Epidemiologic study of cystic fibrosis: Design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosisin the US and Canada

Cystic fibrosis (CF) is a complex illness characterized by chronic lung inf ection leading to deterioration in function and respiratory failure in over 85% of patients. An understanding of the risk factors for that progression and the interaction of these factors with current therapeutic strategies s hould materially improve the prevention of this progressive lung disease. T he Epidemiologic Study of Cystic Fibrosis (ESCF) was therefore designed as a multicenter, longitudinal, observational study to prospectively collect d etailed clinical, therapeutic. microbiologic, and lung function data from a large number of CF treatment sites in the U.S. and Canada. The ESCF also s erves an important role as a phase-IV study of dornase alfa. To be eligible for enrollment, subjects must have the diagnosis of CF and receive the maj ority of their care at an ESCF site. In this paper. the authors present the ESCF study design in detail. Further , enrollment data collected at 194 study sites in 18,411 subjects enrolled from December 1, 1993 to December 31, 1995 are presented in summary form. T his comprehensive study is unique in the detail of clinical data collected regarding patient monitoring and therapeutic practices in CF care. Two comp anion articles present data regarding practice patterns in cystic fibrosis care, including data on resource utilization and prescribing practices. (C) 1999 Wiley-Liss, Inc.