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Endpoints for Clinical Trials in Young Children with Cystic Fibrosis

¹ Division of Pulmonology, Department of Pediatrics, University of North Carolina at Chapel Hill, Chapel Hill, North Carolina; ² Department of Radiology, Cincinnati Children's Hospital, Cincinnati, Ohio; ³ Cystic Fibrosis Therapeutic Development Network Coordinating Center and Department of Biostatistics, University of Washington, Seattle, Washington; and ⁴ Division of Pulmonary Medicine, Children's Hospital and Regional Medical Center and Department of Pediatrics, University of Washington, Seattle, Washington

Correspondence and requests for reprints should be addressed to Stephanie D. Davis, M.D., Division of Pediatric Pulmonology, 130 Mason Farm Road, Campus Box 7220, Chapel Hill, NC 27599-7220. E-mail: sddavis{at}med.unc.edu

ABSTRACT

The availability of sensitive, reproducible, and feasible outcome measures for quantifying lung disease in children with cystic fibrosis (CF) younger than 6 years is critical to the conduct of clinical trials in this important population. Historically, identifying and quantifying the presence of lung disease in very young children with CF was hampered by a lack of reproducible measures of lung function or lung pathology. Over the past 10 years, significant progress has led to physiologic, anatomic, and bronchoscopic measures that may serve as endpoints for future intervention trials. These endpoints include infant and preschool lung function testing, computed tomography of the chest, and bronchoalveolar lavage markers of inflammation and infection. Much progress has occurred in standardizing lung function testing, which is essential for multicenter collaboration. Pulmonary exacerbation has the potential to serve as a clinical endpoint; however, there is currently no standardized definition in children with CF younger than 6 years. Further development of these outcomes measures will enable clinical trials in the youngest CF population with the objective of improving long-term prognosis.

Key Words: infant • child, preschool • respiratory function tests, computed tomography scanners, X-ray • bronchoalveolar lavage

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