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 |
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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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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.</description><identifier>ISSN: 8755-6863</identifier><identifier>EISSN: 1099-0496</identifier><identifier>DOI: 10.1002/(SICI)1099-0496(199910)28:4<231::AID-PPUL1>3.0.CO;2-2</identifier><identifier>PMID: 10497371</identifier><identifier>CODEN: PEPUES</identifier><language>eng</language><publisher>New York: John Wiley & Sons, Inc</publisher><subject>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</subject><ispartof>Pediatric pulmonology, 1999-10, Vol.28 (4), p.231-241</ispartof><rights>Copyright © 1999 Wiley‐Liss, Inc.</rights><rights>1999 INIST-CNRS</rights><rights>Copyright 1999 Wiley-Liss, Inc.</rights><lds50>peer_reviewed</lds50><woscitedreferencessubscribed>false</woscitedreferencessubscribed><cites>FETCH-LOGICAL-c4331-f3af953c4be0f36552de51789fda0999ae1da74d90e5699669dc606ea03e09713</cites></display><links><openurl>$$Topenurl_article</openurl><openurlfulltext>$$Topenurlfull_article</openurlfulltext><thumbnail>$$Tsyndetics_thumb_exl</thumbnail><linktopdf>$$Uhttps://onlinelibrary.wiley.com/doi/pdf/10.1002%2F%28SICI%291099-0496%28199910%2928%3A4%3C231%3A%3AAID-PPUL1%3E3.0.CO%3B2-2$$EPDF$$P50$$Gwiley$$H</linktopdf><linktohtml>$$Uhttps://onlinelibrary.wiley.com/doi/full/10.1002%2F%28SICI%291099-0496%28199910%2928%3A4%3C231%3A%3AAID-PPUL1%3E3.0.CO%3B2-2$$EHTML$$P50$$Gwiley$$H</linktohtml><link.rule.ids>314,776,780,1411,27901,27902,45550,45551</link.rule.ids><backlink>$$Uhttp://pascal-francis.inist.fr/vibad/index.php?action=getRecordDetail&idt=1966603$$DView record in Pascal Francis$$Hfree_for_read</backlink><backlink>$$Uhttps://www.ncbi.nlm.nih.gov/pubmed/10497371$$D View this record in MEDLINE/PubMed$$Hfree_for_read</backlink></links><search><creatorcontrib>Morgan, Wayne J.</creatorcontrib><creatorcontrib>Butler, Steven M.</creatorcontrib><creatorcontrib>Johnson, Charles A.</creatorcontrib><creatorcontrib>Colin, Andrew A.</creatorcontrib><creatorcontrib>FitzSimmons, Stacey C.</creatorcontrib><creatorcontrib>Geller, David E.</creatorcontrib><creatorcontrib>Konstan, Michael W.</creatorcontrib><creatorcontrib>Light, Michael J.</creatorcontrib><creatorcontrib>Rabin, Harvey R.</creatorcontrib><creatorcontrib>Regelmann, Warren E.</creatorcontrib><creatorcontrib>Schidlow, Daniel V.</creatorcontrib><creatorcontrib>Stokes, Dennis C.</creatorcontrib><creatorcontrib>Wohl, Mary Ellen B.</creatorcontrib><creatorcontrib>Kaplowitz, Haley</creatorcontrib><creatorcontrib>Wyatt, Matthew M.</creatorcontrib><creatorcontrib>Scott Stryker MD, DRph, for the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis</creatorcontrib><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><title>Pediatric pulmonology</title><addtitle>Pediatr. 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. 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.</description><subject>Adolescent</subject><subject>Adult</subject><subject>Age Distribution</subject><subject>Biological and medical sciences</subject><subject>Canada - epidemiology</subject><subject>Child</subject><subject>Child, Preschool</subject><subject>cystic fibrosis</subject><subject>Cystic Fibrosis - diagnosis</subject><subject>Cystic Fibrosis - epidemiology</subject><subject>deoxyribonuclease I</subject><subject>Female</subject><subject>forced expiratory volume</subject><subject>Gastroenterology. Liver. Pancreas. Abdomen</subject><subject>growth disorders</subject><subject>human epidemiology</subject><subject>Humans</subject><subject>Incidence</subject><subject>Liver. Biliary tract. Portal circulation. Exocrine pancreas</subject><subject>Longitudinal Studies</subject><subject>lung</subject><subject>Male</subject><subject>Medical sciences</subject><subject>Middle Aged</subject><subject>Other diseases. 