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J Cyst Fibros. 2017 May;16(3):318-326. doi: 10.1016/j.jcf.2017.01.002. Epub 2017 Jan 20.

Use of FEV1 in cystic fibrosis epidemiologic studies and clinical trials: A statistical perspective for the clinical researcher.

Author information

1
Division of Biostatistics & Epidemiology, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA; Division of Pulmonary Medicine, Cincinnati Children's Hospital Medical Center, Cincinnati, OH, USA. Electronic address: Rhonda.Szczesniak@cchmc.org.
2
Department of Pediatrics, University of Washington, Seattle, WA, USA; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, USA.
3
Division of Respiratory Medicine, Hospital for Sick Children, Toronto, ON, Canada.
4
Department of Pediatrics, University of Washington, Seattle, WA, USA; Cystic Fibrosis Foundation Therapeutics Development Network Coordinating Center, Seattle Children's Research Institute, Seattle, WA, USA; Department of Biostatistics, University of Washington, Seattle, WA, USA.

Abstract

BACKGROUND:

Forced expiratory volume in 1s (FEV1) is an established marker of cystic fibrosis (CF) disease progression that is used to capture clinical course and evaluate therapeutic efficacy. The research community has established FEV1 surveillance data through a variety of observational data sources such as patient registries, and there is a growing pipeline of new CF therapies demonstrated to be efficacious in clinical trials by establishing improvements in FEV1.

RESULTS:

In this review, we summarize from a statistical perspective the clinical relevance of FEV1 based on its association with morbidity and mortality in CF, its role in epidemiologic studies of disease progression and comparative effectiveness, and its utility in clinical trials. In addition, we identify opportunities to advance epidemiologic research and the clinical development pipeline through further statistical considerations.

CONCLUSIONS:

Our understanding of CF disease course, therapeutics, and clinical care has evolved immensely in the past decades, in large part due to the thoughtful application of rigorous research methods and meaningful clinical endpoints such as FEV1. A continued commitment to conduct research that minimizes the potential for bias, maximizes the limited patient population, and harmonizes approaches to FEV1 analysis while maintaining clinical relevance, will facilitate further opportunities to advance CF care.

KEYWORDS:

Disease progression; FEV(1) endpoints; Longitudinal; Lung function; Spirometry

PMID:
28117136
PMCID:
PMC5420487
DOI:
10.1016/j.jcf.2017.01.002
[Indexed for MEDLINE]
Free PMC Article

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