Immune tolerance induction in haemophilia A patients with inhibitors - Scientific guideline

This document discusses the treatment of patients with haempophilia A that develop inhibitory antibodies against factor VIII. It addresses the clinical and regulatory aspects of inhibitor patients therapy.

Keywords: Immune tolerance induction, inhibitors, haemophilia A

Current effective version

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

Adopted Reference Number: EMA/CHMP/BPWP/153137/2011 Summary:

In haemophilia A patients, replacement therapy with factor VIII products has become state of the art. However, a serious complication in the treatment of haemophilia A is the development of neutralizing antibodies against FVIII, causing therapy resistance and increased risk of bleeding.

Document history

Overview of comments received on reflection paper on immune-tolerance induction in haemophilia-A patients with inhibitors

Reference Number: EMA/CHMP/BPWP/734487/2012

Draft reflection paper on immune tolerance induction in haemophilia-A patients with inhibitors

Draft: consultation closed Consultation dates: 30/07/2012 to 31/10/2012 Reference Number: EMA/CHMP/BPWP/153137/2011 Summary:

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.