofilia e Trombosi, Napoli, Italia.
de Biasi
R
R
D000293:Adolescent; D000911:Antibodies, Monoclonal / Q000627:therapeutic use; D000941:Antigens / Q000097:blood; D002675:Child, Preschool; D003001:Cloning, Molecular; D005169:Factor VIII / Q000008:administration & dosage* / Q000276:immunology; D006467:Hemophilia A / Q000097:blood / Q000188:drug therapy / Q000276:immunology; D006801:Humans; D007108:Immune Tolerance / Q000187:drug effects*; D011994:Recombinant Proteins / Q000008:administration & dosage / Q000276:immunology; D013997:Time Factors; D014841:von Willebrand Factor / Q000032:analysis / Q000187:drug effects / Q000276:immunology
D000911:Antibodies, Monoclonal; D000941:Antigens; D011994:Recombinant Proteins; D014841:von Willebrand Factor; C078147:F8 protein, human; D005169:Factor VIII