What are treatments for hemophilia?

What are treatments for hemophilia?

The mainstay of treatment is replacement of the blood clotting factors. Clotting factor concentrates can be purified from human donor blood or made in the laboratory using methods that do not use donor blood. This type of therapy is known as replacement therapy. Clotting factor replacement therapy is carried out by infusing the clotting factor concentrates into a vein, much like a blood transfusion. This type of therapy can be administered at home with proper instruction and training.

Depending upon the severity of the condition, replacement therapy may be carried out on an as-needed basis (called demand therapy) or on a regular basis to prevent bleeding episodes (known as prophylactic therapy).

People who have mild cases of hemophilia A are sometimes treated with the drug desmopressin, also known as DDAVP. This drug stimulates release of more clotting factor by the body. It is administered either slowly through the intravenous route (IV) or occasionally, in nasal spray form.

Pain relievers may be prescribed for symptom relief, but pain relievers other than aspirin or non-steroidal anti-inflammatory medications (such asnaproxen, ibuprofen) must be used, since these types of drugs further inhibit the blood's ability to clot. Acetaminophen (Tylenol and others) is often given for pain relief.

What are complications of treatment?

Inhibitors

A major complication of treatment is the development of so-called inhibitors to the clotting factors. Inhibitors (antibodies) are produced because the body sees the factor concentrates used to treat patients to reduce or prevent bleeding, as foreign and activates an immune response in the patient to destroy the foreign substances (factor VIII or factor IX).

Inhibitors to factor VIII are the most common and occur in about one-third of those with severe hemophilia A and about 1 out of every 50 people with mild or moderate hemophilia A. They typically develop in childhood in those with severe hemophilia A and later in life in milder cases. Inhibitors destroy both the replacement factor VIII concentrates as well as any factor VIII that is present in the body. This is a serious complication of treatment because the factor concentrates are no longer effective in treating the condition. The action of inhibitors to destroy factor VIII concentrates shows different degrees of severity among individuals and can even vary over time in the same individual.

In about two-thirds of cases, the inhibitors disappear on their own or with treatment known as immune tolerance therapy (ITT) or immune tolerance induction (ITI). In cases of severe hemophilia A with persistence of inhibitors, other factor concentrates, such as activated prothrombin complex concentrate or recombinant factor VIIa, are administered to attempt to help control bleeding.

The development of inhibitors to factor IX is much less common and occurs in about 1% of those with hemophilia B. However, these can cause a very serious allergic reaction when factor IX concentrates are given. Immune tolerance therapy to eliminate inhibitors is less successful than with hemophilia A.