New Study Shows Increased Development of Inhibitors using Recombinant Factor VIII

A recent trial shows that previously untreated patients with severe hemophilia A are nearly twice as likely to develop inhibitors when using recombinant factor VIII (rFVIII) treatment compared to using a single plasma derived factor VIII (pdFVIII) treatment.

The study found that patients treated with rFVIII have an 87% higher risk of developing inhibitors compared to patients treated with pdFVIII. This difference in developing inhibitors remained even when second generation full length rFVIII concentrate was excluded from the analyses. Of the 126 patients treated with rFVIII, 47 (44.5%) developed inhibitors with 30 of those being high-titre. Of the 125 patients treated with pdFVIII, 29 (26.8%) developed inhibitors and 20 of those were high-titre.

The Survey of Inhibitors in Plasma Product-Exposed Toddlers (SIPPET) randomized trial analyzed 251 previously untreated patients over the course of three years or until inhibitor development and took into account a number of demographic and treatment-related factors including age, ethnicity, family history, treatment regimen, and mutation.

Because of the potential impact of this new study one of the authors, Flora Peyvandi, MD, PhD of the Angelo Bianchi Hemophilia and Thrombosis Centre at the University of Milan, Italy, has been invited to present at the 2016 International Plasma Protein Congress, which will be held March 22-23 in Barcelona.

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