24 July 2017


A clinical trial has found hopeful results for the treatment of haemophilia A with a novel therapy called emicizumab.

A team led by Dr Guy Young of Children's Hospital Los Angeles, USA, recruited 109 boys aged 12 years and older, diagnosed with haemophilia A with inhibitors against factor VIII or IX.

They were given treatment with emicizumab, a novel monoclonal antibody given by injections once a week, or standard treatment. Those on emicizumab experienced 87% fewer bleeding episodes. None of the patients developed antibodies against the drug.

Full results of this multi-centre phase III study were presented at the International Society of Thrombosis and Haemostasis, held in Berlin, Germany, from July 8-13, and published in the New England Journal of Medicine on July 10.

Dr Young and colleagues say that people with haemophilia A experience excessive bleeding and need prophylactic treatment with intravenously administered clotting factor VIII or IX usually three times a week.

But over time this can lead to the development of antibodies called inhibitors, which attack and destroy the necessary factor and limit treatment options.

Co-author, Dr Alan Wayne, said: 'This is a breakthrough for individuals with haemophilia A who no longer respond to conventional clotting therapies.

'The standard treatment for patients with haemophilia A and inhibitors has been to use medications called 'by-passing agents'. This new therapy is dramatically more effective at preventing bleeding.

'Additionally, in comparison to by-passing agents, emicizumab is easier to administer, requires less frequent dosing, and based on this study, appears to have an improved safety profile.'

Dr Young added: 'This is the most significant advancement I have seen during my 20 years working in the field of haemophilia.'


Source: Oldenburg, J. et al. Emicizumab Prophylaxis in Hemophilia A with Inhibitors. New England Journal of Medicine July 10 2017; doi: 10.1056/NEJMoa1703068

Link: http://www.nejm.org/doi/full/10.1056/NEJMoa1703068#t=article


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