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Eur J Haematol. 2019 Dec;103(6):531-541. doi: 10.1111/ejh.13319. Epub 2019 Sep 18.

Autoimmune thrombocytopenia: Current treatment options in adults with a focus on novel drugs.

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Copernicus Memorial Hospital, Lodz, Poland.
Department of Experimental Hematology, Medical University of Lodz, Lodz, Poland.
Department of Hematology, Medical University of Lodz, Lodz, Poland.


Immune thrombocytopenia (ITP) is an autoimmune disease characterized by platelet destruction and reduced platelet production resulting in decreased platelet level and an increased risk of bleeding. Based on the immunologic mechanism of ITP, front-line standard therapy consists of corticosteroids and intravenous immunoglobulins (IVIG). If patients do not respond to the first-line treatment, or if continuous therapy is required, the disorder is called refractory ITP, and second-line therapy is indicated. This treatment may consist of rituximab, thrombopoietin receptor agonists, splenectomy, or cytotoxic drugs. Despite significant advances, many patients do not respond to any the treatments listed below, and new treatment options need to be developed for this relapsed and refractory group. Recent clinical studies have indicated promising outcomes for novel drugs, either as single agents or in combination with traditional drugs. This review discusses the latest and the most promising novel drugs for ITP in adults.


avatrombopag; eltrombopag; immune thrombocytopenia; indirubin; receptor antagonist; rituximab; romiplostim; rozanolixizumab; rozrolimupab; thrombopoietin


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