ABOUT IDIOPATHIC THROMBOCYTOPENIC PURPURA
Idiopathic thrombocytopenic purpura is a rare disorder in which the blood does not clot. It is caused when a person’s platelets are misidentified as foreign objects by the immune system and are subsequently targeted for elimination.
Normal proteins found on the surface of the platelets act as antigens in idiopathic thrombocytopenic purpura-affected patients, thus, signaling the body’s white blood cells to remove the platelets from the bloodstream. As a result, patients with idiopathic thrombocytopenic purpura have an abnormally low platelet count. A healthy person usually has a platelet count of between 150,000 and 400,000 per cu/ml of blood. A count of 30,000 is generally considered safe (i.e., protecting against spontaneous bleeding). Patients with idiopathic thrombocytopenic purpura often have blood counts ranging from zero (in the most severe cases) to about 100,000 per cu/ml of blood (milder case). As a result, idiopathic thrombocytopenic purpura sufferers often bruise easily, suffer from bleeding gums as well as nosebleeds, gingival, gastrointestinal, and/or central nervous system bleeding.
There are two types of idiopathic thrombocytopenic purpura: (1) Acute idiopathic thrombocytopenic purpura, usually lasting less than six months and mainly occurring in children, typically caused by a viral infection (often not requiring treatment and non-recurring), and; (2) Chronic idiopathic thrombocytopenic purpura, which is longer-term and chiefly affects adults, specifically women over 40 years of age.
According to the Platelet Disorder Support Association, idiopathic thrombocytopenic purpura currently affects approximately 200,000 people in the U.S., with women suffering from the disorder at a rate about 3 times greater than men. About 50% of new cases occur in children and roughly 30,000 new cases occur annually. At present, there is no known cure and only a limited number of treatments are available, including chemotherapy, various biologics, hormones, small molecule immunosuppressants or splenectomy. High dose corticosteroids are currently the standard first-line therapy due to ease of use (pill form), acceptable short term efficacy and lower cost. However, corticosteroid use is associated with significant short- and long-term side effects, including swelling of the face, neck, and/or shoulders, fluid retention, heartburn, osteoporosis, premature atherosclerosis, cataracts, glucose intolerance, thinning of the skin and increased risk of infections, among others. Despite these risks and associated drawbacks of other current treatments, the market for idiopathic thrombocytopenic purpura therapy is currently estimated at $750 million to $1 billion.
External Resources: