The treatment approach to haemophilia depends on the type of haemophilia a person has and its severity. There are two forms of this condition, haemophilia A and haemophilia B, although most cases are haemophilia A.
There are two main approaches to the treatment of haemophilia A. These include:
- Preventive treatment - This refers to the regular medications that are taken to prevent episodes of bleeding and subsequent complications such as joint damage.
- Episodic treatment - These treatments are used to manage episodes of prolonged bleeding.
Over recent decades, genetically engineered clotting factor medications have been made available to help replenish clotting factors and prevent prolonged bleeding. This medication is administered as an injection on a regular basis in the case of severe haemophilia. Children with haemophilia A are often taught to give self-injections so as to minimize the need for hospital admissions as they pass into adulthood. An implantable port is sometimes surgically placed under the skin (e.g. Port-a-Cath). This is connected to a blood vessel near the heart and can be used each time injections are given. These preventive medications need to be continued lifelong to prevent bleeding episodes.
For haemophilia A, regular injections of a medication called octocog alfa are administered. This is an engineered version of clotting factor VIII, the clotting factor people with haemophilia A are deficient in. Patients are advised to have an injection every 48 hours. Side effects include an itchy skin rash and redness at the injection site.
In mild or moderate haemophilia, regular preventive treatment is not always required. These patients may only require treatment to manage individual episodes of bleeding. For people with haemophilia A, this may be achieved with injections of octocog alfa or another medication called desmopressin. Desmopressin is a synthetic hormone that stimulates the production of clotting factor VIII.
Reviewed by Sally Robertson, BSc