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Idiopathic Thrombocytopenic Purpura

NORD is very grateful to Kenneth M. Algazy, MD, Clinical Professor of Medicine, University of Pennsylvania School of Medicine, for assistance in the preparation of this report.

Synonyms of Idiopathic Thrombocytopenic Purpura

  • autoimmune thrombocytopenic purpura
  • ITP
  • primary thrombocytopenic purpura

Disorder Subdivisions

  • No subdivisions found.

General Discussion

Idiopathic thrombocytopenic purpura (ITP) is a not infrequent autoimmune bleeding disorder characterized by the abnormally low levels of blood cells called platelets, creating a condition known as thrombocytopenia. Platelets are specialized blood cells that help prevent and stop bleeding by inducing clotting. In many ITP cases, there are no readily apparent causes or underlying disease (idiopathic), but frequently there are associated collagen vascular diseases or underlying neoplasms, most frequently lymphoid. The cells of the immune system, lymphocytes, produce anti-platelet antibodies that attach to the platelets. The presence of antibodies on platelets leads to their destruction in the spleen. The disorder is characterized by abnormal bleeding into the skin resulting in bruising, which is what the term purpura means. Bleeding from mucous membranes also occurs, and may subsequently result in low levels of circulating red blood cells (anemia).

ITP presents as a brief, self-limiting form of the disorder (acute ITP) or a longer-term form (chronic ITP). Acute ITP accounts for about 50% of cases, and chronic ITP accounts for the remainder. Eighty percent (80%) of the children with ITP have the acute form while the chronic form affects mostly adults. The acute form usually resolves without treatment (spontaneously) within three to six months. When thrombocytopenia lasts for more than six to 12 months, ITP is classified as the chronic form. Onset of acute ITP is often rapid, while the onset of the chronic form may be gradual.

Symptoms

A child or adult with idiopathic thrombocytopenic purpura may display no symptoms (asymptomatic). Symptoms of ITP may not appear until the platelet count is extremely low. Such symptoms may include:

- Skin that bruises very easily
- A rash consisting of small red dots (petechiae) that represent small hemorrhages
- Bleeding from any area of the body made obvious by blood in urine or feces
- Bleeding from the gums
- Frequent and long-lasting nose bleeds
- Abnormal menstruation

In some cases, frequent bleeding episodes may result in low levels of circulating red blood cells (anemia), which may produce weakness and fatigue. Additionally, people with this disorder may experience fevers and abnormal enlargement of the spleen (splenomegaly). Some women with ITP may experience prolonged and heavy menstrual bleeding. In rare cases of ITP, a serious condition known as bleeding into the brain (intracranial hemorrhage) may occur.

Causes

Idiopathic thrombocytopenic purpura belongs to a group of disorders in which the body's natural immune defenses inappropriately act against the body's own tissues (autoimmune disorders). In ITP, an abnormal immune reaction appears to lead to destruction of certain blood cells known as platelets. For reasons that remain as yet unknown, lymph tissues and the spleen are stimulated to produce anti-platelet antibodies that mistakenly attach to platelets, forming an "antibody-platelet complex". Antibodies are produced by the body's immune system to react to foreign substances, known as antigens. In many cases, the exact cause of the production of these anti-platelet antibodies is unknown (idiopathic), but in some underling collagen vascular/rheumatologic conditions as well as malignancy may stimulate antibody production

ITP affects normal platelets as they circulate through the spleen. The antibody-platelet complex is recognized as foreign by the immune system, which then goes to work to destroy it. After a while, the platelet count in the blood declines and thrombocytopenia (abnormally low numbers of platelets) ensues.

In children, ITP often occurs following an acute viral infection or upper respiratory illness suggesting that antibodies produced to fight foreign viral substances (antigens) may cross- react with the antigens and, in turn, destroy platelets.

Some cases resembling ITP may result from the use of certain drugs. According to the medical literature, Helicobacter pylori, a bacterium that has been shown to cause stomach ulcers, is associated with the development of ITP in some cases.

Affected Populations

The incidence of idiopathic thrombocytopenic purpura among adults annually in the USA has been estimated at 66 cases per million. The annual incidence among children has been estimated at about 50 cases per million of population. Chronic ITP is thought to make up about 10 cases per million per year.

