Skip to main page content
U.S. flag

An official website of the United States government

Dot gov

The .gov means it’s official.
Federal government websites often end in .gov or .mil. Before sharing sensitive information, make sure you’re on a federal government site.

Https

The site is secure.
The https:// ensures that you are connecting to the official website and that any information you provide is encrypted and transmitted securely.

Access keys NCBI Homepage MyNCBI Homepage Main Content Main Navigation
Randomized Controlled Trial
. 2016 May;15(3):386-91.
doi: 10.1016/j.jcf.2015.10.007. Epub 2015 Nov 4.

Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis

Stephanie D Davis  1 , Felix Ratjen  2 , Lyndia C Brumback  3 , Robin C Johnson  4 , Amy G Filbrun  5 , Gwendolyn S Kerby  6 , Howard B Panitch  7 , Scott H Donaldson  8 , Margaret Rosenfeld  9 ; ISIS Study Group
Affiliations
Randomized Controlled Trial

Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis

Stephanie D Davis et al. J Cyst Fibros. 2016 May.

Abstract

Background: The Infant Study of Inhaled Saline (ISIS) in CF was the first multicenter clinical trial to utilize infant pulmonary function tests (iPFTs) as an endpoint.

Methods: Secondary analysis of ISIS data was conducted in order to assess feasibility of iPFT measures and their associations with respiratory symptoms. Standard deviations were calculated to aid in power calculations for future clinical trials.

Results: Seventy-three participants enrolled, 70 returned for the final visit; 62 (89%) and 45 (64%) had acceptable paired functional residual capacity (FRC) and raised volume measurements, respectively. Mean baseline FEV0.5, FEF75 and FRC z-scores were 0.3 (SD: 1.2), -0.2 (SD: 2.0), and 1.8 (SD: 2.0).

Conclusions: iPFTs are not appropriate primary endpoints for multicenter clinical trials due to challenges of obtaining acceptable data and near-normal average raised volume measurements. Raised volume measures have potential to serve as secondary endpoints in future clinical CF trials.

Keywords: FEV; Forced expiratory flow rates; Pulmonary function tests.

PubMed Disclaimer

References

    1. Sly PD, Brennan S, Gangell C, de Klerk N, Murray C, Mott L, Stick SM, Robinson PJ, Robertson CF, Ranganathan SC. Lung disease at diagnosis in infants with cystic fibrosis detected by newborn screening. Am J Respir Crit Care Med. 2009;180:146–152. - PubMed
    1. Sly PD, Gangell CL, Chen L, Ware RS, Ranganathan S, Mott LS, Murray CP, Stick SM. Risk factors for bronchiectasis in children with cystic fibrosis. The New England journal of medicine. 2013;368:1963–1970. - PubMed
    1. Ranganathan SC, Stocks J, Dezateux C, Bush A, Wade A, Carr S, Castle R, Dinwiddie R, Hoo AF, Lum S, Price J, Stroobant J, Wallis C. The evolution of airway function in early childhood following clinical diagnosis of cystic fibrosis. Am J Respir Crit Care Med. 2004;169:928–933. - PubMed
    1. Linnane BMHG, Nolan G, Brennan S, Stick SM, Sly PD, Robertson CF, Robinson PJ, Franklin PJ, Turner SW, et al. Lung function in infants with cystic fibrosis diagnosed by newborn screening. Am J Respir Crit Care Med. 2008 - PubMed
    1. Pillarisetti NWE, Linnane B, Skoric B, Robertson CF, Robinson P, Massie J, Hall GL, Sly P, Stick S, Ranganathan S. Infection, inflammation, and lung function decline in infants with cystic fibrosis. Am J Respir Crit Care Med. 2011;184:75–81. - PubMed

Publication types

Substances