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

Stephanie D. Davis; Felix Ratjen; Lyndia C. Brumback; Robin C. Johnson; Amy G. Filbrun; Gwendolyn S. Kerby; Howard B. Panitch; Scott H. Donaldson; Margaret Rosenfeld; Stephanie Davis; Albert Faro; Amy Filbrun; Peter Hiatt; Gwendolyn Kerby; Karen McCoy; Karen McDowell; James Acton; Carlos Milla; Howard Panitch; Adrienne Prestridge; Peggy Radford; Clement Ren; Jack Sharp

doi:10.1016/j.jcf.2015.10.007

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

Stephanie D. Davis^*, Felix Ratjen, Lyndia C. Brumback, Robin C. Johnson, Amy G. Filbrun, Gwendolyn S. Kerby, Howard B. Panitch, Scott H. Donaldson, Margaret Rosenfeld, Stephanie Davis, Albert Faro, Amy Filbrun, Peter Hiatt, Gwendolyn Kerby, Karen McCoy, Karen McDowell, James Acton, Carlos Milla, Howard Panitch, Adrienne PrestridgePeggy Radford, Clement Ren, Jack Sharp

^*Corresponding author for this work

Pediatrics

Research output: Contribution to journal › Article › peer-review

12 Scopus citations

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 FEV_0.5, 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 language	English (US)
Pages (from-to)	386-391
Number of pages	6
Journal	Journal of Cystic Fibrosis
Volume	15
Issue number	3
DOIs	https://doi.org/10.1016/j.jcf.2015.10.007
State	Published - May 1 2016

Keywords

FEV
Forced expiratory flow rates
Pulmonary function tests

ASJC Scopus subject areas

Pulmonary and Respiratory Medicine
Pediatrics, Perinatology, and Child Health

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10.1016/j.jcf.2015.10.007

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Davis, S. D., Ratjen, F., Brumback, L. C., Johnson, R. C., Filbrun, A. G., Kerby, G. S., Panitch, H. B., Donaldson, S. H., Rosenfeld, M., Davis, S., Faro, A., Filbrun, A., Hiatt, P., Kerby, G., McCoy, K., McDowell, K., Acton, J., Milla, C., Panitch, H., ... Sharp, J. (2016). Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis. Journal of Cystic Fibrosis, 15(3), 386-391. https://doi.org/10.1016/j.jcf.2015.10.007

@article{b407430c40e040f1b590a88920e93069,

title = "Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis",

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",

author = "Davis, {Stephanie D.} and Felix Ratjen and Brumback, {Lyndia C.} and Johnson, {Robin C.} and Filbrun, {Amy G.} and Kerby, {Gwendolyn S.} and Panitch, {Howard B.} and Donaldson, {Scott H.} and Margaret Rosenfeld and Stephanie Davis and Albert Faro and Amy Filbrun and Peter Hiatt and Gwendolyn Kerby and Karen McCoy and Karen McDowell and James Acton and Carlos Milla and Howard Panitch and Adrienne Prestridge and Peggy Radford and Clement Ren and Jack Sharp",

note = "Publisher Copyright: {\textcopyright} 2015 European Cystic Fibrosis Society.",

year = "2016",

month = may,

day = "1",

doi = "10.1016/j.jcf.2015.10.007",

language = "English (US)",

volume = "15",

pages = "386--391",

journal = "Journal of Cystic Fibrosis",

issn = "1569-1993",

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Davis, SD, Ratjen, F, Brumback, LC, Johnson, RC, Filbrun, AG, Kerby, GS, Panitch, HB, Donaldson, SH, Rosenfeld, M, Davis, S, Faro, A, Filbrun, A, Hiatt, P, Kerby, G, McCoy, K, McDowell, K, Acton, J, Milla, C, Panitch, H, Prestridge, A, Radford, P, Ren, C & Sharp, J 2016, 'Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis', Journal of Cystic Fibrosis, vol. 15, no. 3, pp. 386-391. https://doi.org/10.1016/j.jcf.2015.10.007

TY - JOUR

T1 - Infant lung function tests as endpoints in the ISIS multicenter clinical trial in cystic fibrosis

AU - Davis, Stephanie D.

AU - Ratjen, Felix

AU - Brumback, Lyndia C.

AU - Johnson, Robin C.

AU - Filbrun, Amy G.

AU - Kerby, Gwendolyn S.

AU - Panitch, Howard B.

AU - Donaldson, Scott H.

AU - Rosenfeld, Margaret

AU - Davis, Stephanie

AU - Faro, Albert

AU - Filbrun, Amy

AU - Hiatt, Peter

AU - Kerby, Gwendolyn

AU - McCoy, Karen

AU - McDowell, Karen

AU - Acton, James

AU - Milla, Carlos

AU - Panitch, Howard

AU - Prestridge, Adrienne

AU - Radford, Peggy

AU - Ren, Clement

AU - Sharp, Jack

PY - 2016/5/1

Y1 - 2016/5/1

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.

AB - 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.

KW - FEV

KW - Forced expiratory flow rates

KW - Pulmonary function tests

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U2 - 10.1016/j.jcf.2015.10.007

DO - 10.1016/j.jcf.2015.10.007

M3 - Article

C2 - 26547590

AN - SCOPUS:84964992868

SN - 1569-1993

VL - 15

SP - 386

EP - 391

JO - Journal of Cystic Fibrosis

JF - Journal of Cystic Fibrosis

IS - 3

ER -

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

Abstract

Keywords

ASJC Scopus subject areas

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