| Summary: | Eltrombopag is an agonist that binds to the membrane-bound domain of the thrombopoietin receptor used in immune thrombocytopenic purpura (ITP). We conducted a meta-analysis of randomized controlled trials to assess the efficacy and safety of eltrombopag in adults and children with refractory ITP. Adults who received eltrombopag had a significantly better platelet response (relative risk [RR], 3.65; 95% confidence interval [CI], 2.39–5.55), but there were no differences in the incidence of bleeding (RR, 0.8; 95% CI, 0.52–1.22) and adverse effects (RR, 0.99; 95% CI, 0.55–1.78) compared with the placebo. In children, there was no difference between eltrombopag and placebo for a platelet response >50,000/mm<sup>3</sup> (RR, 3.93; 95% CI, 0.56–27.79) and the number of adverse events (RR, 0.99; 95% CI, 0.25–1.49); however, a lower incidence of bleeding was observed (RR, 0.47; 95% CI, 0.27–0.83). Treatment with eltrombopag protected adults and children from severe disease and death.
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