Safety and Efficacy of Eltrombopag in Patients with Chronic Immune Thrombocytopenia: Meta-Analysis of Randomized Controlled Trials

Abstract

Background: Immune thrombocytopenia (ITP) is a complex autoimmune syndrome associated with low platelet count. Eltrombopag is an oral thrombopoietin receptor agonist that used in the treatment of chronic ITP. The aim: Present meta-analysis is to evaluate the safety and efficiency of Eltrombopag in the prevention and therapy of ITP Methods: Analysis was performed according to the PRISMA guideline with use of Excerpta Medica Database (EMBASE) as well as Web of Science and the Cochrane (CENTAL) databases.  Results: Seven randomized controlled trials (N = 766 patients) were included in the final analysis. Overall platelet response was significantly higher in the Eltrombopag group than in placebo (RR = 3.90; 95%CI [2.89-5.25]; P< 0.00001) showing mild heterogeneity (I² = 45%). Incidences of significant bleeding events in Eltrombopag group (World Health Organization [WHO] grades II-IV); (RR = 0.63; 95% CI: [0.47-0.85]; P = 0.003), showed lower heterogeneity (I² = 18%) in comparison to placebo group. Cases of use of rescue medications in Eltrombopag group compared to placebo group (RR = 0.40; 95% CI: [0.29-0.55]; P < 00001) in all considered studies showed low heterogeneity (I² = 41 %; P = 0.16). Incidences of any bleeding in Eltrombopag group compared to placebo group (RR = 0.77; 95% CI: [0.70-0.86]; P < .00001; I² = 65%), showed substantial heterogeneity. Finally, subgroup analysis of Eltrombopag efficiency revealed significant difference in frequency of bleeding cases between adults (RR= 0.84) and children (RR= 0.51); (P = 0.005). Conclusion: this systematic review presents class one evidence suggesting Eltrombopag as safe and effective drug for therapy of both children and adult patients with ITP.