Mori Makiko
Department of Hematology/Oncology, Saitama Children's Medical Center.
Rinsho Ketsueki. 2024;65(9):1209-1215. doi: 10.11406/rinketsu.65.1209.
The new guidelines for pediatric immune thrombocytopenia (ITP) not only include changes to the name and staging of the disease, but also introduce the modified Buchanan's bleeding score for the assessment of bleeding symptoms. Treatments should aim to improve patients' health-related quality of life (HRQoL) based on a multidimensional assessment of not only platelet counts but also bleeding symptoms, as well as activity level, lifestyle, and access to healthcare. First-line therapy includes intravenous immunoglobulin therapy (IVIG) and short-term corticosteroids. Second-line therapy includes thrombopoietin receptor agonists, rituximab, and splenectomy. Many novel agents are also in development, with splenic-derived tyrosine kinase (Syk), Bruton's kinase (BTK), and fetal Fc receptor (FcRn) attracting attention as target molecules. Future developments in the treatment of pediatric ITP are eagerly awaited.
儿童免疫性血小板减少症（ITP）的新指南不仅包括疾病名称和分期的变化，还引入了改良的布坎南出血评分以评估出血症状。治疗应旨在基于对血小板计数、出血症状、活动水平、生活方式以及医疗保健可及性的多维度评估，改善患者的健康相关生活质量（HRQoL）。一线治疗包括静脉注射免疫球蛋白疗法（IVIG）和短期使用皮质类固醇。二线治疗包括血小板生成素受体激动剂、利妥昔单抗和脾切除术。许多新型药物也在研发中，脾源性酪氨酸激酶（Syk）、布鲁顿激酶（BTK）和胎儿Fc受体（FcRn）作为靶分子备受关注。人们热切期待儿童ITP治疗的未来发展。