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 & 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. Liver. Pancreas. Abdomen</topic><topic>growth disorders</topic><topic>human epidemiology</topic><topic>Humans</topic><topic>Incidence</topic><topic>Liver. Biliary tract. Portal circulation. Exocrine pancreas</topic><topic>Longitudinal Studies</topic><topic>lung</topic><topic>Male</topic><topic>Medical sciences</topic><topic>Middle Aged</topic><topic>Other diseases. Semiology</topic><topic>phase-IV clinical trials</topic><topic>Prospective Studies</topic><topic>Pseudomonas</topic><topic>Risk Factors</topic><topic>Sex Distribution</topic><topic>Survival Rate</topic><topic>United States - epidemiology</topic><toplevel>peer_reviewed</toplevel><toplevel>online_resources</toplevel><creatorcontrib>Morgan, Wayne J.</creatorcontrib><creatorcontrib>Butler, Steven M.</creatorcontrib><creatorcontrib>Johnson, Charles A.</creatorcontrib><creatorcontrib>Colin, Andrew A.</creatorcontrib><creatorcontrib>FitzSimmons, Stacey C.</creatorcontrib><creatorcontrib>Geller, David E.</creatorcontrib><creatorcontrib>Konstan, Michael W.</creatorcontrib><creatorcontrib>Light, Michael J.</creatorcontrib><creatorcontrib>Rabin, Harvey R.</creatorcontrib><creatorcontrib>Regelmann, Warren E.</creatorcontrib><creatorcontrib>Schidlow, Daniel V.</creatorcontrib><creatorcontrib>Stokes, Dennis C.</creatorcontrib><creatorcontrib>Wohl, Mary Ellen B.</creatorcontrib><creatorcontrib>Kaplowitz, Haley</creatorcontrib><creatorcontrib>Wyatt, Matthew M.</creatorcontrib><creatorcontrib>Scott Stryker MD, DRph, for the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis</creatorcontrib><collection>Istex</collection><collection>Pascal-Francis</collection><collection>Medline</collection><collection>MEDLINE</collection><collection>MEDLINE (Ovid)</collection><collection>MEDLINE</collection><collection>MEDLINE</collection><collection>PubMed</collection><collection>CrossRef</collection><collection>MEDLINE - Academic</collection><jtitle>Pediatric pulmonology</jtitle></facets><delivery><delcategory>Remote Search Resource</delcategory><fulltext>fulltext</fulltext></delivery><addata><au>Morgan, Wayne J.</au><au>Butler, Steven M.</au><au>Johnson, Charles A.</au><au>Colin, Andrew A.</au><au>FitzSimmons, Stacey C.</au><au>Geller, David E.</au><au>Konstan, Michael W.</au><au>Light, Michael J.</au><au>Rabin, Harvey R.</au><au>Regelmann, Warren E.</au><au>Schidlow, Daniel V.</au><au>Stokes, Dennis C.</au><au>Wohl, Mary Ellen B.</au><au>Kaplowitz, Haley</au><au>Wyatt, Matthew M.</au><aucorp>Scott Stryker MD, DRph, for the Investigators and Coordinators of the Epidemiologic Study of Cystic Fibrosis</aucorp><format>journal</format><genre>article</genre><ristype>JOUR</ristype><atitle>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</atitle><jtitle>Pediatric pulmonology</jtitle><addtitle>Pediatr. Pulmonol</addtitle><date>1999-10</date><risdate>1999</risdate><volume>28</volume><issue>4</issue><spage>231</spage><epage>241</epage><pages>231-241</pages><issn>8755-6863</issn><eissn>1099-0496</eissn><coden>PEPUES</coden><abstract>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.</abstract><cop>New York</cop><pub>John Wiley & Sons, Inc</pub><pmid>10497371</pmid><doi>10.1002/(SICI)1099-0496(199910)28:4<231::AID-PPUL1>3.0.CO;2-2</doi><tpages>11</tpages></addata></record> |
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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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