La Revue de médecine interne
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Ten years after their licence in France, the use of the two thrombopoietin receptor agonists (TPO-RA), eltrombopag and romiplostim, has deeply modified the landscape of immune thrombocytopenia (ITP) treatment. In this review, we summarise data on efficacy and safety of these treatments during ITP, as well as their use in clinical practice. ⋯ However physician have to keep in mind that thromboembolism rates appear to be higher with TPO-RA treatment in ITP patients at high risk of thrombosis, and that data from "real-life" studies with very long term follow up are not available. Finally, the cost of these treatments should also be evaluated in future therapeutic strategies comparisons.
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New insights into immune thrombocytopenia (ITP) epidemiology in adult patients highlight three main outcomes of morbidity and mortality: bleeding, infection and thrombosis. This review depicts current evidence about incidence and risk factors of bleeding, infection and thrombosis as well as predictors of chronicity, and shows how this assessment impacts the choice of ITP second-line treatment at the individual-level basis.
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Immune thrombocytopenia (ITP) is a rare autoimmune disease due to an immune peripheral destruction of platelets and an inappropriate platelet production. The pathogenesis of ITP is now better understood: it involves a humoral immune response which dependents on the stimulation of B cells by specific T cells called T follicular helper cells, leading to their differentiation into plasma cells that produce antiplatelet antibodies thus promoting the phagocytosis of platelets mainly by splenic macrophages. The deciphering of ITP pathogenesis has led to a better understanding of the inefficiency of treatments such as rituximab, although it has not provided yet the determination of biological predictive factor of response to treatments. Moreover, new therapeutic perspectives have been opened in the last few years with the development of molecules targeting Fcγ receptor signalling such as Syk inhibitor, or molecules increasing the clearance of pathogenic autoantibodies such as inhibitors of the neonatal Fc receptor (FcRn).
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Randomized Controlled Trial
[Expectation of patients and caregivers about patient education for immune thrombocytopenia].
Immune thrombocytopenia (ITP) durably affects quality of life in patients. Patient education aims at improving their self-care and psychosocial skills, allowing them be more autonomous, to prevent avoidable complications, and to maintain or improve quality of life. The aim of this study was to assess patients' and caregivers' expectations regarding patient education in ITP. ⋯ These discrepancies emphasize the differences between patients and caregivers' expectations regarding a patient education program in ITP, and thus the relevance of patient-caregiver co-construction of such programs.