Clinical outcome measures for use in primary ciliary dyskinesia research: a scoping systematic review

2019 ◽  
Author(s):  
Bruna Rubbo ◽  
Florian Gahleitner ◽  
Claire Jackson ◽  
Myrofora Goutaki ◽  
Florian Halbeisen ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Primary Ciliary Dyskinesia ◽  
Ciliary Dyskinesia

scholarly journals Lower airways clinical outcome measures for use in primary ciliary dyskinesia research, a scoping review

2021 ◽  
pp. 00320-2021
Author(s):  
Florian Gahleitner ◽  
James Thompson ◽  
Claire L Jackson ◽  
Jana F Hueppe ◽  
Laura Behan ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Outcome Measures ◽  
Scoping Review ◽  
Primary Ciliary Dyskinesia ◽  
Scoring Systems ◽  
Respiratory Pathogen ◽  
High Resolution Computed Tomography ◽  
Related Quality ◽  
Multiple Breath Washout ◽  
Ciliary Dyskinesia

ObjectivesDisease-specific, well-defined, and validated clinical outcome measures are essential in designing research studies. Poorly defined outcome measures hamper pooling of data and comparisons between studies. We aimed to identify and describe pulmonary outcome measures that could be used for follow-up of patients with primary ciliary dyskinesia (PCD).MethodsWe conducted a scoping review by systematically searching Medline, Embase and Cochrane Systematic Review online databases for studies published from 1996 to 2020 that included ≥10 PCD adult and/or paediatric patients.ResultsWe included 102 studies (7289 patients). Eighty-three studies reported on spirometry, 11 on body plethysmography, 15 on multiple breath washout, 36 on high-resolution computed tomography (HRCT), 57 on microbiology, and 17 on health-related quality of life. Measurement and reporting of outcomes varied considerably between studies (e.g. different scoring systems for chest HRCT scans). Additionally, definitions of outcome measures varied (e.g. definition of chronic colonisation by respiratory pathogen), impeding direct comparisons of results.ConclusionsThis review highlights the need for standardisation of measurements and reporting of outcome measures to enable comparisons between studies. Defining a core set of clinical outcome measures is necessary to ensure reproducibility of results and for use in future trials and prospective cohorts.

scholarly journals Clinical outcome measures and scoring systems used in prospective studies of port wine stains: A systematic review

PLoS ONE ◽  
2020 ◽  
Vol 15 (7) ◽  
pp. e0235657 ◽  
Author(s):  
M. Ingmar van Raath ◽  
Sandeep Chohan ◽  
Albert Wolkerstorfer ◽  
Chantal M. A. M. van der Horst ◽  
Jacqueline Limpens ◽  
...  
Keyword(s):  
Systematic Review ◽  
Clinical Outcome ◽  
Outcome Measures ◽  
Prospective Studies ◽  
Scoring Systems ◽  
Port Wine ◽  
Port Wine Stains

scholarly journals Primary ciliary dyskinesia in Japan: systematic review and meta-analysis

2019 ◽  
Vol 19 (1) ◽  
Author(s):  
Atsushi Inaba ◽  
Masanori Furuhata ◽  
Kozo Morimoto ◽  
Mahbubur Rahman ◽  
Osamu Takahashi ◽  
...  
Keyword(s):  
Systematic Review ◽  
Primary Ciliary Dyskinesia ◽  
Meta Analysis ◽  
Ciliary Dyskinesia

scholarly journals Clinical manifestations in primary ciliary dyskinesia: systematic review and meta-analysis

2016 ◽  
Vol 48 (4) ◽  
pp. 1081-1095 ◽  
Author(s):  
Myrofora Goutaki ◽  
Anna Bettina Meier ◽  
Florian S. Halbeisen ◽  
Jane S. Lucas ◽  
Sharon D. Dell ◽  
...  
Keyword(s):  
Systematic Review ◽  
Primary Ciliary Dyskinesia ◽  
Clinical Symptoms ◽  
Meta Analysis ◽  
Age Groups ◽  
Clinical Manifestations ◽  
Explanatory Factors ◽  
Symptom Prevalence ◽  
Meta Analyses ◽  
Ciliary Dyskinesia

Few original studies have described the prevalence and severity of clinical symptoms of primary ciliary dyskinesia (PCD). This systematic review and meta-analysis aimed to identify all published studies on clinical manifestations of PCD patients, and to describe their prevalence and severity stratified by age and sex.We searched PubMed, Embase and Scopus for studies describing clinical symptoms of ≥10 patients with PCD. We performed meta-analyses and meta-regression to explain heterogeneity.We included 52 studies describing a total of 1970 patients (range 10–168 per study). We found a prevalence of 5% for congenital heart disease. For the rest of reported characteristics, we found considerable heterogeneity (I2 range 68–93.8%) when calculating the weighted mean prevalence. Even after taking into account the explanatory factors, the largest part of the between-studies variance in symptom prevalence remained unexplained for all symptoms. Sensitivity analysis including only studies with test-proven diagnosis showed similar results in prevalence and heterogeneity.Large differences in study design, selection of study populations and definition of symptoms could explain the heterogeneity in symptom prevalence. To better characterise the disease, we need larger, multicentre, multidisciplinary, prospective studies that include all age groups, use uniform diagnostics and report on all symptoms.

scholarly journals The international primary ciliary dyskinesia cohort (iPCD Cohort): methods and first results

2016 ◽  
Vol 49 (1) ◽  
pp. 1601181 ◽  
Author(s):  
Myrofora Goutaki ◽  
Elisabeth Maurer ◽  
Florian S. Halbeisen ◽  
Israel Amirav ◽  
Angelo Barbato ◽  
...  
Keyword(s):  
Systematic Review ◽  
Primary Ciliary Dyskinesia ◽  
Clinical Phenotype ◽  
Clinical Information ◽  
Age Group ◽  
The European Union ◽  
European Respiratory Society ◽  
Task Forces ◽  
First Results ◽  
Ciliary Dyskinesia

Data on primary ciliary dyskinesia (PCD) epidemiology is scarce and published studies are characterised by low numbers. In the framework of the European Union project BESTCILIA we aimed to combine all available datasets in a retrospective international PCD cohort (iPCD Cohort).We identified eligible datasets by performing a systematic review of published studies containing clinical information on PCD, and by contacting members of past and current European Respiratory Society Task Forces on PCD. We compared the contents of the datasets, clarified definitions and pooled them in a standardised format.As of April 2016 the iPCD Cohort includes data on 3013 patients from 18 countries. It includes data on diagnostic evaluations, symptoms, lung function, growth and treatments. Longitudinal data are currently available for 542 patients. The extent of clinical details per patient varies between centres. More than 50% of patients have a definite PCD diagnosis based on recent guidelines. Children aged 10–19 years are the largest age group, followed by younger children (≤9 years) and young adults (20–29 years).This is the largest observational PCD dataset available to date. It will allow us to answer pertinent questions on clinical phenotype, disease severity, prognosis and effect of treatments, and to investigate genotype–phenotype correlations.

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