Therapeutic treatment
Today haemophilia cannot be cured. It can however be treated by replacing the clotting factor that is missing or is in low levels in the blood. This process involves the intravenous administration of the regimen containing that particular factor.
Αemophilia A, alternatively, can be treated by subcutaneous administration of a molecule that mimics the activity of factor VIII.
There are two ways of treating haemophilia:
Symptomatic or on demand therapy
Symptomatic or on demand therapy is given after a severe bleeding event (e.g. following an injury or bleeding into joints), in order to raise the coagulation factor levels in the blood and to stop the bleeding rapidly.
It is extremely important to administer the therapy as soon as possible after the onset of bleeding, to avoid further damage to afflicted tissue. If bleeding is not treated immediately, can cause long-term damage to the tissues (e.g. distortion or destruction of the joints) or even threaten the life of the person (e.g. in case of intracranial bleeding).
Prophylactic therapy
As the word denotes, prophylactic therapy aims to prevent bleeding episodes, i.e. to prevent bleeding through the long-term retention of satisfactory levels of coagulation factor in the blood. This is achieved by regular intravenous administration of treatment containing the relevant agent, according to physician’s instructions.
Alternatively, prophylactic therapy may include subcutaneous administration of a molecule that mimics the effect of factor VIII.
Prophylactic therapy helps reduce the incidence of bleeding in the joints, minimizing the impact of hemophilic arthropathy.
What is an inhibitor?
A major complication of replacement therapy is the development of inhibitors against coagulation factors administered as treatment. Inhibitors are antibodies developed by the body against the coagulation factors administered as treatment. In this way, the body “fights” the agent that receives through intravenous treatment (factor VIII or IX) because it recognizes it as “intruder” and develops antibodies to “inactivate” it.
Inhibitors of factor VIII are the most common and occur in about a third of people with severe haemophilia A and in about 1 in 50 people with mild or moderate haemophilia A.
Typically, this is developed during childhood in individuals with severe haemophilia A and later in life to those with milder conditions. Inhibitors destroy both the coagulation factor administered as treatment and any other coagulation factor already present in the body.
This is a serious complication of treatment, because if, for example, a person with haemophilia A develops an inhibitor of factor VIII, inhibitors will “inactivate” the total amount of factor VIII present in the body and, consequently, the efficacy of treatment will be reduced.
So, aren’t there any good news for people with inhibitors?
Of course, there are! In about two-thirds of the cases, the inhibitor disappears on its own or by the application of a treatment known as immune tolerance therapy (ITT) or immune tolerance induction (ITI). In cases of severe haemophilia A with persistent inhibitors in an effort to control bleeding, bypassing agents are administered, which contain other factors that can stimulate the formation of a clot and stop the bleeding.
Alternatively, haemophilia A patients with inhibitors against factor VIII may receive a subcutaneous prophylactic therapy with a molecule that mimics the activity of factor VIII.
Key milestones in the treatment of haemophilia
References:
1.WFH. Guidelines for the management of haemophilia. 2012.
