Eltrombopag and high-dose dexamethasone as first-line treatment in children with newly diagnosed primary immune thrombocytopenia

Introduction: Novel treatment strategies for newly diagnosed immune thrombocytopenia (ndITP) pediatric patients are required. Material and methods: The aim of this study was to analyze the safety and efficacy of eltrombopag and dexamethasone when used as the first-line treatment in children with ndITP. Inclusion criteria: age 5–18 years, and ndITP with bleeding manifestation. Treatment course: 28 days of eltrombopag with oral dexamethasone in three repeated courses. Results: A complete response was achieved in 90% of patients after the first week of treatment, and in all patients after the end of the treatment course. Durable and sustained platelet response was observed in 90% of patients after 12 months of follow-up. Conclusions: Our finding support safety and efficacy of eltrombopag and dexamethasone as combined first-line therapy of ndITP in children.