Epidemiologic study of cystic fibrosis: Design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosis in the U.S. and Canada

Cystic fibrosis (CF) is a complex illness characterized by chronic lung infection 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...

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Veröffentlicht in:Pediatric pulmonology 1999-10, Vol.28 (4), p.231-241
Hauptverfasser: Morgan, Wayne J., Butler, Steven M., Johnson, Charles A., Colin, Andrew A., FitzSimmons, Stacey C., Geller, David E., Konstan, Michael W., Light, Michael J., Rabin, Harvey R., Regelmann, Warren E., Schidlow, Daniel V., Stokes, Dennis C., Wohl, Mary Ellen B., Kaplowitz, Haley, Wyatt, Matthew M.
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container_end_page 241
container_issue 4
container_start_page 231
container_title Pediatric pulmonology
container_volume 28
creator Morgan, Wayne J.
Butler, Steven M.
Johnson, Charles A.
Colin, Andrew A.
FitzSimmons, Stacey C.
Geller, David E.
Konstan, Michael W.
Light, Michael J.
Rabin, Harvey R.
Regelmann, Warren E.
Schidlow, Daniel V.
Stokes, Dennis C.
Wohl, Mary Ellen B.
Kaplowitz, Haley
Wyatt, Matthew M.
description Cystic fibrosis (CF) is a complex illness characterized by chronic lung infection 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 should materially improve the prevention of this progressive lung disease. The Epidemiologic Study of Cystic Fibrosis (ESCF) was therefore designed as a multicenter, longitudinal, observational study to prospectively collect detailed clinical, therapeutic, microbiologic, and lung function data from a large number of CF treatment sites in the U.S. and Canada. The ESCF also serves 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 majority 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. This comprehensive study is unique in the detail of clinical data collected regarding patient monitoring and therapeutic practices in CF care. Two companion articles present data regarding practice patterns in cystic fibrosis care, including data on resource utilization and prescribing practices. Pediatr Pulmonol. 1999; 28:231–241. © 1999 Wiley‐Liss, Inc.
doi_str_mv 10.1002/(SICI)1099-0496(199910)28:4<231::AID-PPUL1>3.0.CO;2-2
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An understanding of the risk factors for that progression and the interaction of these factors with current therapeutic strategies should materially improve the prevention of this progressive lung disease. The Epidemiologic Study of Cystic Fibrosis (ESCF) was therefore designed as a multicenter, longitudinal, observational study to prospectively collect detailed clinical, therapeutic, microbiologic, and lung function data from a large number of CF treatment sites in the U.S. and Canada. The ESCF also serves 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 majority 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. 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Pulmonol</addtitle><description>Cystic fibrosis (CF) is a complex illness characterized by chronic lung infection 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 should materially improve the prevention of this progressive lung disease. The Epidemiologic Study of Cystic Fibrosis (ESCF) was therefore designed as a multicenter, longitudinal, observational study to prospectively collect detailed clinical, therapeutic, microbiologic, and lung function data from a large number of CF treatment sites in the U.S. and Canada. The ESCF also serves 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 majority of their care at an ESCF site. In this paper, the authors present the ESCF study design in detail. 