scholarly journals Outcome Measures for Respiratory Physiotherapy in Cystic Fibrosis — Challenges and Advances

10.5772/60674 ◽  
2015 ◽  
Author(s):  
Alda Marques ◽  
Joana Cruz ◽  
Cristina Jácome ◽  
Ana Oliveira
Keyword(s):  
Cystic Fibrosis ◽  
Outcome Measures ◽  
Respiratory Physiotherapy

ePS5.02 Development of mental health quality outcome measures in the Swedish Cystic Fibrosis Registry - pitfalls and possibilities

2020 ◽  
Vol 19 ◽  
pp. S49
Author(s):  
L. Backström Eriksson ◽  
C. Laine ◽  
I. de Monestrol ◽  
A. Lindblad ◽  
E. Bergenmar-Ivarsson ◽  
...  
Keyword(s):  
Mental Health ◽  
Cystic Fibrosis ◽  
Outcome Measures ◽  
Health Quality

scholarly journals Oral cysteamine as an adjunct treatment in cystic fibrosis pulmonary exacerbations: An exploratory randomized clinical trial

PLoS ONE ◽  
2020 ◽  
Vol 15 (12) ◽  
pp. e0242945
Author(s):  
Graham Devereux ◽  
Danielle Wrolstad ◽  
Stephen J. Bourke ◽  
Cori L. Daines ◽  
Simon Doe ◽  
...  
Keyword(s):  
Clinical Trial ◽  
Cystic Fibrosis ◽  
Randomized Clinical Trial ◽  
Outcome Measures ◽  
Leukocyte Count ◽  
Bacterial Load ◽  
Adjunct Treatment ◽  
Dosing Regimens ◽  
Pulmonary Exacerbations ◽  
Blood Leukocyte

Background Emerging data suggests a possible role for cysteamine as an adjunct treatment for pulmonary exacerbations of cystic fibrosis (CF) that continue to be a major clinical challenge. There are no studies investigating the use of cysteamine in pulmonary exacerbations of CF. This exploratory randomized clinical trial was conducted to answer the question: In future pivotal trials of cysteamine as an adjunct treatment in pulmonary exacerbations of CF, which candidate cysteamine dosing regimens should be tested and which are the most appropriate, clinically meaningful outcome measures to employ as endpoints? Methods and findings Multicentre double-blind randomized clinical trial. Adults experiencing a pulmonary exacerbation of CF being treated with standard care that included aminoglycoside therapy were randomized equally to a concomitant 14-day course of placebo, or one of 5 dosing regimens of cysteamine. Outcomes were recorded on days 0, 7, 14 and 21 and included sputum bacterial load and the patient reported outcome measures (PROMs): Chronic Respiratory Infection Symptom Score (CRISS), the Cystic Fibrosis Questionnaire–Revised (CFQ-R); FEV1, blood leukocyte count, and inflammatory markers. Eighty nine participants in fifteen US and EU centres were randomized, 78 completed the 14-day treatment period. Cysteamine had no significant effect on sputum bacterial load, however technical difficulties limited interpretation. The most consistent findings were for cysteamine 450mg twice daily that had effects additional to that observed with placebo, with improved symptoms, CRISS additional 9.85 points (95% CI 0.02, 19.7) p = 0.05, reduced blood leukocyte count by 2.46x109 /l (95% CI 0.11, 4.80), p = 0.041 and reduced CRP by geometric mean 2.57 nmol/l (95% CI 0.15, 0.99), p = 0.049. Conclusion In this exploratory study cysteamine appeared to be safe and well-tolerated. Future pivotal trials investigating the utility of cysteamine in pulmonary exacerbations of CF need to include the cysteamine 450mg doses and CRISS and blood leukocyte count as outcome measures. Clinical trial registration NCT03000348; www.clinicaltrials.gov.

Mucus plugging, air trapping, and bronchiectasis are important outcome measures in assessing progressive childhood cystic fibrosis lung disease

2020 ◽  
Vol 55 (4) ◽  
pp. 929-938 ◽  
Author(s):  
Terry E. Robinson ◽  
Michael L. Goris ◽  
Richard B. Moss ◽  
Lu Tian ◽  
Peiyi Kan ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Lung Disease ◽  
Outcome Measures ◽  
Cystic Fibrosis Lung ◽  
Air Trapping ◽  
Cystic Fibrosis Lung Disease

scholarly journals A systematic review of patient-reported outcome measures (PROMs) in cystic fibrosis

BMJ Open ◽  
2020 ◽  
Vol 10 (10) ◽  
pp. e033867
Author(s):  
Irushi Ratnayake ◽  
Susannah Ahern ◽  
Rasa Ruseckaite
Keyword(s):  
Quality Of Life ◽  
Cystic Fibrosis ◽  
Outcome Measures ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Cochrane Library ◽  
Life Questionnaire ◽  
Health Measurement ◽  
Patient Reported

