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

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。

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 sample 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 research in children,we provide a comprehensive review of the efficacy and safety of eltrombopag in ITP children,in order to provide more evdidence-based basis for the individuation and rational application of eltrombopag in ITP children.