¿How is hemophilia treated?
To treat hemophilia, the missing factor is injected into the bloodstream with a needle.. Bleeding stops when enough clotting factor reaches the place of the hemorrhage. When bleeding occurs into a joint cavity (hemarthrosis), immediate treatment must be provided in order to avoid long-term damage.
Factor concentrates that are made of human blood are known as plasma derivedproducts while the ones that are made with genetically designed cells that carry the gene of human factor are called recombinant factor concentrates. People with mild hemophilia A sometimes use desmopressin (also called DDAVP), a synthetic hormone that stimulates factor VIII release.
With the right treatment and adequate care, people with hemophilia can live a perfectly normal life. If treatment is not administered, hemophilia can cause atrophying pain, severe damage to the joints, disability and early mortality. Unfortunately, only near the 25 % of the people with hemophilia around the world receive proper treatment. The World Federation of Hemophilia’s mission is to improve care and treatment for people with bleeding disorders worldwide. In our country, most people with hemophilia have access to proper treatment and are registered in the Hemophilia Foundation.
¿Where can bleeding occur?
Most bleeding episodes are internal and occur into the joints, which is where complications can frequently be encountered. Hemarthrosis can cause pain, immobility and, if time passes and the right treatment is not administered, it can lead to deformity. Knees, elbows, ankles, shoulders and hips are usually the most affected sites. Recurrent hemarthrosis that are not immediately treated can damage cartilage and bone, which can lead to chronic arthritis or disability. Muscles can also be affected. Muscle bleeding can cause swelling, pain and redness. The excessive amount of blood can increase the pressure on tissue and nerves, which can lead to permanent damage or deformity. Soft tissue bleeding occurs most commonly in arms, forearms, thighs, calves and iliopsoas, located in the pelvis area. Some bleeding can be life-threatening –such as head, throat, abdominal and iliopsoas bleeding– and require immediate treatment.
¿What is profylaxis?
Profylaxis is the regular infusion of clotting factor concentrate in order to prevent bleeding episodes. Two or three times a week factor is injected into the bloodstream to keep a constant level of factor VIII or IX. It helps to reduce and prevent joint damage. In countries where access to factor concentrates is usual, this treatment is becoming the norm for young patients and can be started once the veins are well developed (generally, between the first two and four years of life). Several years ago, in Argentina, the Resolution 2048/2003 was passed, which approved coverage of clotting factors for patients with severe hemophilia A and B. Even though a good prophylactic practice leads to a 300 % increase in factor usage, it is a fact that it brings a 100 % reduction of corrective surgery and a 70 % reduction of hospital admission and medical consultation. In a few words, profylaxis translates into an improved quality of life.
¿What is an inhibitor?
Inhibitors are antibodies that attack and destroy factor VIII and IX proteins that are contained in the clotting factor concentrates and render the treatment ineffective. They are developed almost exclusively in people with severe hemophilia. The actual incidence of inhibitor development is a matter of controversy. However it is possible to say that approximately 15 % of people with severe hemophilia A will develop inhibitors, while people with hemophilia B will rarely do so: the incidence is less than 1 %. Most inhibitors appear after rather few factor infusions. Generally, the more factor replacement therapy a person has received without the presence of inhibitors, the less chances that person has of developing them.
People with hemophilia do not bleed any faster than normal, but they can bleed for a longer time. They quickly respond to replacement therapy and generally know when bleeding started much before showing a visible sign like, for example, joint swelling.
A small cut or bump in a person with hemophilia must be treated like any minor accident as they do not carry a risk of complications. In the case this happens to a child, parents must be notified, while the wound is handled without specific treatment.
In the case of an emergency, the same steps that are usually followed with any person should be taken. The Hemophilia Foundation is the specialized treatment center that must be turned to. People with hemophilia should not take aspirin (ASA or acetylsalicyclic acid), or anything containing aspirin because it interferes with the stickiness of platelets and can worsen the hemorrhage. Paracetamol (acetominophen) is a perfectly safe alternative to aspirin to relieve pain.