Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents.

Background: Chronic ITP is a very common disease in children. Most treatment options have focused on slowing platelet destruction like corticosteroids and IVIG. These treatments are not always effective, or have only a transient effect, and treatment related adverse events often restrict further use. Impaired platelet production has been evolved as a potential disease mechanism for ITP. As a result, growth factor and growth factor analogue stimulation of megakaryopoiesis has also been investigated. Objective: is to evaluate the efficacy and safety of eltrombopag for the treatment of children and adolescents with chronic ITP. Subjects and methods: It was a prospective cohort study which was conducted on forty patients (22 males and 18 females) with chronic ITP. They were treated with eltrombopag for a period ranged from 6 months to 2 years after failure of first line of treatment (steroids, IVIG or both). Patients started eltrombopag with mean initial platelet count 14.4 X 103/ uL and were evaluated after at least 6 months of therapy. The study was approved by the academic and medical ethics committee of Zagazig University and a written informed consent was obtained from all patients. Results: Thirty patients (75%) responded to eltrombopag with a mean platelet count of 100.9 X103/ uL while 10 patients (25%) did not respond to the drug. Mean platelet count was significantly higher after eltrombopag therapy (100.9 ± 65.97 versus 14.4 ± 8.87, P < 0.001). The longer the duration of treatment, the higher the mean platelet count after treatment (r = 0.4, P < 0.001). Only 43.3% of responders received combined treatment with IVIG and steroids versus 20% of non-responders (p = 0.006). Conclusion: Eltrombopag is an effective, well tolerated and safe drug to restore platelet counts at levels high enough to prevent bleeding events in children with chronic ITP. [ABSTRACT FROM AUTHOR]