Both preventive and on-demand treatment can be administered at home. Home treatment is the ideal method of treatment from a medical viewpoint as a minimum amount of time is lost between the recognition of a bleed and treatment. This has many advantages.
It reduces the disruption caused by a bleeding episode to the person with haemophilia and his family and the patient feels more able to control his condition. The benefits of self-infusion at home not only include increased independence and the bonus of not having to travel to the hospital at all hours for treatment, but school and work attendance is more regular. If bleeds are treated promptly, the period of incapacity caused by each episode can be reduced.
In adults and teenagers, home treatment is usually carried out by the affected person. From a young age, children will be taught how to self-infuse, alternatively, a device called a Port-a-Cath can be used to facilitate venous access.
It is recommended that all children with severe haemophilia who are over two years of age or who have had two joint bleeds receive regular doses of coagulation factor concentrate to prevent spontaneous bleeding and reduce the risk of long-term side effects. This is called prophylaxis. For children with severe factor VIII and factor IX deficiency, this involves an injection of coagulation factor concentrate into the veins on a regular basis, usually one to two times per week. In Ireland, the factor concentrates currently used are extended half life factor concentrates which are infused less regularly then regular factor concentrates and offer more protection against bleeding episodes.
This treatment regime, although time-consuming and sometimes difficult to learn, will prevent joint damage and lead to an improved quality of life.
From late 2019, a new generation of treatment for people with severe factor VIII deficiency is available as an option in Ireland. This treatment, called Hemlibra, is a monoclonal antibody which mimics the effect of factor VIII. It can be infused weekly or every 2 weeks subcutaneously (under the skin) thereby avoiding the requirement for regular intravenous injections. This treatment offers a high degree of protection against bleeding but can not be used to treat bleeding episodes. In the event of a bleeding episode, intravenous infusion with FVIII concentrate will continue to be required. This subcutaneous therapy can not be used for factor IX deficiency so, in this case, the routine treatment will continue to be extended half life factor IX concentrate.