Immune thrombocytopenia

Immune thrombocytopenia (ITP), formerly known as idiopathic thrombocytopenic purpura, is an acquired autoimmune disorder characterized by isolated low platelet counts (thrombocytopenia) resulting from immune-mediated destruction of platelets and impaired platelet production. In ITP, the immune system produces autoantibodies—primarily directed against platelet surface glycoproteins such as GPIIb/IIIa and GPIb/IX—that target platelets for premature destruction in the spleen and liver. Additionally, these autoantibodies can impair megakaryocyte function in the bone marrow, reducing platelet production. The condition primarily affects the hematologic system but can have secondary effects on any organ system due to bleeding risk. Key symptoms include easy bruising (ecchymoses), petechiae (small red or purple spots on the skin), purpura, mucosal bleeding such as nosebleeds (epistaxis) and gum bleeding, heavy menstrual periods (menorrhagia), and in severe cases, gastrointestinal or intracranial hemorrhage. Many patients with mild thrombocytopenia may be asymptomatic. ITP can be classified as primary (no identifiable underlying cause) or secondary (associated with other conditions such as autoimmune diseases, infections, or lymphoproliferative disorders). It is further categorized by duration: newly diagnosed (up to 3 months), persistent (3–12 months), and chronic (lasting more than 12 months). ITP can occur in both children and adults. In children, it often follows a viral infection and frequently resolves spontaneously within weeks to months. In adults, the course tends to be more chronic. Treatment depends on severity and bleeding risk. First-line therapies include corticosteroids (such as prednisone or dexamethasone) and intravenous immunoglobulin (IVIG). For patients who do not respond to initial treatment, second-line options include thrombopoietin receptor agonists (such as romiplostim and eltrombopag), the anti-CD20 monoclonal antibody rituximab, the spleen tyrosine kinase inhibitor fostamatinib, and splenectomy. Emergency treatment for life-threatening bleeding may involve platelet transfusions combined with IVIG and high-dose corticosteroids.

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

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