儿童使用艾曲泊帕的安全性分析: 揭示未见的药物不良反应信号

doi:10.5246/jcps.2025.11.077

中国药学(英文版) ›› 2025, Vol. 34 ›› Issue (11): 1033-1040.DOI: 10.5246/jcps.2025.11.077

儿童使用艾曲泊帕的安全性分析: 揭示未见的药物不良反应信号

涂颖秋¹, 胥甜甜¹, 钟楠², 赖鑫¹^,^*()

^1. 南昌大学第一附属医院药学部, 江西南昌 330006
^2. 南昌大学第一附属医院儿科, 江西南昌 330006

收稿日期:2025-08-21 修回日期:2025-09-12 接受日期:2025-09-27 出版日期:2025-12-02 发布日期:2025-12-02
通讯作者: 赖鑫

Eltrombopag in pediatrics: revealing hidden signals of adverse drug events

Yingqiu Tu¹, Tiantian Xu¹, Nan Zhong², Xin Lai¹^,^*()

¹ Department of Pharmacy, The First Affiliated Hospital of Nanchang University, Nanchang 330006, Jiangxi, China
² Pediatrics Department, The First Affiliated Hospital of Nanchang University, Nanchang 330006, Jiangxi, China

Received:2025-08-21 Revised:2025-09-12 Accepted:2025-09-27 Online:2025-12-02 Published:2025-12-02
Contact: Xin Lai

摘要/Abstract

摘要：

本研究旨在利用美国食品药品管理局不良事件报告系统(FAERS)数据库, 分析0–18岁儿童用药信号的不良事件(ADEs), 为临床药物安全性提供有价值的参考。本文检索了2004年第一季度至2023年第四季度0–18岁儿童使用FAERS的数据。采用报告优势比(ROR)和比例报告比(PRR)进行信号检测。ADEs根据监管活动医学词典(MedDRA)第25.0版中的系统器官类(SOC)进行分类。共检索到582份0–18岁儿童使用艾曲泊帕的ADE报告, 涉及21个SOC类别。在临床使用时, 不仅要注意常见的不良事件, 还要注意药品标签上未提及的不良事件。所提供的说明中未记录的药物不良事件信号包括血小板计数异常、血小板增多、抗磷脂综合征、骨髓纤维化、血铁水平降低、骨髓增生异常综合征、肝脏感染和其他相关情况。此外, 建议在临床使用艾曲泊帕期间, 定期监测血清铁和血铁蛋白水平, 特别是对接受长期治疗的患者, 以减轻患缺铁性贫血的风险。

关键词: 儿童, 超说明书用药, 艾曲泊帕, 信号挖掘, 药物不良事件

Abstract:

This study sought to investigate adverse drug event (ADE) signals associated with eltrombopag use in pediatric patients aged 0–18 years, utilizing data from the U.S. Food and Drug Administration Adverse Event Reporting System (FAERS). By analyzing this extensive pharmacovigilance database, the study aimed to offer meaningful insights for improving the clinical safety of eltrombopag in children. Data covering eltrombopag-related ADEs from Q1 2004 to Q4 2023 were extracted from FAERS, and signal detection was conducted using both the reporting odds ratio (ROR) and proportional reporting ratio (PRR) methods. ADEs were categorized based on the System Organ Class (SOC) classification in MedDRA version 25.0. A total of 582 reports involving pediatric patients receiving eltrombopag were identified, encompassing 21 SOC categories. The analysis revealed that, in addition to the known ADEs listed in the drug label, clinicians should remain vigilant for potential off-label ADE signals. These included abnormal platelet counts, thrombocytosis, antiphospholipid syndrome, myelofibrosis, reduced serum iron levels, myelodysplastic syndrome, hepatic infections, and other related conditions. Given these findings, it is strongly recommended that serum iron and ferritin levels should be routinely monitored in pediatric patients undergoing eltrombopag therapy, particularly during long-term treatment. Such proactive surveillance may help prevent the onset of iron deficiency anemia and enhance overall treatment safety.

Key words: Children, Off-label medication, Eltrombopag, Signal mining, Adverse drug events

Supporting:

涂颖秋, 胥甜甜, 钟楠, 赖鑫. 儿童使用艾曲泊帕的安全性分析: 揭示未见的药物不良反应信号[J]. 中国药学(英文版), 2025, 34(11): 1033-1040.

Yingqiu Tu, Tiantian Xu, Nan Zhong, Xin Lai. Eltrombopag in pediatrics: revealing hidden signals of adverse drug events[J]. Journal of Chinese Pharmaceutical Sciences, 2025, 34(11): 1033-1040.

图/表 8

Table 1. Proportional imbalance method table.

Table 2. Basic information of ADE reports for eltrombopag (n).

Figure 1. The 21 SOC Classifications of Eltrombopag.

Table 3. The 30 most frequently reported ADEs for eltrombopag.

Table 4. Aggregate ADE reporting rates for investigation associated with eltrombopag use in pediatric patients aged 0–18 years.

Table 5. Aggregate ADE reporting rates for general disorders and administration site conditions associated with eltrombopag use in pediatric patients aged 0–18 years.

Table 6. Aggregate ADE reporting rates for gastrointestinal disorders with eltrombopag use in pediatric patients aged 0–18 years.

Table 7. Incidence of PT associated with eltrombopag in children aged 0–1 year.

参考文献 13

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儿童使用艾曲泊帕的安全性分析: 揭示未见的药物不良反应信号

Eltrombopag in pediatrics: revealing hidden signals of adverse drug events

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