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Immune thrombocytopenia (ITP) is an acquired disorder characterized by immune-mediated thrombocytopenia. ITP with a duration of less than three months from onset is classified as newly diagnosed ITP (ND-ITP). First-line treatment involves corticosteroids and intravenous immunoglobulin (IVIG). Thrombopoietin receptor agonists (TPO-RAs) are therapeutic options for patients unresponsive to these initial treatments. However, reports on the use of TPO-RAs in pediatric patients are limited, and their efficacy and optimal administration strategies during the acute phase of ND-ITP remain unclear. We report a 10-year-old girl with ND-ITP who showed no response to corticosteroids or IVIG in the acute phase. Eltrombopag was introduced and subsequently increased in dosage, resulting in rapid platelet recovery and discharge without any major bleeding complications. This case suggests that the early use and dose escalation of eltrombopag may be an effective treatment approach even in the acute phase of pediatric ND-ITP. Further accumulation of clinical experience is essential to establish optimal dosing strategies for eltrombopag in acute settings.
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Morikawa et al. (2025) studied this question.
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