Pathophysiology and management of chronic immune thrombocytopenia: focusing on what matters

Abstract

SummaryImmune thrombocytopenia (ITP) is a common autoimmune disease characterized by low platelet counts and an increased risk of bleeding. Antibody-mediated platelet destruction has been the prevailing hypothesis to explain ITP pathogenesis, supported by the efficacy of B-cell depletion therapy; however, the recent success of thrombopoietin receptor agonists lends support to the notion that platelet production is also insufficient. Best practice for the management of chronic ITP has not yet been established because data from comparative trials are lacking. Despite renewed interest in novel drugs capable of increasing platelet counts, ultimate treatment goals for ITP patients must be kept in mind: to improve patients' health and well-being. In this article, the pathophysiology of ITP is reviewed and key remaining questions about mechanism are explored. A rational approach to the management of ITP in adults is outlined, acknowledging evidence and evidence gaps, and highlighting the need for clinically important endpoints in future clinical trials.

Successful discontinuation of eltrombopag after complete remission in patients with primary immune thrombocytopenia

“…Primary immune thrombocytopenia (ITP) is an autoimmune disease characterized by increased destruction and impaired production of platelets caused by autoantibodies directed against the platelets and megakaryocytes and an increased risk of bleeding [1].…”

Eltrombopag and high-dose dexamethasone as frontline treatment of newly diagnosed immune thrombocytopenia in adults

“…1 A platelet count (Plt) ,30 3 10 9 /L generally indicates severe disease. Newly diagnosed ITP is considered as lasting up to 3 months, followed by persistent (3-12 months) and chronic disease (.12 months).…”

Splenectomy as a curative treatment for immune thrombocytopenia: a retrospective analysis of 233 patients with a minimum follow up of 10 years

“…6 Splenectomy has been shown to induce durable responses in a substantial proportion of patients, with an acceptable risk of complications, and was, therefore, long regarded as the gold standard for treatment of chronic ITP patients in need of second-line therapy. [2][3][4][5]7,8 However, the recent introduction of new drugs has offered new perspectives of treatment and has enhanced the generalized tendency to avoid or delay splenectomy. Rituximab has been proved to induce responses sustained at five years in approximately 20-30% of cases; its administration should be decided and managed with caution as severe infectious complications have been reported.…”

“…Circulating antibody-coated platelets are cleared by mononuclear phagocytic mass present in the spleen, liver and bone marrow. [2][3][4] Corticosteroids are the standard first-line therapy of ITP, although durable responses are achieved in only 20-30% of cases. 5 Second-line treatment of ITP patients (at high risk for bleeding or symptomatic) is controversial.…”