Thrombopoetin receptor agonist therapy in thrombocytopenia: ITP and beyond

Abstract

Eltrombopag is well established in treatment of severe immune thrombocytopenia (ITP) and is increasingly commonplace in second-line management. A role is also suggested for both bridging therapy for surgery, as well as treating thrombocytopenia due to non-immune aetiologies. We present the largest single-centre experience with eltrombopag, with our cohort of 62 patients. Patients with severe ITP (n = 34) had 91·2% response, which was sustained over a median of 18·5 months. In 41·4% of ITP cases (n = 14), complete response (CR- platelet count >100 × 10 /l) was achieved and in 2 cases, therapy was stopped and CR maintained. In our bridging group (n = 15) with a higher baseline platelet count, 93·3% achieved a CR. In the non-ITP group (n = 13), a response was achieved in 76·9%. In all groups, side effects were transient, with the drug discontinued in 2 patients due to minor complications (rash, nausea, diarrhoea). We conclude that eltrombopag is both effective and well tolerated as therapy in severe ITP. It is also advantageous in ITP patients who do not normally require therapy, but need a temporary platelet count boost pre-procedure. Furthermore, there are potentially far wider implications for the use of eltrombopag in counteracting thrombocytopenia beyond ITP, which merit further investigation.

Updated international consensus report on the investigation and management of primary immune thrombocytopenia

“…Several case series reported successful use of eltrombopag or romiplostim as a "bridging" therapy to increase platelet counts prior to surgery or another procedure (evidence level III). [52][53][54] Consensusbased recommendations for patients with ITP undergoing surgery are presented in Table 6. and death in patients older than 60 years), [55][56][57][58] patients with other comorbidities, or those on anticoagulants.…”

Managing Preoperative Hemostasis in Patients with Inherited and Acquired Bleeding Disorders

“…45,46 If treatment is needed to achieve these levels, options include corticosteroids (prednisolone 0.5-1 mg/kg/d or dexamethasone 20-40 mg/d for 4 days), intravenous immunoglobulin 1 g/kg, and the thrombopoietin receptor agonists (TPO-RAs) romiplostim and eltrombopag. 47,48 High-dose methylprednisolone may also be useful in this setting. Intravenous tranexamic acid (TXA) can be used alone or as an adjunct to other treatment for both prevention and management of bleeding and platelet transfusions help with immediate control of bleeding in some cases, 49 although are unlikely to be useful for prevention due to their short survival in this setting.…”

Effect of Cytokine Gene Polymorphisms on Eltrombopag Reactivity in Japanese Patients with Immune Thrombocytopenia

“…47 Eltrombopag is a well-established treatment for ITP and is increasingly used in second-line management. 17,19,51,52 However, the relationship between eltrombopag and cytokine gene polymorphisms has yet been reported, which we addressed in the present study. A total of 62 patients were treated with eltrombopag; 32 patients responded and 30 did not.…”

“…10 These drugs contribute to improvements in the prognosis of refractory ITP. [17][18][19] It is known that autoimmune diseases have genetic factors. 20 As previously mentioned, several cytokines (interleukin [IL]-1, IL-2, IL-3 and IL-17) are very important in the pathophysiology of ITP.…”