Pathogenetic justification for the therapy of idiopathic thrombocytopenic purpura (primary immune thrombocytopenia) in adults

Abstract

The review presents current data on key mechanisms of the pathogenesis of idiopathic thrombocytopenic purpura and comparative characteristics of main therapy methods. In recent years, the interest in studying this long known disease has significantly increased, and basic approaches to diagnosis and treatment have been revised. Recognition of the importance of immune-mediated mechanism of development of this disease led to the replacement of the term used for many years «idiopathic thrombocytopenic purpura» to «immune thrombocytopenia». Moreover, development of hemorrhagic manifestations (purpura) is known to be characteristic not for all patients. The basis for the disease development is imbalance between the process of platelet production and destruction, as reflected in decrease of platelet production and increase of their elimination. Conventional treatment methods such as corticosteroids and splenectomy are directed at the suppression of a complex of cell interactions that lead to increased platelet destruction. Modern therapy for idiopathic thrombocytopenic purpura - thrombopoietin receptor agonists, on the contrary, stimulate the platelet production and are recommended for the use when loss or lack of response to previous therapy are observed. Most likely the efficacy of these drugs in resistant idiopathic thrombocytopenic purpura is associated with a fundamentally different, alternative mechanism of action. The idiopathic thrombocytopenic purpura group of patients is heterogeneous both in the character of the disease course and possible response to treatment. A limited number of clinical trials of some treatment methods for idiopathic thrombocytopenic purpura and differing criteria for assessing the response to therapy complicate their direct comparison. The imperfection of certain treatment options, due to development of adverse events, and unpredictability of response to treatment necessitate the search for new approaches to the selection of the optimal variant of treatment of idiopathic thrombocytopenic purpura taking into account the individual characteristics of patients.

Keywords

immune thrombocytopenia / megakaryocytopoiesis / thrombopoietin / line of therapy

RIGHTS & PERMISSIONS

Zotova I.I., Gritsaev S.V.