The incidence varies from country to country with studies in Denmark reporting 10-40 cases per million per year. Other studies in Kuwait report 125 cases per million per year.

Among adults diagnosed with chronic ITP, there are 2.6 cases among women for every case involving a male. Among children diagnosed with acute ITP, the male to female ratio is almost equal, with 52% male to 48% female.

Among adults, the incidence is greatest at ages 20 to 50 years. Among children, the incidence is greatest at ages 2 to 4 years. About 40% of all patients diagnosed with one form of ITP are children younger than 10 years of age.

Related Disorders

Symptoms of the following disorders can be similar to those of ITP. Comparisons may be useful for a differential diagnosis:

Thrombocytopenia may be caused by other blood (hematological) disorders or secondary to a collagen vascular disorder such as systemic lupus erythematosus. Adverse drug reactions (drug-induced thrombocytopenia) are also potential causes of thrombocytopenia. Quinidine, quinine and heparin are three drugs often associated with the development of immune thrombocytopenia.

Thrombotic thrombocytopenia purpura (TTP) is a rare, serious blood disease. Major symptoms may include a severe decrease in the number of blood platelets (thrombocytopenia), abnormal destruction of red blood cells (hemolytic anemia), and disturbances in the nervous system. Affected individuals also exhibit red rash-like areas of skin or patches of purplish discoloration (purpura) resulting from abnormal bleeding into the mucous membranes (the thin, moist layer lining the body's cavities) and into the skin similar to ITP. The cause of thrombotic thrombocytopenia purpura is also currently thought to be antibody production in the majority of cases . (For more information on this disorder, choose "thrombotic thrombocytopenia purpura" as your search term in the Rare Disease Database.)

Henoch-Schonlein purpura is a rare inflammatory disease of the small blood vessels (capillaries) and is usually a self-limited disease. It is the most common form of childhood vascular inflammation (vasculitis) and results in inflammatory changes in small blood vessels. The symptoms of Henoch-Schonlein purpura usually begin suddenly and may include headache, fever, loss of appetite, cramping abdominal pain, and joint pain. Red or purple spots typically appear on the skin (petechial purpura). Inflammatory changes associated with Henoch-Schonlein purpura can also develop in the joints, kidneys, digestive system, and, in rare cases, the brain and spinal cord (central nervous system). Thrombocytopenia is not present. The exact cause of Schonlein-Henoch purpura is not fully understood, although research suggests that it may be an autoimmune disease or, in some rare cases, an extreme allergic reaction to certain offending substances (e.g., foods or drugs). (For more information on this disorder, choose "Henoch-Schonlein purpura" as your search term in the Rare Disease Database.)

Heparin-induced thrombocytopenia is the most common drug-induced, antibody-mediated thrombocytopenic disorder. Heparin is a drug often used in hospitals that prevents blood clotting (anticoagulant). Heparin-induced thrombocytopenia is characterized by serious thrombocytopenia and the formation of blood clots (thrombosis) often affecting the limbs. In some cases, serious complications may develop including stoke or the formation of blood clots in the lungs (pulmonary emboli) resulting in episodes of chest pain, coughing, difficult or labored breathing (dypsnea ), and coughing up blood (hemoptysis). Skin ulcerations may develop in some cases of heparin-induced thrombocytopenia.

Hypersplenism, an overactive spleen usually from liver disease, may mimic ITP.

Anticardiolipnin antibodies may also produce thrombobcytopenia but rather than bleeding, are associated with thrombosis. They frequently are found in adults with cardiovascular diseases.

Standard Therapies

Diagnosis
The diagnosis of ITP is usually made by excluding other causes of thrombocytopenia, including certain medications or the presence of disorders such as acute leukemia and aplastic anemia. The disorder is commonly without apparent symptoms (asymptomatic). Low platelet counts may be found after routine blood tests ordered for other purposes.

Blood tests to determine platelet counts and the presence of platelet antibodies are commonly ordered. Normal appearing red blood cells or white blood cells will rule out or exclude leukemia and/or aplastic anemia from the diagnosis. The presence of unusual cells in the blood will call for the use of a needle biopsy of the bone marrow.

