United Kingdom Cystic Fibrosis Gene Therapy Consortium Multidose Trial: The Proposed Use of Computed Tomography

Abstract

Listen

Translate article

Interest in the role of computed tomography (CT) as a monitoring tool in cystic fibrosis (CF) research has gained momentum in the past 5 years. This is a consequence of the rapid development of therapeutic options for CF lung disease and hence the need for sensitive outcome measures that can be applied to clinical intervention trials. The following article summarizes the proposed use of CT in a forthcoming multidose gene therapy trial. Issues surrounding dose, timing of scans, scoring, and quantification of CT morphologic features are discussed. The literature which supports the use of CT as a surrogate outcome measure is also summarized.