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The role of caplacizumab in the treatment of thrombotic thrombocytopenic purpura remains controversial given its high cost and limited benefit, despite its inclusion in 2020 guidelines.

Careful monitoring of the ADAMTS-13 activity and inhibitor status and use of rituximab can prevent dangerous relapses.



  • Isolated thrombocytopenia (rule out pseudothrombocytopenia by review of peripheral smear).

  • Assess for any new causative medications and HIV, hepatitis B, hepatitis C, and Helicobacter pylori infections.

  • ITP is a diagnosis of exclusion.

General Considerations

ITP is an autoimmune condition in which pathogenic antibodies bind platelets, accelerating their clearance from the circulation. Growing evidence suggests additional pathophysiologic mechanisms, including a role for T cells. Many patients with ITP also lack appropriate compensatory platelet production, thought, at least in part, to reflect the antibody’s effect on megakaryocytopoiesis and thrombopoiesis. ITP is primary (idiopathic) in most adult patients, although it can be secondary (ie, associated with autoimmune disease, such as systemic lupus erythematosus [SLE]; lymphoproliferative disease, such as lymphoma; medications; and infections caused by hepatitis C virus, HIV, and H pylori). Antiplatelet antibody targets include glycoproteins IIb/IIIa and Ib/IX on the platelet membrane, although antibodies are demonstrable in only two-thirds of patients; testing for such antibodies is not standard of care given the significant false-positive and false-negative results. In addition to production of antiplatelet antibodies, HIV and hepatitis C virus may lead to thrombocytopenia through additional mechanisms (for instance, by direct suppression of platelet production [HIV] and cirrhosis-related decreased thrombopoietin [TPO] production and secondary splenomegaly [hepatitis C virus]).

Clinical Findings

A. Symptoms and Signs

Mucocutaneous bleeding may be present, depending on the platelet count. Clinically relevant spontaneous bruising, epistaxis, gingival bleeding, or other types of hemorrhage generally do not occur until the platelet count has fallen below 10,000–20,000/mcL (10–20 × 109/L). Individuals with secondary ITP (such as due to autoimmune disease, HIV or HCV infection, SLE, or lymphoproliferative malignancy) may have additional disease-specific findings.

B. Laboratory Findings

Typically, patients have isolated thrombocytopenia. If substantial bleeding has occurred, anemia may also be present. Hepatitis B and C viruses and HIV infections should be excluded by serologic testing. H pylori infections can sometimes cause isolated thrombocytopenia.

Bone marrow should be examined in patients with unexplained cytopenias in two or more lineages, in patients older than 40 years with isolated thrombocytopenia, or in those who do not respond to primary ITP-specific therapy. A bone marrow biopsy is not necessary in all cases to make an ITP diagnosis in younger patients. Megakaryocyte morphologic abnormalities and hypocellularity or hypercellularity are not characteristic of ITP. ITP patients often have increased numbers of bone marrow megakaryocytes. If there are clinical findings suggestive of a lymphoproliferative malignancy, a CT ...

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