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Semiology</subject><subject>phase-IV clinical trials</subject><subject>Prospective Studies</subject><subject>Pseudomonas</subject><subject>Risk Factors</subject><subject>Sex Distribution</subject><subject>Survival Rate</subject><subject>United States - epidemiology</subject><issn>8755-6863</issn><issn>1099-0496</issn><fulltext>true</fulltext><rsrctype>article</rsrctype><creationdate>1999</creationdate><recordtype>article</recordtype><sourceid>EIF</sourceid><recordid>eNqFkV1v0zAUhiMEYqXwF5AvENqkpdhx4sTlQ5rSbVSqaKWuIHFz5CYnm0e-iNON_id-JE5TdQiQuIpy9Pg9r_04zntGR4xS783xchpPTxiV0qW-FMdMSsnoiReN_XceZ-Px2XTiLharGfvAR3QUz996rvfIGRxOPHYGURgErogEP3KeGXNLKe1CnjpHzAIhD9nA-Xle6xQLXeXVtU6IaTfpllQZSbamtf-ZXjeV0WZMJmj0dUlUmRJd1DkWWLaq1VXZ0YrUFqsxafUdnpJik9vDFsDmlFRrg83dDlX5w4LaTixhyL1ub_5cR3RJ2hskq9FytFsZq1Kl6rnzJFO5wRf779BZXZxfxR_d2fxyGp_N3MTnnLkZV5kMeOKvkWZcBIGXYsDCSGap6l5AIUtV6KeSYiCkFEKmiaACFeVIZcj40Hnd59pbfd-gaaHQJsE8VyVWGwMhDcOIsogfCiS2tmkwg7rRhWq2wCh0GgE6jdBJgU4K9BrBi8AHqxHAaoSdRuBAIZ6DZ-dD5-W-wGZdYPpbau_NAq_2gDKJyrNGlYk2D5y9lKDcYp977F7nuP2r3H-6_ataP7DBbh-sTYs_DsGq-QbCFgzgy6dLWC6CC39y9dWG_QLSRteA</recordid><startdate>199910</startdate><enddate>199910</enddate><creator>Morgan, Wayne J.</creator><creator>Butler, Steven M.</creator><creator>Johnson, Charles A.</creator><creator>Colin, Andrew A.</creator><creator>FitzSimmons, Stacey C.</creator><creator>Geller, David E.</creator><creator>Konstan, Michael W.</creator><creator>Light, Michael J.</creator><creator>Rabin, Harvey R.</creator><creator>Regelmann, Warren E.</creator><creator>Schidlow, Daniel V.</creator><creator>Stokes, Dennis C.</creator><creator>Wohl, Mary Ellen B.</creator><creator>Kaplowitz, Haley</creator><creator>Wyatt, Matthew M.</creator><general>John Wiley &amp; Sons, Inc</general><general>Wiley-Liss</general><scope>BSCLL</scope><scope>IQODW</scope><scope>CGR</scope><scope>CUY</scope><scope>CVF</scope><scope>ECM</scope><scope>EIF</scope><scope>NPM</scope><scope>AAYXX</scope><scope>CITATION</scope><scope>7X8</scope></search><sort><creationdate>199910</creationdate><title>Epidemiologic study of cystic fibrosis: Design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosis in the U.S. and Canada</title><author>Morgan, Wayne J. ; Butler, Steven M. ; Johnson, Charles A. ; Colin, Andrew A. ; FitzSimmons, Stacey C. ; Geller, David E. ; Konstan, Michael W. ; Light, Michael J. ; Rabin, Harvey R. ; Regelmann, Warren E. ; Schidlow, Daniel V. ; Stokes, Dennis C. ; Wohl, Mary Ellen B. ; Kaplowitz, Haley ; Wyatt, Matthew M.</author></sort><facets><frbrtype>5</frbrtype><frbrgroupid>cdi_FETCH-LOGICAL-c4331-f3af953c4be0f36552de51789fda0999ae1da74d90e5699669dc606ea03e09713</frbrgroupid><rsrctype>articles</rsrctype><prefilter>articles</prefilter><language>eng</language><creationdate>1999</creationdate><topic>Adolescent</topic><topic>Adult</topic><topic>Age Distribution</topic><topic>Biological and medical sciences</topic><topic>Canada - epidemiology</topic><topic>Child</topic><topic>Child, Preschool</topic><topic>cystic fibrosis</topic><topic>Cystic Fibrosis - diagnosis</topic><topic>Cystic Fibrosis - epidemiology</topic><topic>deoxyribonuclease I</topic><topic>Female</topic><topic>forced expiratory volume</topic><topic>Gastroenterology. 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subjects Adolescent
Adult
Age Distribution
Biological and medical sciences
Canada - epidemiology
Child
Child, Preschool
cystic fibrosis
Cystic Fibrosis - diagnosis
Cystic Fibrosis - epidemiology
deoxyribonuclease I
Female
forced expiratory volume
Gastroenterology. Liver. Pancreas. Abdomen
growth disorders
human epidemiology
Humans
Incidence
Liver. Biliary tract. Portal circulation. Exocrine pancreas
Longitudinal Studies
lung
Male
Medical sciences
Middle Aged
Other diseases. Semiology
phase-IV clinical trials
Prospective Studies
Pseudomonas
Risk Factors
Sex Distribution
Survival Rate
United States - epidemiology
title Epidemiologic study of cystic fibrosis: Design and implementation of a prospective, multicenter, observational study of patients with cystic fibrosis in the U.S. and Canada
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