Both diseases are rare bleeding disorders in which blood clotting—the natural process that
allows wounds to heal and prevents excessive bleeding—is defective. Yet while most people
have likely heard of hemophilia, the so-called “royal disease” that afflicted the young son
of Czar Nicholas II at the time of the Russian Revolution, immune thrombocytopenia purpura
(ITP) is much less well-known.
Although they are both bleeding disorders, hemophilia and ITP do have striking differences. In ITP, the defective clotting is associated with a low number of platelets (thrombocytopenia) in the blood. In hemophilia, the defect is the absence of certain blood clotting proteins (factor VIII or XI). ITP is an autoimmune disease, a disorder in which the immune system attacks healthy cells—in this case, platelets—as “intruders.” In contrast, hemophilia is a genetic disorder that usually is inherited but also can be caused by a spontaneous genetic mutation. Sudden and severe loss of blood and internal bleeding are possible in both diseases, although these complications are much less likely to occur with ITP than with hemophilia.
September is National Immune (or Idiopathic) Thrombocytopenic Purpura Awareness Month,
and purple is the designated color for showing your support. At Connexion Healthcare, we aim to help raise awareness about rare diseases, and during the month of September we are focusing public attention on ITP. ITP affects around 120,000 Americans and is over ten times more prevalent than hemophilia.
Although the exact cause of ITP is still unknown, it is thought that during a viral infection, when the body creates antibodies to fight off foreign substances, antibodies are accidentally created that stick to the platelet cells. These cells are then recognized as “foreign” by the immune system and are destroyed in the spleen.
Children with ITP usually recover fully without treatment. In adults, ITP may become a long-term condition requiring pharmacotherapy with corticosteroids or other drugs. Surgery to remove the spleen in patients with ITP is rare but sometimes necessary for severe menorrhagia or life-threatening hemorrhage.
ITP is called chronic when it is present for longer than 1 year and is called acute when it is short-lived. The clinical onset may be gradual or rapid. About 85% of children recover within 1 year, with no recurrence of ITP. More than 50% of adults have chronic ITP. ITP is called primary when it develops for no known reason. Secondary ITP is associated with other illnesses such as an autoimmune disease or an infection, or occurs after the use of certain drugs or after a transfusion.
A common sign of ITP is easy bruising, often in the form of small purple spots where blood has leaked (purpura) or tiny red pinpoints on this skin (petechiae) that may look like a rash. Other symptoms may include nosebleeds, bleeding in the mouth or gums, and blood found in vomit, urine, or stool.
If you experience any of these issues, it is best to visit your doctor to have a complete blood count (CBC) done. Normally, blood contains 150,000 to 450,000 platelets, but in those with ITP, the platelet count is usually below 100,000. The lower the platelet count drops, the greater the risk of serious bleeding.
During National ITP Awareness Month,
please join us in raising public understanding of this rare disease and in showing our support for those who have ITP.
ITP Foundation: http://www.itpfoundation.org/
PDSA (Platelet Disorder Support Association): https://www.pdsa.org/
NORD (National Organization for Rare Diseases): http://rarediseases.org/rare-diseases/immune-thrombocytopenia/
About Connexion Healthcare
Connexion Healthcare is a full-service global provider of strategic medical and scientific communications with decades of experience providing exceptional service to the pharmaceutical industry. We provide services through 2 Centers of Excellence—Oncology and Rare Disease—and possess core expertise in these therapeutic areas and their marketplaces. We synthesize scientifically complex information and distill it into focused and highly compelling programs. In addition, we develop communication platforms that effectively influence clinical behavior and drive strategic initiatives forward. Our esteemed medical directors and content development team, along with an accounts team rooted in science and best practices, are poised and ready to support brands anytime from phase 2 development on.
For more information on how Connexion Healthcare can assist you, please contact:
Susan Stein, MPH