Patient-reported respiratory symptoms in cystic fibrosis

Goss, Christopher H.; Edwards, Todd C.; Ramsey, Bonnie W.; Aitken, Moira L.; Patrick, DL

doi:10.1016/j.jcf.2009.04.003

Cited by 114 publications

(112 citation statements)

References 48 publications

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“…Study assessments conducted in all enrolled subjects (i.e., the "core" study) included spirometry; body weight; sweat chloride; and the patient-reported outcome instruments Cystic Fibrosis QuestionnaireRevised (CFQ-R) (14), Cystic Fibrosis Respiratory Symptom Diary (15), and SinoNasal Outcome Test-20 (16). Samples collected and subsequently stored at the Cystic Fibrosis Foundation Therapeutics Biorepository for future research included plasma, serum, buffy coat, urine, and expectorated sputum.…”

Section: Methodsmentioning

confidence: 99%

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

Rowe

Heltshe

Gonska

et al. 2014

Am J Respir Crit Care Med

447

429

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Rationale: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator recently approved for patients with CF age 6 and older with the G551D mutation.Objectives: To evaluate ivacaftor in a postapproval setting and determine mechanism of action and response of clinically relevant markers.Methods: We conducted a longitudinal cohort study in 2012-2013 in G551D CF patients age 6 and older with no prior exposure to ivacaftor. Study assessments were performed at baseline, 1, 3, and 6 months after ivacaftor initiation. Substudies evaluated mucociliary clearance, b-adrenergic sweat secretion rate, gastrointestinal pH, and sputum inflammation and microbiology Measurements and Main Results: A total of 151 of 153 subjects were prescribed ivacaftor and 88% completed the study through 6 months. FEV 1 % predicted improved from baseline to 6 months (mean absolute change, 6.7%; P , 0.001). Similarly, body mass index improved from baseline to 6 months (mean change, 0.8 kg/m 2 ; P , 0.001). Sweat chloride decreased from baseline to 6 months (mean change, 253.8 mmol/L; 95% confidence interval, 257.7 to 249.9; P , 0.001), reflecting augmented CFTR function. There was significant improvement in hospitalization rate (P , 0.001) and Pseudomonas aeruginosa burden (P , 0.01). Significant improvements in mucociliary clearance (P , 0.001), gastrointestinal pH (P = 0.001), and microbiome were also observed, providing clinical mechanisms underlying the therapeutic benefit of ivacaftor.Conclusions: Significant clinical and physiologic improvements were observed on initiation of ivacaftor in a broad patient population, including reduced infection with P. aeruginosa. Biomarker studies substantially improve the understanding of the mechanistic consequences of CFTR modulation on pulmonary and gastrointestinal physiology.

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Section: Methodsmentioning

confidence: 99%

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

Rowe

Heltshe

Gonska

et al. 2014

Am J Respir Crit Care Med

447

429

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“…The LRSQ has potential advantages as it has been designed as an instrument for parental completion, is partially validated in those aged f5 yrs and covers an extensive number of symptoms, whilst maintaining acceptability. Unlike other respiratory conditions, such as asthma [20], much of the focus in the development of respiratory symptom scores for CF has been on creating shorter term recall tools that are capable of identifying pulmonary exacerbations [2,6]. In contrast, the LRSQ is not designed to be used as a pulmonary exacerbation score.…”

Section: Discussionmentioning

confidence: 99%

“…Those tools that have been validated in children focus on the recognition of acute pulmonary exacerbations or short-term trial outcomes, rather than a longer term reflection of parent-reported respiratory condition. The Cystic Fibrosis Questionnaire Revised (CFQ-R) respiratory symptom scale has been well validated in the 6-to 13-yr-old age group, but is focussed on a 2-week recall period and has mainly been used in clinical trial outcomes rather than clinical monitoring [6][7][8][9][10].…”

mentioning

confidence: 99%

Assessing the Liverpool Respiratory Symptom Questionnaire in children with cystic fibrosis

