The new Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry: design, rationale, and characteristics of patients enrolled in the first 12 months.
The new Childhood Arthritis and Rheumatology Research Alliance (CARRA) registry: design, rationale, and characteristics of patients enrolled in the first 12 months.
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DOI：
10.1186/s12969-017-0160-6
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发表时间：
2017-04-17
期刊：
影响因子：
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通讯作者：
CARRA Registry Investigators
中科院分区：
文献类型：
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作者：
Beukelman T;Kimura Y;Ilowite NT;Mieszkalski K;Natter MD;Burrell G;Best B;Jones J;Schanberg LE;CARRA Registry Investigators
在此，我们描述了新的儿童关节炎和流变学研究联盟（CARRA）登记的历史，设计和基本原理，并介绍了在手术前12个月登记的幼年特发性关节炎（JIA）患者的特征。CARRA登记研究于2015年7月开始在美国和加拿大前瞻性收集数据，以评估治疗药物在儿童期发作的风湿性疾病患者中的安全性，最初仅限于JIA。次要目的包括评估疾病结局及其与药物使用和其他因素的相关性。每6个月收集一次数据，包括临床评估、详细的药物使用、患者报告的结局和安全性事件。计划对每位参与者进行至少10年的随访，并通过电话呼叫中心提供便利。截至2016年7月，CARRA登记研究在49家临床试验机构招募了1192例JIA患者。入组时，他们的中位年龄为12.4岁，中位病程为2.6年。由于优先入组，与典型临床实践中的患者相比，全身性JIA患者（13%）和多关节病程患者（75%）的代表性过高。大约49%的患者目前正在使用生物制剂，口服糖皮质激素也很常见（47%）。CARRA注册中心为制药公司提供安全性监测数据，以满足其监管要求，目前正在进行几项使用注册中心基础设施的独立资助的子研究。新的CARRA登记研究在其运行的前12个月内成功招募了近1200名JIA参与者。从多个来源获得了可持续的资金。CARRA登记处可以作为其他罕见疾病研究的模型。
Herein we describe the history, design, and rationale of the new Childhood Arthritis and Rheumatology Research Alliance (CARRA) Registry and present the characteristics of patients with juvenile idiopathic arthritis (JIA) enrolled in the first 12 months of operation. The CARRA Registry began prospectively collecting data in the United States and Canada in July 2015 to evaluate the safety of therapeutic agents in persons with childhood-onset rheumatic disease, initially restricted to JIA. Secondary objectives include the evaluation of disease outcomes and their associations with medication use and other factors. Data are collected every 6 months and include clinical assessments, detailed medication use, patient-reported outcomes, and safety events. Follow-up is planned for at least 10 years for each participant and is facilitated by a telephone call center. As of July 2016, 1192 patients with JIA were enrolled in the CARRA Registry at 49 clinical sites. At enrollment, their median age was 12.4 years old and median disease duration was 2.6 years. Owing to preferential enrollment, patients with systemic JIA (13%) and with a polyarticular course (75%) were over-represented compared to patients in typical clinical practice. Approximately 49% were currently using biologic agents and ever use of oral glucocorticoids was common (47%). The CARRA Registry provides safety surveillance data to pharmaceutical companies to satisfy their regulatory requirements, and several independently-funded sub-studies that use the Registry infrastructure are underway. The new CARRA Registry successfully enrolled nearly 1200 participants with JIA in the first 12 months of its operation. Sustainable funding has been secured from multiple sources. The CARRA Registry may serve as a model for the study of other uncommon diseases.