TY - JOUR
T1 - Epidemiologic Study of Cystic Fibrosis
T2 - 25 years of observational research
AU - Scientific Advisory Group and the Investigators and Coordinators of ESCF
AU - Konstan, Michael W.
AU - Pasta, David J.
AU - VanDevanter, Donald R.
AU - Wagener, Jeffrey S.
AU - Morgan, Wayne J.
N1 - Funding Information:
The authors gratefully acknowledge the participation of the more 400 site investigators and coordinators of ESCF and thank the Cystic Fibrosis Foundation for the use of CFFPR genotype and mortality data. They also thank the patients and care providers throughout the United States and Canada for their contributions to ESCF and the CFFPR. The authors thank their coauthors and especially the North American Scientific Advisory Group members as well as the statistical analysts who have made these studies possible. Special thanks are due to Stefanie Millar from ICON Clinical Research for her more than 15 years of analytic contributions. Finally, the authors thank Genentech, Inc., who have generously supported ESCF from its inception. The authors acknowledge that coauthor Dr. Jeffrey S. Wagener, who died on October 28, 2018, contributed substantially to the writing of this manuscript including the near‐final draft before his death, and was thus included posthumously in the author list. The Epidemiologic Study of Cystic Fibrosis was funded by Genentech, Inc.
Funding Information:
The authors gratefully acknowledge the participation of the more 400 site investigators and coordinators of ESCF and thank the Cystic Fibrosis Foundation for the use of CFFPR genotype and mortality data. They also thank the patients and care providers throughout the United States and Canada for their contributions to ESCF and the CFFPR. The authors thank their coauthors and especially the North American Scientific Advisory Group members as well as the statistical analysts who have made these studies possible. Special thanks are due to Stefanie Millar from ICON Clinical Research for her more than 15 years of analytic contributions. Finally, the authors thank Genentech, Inc., who have generously supported ESCF from its inception. The authors acknowledge that coauthor Dr. Jeffrey S. Wagener, who died on October 28, 2018, contributed substantially to the writing of this manuscript including the near-final draft before his death, and was thus included posthumously in the author list. The Epidemiologic Study of Cystic Fibrosis was funded by Genentech, Inc.
Publisher Copyright:
© 2021 Wiley Periodicals LLC
PY - 2021/5
Y1 - 2021/5
N2 - 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.
AB - 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.
KW - cystic fibrosis
KW - epidemiology
KW - lung function
KW - pulmonary exacerbation
UR - http://www.scopus.com/inward/record.url?scp=85099393654&partnerID=8YFLogxK
UR - http://www.scopus.com/inward/citedby.url?scp=85099393654&partnerID=8YFLogxK
U2 - 10.1002/ppul.25248
DO - 10.1002/ppul.25248
M3 - Review article
C2 - 33434406
AN - SCOPUS:85099393654
SN - 8755-6863
VL - 56
SP - 823
EP - 836
JO - Pediatric pulmonology
JF - Pediatric pulmonology
IS - 5
ER -