New treatment for people with haemophilia

Will offer more protection
  • Deborah Condon

A innovative new treatment for people with haemophilia is to be provided in Ireland, the Irish Haemophilia Society has said.

The news was confirmed at the society's annual conference at the weekend. Agreement has been reached for the provision of the treatment, which will provide greater protection for those with the condition.

Haemophilia is a group of inherited blood disorders that affect the blood's ability to form clots. Blood contains many proteins called clotting factors, and these work to stop bleeding. An absence or lack of clotting factor causes people with haemophilia to bleed for longer periods of time than those whose clotting factor levels are normal.

There are two main types of haemophilia - A and B. Haemophilia A is the most common type.

The standard treatment for the condition for the last 40 years has been intravenous injections of the missing clotting factor. In order to prevent joint damage, these injections were administered three times per week. However last year, an improved form of the intravenous injection was introduced.

The new treatment, Emicizumab (Hemlibra), which has been licenced by the European Medicines Agency, mimics the effect of the clotting factor VIII injections for patients with haemophilia A.

It offers more protection against bleeding and rather than requiring vein access, it is injected subcutaneously (under the skin) once a week or once every two weeks.

"This new treatment is a very significant step forward. It will provide greater protection for people with haemophilia from the risk of bleeding and it will be much easier to administer, without the need for regular intravenous injections,' explained the society's CEO, Brian O'Mahony.

The product will be made available to all people with the severe form of haemophilia A.

"The burden of venous access and central venous access devices, with their potential for infective, mechanical and other complications, will be lifted from families of children with severe factor VIII deficiency using Hemlibra for prophylaxis," commented Dr Beatrice Nolan, a consultant haematologist and director of the National Paediatric Haemophilia Treatment Centre at Our Lady's Children's Hospital, Crumlin.

The product has been made available following agreement with the Haemophilia Product Selection and Monitoring Advisory Board, which procures medications for haemophilia in Ireland. The board includes the society, haemophilia specialists and officials from the Department of Health.

For more information on the Irish Haemophilia Society, click here.


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