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Gone are the days when聽a hemophilia diagnosis meant you could not live a normal life. Now more hemophilia treatments are approved by the Food and Drug Administration (FDA), and people with the condition can better manage bleeding. That鈥檚 good news as thousands observe World Hemophilia Day on April 17, 2014.

Hemophilia is a rare bleeding dis- order. It is usually hereditary, but it can be acquired in rare cases if a person鈥檚 body develops antibodies that attack clotting factors in the bloodstream. Hereditary hemophilia usually occurs in males and currently affects about 23,500 Americans.

People with hemophilia are lacking one or more important clotting factors, which are proteins needed for blood clotting. Several types of clotting factors exist and there are two main types of hemophilia, says Nisha Jain, M.D., chief of the Clinical Review Branch in FDA鈥檚 Office of Blood Research and Review. Hemophilia A occurs when people have low levels, or missing, clotting factor VIII (8). Hemophilia B occurs when people have low, or missing, clotting factor IX (9).

People with hemophilia may bleed for a longer time than others after injury or surgery. They may also have internal bleeding鈥攅specially in knees, ankles, and elbows鈥攖hat can damage organs and tissues and even be life-threatening.

With appropriate medical care, people with hemophilia are living healthier lives.

锟糡reatments for Hemophilia

At present hemophilia is not curable, but treatments have come a long way, Jain says.

Hemophilia is sometimes called the 鈥淩oyal Disease鈥 because it affected the royal family of England during the 1800s. By the 1960s, hemophilia was treated with whole blood or fresh plasma. But these treatments didn鈥檛 have enough factor VIII or IX proteins to stop serious internal bleeding.

Now the primary type of hemo- philia treatment is replacement therapy: Concentrates of clotting聽factor VIII (for hemophilia A) or clotting factor IX (for hemophilia B) are injected into a patient鈥檚 vein to replace low or missing factor.聽These concentrates have traditionally been made from human聽blood. Today, an increasing number聽are made using recombinant DNA聽Art should be large on the first聽technology (a form of artificial DNA),聽page, so it shows up clearly when聽with some made without any material聽the PDF is made into a small JPEG.聽sourced from humans or animals. The final product is a powder that is mixed with sterile water before use.

Some people have regular preventive or 鈥減rophylactic鈥 therapy to prevent聽bleeding. This can be done in less than five minutes, excluding prep time, Jain explains. Others have therapy only as needed.

The type and frequency of treatment varies depending on the severity of hemophilia, which can be classified as mild, moderate or severe.

People with mild hemophilia have 6% to 49% of the normal levels of clotting factors in their blood. They generally only have bleeding problems after serious injury or surgery.

Those with moderate hemophilia鈥 about 15 percent of the hemophilia population, according to the National Hemophilia Foundation聽 have 1% to 5% of normal clotting factor levels. They聽can have bleeding problems after injury and spontaneously.

Those with severe hemophilia鈥 about 60% of the hemophilia population, per NHF鈥攈ave less than 1% of normal clotting factor levels. They bleed after injury and may have frequent spontaneous bleeding, including bleeds into joints and muscles. In addition to factor replacement, pain medication and physical therapy are also used to lessen pain and swelling if joint bleeds occur.

鈥淭he mild patients rarely need treatment. And moderate patients are the same,鈥 explains Jain. In certain situations, such as before dental work, patients with mild hemophilia receive Desmopressin (DDAVP) a man-made hormone that increases the level of factor in the blood. 鈥淧atients with severe hemophilia are those who really need treatment to prevent or resolve bleeds.鈥

A Shift Toward Prevention

Doctors, particularly hematologists who specialize in the study of blood, tend to identify people with severe hemophilia early. 鈥淧atients can be diagnosed as infants during circum- cision,鈥 says Jain. Then families can work with hematologists and hema- tology treatment centers.

鈥淚n recent times, we have seen shifting toward the prevention of bleeds,鈥 Jain says. 鈥淵ou want to prevent bleeding that causes joint damage. Once joint damage starts, it is difficult to stop progression unless bleeding into joints is reduced.鈥

The treatments for hemophilia continue to improve, and FDA has approved many replacement factors in recent years. For instance, in

March 2014 it approved Alprolix, the first Hemophilia B treatment designed to require less frequent injections when used to prevent or reduce the frequency of bleeding. It also recently approved Rixubis鈥攁 factor IX product鈥攖o control, prevent and reduce bleeding associated with hemophilia B. And the agency approved Novoeight, a factor VIII product, for control of bleeding and a routine prophylaxis treatment for adults, adolescents and children with hemophilia A.

Of course, the agency continues to carry out its broad responsibility to regulate medicines made from blood and blood components,聽including clotting factors. Today, due to strengthened FDA safeguards and oversight, these products are safer than they have ever been.

鈥淧atients should stay informed about various treatment options,鈥 Jain adds, 鈥渁nd should consult with their health care providers to obtain, and follow, a comprehensive management plan.鈥

She says those with hemophilia tend to be well-educated about their health and notes, 鈥淲ith appropriate treatment and care, people with hemophilia can, and do, live normal lives.鈥

Click HERE to read the FDA’s article in its entirety.

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