The Cystic Fibrosis Foundation Patient Registry Design and Methods of a National Observational Disease Registry
The Cystic Fibrosis Foundation Patient Registry Design and Methods of a National Observational Disease Registry
复制标题
DOI：
10.1513/annalsats.201511-781oc
复制
发表时间：
2016-07-01
影响因子：
8.3
通讯作者：
Marshall, Bruce C.
中科院分区：
文献类型：
--
作者：
Knapp, Emily A.;Fink, Aliza K.;Marshall, Bruce C.
理由：囊性纤维化基金会患者登记处 (CFFPR) 是一项正在进行的患者登记研究，收集美国囊性纤维化 (CF) 患者的纵向人口统计、临床和治疗信息。 CF 是一种缩短寿命的遗传性疾病，在美国，大约每 3,500 名新生儿中就有 1 人患有 CF。高质量的观察数据对于临床研究、质量改进和临床管理非常重要。目标：描述 CFFPR 的数据收集、患者群体和主要局限性。方法：CFFPR 的纳入标准包括诊断为 CF 或 CFTR 相关疾病、在经认可的护理中心计划进行护理以及提供知情同意书。诊所就诊和住院的数据是通过安全网站收集的。使用多种方法检查了随访损失和普遍性。通过对 2012 年 CFFPR 数据与医疗记录进行比较来评估 CFFPR 数据的准确性。测量和主要结果：自 1986 年以来，CFFPR 包含 48,463 名 CF 患者的记录。在经认可的护理中心就诊的个人的参与度很高，而且失访率很低。对 2012 年 CFFPR 数据的审计表明，CFFPR 包含这些患者病历中 95% 的就诊情况和 90% 的住院情况，而且几乎所有审计字段都高度准确。结论：CFFPR 等登记系统是研究、临床护理以及跟踪发病率、死亡率和人口趋势的重要工具。
Rationale: The Cystic Fibrosis Foundation Patient Registry (CFFPR) is an ongoing patient registry study that collects longitudinal demographic, clinical, and treatment information about persons with cystic fibrosis (CF) in the United States. CF is a life-shortening genetic disorder that occurs in approximately 1 in 3,500 births in the United States. High-quality observational data is important for clinical research, quality improvement, and clinical management.Objectives: To describe the data collection, patient population, and key limitations of the CFFPR.Methods: Inclusion criteria for the CFFPR include diagnosis with CF or a CFTR-associated disorder, care at an accredited care center program, and provision of informed consent. Data from clinic visits and hospitalizations are collected through a secure website. Loss to follow-up and generalizability were examined using several methods. The accuracy of CFFPR data was evaluated with an audit of 2012 CFFPR data compared to the medical record.Measurements and Main Results: Since 1986, the CFFPR contains the records of 48,463 individuals with CF. Participation among individuals seen at accredited care centers is high, and loss to follow-up is low. An audit of 2012 CFFPR data suggests that the CFFPR contains 95% of clinic visits and 90% of hospitalizations found in the medical record for these patients, and nearly all of the audited fields were highly accurate.Conclusions: Registries such as the CFFPR are important tools for research, clinical care, and tracking incidence, mortality and population trends.