Idiopathic Thrombocytopenic Purpura

Idiopathic thrombocytopenic purpura (ITP) is a bleeding disorder characterized by too few platelets in the blood. This is because platelets are being destroyed by the immune system. Symptoms may include bruising, nosebleed or bleeding in the mouth, bleeding into the skin, and abnormally heavy menstruation. With treatment, the chance of remission (a symptom-free period) is good. Rarely, ITP may become a chronic ailment in adults and reappear, even after remission.

The medication(s) listed below have been approved by the Food and Drug Administration (FDA) as orphan products for treatment of this condition.

Immune Globulin Injection (Human) 10% Caprylate/Chromatography Purified(Brand name: Gammaked) – Manufactured by Kedrion BiopharmaFDA-approved indication: The treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse, primary humoral immunodeficiency (PI) in patients 2 years of age and older, and idiopathic thrombocytopenic purpura (ITP) to raise platelet counts to prevent bleeding or to allow a patient with ITP to undergo surgery.

Human immunoglobulin (human)(Brand name: Gammaplex) – Manufactured by Bio Products Laboratory. FDA-approved indication: In adults for treatment of chronic immune thrombocytopenic purpura (ITP)

Immune globulin injection [human], 10% caprylate/chromatography purified(Brand name: Gamunex-C) – Manufactured by Grifols. FDA-approved indication: Treatment of chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment and for maintenance therapy to prevent relapse. Also for use for the treatment of primary immunodeficiency disease (PIDD) in patients 2 years of age and older and idiopathic thrombocytopenic purpura (ITP).

Romiplstim(Brand name: Nplate) – Manufactured by Amgen. FDA-approved indication: Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy.

Immune globulin intravenous (human), 10% liquid(Brand name: Privigen) – Manufactured by CSL Behring. FDA-approved indication: For the treatment of adults with chronic inflammatory demyelinating polyneuropathy (CIDP) to improve neuromuscular disability and impairment, however not studied for use longer than 6 months. Also for the treatment of patients age 15 years and older with chronic immune thrombocytopenic purpura (ITP) to raise platelet counts, and as replacement therapy for primary humoral immunodeficiency (PI).

Eltrombopag(Brand name: Promacta) – Manufactured by GlaxoSmithKline. FDA-approved indication: Treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Treatment of patients with severe aplastic anemia who have had an insufficient response to immunosuppressive therapy.

Rho (D) immunoglobulin intravenous (human)(Brand name: WinRho SD) – Manufactured by Baxter. FDA-approved indication: Treatment of adults and children with chronic and acute immune thrombocytopenic purpura.