Potential new treatment for haemophilia developed by Cambridge researchers

Cambridge: A new treatment that might one day help all patients with haemophilia, including those that become resistant to existing therapies, has been developed by researchers at the University of Cambridge. Around 400,000 individuals around the world are affected by haemophilia, a genetic disorder that causes uncontrolled bleeding. Haemophilia is the result of a deficiency in proteins required for normal blood clotting – factor VIII for haemophilia A and factor IX for haemophilia B. Currently, the standard treatment is administration of the missing clotting factor. However, this requires regular intravenous injections, is not fully effective, and in about a third of patients results in the development of inhibitory antibodies. Nearly three-quarters of haemophilia sufferers have no access to treatment and have a life-expectancy of only 10 years.

Simoctocog alfa for haemophilia A: no suitable data

IQWIG. Germany: Simoctocog alfa (trade name Nuwiq) has been approved since July 2014 for people with type A haemophilia, an inherited disorder that impairs blood clotting. The German Institute for Quality and Efficiency in Health Care (IQWiG) examined in a dossier assessment whether this new drug offers an added benefit over the appropriate comparator therapy. Such an added benefit cannot be derived from the dossier, however, because the drug manufacturer did not submit any suitable data.

Preventing bleeding in people with congenital bleeding disorders during and after surgery

Cochrane: In haemophilia and other congenital bleeding disorders blood does not clot properly, which can cause excessive bleeding. This is particularly relevant during surgery, when the risk of bleeding depends on the type and severity of the clotting disorder and on the type of surgery. Therefore, during and after surgery, these individuals should receive treatment to improve the ability of their blood to clot and so prevent bleeding. Clotting factor concentrates (when available and appropriate in those individuals missing specific clotting proteins) or other non-specific drugs for clotting, or a combination of both, are administered. It is not known what is the optimal dose or duration or method of administration of these treatments in these circumstances.