Hemophilia cannot be cured but when the condition is managed with medication, many patients experience a normal quality of life.
The treatment approach depends on the type of hemophilia a child has, as well as how severe the condition is. There are two forms of this condition, hemophilia A and hemophilia B, although most cases are hemophilia A.
The two main approaches to treatment are:
- Preventive treatment, which refers to the regular medications that are taken to prevent episodes of bleeding and subsequent complications such as joint damage.
- Episodic treatments, which are used to manage episodes of prolonged bleeding.
Hemophiliacs lack certain clotting factors that help to coagulate the blood and make it sticky so that bleeding eventually stops after an injury. Genetically engineered clotting factor medications are now available to help replenish clotting factors and prevent prolonged bleeding. These clotting factors can be administered as injections, which may be needed on a regular basis in cases of severe hemophilia. Most patients are taught to administer these injections at home so that regular hospital visits can be avoided. 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.
For hemophilia A, regular injections of a medication called octocog alfa are administered, which is an engineered version of clotting factor VIII. Patients are advised to have an injection every 48 hours. Side effects include an itchy skin rash and redness at the injection site.
For hemophilia B, regular injections of a medication called nonacog alfa are administered. This is a genetically engineered version of clotting factor IX and injections are usually administered twice a week.
Other treatments include regular joint strengthening exercises to prevent joint damage. Hemophiliacs should also avoid high impact sports and situations that could lead to trauma and bleeding.
In mild or moderate hemophilia, 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