Immune tolerance induction: a role for recombinant activated factor VII (rFVIIa)?

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Abstract
The development of factor VIII (FVIII) and FIX inhibitors is one of the most serious complications of repeated transfusions in patients with haemophilia. Management of patients with haemophilia with inhibitors must be separated into 2 concepts: the control of acute bleeding episodes and, in the long term, the treatment of the inhibitor itself. This paper will discuss both aspects of the management of these patients, focusing in particular on the therapeutic options available for the treatment of bleeding episodes during immune tolerance induction (ITI) therapy. The second part of the paper will review the management and outcomes of 10 patients with severe haemophilia in whom recombinant activated FVII (rFVIIa; NovoSeven®, Novo Nordisk, Bagsværd, Denmark) has been used to treat bleeding episodes in the Haemophilia Centre at Bonn University; 6 patients were treated with rFVIIa for bleeding episodes while undergoing ITI. The results obtained demonstrate that rFVIIa is a safe and effective treatment for bleeding episodes and haemostatic cover for surgical procedures in patients with inhibitors. Inhibitor titres were not boosted in patients with haemophilia and inhibitors on treatment with rFVIIa. Therefore, rFVIIa is also a suitable treatment for bleeding episodes or control of haemostasis during surgery in patients prior to initiation of ITI therapy.