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Expert Rev Hematol. 2011 Feb;4(1):107-18. doi: 10.1586/ehm.10.76.

Current treatment options for primary immune thrombocytopenia.

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  • 1Institut für Transfusionsmedizin, Charité-Universitätsmedizin Berlin, Augustenburger Platz 1, D-13353 Berlin, Germany.


Traditional treatment of primary (idiopathic) immune thrombocytopenia (ITP) predominantly consists of immune suppression and/or modulation. In addition, many treated patients develop severe adverse effects, and approximately a third of patients do not respond. Two of the newly developed thrombopoietin-receptor agonists, romiplostim and eltrombopag, are now available for the treatment of ITP. Both drugs have been shown to increase the production of platelets in a dose-dependent manner, and to compensate, at least partly, for thrombocytopenia in the majority of ITP patients. The reported adverse effects are predominantly mild, although serious and long-term side effects cannot be excluded. Nevertheless, these drugs are increasingly used in the treatment of patients with thrombocytopenias. Thrombopoietin-receptor agonists do not appear to stop either the production of autoantibodies or the accelerated platelet destruction observed in ITP. Thus, the need for a specific therapy is essential, and the ultimate solution is to clarify and halt the mechanism(s) that lead to the development of ITP.

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