病例报告：一例对阿伐曲泊帕有反应的儿童持续性难治性免疫性血小板减少症。
Case Report: A case of pediatric persistent refractory ITP responsive to avatrombopag.
作者信息
Sau Antonella, Di Prinzio Gianfranco, Onofrillo Daniela, Russo Anna Caterina, Santoro Nicole, Di Ianni Mauro
机构信息
Department of Hematology and Oncology, Santo Spirito Hospital, Pescara, Italy.
Department of Medicine and Sciences of Aging, G. d'Annunzio University of Chieti and Pescara, Chieti, Italy.
出版信息
Front Immunol. 2025 Apr 15;16:1576053. doi: 10.3389/fimmu.2025.1576053. eCollection 2025.
Immune thrombocytopenia (ITP) is a rare hematologic disorder characterized by low platelet counts due to an immune-mediated destruction of platelets. While corticosteroids, intravenous immunoglobulin (IVIG) are the mainstays of treatment, a subset of patients may remain refractory to these therapies. Here, we present a case of a 6-year-old girl diagnosed with refractory ITP, who failed to respond to standard therapies but showed a remarkable clinical improvement with avatrombopag, a thrombopoietin receptor agonist.
免疫性血小板减少症（ITP）是一种罕见的血液系统疾病，其特征是由于免疫介导的血小板破坏导致血小板计数降低。虽然皮质类固醇、静脉注射免疫球蛋白（IVIG）是主要的治疗方法，但一部分患者可能对这些治疗仍无反应。在此，我们报告一例6岁女童诊断为难治性ITP的病例，她对标准治疗无反应，但使用血小板生成素受体激动剂阿伐曲泊帕后临床症状有显著改善。
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