EMA: Reflection paper on immune tolerance induction in haemophilia-A patients with inhibitors

In haemophilia-A patients, replacement therapy with factor-VIII (FVIII) products has become state of the art. However, a serious complication in the treatment of haemophilia A is the development of neutralising antibodies against FVIII, causing therapy resistance and increased risk of bleeding. This reflection paper dicsusses several factors (e.g. genetic and environmental) as possible contributors to inhibitor development.