Treatment
In some cases, especially with the acute from of ITP, no therapy may be necessary and the disorder may resolve itself (spontaneous resolution). When therapy is necessary, treatment with corticosteroid drugs (e.g., prednisone) is usually administered, and this is the mainstay of therapy. If platelet levels do not improve after corticosteroid treatment, affected individuals may require ongoing treatment with intravenous immunoglobulins (IVIG), usually through monthly infusions, but this does not lead to a cure.

The orphan drug anti-D (WinRho SDF), a form of gammaglobulin, was approved by the Food and Drug Administration (FDA) for the treatment of individuals with ITP who are RH positive and have not received a splenectomy. The drug may be used repeatedly in affected individuals, particularly children, who have the acute or chronic form of ITP. For information about this drug, contact:

1-800-4WINRHO (1-800-494-6746)
Cangene bioPharma, Inc.
Baltimore, MD 21230
Email: CustomerService@cangene.com
Product Website: www.winrho.com

Anti CD20 antibody, Rituximab (Rituxn) reduces IgG antibody production and is frequently used in patients refractory to other therapies.

Because the spleen plays a role in destroying antibody-covered platelets, surgical removal of the spleen (splenectomy) may be recommended in cases where affected individuals fail to respond to steroids or who fail to maintain a remission when steroids are discontinued. Splenectomy improves platelet counts in approximately 70 percent of cases and can achieve a remission in 50 to 60 percent.

If the patient has antibodies or evidence of Helicobacter pylori infection, antibiotics and proton pump inhibitors may ameliorate the condition. (This is much more common in Asia)

In 2008, the FDA approved Promacta manufactured by GlaxoSmithKline (GSK) for patients with chronic immune thrombocytopenic purpura to increase platelet counts and reduce bleeding. According to GSK, Promacta is the first pill to treat the condition. Other medications are available from different manufactures with the same mechanism of action.

In 2008, the FDA approved romiplostim for subcutaneous injection (Nplate, made by Amgen Inc.) for the treatment of thrombocytopenia in patients with ITP who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Romiplostim is a thrombopoietin (TPO) receptor agonist that stimulates bone marrow megakaryocytes to produce platelets.

Investigational Therapies

Information on current clinical trials is posted on the Internet at www.clinicaltrials.gov. All studies receiving U.S. government funding, and some supported by private industry, are posted on this government web site.

For information about clinical trials being conducted at the NIH Clinical Center in Bethesda, MD, contact the NIH Patient Recruitment Office:

Tollfree: (800) 411-1222
TTY: (866) 411-1010
Email: prpl@cc.nih.gov

For information about clinical trials sponsored by private sources, contact:
www.centerwatch.com

Idiopathic Thrombocytopenic Purpura Resources

NORD Member Organizations:

(To become a member of NORD, an organization must meet established criteria and be approved by the NORD Board of Directors. If you're interested in becoming a member, please contact Susan Olivo, Membership Manager, at solivo@rarediseases.org.)

Other Organizations:

References

TEXTBOOKS
Algazy KM. Idiopathic Thrombocytopenic Purpura. In: NORD Guide to Rare Disorders. Lippincott Williams & Wilkins. Philadelphia, PA. 2003:415-16.

Beers MH, Berkow R, eds. The Merck Manual, 17th ed. Whitehouse Station, NJ: Merck Research Laboratories; 1999:923.

Fauci AS, et al, eds. Harrison's Principles of Internal Medicine, 14th Ed. New York, NY: McGraw-Hill, Inc; 1998:732.

Bennett JC, Plum F, eds. Cecil Textbook of Medicine. 20th ed. Philadelphia, PA: W.B. Saunders Co; 1996:980-2.

Frank MM, et al. Samter's Immunologic Diseases, 5th ed. Boston, MA: Little, Brown and Company; 1995:919-25.

JOURNAL ARTICLES
Chouhan JD, Herrington JD. Treatment options for chronic refractory idiopathic thrombocytopenic purpura in adults: focus on romiplostim and eltrombopag. Pharmacotherapy. 2010;30(7):666-83.

Deane S, Teuber SS, Gershwin ME. The geoepidemiology of immune thrombocytopenic purpura. Autoimmun Rev. 2010;9(5):A34

Bussel JB. Traditional and new approaches to the management of immune thrombocytopenia: issues of when and who to treat. Hematol Oncol Clin North Am. 2009;23(6):1329-41.

