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

Stephanie Davis, Felix Ratjen, Lyndia C. Brumback, Robin C. Johnson, Amy G. Filbrun, Gwendolyn S. Kerby, Howard B. Panitch, Scott H. Donaldson, Margaret Rosenfeld

Research output: Contribution to journalArticle

6 Citations (Scopus)

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.

Original languageEnglish (US)
JournalJournal of Cystic Fibrosis
DOIs
StateAccepted/In press - Jun 18 2015

Fingerprint

Respiratory Function Tests
Cystic Fibrosis
Multicenter Studies
Clinical Trials
Functional Residual Capacity

Keywords

  • FEV
  • Forced expiratory flow rates
  • Pulmonary function tests

ASJC Scopus subject areas

  • Pulmonary and Respiratory Medicine
  • Pediatrics, Perinatology, and Child Health

Cite this

Davis, S., Ratjen, F., Brumback, L. C., Johnson, R. C., Filbrun, A. G., Kerby, G. S., ... Rosenfeld, M. (Accepted/In press). Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis. Journal of Cystic Fibrosis. https://doi.org/10.1016/j.jcf.2015.10.007

Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis. / Davis, Stephanie; Ratjen, Felix; Brumback, Lyndia C.; Johnson, Robin C.; Filbrun, Amy G.; Kerby, Gwendolyn S.; Panitch, Howard B.; Donaldson, Scott H.; Rosenfeld, Margaret.

In: Journal of Cystic Fibrosis, 18.06.2015.

Research output: Contribution to journalArticle

Davis, S, Ratjen, F, Brumback, LC, Johnson, RC, Filbrun, AG, Kerby, GS, Panitch, HB, Donaldson, SH & Rosenfeld, M 2015, 'Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis', Journal of Cystic Fibrosis. https://doi.org/10.1016/j.jcf.2015.10.007
Davis, Stephanie ; Ratjen, Felix ; Brumback, Lyndia C. ; Johnson, Robin C. ; Filbrun, Amy G. ; Kerby, Gwendolyn S. ; Panitch, Howard B. ; Donaldson, Scott H. ; Rosenfeld, Margaret. / Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis. In: Journal of Cystic Fibrosis. 2015.
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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.",
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AU - Kerby, Gwendolyn S.

AU - Panitch, Howard B.

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AU - Rosenfeld, Margaret

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N2 - 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.

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