Determination of the Minimal Clinically Important Difference Scores for the Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Scale in Two Populations of Patients With Cystic Fibrosis and Chronic Pseudomonas aeruginosa Airway Infection
Alexandra L. Quittner,Avani C. Modi,Claire E. Wainwright,Kelly L. Otto,Jean Kirihara,A. Bruce Montgomery +5 more
TLDR
In this article, the authors used a global rating-of-change questionnaire (GRCQ) to assess patients' perceptions of change in their respiratory symptoms after TIS treatment, and mapped the mean change from baseline CFQ-R-Respiratory scores onto the GRCQ to estimate the minimal clinically important difference (MCID).About:
This article is published in Chest.The article was published on 2009-06-01 and is currently open access. It has received 292 citations till now. The article focuses on the topics: Minimal clinically important difference & Exacerbation.read more
Citations
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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
Bonnie W. Ramsey,Jane C. Davies,N. Gerard McElvaney,Elizabeth Tullis,Scott C. Bell,Pavel Dř evínek,Matthias Griese,Edward F. McKone,Claire E. Wainwright,Michael W. Konstan,Richard B. Moss,Felix Ratjen,Isabelle Sermet-Gaudelus,Steven M. Rowe,Qunming Dong,Sally Rodriguez,Karl Yen,Claudia L. Ordoñez,J. Stuart Elborn +18 more
TL;DR: Ivacaftor was associated with improvements in lung function at 2 weeks that were sustained through 48 weeks and substantial improvements were also observed in the risk of pulmonary exacerbations, patient-reported respiratory symptoms, weight, and concentration of sweat chloride.
Journal ArticleDOI
Lumacaftor–Ivacaftor in Patients with Cystic Fibrosis Homozygous for Phe508del CFTR
Jennifer L. Taylor-Cousar,Anne Munck,Edward F. McKone,Cornelis K. van der Ent,Alexander Moeller,Christopher Simard,Linda T Wang,Edward P. Ingenito,Charlotte M. McKee,Yimeng Lu,J Lekstrom-Himes,J. Stuart Elborn +11 more
TL;DR: These data show that lumacaftor in combination with ivacaftors provided a benefit for patients with cystic fibrosis homozygous for the Phe508del CFTR mutation.
Journal ArticleDOI
Effect of VX-770 in Persons with Cystic Fibrosis and the G551D-CFTR Mutation
Frank J. Accurso,Steven M. Rowe,John P. Clancy,Michael P. Boyle,Jordan M. Dunitz,Peter R. Durie,Scott D. Sagel,Douglas B. Hornick,Michael W. Konstan,Scott H. Donaldson,Richard B. Moss,Joseph M. Pilewski,Ronald C. Rubenstein,Ahmet Uluer,Moira L. Aitken,Steven D. Freedman,Lynn M. Rose,Nicole Mayer-Hamblett,Qunming Dong,Jiuhong Zha,Anne Stone,Eric R. Olson,Claudia L. Ordoñez,Preston W. Campbell,Melissa A. Ashlock,Bonnie W. Ramsey +25 more
TL;DR: This study showed that VX-770 was associated with within-subject improvements in CFTR and lung function and provides support for further studies of pharmacologic potentiation of CFTR as a means to treat cystic fibrosis.
Journal ArticleDOI
Efficacy and safety of the elexacaftor plus tezacaftor plus ivacaftor combination regimen in people with cystic fibrosis homozygous for the F508del mutation : a double-blind, randomised, phase 3 trial
Harry G.M. Heijerman,Edward F. McKone,Damian G. Downey,Eva Van Braeckel,Steven M. Rowe,Elizabeth Tullis,Marcus A. Mall,John Welter,Bonnie W. Ramsey,Charlotte M. McKee,Gautham Marigowda,Samuel M. Moskowitz,David Waltz,Patrick R Sosnay,Christopher Simard,Neil Ahluwalia,Fengjuan Xuan,Yaohua Zhang,Jennifer L. Taylor-Cousar,Karen McCoy +19 more
TL;DR: Elexacaftor plus tezacaftorplus ivacaftors provided clinically robust benefit compared with tezacftor plus ivACaftor alone, with a favourable safety profile, and shows the potential to lead to transformative improvements in the lives of people with cystic fibrosis who are homozygous for the F508del mutation.
Journal ArticleDOI
Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation
John P. Clancy,Steven M. Rowe,Frank J. Accurso,Moira L. Aitken,Raouf S. Amin,Melissa A. Ashlock,Manfred Ballmann,Michael P. Boyle,Inez Bronsveld,Preston W. Campbell,Kris De Boeck,Scott H. Donaldson,Henry L. Dorkin,Jordan M. Dunitz,Peter R. Durie,Manu Jain,Anissa Leonard,Karen McCoy,Richard B. Moss,Joseph M. Pilewski,Daniel B. Rosenbluth,Ronald C. Rubenstein,Michael S. Schechter,Martyn Botfield,Claudia L. Ordoñez,George T. Spencer-Green,Laurent Vernillet,Steve Wisseh,Karl Yen,Michael W. Konstan +29 more
TL;DR: In this study, VX-809 had a similar adverse event profile to placebo for 28 days in F508del-CFTR homozygous patients, and demonstrated biological activity with positive impact on CFTR function in the sweat gland.
References
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Standardization of Spirometry, 1994 Update. American Thoracic Society.
R. Crapo,John L. Hankinson,Charles G. Irvin,Neil R. MacIntyre,K. Z. Voter,Robert A. Wise,Brian L. Graham,C. O'Donnell,P. Paoletti,J. Roca,G. Veigi +10 more
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Cystic Fibrosis
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Measurement of health status: Ascertaining the minimal clinically important difference
TL;DR: An approach to elucidating the significance of changes in score in quality of life instruments by comparing them to global ratings of change is developed, and a plausible range within which the minimal clinically important difference (MCID) falls is established.
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Changes in the normal maximal expiratory flow-volume curve with growth and aging.
TL;DR: Improved prediction equations for each sex by age group for 5 spirometric and flow-volume variables are derived and "Normal" limits are proposed that take into consideration the between-subject variability and non-Gaussian distribution of the various measurements.
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Determining a minimal important change in a disease-specific quality of life questionnaire
TL;DR: The observation that the minimal important difference is consistent across domains and for both improvement and deterioration will facilitate interpretation of results of studies examining quality of life.
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A CFTR potentiator in patients with cystic fibrosis and the G551D mutation
Bonnie W. Ramsey,Jane C. Davies,N. Gerard McElvaney,Elizabeth Tullis,Scott C. Bell,Pavel Dř evínek,Matthias Griese,Edward F. McKone,Claire E. Wainwright,Michael W. Konstan,Richard B. Moss,Felix Ratjen,Isabelle Sermet-Gaudelus,Steven M. Rowe,Qunming Dong,Sally Rodriguez,Karl Yen,Claudia L. Ordoñez,J. Stuart Elborn +18 more