Department of Pediatrics, Case Western Reserve University School of Medicine, Cleveland, Ohio, USA.
UH Rainbow Babies and Children's Hospital, Cleveland, Ohio, USA.
Pediatr Pulmonol. 2021 May;56(5):823-836. doi: 10.1002/ppul.25248. Epub 2021 Jan 12.
The Epidemiologic Study of Cystic Fibrosis (ESCF) was a prospective observational study of over 32,000 people with cystic fibrosis (CF) from 250 clinical care sites in North America from 1994 to 2005. Begun as a pharmacovigilance study in connection with the approval of dornase alfa in 1993, ESCF was open to all people with CF treated at any participating site in the United States or Canada. In addition to obtaining safety and effectiveness data on dornase alfa, ESCF collected encounter-based data to characterize the natural history and management of CF with a special focus on lung disease. During the study, 32,178 patients reported at least one encounter, contributing 869,136 encounters, 622,592 pulmonary function tests, 432,896 cultures, and 118,563 pulmonary exacerbations treated with intravenous antibiotics. Although ESCF data collection concluded in 2005, through a collaboration with the U.S. Cystic Fibrosis Foundation Patient Registry, additional follow-up data through 2017 was available for two-thirds of patients. This allowed for updating of CF genotype and survival information. Fifty-six peer-reviewed publications (cited over 3600 times) resulted from this study. In this manuscript we summarize the published ESCF manuscripts in thematic groups with key study findings and brief comments, and speculate on how ESCF findings will inform future data registries and patient care practices.
囊性纤维化的流行病学研究（ESCF）是一项针对北美 250 个临床护理点的超过 32000 名囊性纤维化（CF）患者的前瞻性观察性研究，从 1994 年至 2005 年进行。该研究始于 1993 年多酶制剂（dornase alfa）获得批准后的药物警戒研究，向所有在美国或加拿大任何参与地点接受治疗的 CF 患者开放。除了获取多酶制剂（dornase alfa）的安全性和有效性数据外，ESCF 还收集了基于就诊的资料，以特别关注肺部疾病，来描述 CF 的自然史和管理。在研究期间，32178 名患者报告了至少一次就诊，共贡献了 869136 次就诊、622592 次肺功能测试、432896 次培养和 118563 次用静脉抗生素治疗的肺部恶化。尽管 2005 年结束了 ESCF 的数据收集，但通过与美国囊性纤维化基金会患者登记处的合作，三分之二的患者可以获得额外的随访数据至 2017 年。这使得 CF 基因型和生存信息得以更新。该研究产生了 56 篇经过同行评审的出版物（被引用超过 3600 次）。在本文中，我们按主题组总结了已发表的 ESCF 手稿，介绍了关键的研究结果和简要评论，并推测 ESCF 的研究结果将如何为未来的数据登记处和患者护理实践提供信息。
