Personalized Medication Management
for Bleeding Disorders (Hemophilia)

Mayo Clinic Specialty Pharmacy offers pharmacy services to people with hemophilia who need long-term clotting-factor drug therapy. Experienced pharmacy professionals work closely with the patient's Mayo Clinic care team to develop a personalized medication management program at no additional cost. The Specialty Pharmacy helps coordinate all billing and claims, relieving the patient of up-front costs and the headaches of completing insurance forms and filing claims.

About Hemophilia
The Role of Medications in Managing Hemophilia
Side Effects, Cautions and Special Requirements
Always Ask Your Pharmacist

About Hemophilia

Hemophilia is a bleeding disorder characterized by a deficiency of select proteins in the blood-clotting system. Clotting is the process by which blood changes from a liquid to a solid state in order to stop bleeding. The clotting process makes use of blood particles (platelets) and clot-forming proteins (clotting factors). Blood has 13 clotting factors, identified by Roman numerals, which are involved in the clotting process.

Hemophilia occurs in three types — A, B and C — depending on which clotting factor is deficient. All types can cause prolonged bleeding. A person with hemophilia who gets a cut will bleed longer than a person with normal clotting. Small cuts usually aren't a significant problem. The greater problem is deep internal bleeding and bleeding into joints.

About 18,000 Americans have hemophilia. Most cases are either hemophilia A or B, and hemophilia A accounts for about 80 percent of cases. Hemophilia C is rare, occurring in approximately one in 100,000 Americans.

Hemophilia A and B almost always occur in boys. Generally, hemophilia A and B pass from mother to son through one of the mother's genes. Everyone has two sex chromosomes, one from each parent. Females inherit an X chromosome from their mother and an X chromosome from their father. Males inherit an X chromosome from their mother and a Y chromosome from their father. The gene that causes hemophilia A or B is located on the X chromosome. This is why men can't pass along the gene that causes hemophilia to their sons. Most women who have the defective gene are simply carriers and exhibit no signs or symptoms of hemophilia. It is also possible for hemophilia A or B to occur through spontaneous gene mutation.

Hemophilia C can occur in both boys and girls. The defective gene that causes hemophilia C can also be passed on to children by mothers and fathers, but it follows an inheritance pattern different from that which occurs with hemophilia A and B.

Complications
Complications can occur from hemophilia or from the disease treatment:

Deep internal bleeding. Hemophilia can cause deep-muscle bleeding. This deep internal bleeding may cause an arm or leg to swell and create pressure on nerves, leading to numbness and pain.

Damage to joints. Internal bleeding may also put pressure on and damage joints. Pain sometimes may be severe, and a patient may be reluctant to use an arm or a leg or move a joint. If bleeding occurs frequently and the patient doesn't receive adequate treatment, the irritation may lead to destruction of the joint or to the development of arthritis.

Infection. People with hemophilia are more likely to receive blood transfusions and are at greater risk of receiving contaminated blood products. Until the mid-1980s, it was more common for people with hemophilia to become infected with the human immunodeficiency virus (HIV) or hepatitis through contaminated blood products. Since then, blood products are much safer due to steps taken to purify and screen the supply of donated blood. The risk of infection through blood products has decreased substantially since the introduction of genetically engineered clotting products (recombinant factors), which are not derived from human blood. However, it's possible for people who rely on blood products to contract other diseases. A patient with hemophilia should consider receiving immunization against hepatitis A and B.

Adverse reaction to clotting-factor treatment. Some people with hemophilia develop proteins in their blood that inactivate the clotting factors used to treat bleeding.

The Role of Medications in Managing Hemophilia

Hemophilia is a lifelong disease. But with medication, replacement of deficient clotting factors and proper self-care, most people with hemophilia can live an active, productive lifestyle.

Treatment of hemophilia varies depending on the severity of the condition:

Mild hemophilia A.
Mild hemophilia A is treated with an injection of the hormone desmopressin (DDAVP) into a vein to stimulate a release of more clotting factors to stop bleeding. Occasionally, desmopressin is given as a nasal medication.

Severe hemophilia A or hemophilia B.
Bleeding may stop only after an infusion of clotting factor derived from donated human blood or from genetically engineered products (recombinant clotting factors). A patient may need repeated infusions if internal bleeding is serious. The patient's doctor may also suggest preventive use of clotting factor to avoid bleeding episodes.

Hemophilia C
The patient needs plasma infusions to stop bleeding episodes.

Infusions of a clotting factor two or three times a week may help prevent bleeding. This approach may reduce time spent in the hospital and away from home, work, or school, and limit side effects such as damage to joints. The doctor can train the patient to do infusions of desmopressin or clotting factor at home, work or school.

Aminocaproic acid (Amicar) is a drug used to hold a clot in place once it has formed in the bloodstream. Aminocaproic acid is useful for people with hemophilia before they undergo dental work, for mouth, nose and minor intestinal bleeding and for hemophilia carriers (women who have the gene responsible for causing hemophilia but do not have hemophilia) with heavy, prolonged menstrual bleeding.

Side Effects, Cautions and Special Requirements

Clotting factor products may cause flushing, nausea, fever, chills, mild fatigue, nosebleeds or hives. Desmopressin (DDAVP) may cause facial flushing, mild headaches, nausea and abdominal cramps. Aminocaproic acid (Amicar) can cause nausea, drowsiness or dizziness, diarrhea and stomach pain.

Drugs that can increase the risk of bleeding include aspirin and nonsteroidal anti-inflammatory drugs (for example, ibuprofen (Advil, Motrin, others). Instead, acetaminophen (Tylenol, Panadol, others), is a safe alternative for mild pain relief. Also avoid certain blood-thinning medications, such as heparin and warfarin (Coumadin), which prevent blood from clotting.

Most clotting factor products should be refrigerated; however many can be stored at room temperature for up to six months. Check with a pharmacist regarding the proper storage of prescribed clotting-factor products. Mayo Clinic Specialty Pharmacy ships products that require refrigeration with ice packs by overnight delivery.

Always Ask Your Pharmacist

A pharmacist is a reliable source of information about hemophilia management and can help the patient monitor the condition, maximize the benefits of medications, limit side effects and identify drug-drug or drug-disease interactions. The patient's Specialty Pharmacy pharmacist will work closely with the Mayo Clinic Comprehensive Hemophilia Treatment Center to create a safe and appropriate care plan.

Optimal control of hemophilia requires the coordinated care of the patient's health care team. Doctors, nurses and other caregivers must work closely with pharmacists and others to ensure therapy is safe and effective. Incomplete management of hemophilia or another medical condition may lead to complications or limit the success of treatment.