艾曲泊帕治疗儿童免疫性血小板减少症的临床研究进展
Clinical research progress on eltrombopag in the treatment of children with immune thrombocytopenia
王南 1王志法 2王晓玲 3吴润晖 2成晓玲3
作者信息
- 1. 国家儿童医学中心,首都医科大学附属北京儿童医院 药学部,北京 100045;中国药科大学基础医学与临床药学学院,江苏 南京 211198
- 2. 国家儿童医学中心,首都医科大学附属北京儿童医院 血液二科,北京 100045
- 3. 国家儿童医学中心,首都医科大学附属北京儿童医院 药学部,北京 100045
- 折叠
摘要
艾曲泊帕(Eltrombopag)作为非肽类、小分子血小板生成素受体激动剂(Thrombopoietin receptor agonists,TPO-RAs),被美国食品药品监督管理局批准用于1 岁及以上儿童和成人免疫性血小板减少症(Immune thrombocytopenia,ITP)的治疗.作为在国内外ITP临床实践中应用最为广泛的TPO-RAs,近几年对艾曲泊帕在儿童ITP群体中的报道局限在个例患者的病例报告、小样本量群体的回顾性研究和短期应用的临床疗效报道等,其在儿童患者中长期使用的有效性和安全性缺乏报道.因此,本文从儿童临床试验研究和真实世界研究两个阶段入手,对艾曲泊帕治疗儿童ITP的临床效果进行综述,以期为艾曲泊帕在儿童ITP群体中的个体化与合理应用提供循证依据.
Abstract
Eltrombopag,as non-peptidic,micromolecular thrombopoietin receptor agonists(TPO-RAs),have been approved by FDA for immune thrombocytopenia(ITP)treatment in children being or over 1 year old and adults.As the most widely used TPO-RAs in domestic and international ITP clinical practice,recent reports of eltrombopag in the pediatric ITP population have been limited in case reports of individual patients,retrospective studies of small sam-ple size groups and case observations of short-term applications.There is a lack of reports on the efficacy and safety of long term use of eltrombopag in pediatric patients.Starting with two stages,clinical trial research and real-world re-search in children,we provide a comprehensive review of the efficacy and safety of eltrombopag in ITP children,in or-der to provide more evdidence-based basis for the individuation and rational application of eltrombopag in ITP children.
关键词
儿童/免疫性血小板减少症/艾曲泊帕/有效性/安全性Key words
Children/Immune thrombocytopenia(ITP)/Eltrombopag/Efficacy/Safety引用本文复制引用
基金项目
首都卫生发展科研专项(首发-2022-2Z-2099)
首都临床特色诊疗技术研究及转化应用(Z221100007422067)
北京市属医院科研培育计划(PX2022049)
出版年
2023
NETL
NSTL
维普
万方数据