在接受生物制剂或合成药物治疗的幼年特发性关节炎患者中的药物警戒：来自 Pharmachild 和国家登记处的超过 15000 名患者的合并数据。
Pharmacovigilance in juvenile idiopathic arthritis patients treated with biologic or synthetic drugs: combined data of more than 15,000 patients from Pharmachild and national registries.
机构信息
Department of Pediatric Immunology and Rheumatology, Wilhelmina Children's Hospital, Lundlaan, 6 PO box 85090, Utrecht, The Netherlands.
IRCCS Istituto Giannina Gaslini, Clinica Pediatrica e Reumatologia, PRINTO, Via Gaslini, 5, 16147, Genoa, Italy.
出版信息
Arthritis Res Ther. 2018 Dec 27;20(1):285. doi: 10.1186/s13075-018-1780-z.
BACKGROUND
The availability of methotrexate and the introduction of multiple biological agents have revolutionized the treatment of juvenile idiopathic arthritis (JIA). Several international and national drug registries have been implemented to accurately monitor the long-term safety/efficacy of these agents. This report aims to present the combined data coming from Pharmachild/PRINTO registry and the national registries from Germany (BiKeR) and Sweden.
METHODS
Descriptive statistics was used for demographic, clinical data, drug exposure, adverse events (AEs) and events of special interest (ESIs). For the Swedish register, AE data were not available.
RESULTS
Data from a total of 15,284 patients were reported: 8274 (54%) from the Pharmachild registry and 3990 (26%) and 3020 (20%) from the German and the Swedish registries, respectively. Pharmachild children showed a younger age (median of 5.4 versus 7.6 years) at JIA onset and shorter disease duration at last available visit (5.3 versus 6.1-6.8) when compared with the other registries. The most frequent JIA category was the rheumatoid factor-negative polyarthritis (range of 24.6-29.9%). Methotrexate (61-84%) and etanercept (24%-61.8%) were the most frequently used synthetic and biologic disease-modifying anti-rheumatic drugs (DMARDs), respectively. There was a wide variability in glucocorticoid use (16.7-42.1%). Serious AEs were present in 572 (6.9%) patients in Pharmachild versus 297 (7.4%) in BiKeR. Infection and infestations were the most frequent AEs (29.4-30.1%) followed by gastrointestinal disorders (11.5-19.6%). The most frequent ESIs were infections (75.3-89%).
CONCLUSIONS
This article is the first attempt to present a very large sample of data on JIA patients from different national and international registries and represents the first proposal for data merging as the most powerful tool for future analysis of safety and effectiveness of immunosuppressive therapies in JIA.
REGISTRY REGISTRATION
The Pharmachild registry is registered at ClinicalTrials.gov ( NCT01399281 ) and at the European Network of Centres for Pharmacoepidemiology and Pharmacovigilance (ENCePP) ( http://www.encepp.eu/encepp/viewResource.htm?id=19362 ). The BiKeR registry is registered at ENCePP ( http://www.encepp.eu/encepp/viewResource.htm?id=20591 ).
背景
甲氨蝶呤的出现和多种生物制剂的引入彻底改变了幼年特发性关节炎（JIA）的治疗方法。已经实施了几个国际和国家药物登记处，以准确监测这些药物的长期安全性/疗效。本报告旨在介绍 Pharmachild/PRINTO 登记处和德国（BiKeR）和瑞典国家登记处的数据。
方法
描述性统计用于人口统计学、临床数据、药物暴露、不良事件（AE）和特殊关注事件（ESI）。对于瑞典登记处，AE 数据不可用。
结果
共报告了 15284 名患者的数据：Pharmachild 登记处 8274 名（54%），德国登记处 3990 名（26%）和瑞典登记处 3020 名（20%）。Pharmachild 儿童在 JIA 发病时年龄更小（中位数为 5.4 岁，而其他登记处为 7.6 岁），在最后一次就诊时疾病持续时间更短（5.3 岁，而其他登记处为 6.1-6.8 岁）。最常见的 JIA 类别是类风湿因子阴性多关节炎（范围为 24.6-29.9%）。甲氨蝶呤（61-84%）和依那西普（24%-61.8%）是最常用的合成和生物性疾病修饰抗风湿药物（DMARDs）。糖皮质激素的使用差异很大（16.7-42.1%）。Pharmachild 中有 572 名（6.9%）患者出现严重不良事件，而 BiKeR 中有 297 名（7.4%）。感染和寄生虫感染是最常见的 AE（29.4-30.1%），其次是胃肠道疾病（11.5-19.6%）。最常见的 ESI 是感染（75.3-89%）。
结论
本文首次尝试从不同的国家和国际登记处提供大量 JIA 患者的数据，代表了数据合并的首次尝试，这是未来分析 JIA 免疫抑制治疗安全性和有效性的最有力工具。
登记处注册
Pharmachild 登记处在 ClinicalTrials.gov（NCT01399281）和欧洲药物流行病学和药物警戒网络（ENCePP）注册（http://www.encepp.eu/encepp/viewResource.htm?id=19362）。BiKeR 登记处在 ENeCPP 注册（http://www.encepp.eu/encepp/viewResource.htm?id=20591）。
Zotero 插件