Journal Article

Outcomes of Eltrombopag Treatment and Development of Iron Deficiency in Children with Immune Thrombocytopenia in Turkey

Karaman, Serap, Akbayram, Sinan, Yılmaz, Şebnem, Gürlek Gökçebay, Dilek, Özbek, Namık Yaşar, Karakaş, Zeynep, Tokgöz, Hüseyin, Şaşmaz, İlgen, Ören, Hale, Özdemir, Gül Nihal, Yaralı, Neşe, Erduran, Erol, Bör, Özcan, Albayrak, Meryem, Söker, Murat, Koca Yozgat, Ayça, Malbora, Barış, Çınar Özel, Simge, Koçak, Ülker, Ünüvar, Ayşegül, Zengin, Emine, Leblebisatan, Göksel, Çakmaklı, Hasan Fatih, Ünal, Şule, Celkan, Tiraje, Oymak, Yeşim

2020

Overview

Immune thrombocytopenia (ITP) is a rare autoimmune disease and hematologic disorder characterized by reduced platelet counts that can result in significant symptoms, such as bleeding, bruising, epistaxis, or petechiae. The thrombopoietin receptor agonist eltrombopag (EPAG) is a second-line agent used to treat chronic ITP purpura in adults and children. The present retrospective study evaluated the efficacy, safety, and side effects of EPAG treatment in pediatric patients with acute refractory and chronic immune thrombocytopenia, particularly focusing on iron-deficiency anemia. The diagnosis was chronic ITP in 89 patients and acute refractory ITP in 16 patients. The mean age of patients was 9.5±4.5 years (minimum-maximum: 1.2-18 years) at the beginning of EPAG treatment. The overall response rate was 74.3% (n=78). The mean time for platelet count of ≥50x109/L was 11.6±8 weeks (range: 1-34 weeks). The treatment was stopped for 27 patients (25.7%) at an average of 6.8±9 months (range: 1-38 months). The reason for discontinuation was lack of response in 18 patients, nonadherence in 4 patients, and hepatotoxicity in 2 patients. Response to treatment continued for an average of 4 months after cessation of EPAG in 3 patients. Results of the current study imply that EPAG is an effective therapeutic option in pediatric patients with acute refractory and chronic ITP. However, patients must be closely monitored for response and side effects during treatment, and especially for iron deficiency.

• Publisher: Türk Hematoloji Derneği,Galenos Publishing House,Galenos Publishing,Turkish Society of Hematology
• Subject: Administration, Oral / Adolescent / Anemia, Iron-Deficiency - diagnosis / Anemia, Iron-Deficiency - drug therapy / Benzoates - administration & dosage / Benzoates - adverse effects / Benzoates - therapeutic use / Child / Child, Preschool / eltrombopag / Female / Humans / Hydrazines - administration & dosage / Hydrazines - adverse effects / Hydrazines - therapeutic use / immune thrombocytopenia / Infant / Iron / iron deficiency / Male / Pediatrics / Purpura, Thrombocytopenic, Idiopathic - diagnosis / Purpura, Thrombocytopenic, Idiopathic - drug therapy / Pyrazoles - administration & dosage / Pyrazoles - adverse effects / Pyrazoles - therapeutic use / Retrospective Studies / Thrombocytopenia / Tıp / Treatment Outcome / Turkey