BackgroundTo determine patient-reported outcome measures (PROMs) which may be suitable for incorporation into the Australian Cystic Fibrosis Data Registry (ACFDR) by identifying PROMs administered in adult and paediatric cystic fibrosis (CF) populations in the last decade.MethodsWe searched MEDLINE, EMBASE, Scopus, CINAHL, PsycINFO and Cochrane Library databases for studies published between January 2009 and February 2019 describing the use of PROMs to measure health-related quality of life (HRQoL) in adult and paediatric patients with CF. Validation studies, observational studies and qualitative studies were included. The search was conducted on 13 February 2019. The COnsensus-based Standards for the selection of health Measurement INstruments Risk of Bias Checklist was used to assess the methodological quality of included studies.ResultsTwenty-seven different PROMs were identified. The most commonly used PROMs were designed specifically for CF. Equal numbers of studies were conducted on adult (32%, n=31), paediatric (35%, n=34) and both (27%, n=26) populations. No PROMs were used within a clinical registry setting previously. The two most widely used PROMs, the Cystic Fibrosis Questionnaire—Revised (CFQ-R) and the Cystic Fibrosis Quality of Life Questionnaire (CFQoL), demonstrated good psychometric properties and acceptability in English-speaking populations.DiscussionWe found that although PROMs are widely used in CF, there is a lack of reporting on the efficacy of methods and timepoints of administration. We identified the CFQ-R and CFQoL as the most suitable for incorporation in the ACFDR as they captured significant effects of CF on HRQoL and were reliable and valid in CF populations. These PROMs will be used in a further qualitative study assessing patients’ with CF and clinicians’ perspectives toward the acceptability and feasibility of incorporating a PROM in the ACFDR.PROSPERO registration numberCRD42019126931.

scholarly journals Cystic fibrosis and the role of gastrointestinal outcome measures in the new era of therapeutic CFTR modulation

2015 ◽  
Vol 14 (2) ◽  
pp. 169-177 ◽  
Author(s):  
Frank A.J.A. Bodewes ◽  
Henkjan J. Verkade ◽  
Jan A.J.M. Taminiau ◽  
Drucy Borowitz ◽  
Michael Wilschanski
Keyword(s):  
Cystic Fibrosis ◽  
Outcome Measures ◽  
New Era

scholarly journals P72 Feasibility of recruiting newborn babies with cystic fibrosis diagnosed by newborn screening to a clinical study with invasive outcome measures

Thorax ◽  
2010 ◽  
Vol 65 (Suppl 4) ◽  
pp. A107-A108
Author(s):  
J. Chudleigh ◽  
A.-F. Hoo ◽  
S. A. Prasad ◽  
D. Ahmed ◽  
A. Bush ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Clinical Study ◽  
Newborn Screening ◽  
Outcome Measures

Animal models for cystic fibrosis: A systematic search and mapping review of the literature – Part 1: genetic models

2019 ◽  
Vol 54 (4) ◽  
pp. 330-340 ◽  
Author(s):  
Cathalijn HC Leenaars ◽  
Rob BM De Vries ◽  
Anna Heming ◽  
Damian Visser ◽  
David Holthaus ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Animal Model ◽  
Animal Models ◽  
Outcome Measures ◽  
Reporting Quality ◽  
Genetic Models ◽  
Suitable Model ◽  
Objective Evidence ◽  
New Research ◽  
New Treatments

Animal models for cystic fibrosis (CF) have enhanced our understanding of the pathology and contributed to the development of new treatments. In the field of CF, many animal models have been developed and described. To our knowledge, thus far, none of the reviews of CF animal models has used a systematic methodology. A systematic approach to creating model overviews can lead to an objective, evidence-based choice of an animal model for new research questions. We searched Pubmed and Embase for the currently available animal models for CF. Two independent reviewers screened the results. We included all primary studies describing an animal model for CF. After duplicate removal, 12,304 publications were left. Because of the large number of models, in the current paper, only the genetic models are presented. A total of 636 publications were identified describing genetic animal models for CF in mice, pigs, ferrets, rats and zebrafish. Most of these models have an altered Cftr gene. An overview of basic model characteristics and outcome measures for these genetic models is provided, together with advice on using these data. As far as the authors are aware, this is one of the largest systematic mapping reviews on genetic animal models for CF. It can aid in selecting a suitable model and outcome measures. In general, the reporting quality of the included publications was poor. Further systematic reviews are warranted to determine the quality and translational value of these models further.

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