Idiopathic Thrombocytopenic Purpura Medication

Updated: Jul 22, 2016
  • Author: Michael A Silverman, MD, MD; Chief Editor: Gil Z Shlamovitz, MD, FACEP  more...
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Medication

Medication Summary

Glucocorticoids and intravenous immunoglobulin (IVIg) are the mainstays of medical therapy for idiopathic thrombocytopenic purpura (ITP). Indications for use, dosage, and route of administration are based on the patient's clinical condition, the absolute platelet count, and the degree of symptoms. Consultation with a hematologist may be needed prior to starting therapy.

In children with ITP who have no bleeding or mild bleeding (eg, cutaneous manifestations such as bruising and petechiae), the American College of Hematology (ACH) recommends management with observation alone, regardless of the platelet count. [3] A retrospective review by Schultz et al found that this approach did not lead to an increase in later treatment or an increase in delayed bleeding symptoms. [4]

Adults with platelet counts >50,000/mm3 do not require treatment. Treatment is indicated for adults with counts < 50,000/mm3 with significant mucous membrane bleeding. Treatment also is indicated for those adults with risk factors for bleeding (eg, hypertension, peptic ulcer disease, vigorous lifestyle) and in patients with a platelet count < 20,000-30,000/mm3.

IV Rho immunoglobulin (RhIG) is generally less toxic than IV steroids, but is more expensive, and has been associated with acute intravascular hemolysis, with an estimated incidence of one in 1115 patients. The ACH advises against the use of IV RhIG in splenectomized children, in those with a hemoglobin concentration that is decreased because of bleeding, or in those with evidence of autoimmune hemolysis. However, the ACH suggests that a single dose of IV RhIG can be used as first-line treatment in Rh-positive, nonsplenectomized children who require treatment. [3]

Steroid use and immunosuppressives and splenectomy may be undesirable because of their associated complications. For long-term steroid use, this includes osteoporosis, glaucoma, cataracts, loss of muscle mass, and an increased risk of infection. For immunosuppressive therapy and splenectomy, risks include worsening immunosuppression and infection or sepsis. Studies of the use of multiagent therapies in refractory patients are ongoing. Some small studies have shown limited success. According to one study [5] , using a combination of weekly vincristine, weekly methylprednisolone, both until platelet counts reached 50,000/mm3, and cyclosporine orally twice daily until the platelet count is normal for 3-6 months seems promising, though larger prospective studies are needed.

Other therapies, such as cyclophosphamide, danazol, dapsone, interferon alfa, azathioprine, vinca alkaloids, accessory splenectomy, and splenic radiation have been studied. Many case series discussing these treatments are too small to show sufficient evidence of a clinically significant reduction in bleeding or mortality rate; however, they serve as additional therapeutic measures in ITP refractory-to-primary therapy (eg, glucocorticoids, IVIg immunoglobulin, splenectomy). Newer studies on rituximab suggest that this agent is an effective treatment option in splenectomized refractory or relapsed ITP patients. [6, 7]

Clinical trials have shown promise for agents that directly stimulate platelet production, such as thrombopoietin (TPO) receptor-binding agents. Two new agents, eltrombopag and romiplostim, are available to patients with chronic ITP who have failed other therapies. [8, 9] Both of these agents require registration in a database. While they show promise for raising platelet counts, there are potential safety concerns such as thrombocytosis and rebound thrombocytopenia. It is unlikely that emergency physicians should be prescribing these agents without being under the recommendation of a hematologist.

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Glucocorticoids

Class Summary

These agents are used to treat idiopathic and acquired autoimmune disorders. They have been shown to increase platelet count in ITP.

Prednisone (Deltasone, Orasone, Sterapred)

Useful in treating inflammatory and allergic reactions; may decrease inflammation by reversing increased capillary permeability and suppressing PMN activity. DOC for all adult patients with platelet counts < 50,000/mm3. Asymptomatic patients with platelet counts >20,000/mm3, or patients with counts 30,000-50,000/mm3 with only minor purpura, may not need therapy; withholding medical therapy may be appropriate for asymptomatic patients, regardless of count.

Methylprednisolone (Solu-Medrol, Depo-Medrol)

Decreases inflammation by suppressing migration of polymorphonuclear leukocytes and reversing increased permeability. Used as alternative glucocorticoid of choice for all patients with severe, life-threatening bleeding or children with platelet counts < 30,000/mm3. Careful observation without medical treatment may be appropriate in some asymptomatic children.

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Blood products

Class Summary

Administration of IVIg may temporarily increase platelet counts in some children and adults with ITP. Consider IVIg if the situation requires a rapid, temporary rise in platelet count.

Intravenous immune globulin (IVIg)

DOC for severe, life-threatening bleeding or for children with platelet counts < 20,000/mm3 with minor purpura; can be used alone or in addition to glucocorticoid therapy.

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Thrombopoietic Agent

Class Summary

These agents directly stimulates bone marrow platelet production. [10]

Eltrombopag (Promacta)

Oral thrombopoietin (TPO) receptor agonist. Interacts with transmembrane domain of human TPO receptor and induces megakaryocyte proliferation and differentiation from bone marrow progenitor cells. Indicated for thrombocytopenia associated with chronic idiopathic thrombocytopenic purpura in patients experiencing inadequate response to corticosteroids, immunoglobulins, or splenectomy. Not for use to normalize platelet counts but used when clinical condition increases bleeding risk.

Prescribers must enroll in Promacta Cares program. Only available through restricted distribution program. Program phone number is (877) 9-PROMACTA (877-977-6622).

Romiplostim (Nplate)

An Fc-peptide fusion protein (peptibody) that increases platelet production through binding and activation of the thrombopoietin (TPO) receptor, a mechanism similar to endogenous TPO. Indicated for chronic immune (idiopathic) thrombocytopenic purpura in patients who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Only available through the Nplate NEXUS (Network of Experts Understanding and Supporting Nplate) program, a program designed to promote informed risk-benefit decisions before initiating treatment. For more information, see www.nplate.com or call (877) NPLATE1 (877-675-2831).

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