Bennett CM, Tarantino M. Chronic immune thrombocytopenia in children: epidemiology and clinical presentation. Hematol Oncol Clin North Am. 2009;23(6):1223-38.

Fogarty PF. Chronic immune thrombocytopenia in adults: epidemiology and clinical presentation. Hematol Oncol Clin North Am. 2009;23(6):1213-21.

Cines DB, Liebman HA. The immune thrombocytopenia syndrome: a disorder of diverse pathogenesis and clinical presentation. Hematol Oncol Clin North Am. 2009;23(6):1155-61.

Ahn YS, Horstman LL. Idiopathic thrombocytopenia purpura: pathophysiology and management. Int J Hematol. 2002;76:123-31.

Todisco M, Rossi N. Melatonin for refractory idiopathic thrombocytopenic purpura: a report of 3 cases. Am J Ther. 2002;9:524-6.

Ancona KG, et al. Randomized trial of high-dose methylprednisolone versus intravenous immunoglobulin for the treatment of acute idiopathic thrombocytopenic purpura in children. J Pediatr Hematol Oncol. 2002;24:540-4.

Giagounidis AA, et al. Treatment of relapsed idiopathic thrombocytopenic purpura with the anti-CD20 monoclonal antibody rituximab: a pilot study. Eur J Haematol. 2002;69:95-100.

Pamuk GE, et al. Overview of 321 patients with idiopathic thrombocytopenic purpura. Retrospective analysis of the clinical features and response to therapy. Ann Hematol. 2002;81:436-40.

Yenicesu I, et al. Virus-associated immune thrombocytopenic purpura in childhood. Pediatr Hematol Oncol. 2002;19:433-7.

Michel M, et al. Autoimmune thrombocytopenic purpura and helicobacter pylori infection. Arch Intern Med. 2002;162:1033-6.

Adams JR, et al. Pharmacoeconomics of therapy for ITP; steroids, i.v.Ig, anti-D, and splenectomy. Blood Rev. 2002;16:65-7.

McMillan R. Classical management of refractory adult immune (idiopathic) thrombocytopenic purpura. Blood Rev. 2002;16:51-5.

Blanchette V. Childhood chronic immune thrombocytopenic purpura (ITP). Blood Rev. 2002;16:23-6.

Caplin C, et al. Is bone marrow aspiration needed in acute childhood idiopathic thrombocytopenic purpura to rule out leukemia? Arch Pediatr Adolesc Med. 1998;152:345-57.

Law C, et al. High-dose intravenous immune globulin and the response to splenectomy in patients with idiopathic thrombocytopenic purpura. N Engl J Med. 1997;336:1494-8.

Demiroglu H, et al. High-dose pulsed dexamethasone for immune thrombocytopenia. N Engl J Med. 1997;337:425-7.

Figueroa M, et al. Combination chemotherapy in refractory immune thrombocytopenic purpura. N Engl J Med. 1993;328:1226-9.

FROM THE INTERNET
Kessler CM, Sandler SG, Bhanji R. Immune thrombocytopenic purpura. eMedicine. http://emedicine.medscape.com/article/202158-overview. Updated December 28, 2010. Accessed February 15, 2011.

Cohen EW. Idiopathic thrombocytopenic purpura (ITP). Medical Encyclopedia, MEDLINEplus. Update Date: 3/28/2010. 3pp.
www.nlm.nih.gov/medlineplus/ency/article/000535.htm. Accessed February 15, 2011.

McKusick VA, ed. Online Mendelian Inheritance in Man (OMIM). The Johns Hopkins University. Thrombocytopenic Purpura, Autoimmune. Entry Number; 188030: Updated 3/20/2009. Accessed February 15, 2011.

National Cancer Institute/Cancer Drug Information. http://www.cancer.gov/cancertopics/druginfo/fda-romiplostim. Reviewed November 26, 2010. Accessed February 15, 2011.

The information in NORD’s Rare Disease Database is for educational purposes only. It should never be used for diagnostic or treatment purposes. If you have questions regarding a medical condition, always seek the advice of your physician or other qualified health professional. NORD’s reports provide a brief overview of rare diseases. For more specific information, we encourage you to contact your personal physician or the agencies listed as “Resources” on this report.

Report last updated: 2011/02/18 00:00:00 GMT+0

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