Safety and Efficacy of Eltrombopag in Children and Adults with Immune Thrombocytopenia: A Systematic Review and Meta-Analysis

Abstract

: Immune thrombocytopenia is an immune condition where antibodies are produced against platelets. Eltrombopag is a thrombopoietin receptor agonist that stimulates and promotes platelet production approved for treating thrombocytopenia in patients with chronic immune thrombocytopenia, where other treatments as corticosteroids, splenectomy or immunoglobulins are inadequate. The aim of this meta-analysis was to evaluate the efficacy and safety of the eltrombopag in adults and children with immune thrombocytopenia. We included 7 studies with a total of 765 patients (606 adults and 159 children). We evaluated the number of patients that achieved a post treatment platelet count equal or above 50x109 /L (primary result-target) without the need of rescue treatment for at least 4 weeks. Our data showed that patients who received eltrombopag were almost 4 times more probable in achieving the primary target when compared to patients that received placebo (RR 3.84, 95% CI 2.39 to 6.14; I2 = 46%). The number of patients that needed rescue treatment and the number of bleeding incidents were reduced in the group that received eltrombopag when compared to those who received placebo (RR 0.40, 95% CI 0.25 to 0.62; I2 = 40%) (RR 0.74, 95% CI 0.62 to 0.89; I2 = 68%). The total number of side effects did not statistically differ between the two groups (RR 0.99, 95% CI 0.90 to 1.08; I2 = 14%). Our findings were similar to previously published studies and confirm that eltrombopag is safe and efficient in immune thrombocytopenia. However more clinical trials are needed in order to enhance our findings.

Eltrombopag for Adults and Children with Immune-Refractory Thrombocytopenic Purpura: A Systematic Review

“…In Brazil, its use is authorized for children > 6 years of age. Previous reviews have investigated the effectiveness of eltrombopag with refractory ITP: Massaro et al [ 6 ] investigated children and Wang et al [ 13 ], Elgebaly et al [ 14 ], and Kolanis et al [ 15 ] published reports on a mix of children and adults. However, these reviews have some methodologic flaws, including the lack of registration, lack of an appropriate assessment of the overall quality of evidence, or the lack of assessment of statistical heterogeneity for the meta-analysis.…”