Hassan, T., Mohamed, M., Nada, S. (2019). Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents. The Egyptian Journal of Hospital Medicine, 77(2), 5068-5073. doi: 10.21608/ejhm.2019.49264
Tamer Hasan Hassan; Marwa Zakaria Mohamed; Sameh Nabil Khalil Nada. "Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents". The Egyptian Journal of Hospital Medicine, 77, 2, 2019, 5068-5073. doi: 10.21608/ejhm.2019.49264
Hassan, T., Mohamed, M., Nada, S. (2019). 'Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents', The Egyptian Journal of Hospital Medicine, 77(2), pp. 5068-5073. doi: 10.21608/ejhm.2019.49264
Hassan, T., Mohamed, M., Nada, S. Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents. The Egyptian Journal of Hospital Medicine, 2019; 77(2): 5068-5073. doi: 10.21608/ejhm.2019.49264
Efficacy and Safety of Eltrombopag for the Treatment of Chronic Idiopathic Thrombocytopenic Purpura in Children and Adolescents
Department of Pediatrics, Faculty of Medicine, Zagazig University
Abstract
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.