Introduction of the treatment with recombinant FVIII has not increased the inhibitor incidence rate in Czech PUPs with haemophilia A
Abstract
Both relative and absolute incidence of inhibitors against FVIII in previously untreated patients (PUPs) receiving only rFVIII in the Czech Republic between 2003 and 2012 were evaluated based on the Czech National Haemophilia Programme (CNHP) registry data. These findings were correlated with the treatment regimens used and relevant relationships were investigated.
Incidence of inhibitors against FVIII in PUPS treated with rFVIII and pdFVIII was compared as well. From 2003-2012, 41 PUPs were treated for 118 treatment years with rFVIII in the Czech Republic.
None of the children with mild/moderate haemophilia A developed inhibitors. Relative incidence of inhibitors in PUPS was 5.2 per 100 treatment years (5,2%) in rFVIII treated and 4,7 per 100 treatment years (4,7%) in pdFVIII treated PUPs.
All of these PUPS were children with severe haemophilia A. Relative incidence of inhibitors in PUPs thus did not differ significantly between the groups treated with rFVIII and with pdFVIII.
Absolute incidence of inhibitors was 20% for rFVIIa and 26% for pdFVIII. This difference was not statistically significant either.
We thus conclude that the incidence of inhibitors in Czech PUPs is low and introduction of rFVIII in 2003 has not led an increase in inhibitor development risk in our country to date.