Trinick

Southern²,

McNamara³

2011

Eur Respir J

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Monitoring respiratory status in cystic fibrosis (CF) is challenging, particularly in young children. We aimed to test whether the Liverpool Respiratory Symptom Questionnaire (LRSQ) could distinguish well, pre-school and older children with and without CF, whether it could distinguish well and unwell children with CF and, finally, whether LRSQ scores in older children with CF correlated with established measures of disease severity.20 stable pre-school children with CF had significantly higher total LRSQ scores than 51 preschool controls, and higher scores in two out of eight domains. Similarly, 21 stable 6-to 12-yr-old children with CF had higher total scores than 97 6-to 12-yr-old controls, and higher scores in seven out of eight domains.In older children with CF, LRSQ scores correlated negatively with Shwachman score and forced expiratory volume in 1 s (r5 -0.58, p,0.001, n531; and r5 -0.46, p,0.010, n534, respectively). Within the CF group, patients who cultured Pseudomonas aeruginosa, who used more ''back-up'' antibiotics or whose school attendance was lower also had higher LRSQ scores.The LRSQ differentiates well children from those with CF in both pre-school and the 6-to 12-yrold age group, even at a point of stability. It also differentiates stable from unwell children with CF, and scores correlate with other measures of respiratory disease, highlighting its potential as a clinical monitoring tool in paediatric CF.

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“…Meditative movement is also likely to improve flexibility and posture: a symptom and aggravator of CF. 6 The mindful awareness of the movements can reduce the body's stress level and improve quality of life and well-being.…”

Section: Clinical/practical Implicationsmentioning

confidence: 99%

“…5 They may also suffer stress, frustration, depression, irritability, worry, insomnia,/behavioral issues, and poor posture, and as a result miss school or work. 4,[6][7][8] Meditative movement is a recently coined term for gentle exercises that incorporate meditation, breathing, and relaxation. 9 Meditative movement (also called complementary/alternative exercise or mind-body exercise) as a group of therapies is increasingly the focus of studies and reviews, with evidence identified for fibromyalgia 10,11 and cognitive impairment.…”

Section: Introductionmentioning

confidence: 99%

Meditative Movement for Respiratory Function: A Systematic Review

et al. 2013

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BACKGROUND:Meditative movement, such as tai chi, yoga, and qi gong, may benefit people with cystic fibrosis (CF), as a form of gentle exercise incorporating meditation, breathing, and relaxation. Respiratory function is the most common issue in CF. In this systematic review we synthesized the evidence on the effect of meditative movement on respiratory function in patients with CF. METHODS: We searched Chinese and English language databases with terms relating to tai chi/yoga/qi gong, and respiratory function/cough/dyspnea. Articles were screened and selected by 2 researchers. We included controlled studies published in English or Chinese after 1980, and extracted data using a specially designed spreadsheet. Two researchers independently evaluated study quality and reporting, using 3 standardized checklists. Meta-analysis was not possible due to heterogeneous methods. RESULTS: We found 1,649 papers, included 43 (30 in English, 13 in Chinese), 23 of which were randomized controlled trials, and 20 were non-randomized trials. No studies were concerned with CF. Eleven studies included patients with respiratory disorders, and 27 included healthy people. Very few studies were high quality. The main problems with the randomized controlled trials was the randomization and non-random and/or poorly reported sampling. The main problems with the non-randomized studies were poor reporting of samples and non-equivalent groups. Although no clinically important changes were found, meditative movement may improve FEV 1 in healthy people, compared to no treatment/exercise (the intervention groups showed effect-size changes from 0.07 to 0.83), but meditative movement did not appear to affect FEV 1 /FVC in subjects with COPD. Key study limitations were: poor reporting of sampling or methods; inadequate sample size; non-randomized design; inadequate description of randomization; randomization by center; no blinding; lack of reporting of important aspects of meditative movement; and short-term follow-up.

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Patient-reported respiratory symptoms in cystic fibrosis

Abstract: Using a qualitative inductive methodology, we have obtained patient centered data regarding pulmonary symptoms and burdens and have created a novel patient reported outcome measure for CF. Future studies will assess the validity of the instruments.

Cited by 114 publications

References 48 publications

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

Clinical Mechanism of the Cystic Fibrosis Transmembrane Conductance Regulator Potentiator Ivacaftor in G551D-mediated Cystic Fibrosis

Assessing the Liverpool Respiratory Symptom Questionnaire in children with cystic fibrosis

Meditative Movement for Respiratory Function: A Systematic Review

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