Rare Disease Report

April 17 is World Hemophilia Day

APRIL 16, 2017
RDR Staff
Its World Hemophilia Day and this year’s theme is to share your story so that others can learn about the condition.  By sharing stories, it is hoped that others will be tested to see if they have a bleeding disorder or if they are carriers. Most hemophilias are genetic conditions and many people may not know that they are carriers.

Different Types of Hemophilia

There are many different types of bleeding disorders, including hemophilia (types A and B), other clotting factor deficiencies, and platelet disorders. Most are rare conditions. Individuals with these conditions have clotting factor proteins or platelets that work poorly, if at all.
The severity of symptoms varies based on the amount of clotting factor involved and its degree of functioning. Without proper treatment, affected individuals may suffer from severe pain or permanent joint damage. Severe bleeds into the brain or other organs can cause death.
Hemophilia A results in impaired clotting mechanisms due to missing or reduced levels of factor VIII. People with hemophilia A experience recurrent and extended bleeding episodes that cause pain and irreversible joint damage. Some of these bleeding episodes can be life threatening. There are an estimated 16,000 – 20,000 people in the United States with hemophilia A (and 142,000 world wide). 
AdvateKogenate and Kovaltry - are approved for prophylactic use in patients with hemophilia A.
Hemophilia B is a genetic disorder caused by missing or defective factor IX. Although it is passed down from parents to children, about 1/3 of cases are caused by a spontaneous mutation, a change in a gene. There are more than 28,000 hemophilia B patients worldwide, including 4,000 patients in the United States.
Current standard of care for patients with hemophilia B involves chronic replacement of FIX protein through intravenous infusion.
Idelvion and Ixinity are approved for prophylactic use in patients with hemophilia B.

History of World Hemophilia Day

World Hemophilia Day was started by the World Federation of Hemophilia (WFH) in 1989 to increase global awareness of hemophilia and other bleeding disorders. April 17th was selected in honor of the birthday of Frank Schnabel, founder of WFH.  Each year, numerous landmarks change their lighting to red, the color associated with awareness of the disease. 
More information about bleeding disorders and World Hemophilia Day is available at www.wfh.org.

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