Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study

Scott H. Donaldson; Beth L. Laube; Peter Mogayzel; Timothy E. Corcoran; Joseph M. Pilewski; Agathe Ceppe; Jihong Wu; Pradeep G. Bhambhvani; Felix Ratjen; Scott D. Sagel; J. P. Clancy; Steven M. Rowe; William D. Bennett

doi:10.1016/j.jcf.2021.05.004

Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study

Scott H. Donaldson, Beth L. Laube, Peter Mogayzel, Timothy E. Corcoran, Joseph M. Pilewski, Agathe Ceppe, Jihong Wu, Pradeep G. Bhambhvani, Felix Ratjen, Scott D. Sagel, J. P. Clancy, Steven M. Rowe, William D. Bennett

School of Medicine

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

Abstract

CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV₁ or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.

Original language	English (US)
Pages (from-to)	143-145
Number of pages	3
Journal	Journal of Cystic Fibrosis
Volume	21
Issue number	1
DOIs	https://doi.org/10.1016/j.jcf.2021.05.004
State	Published - Jan 2022

ASJC Scopus subject areas

Pulmonary and Respiratory Medicine
Pediatrics, Perinatology, and Child Health

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Donaldson, S. H., Laube, B. L., Mogayzel, P., Corcoran, T. E., Pilewski, J. M., Ceppe, A., Wu, J., Bhambhvani, P. G., Ratjen, F., Sagel, S. D., Clancy, J. P., Rowe, S. M., & Bennett, W. D. (2022). Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study. Journal of Cystic Fibrosis, 21(1), 143-145. https://doi.org/10.1016/j.jcf.2021.05.004

Donaldson, SH, Laube, BL , Mogayzel, P, Corcoran, TE, Pilewski, JM, Ceppe, A, Wu, J, Bhambhvani, PG, Ratjen, F, Sagel, SD, Clancy, JP, Rowe, SM & Bennett, WD 2022, 'Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study', Journal of Cystic Fibrosis, vol. 21, no. 1, pp. 143-145. https://doi.org/10.1016/j.jcf.2021.05.004

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abstract = "CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV1 or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.",

author = "Donaldson, {Scott H.} and Laube, {Beth L.} and Peter Mogayzel and Corcoran, {Timothy E.} and Pilewski, {Joseph M.} and Agathe Ceppe and Jihong Wu and Bhambhvani, {Pradeep G.} and Felix Ratjen and Sagel, {Scott D.} and Clancy, {J. P.} and Rowe, {Steven M.} and Bennett, {William D.}",

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doi = "10.1016/j.jcf.2021.05.004",

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AU - Donaldson, Scott H.

AU - Laube, Beth L.

AU - Mogayzel, Peter

AU - Corcoran, Timothy E.

AU - Pilewski, Joseph M.

AU - Ceppe, Agathe

AU - Wu, Jihong

AU - Bhambhvani, Pradeep G.

AU - Ratjen, Felix

AU - Sagel, Scott D.

AU - Clancy, J. P.

AU - Rowe, Steven M.

AU - Bennett, William D.

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N2 - CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV1 or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.

AB - CFTR function is required for normal mucociliary clearance (MCC) and cough-assisted clearance (CC). Lumacaftor-ivacaftor is approved for use in people with cystic fibrosis (CF) carrying two copies of F508del-CFTR. In this observational study performed at four study sites, we characterized the effect of lumacaftor-ivacaftor on mucociliary and cough clearance and related this to other clinical and research endpoints after one month of treatment. Twenty-five adolescents and adults were enrolled. No effect on whole lung MCC was observed, but CC was significantly increased. Sweat chloride improved by 18 mEq/L in this group, indicating a modest restoration of CFTR activity, but no demonstrable change in FEV1 or lung clearance index was observed. We speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to yield an improvement in MCC.

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Effect of lumacaftor-ivacaftor on mucociliary clearance and clinical outcomes in cystic fibrosis: Results from the PROSPECT MCC sub-study

Abstract

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