scholarly journals Pulmonary exacerbation for children and adults with primary ciliary dyskinesia (PCD) participating in clinical research

2019 ◽  
Author(s):  
Jane S. Lucas ◽  
Florian Gahleitner ◽  
Adelina Amorim ◽  
Mieke Boon ◽  
Philippa Brown ◽  
...  
Keyword(s):  
Clinical Research ◽  
Primary Ciliary Dyskinesia ◽  
Pulmonary Exacerbation ◽  
Ciliary Dyskinesia

scholarly journals Collecting clinical data in primary ciliary dyskinesia- challenges and opportunities

F1000Research ◽  
2016 ◽  
Vol 5 ◽  
pp. 2031 ◽  
Author(s):  
Israel Amirav ◽  
Mary Roduta Roberts ◽  
Huda Mussaffi ◽  
Avigdor Mandelberg ◽  
Yehudah Roth ◽  
...  
Keyword(s):  
Data Collection ◽  
Clinical Research ◽  
Clinical Data ◽  
Primary Ciliary Dyskinesia ◽  
Completion Rates ◽  
End User ◽  
Revision Process ◽  
Data Collection And Analysis ◽  
Challenges And Opportunities ◽  
Ciliary Dyskinesia

Rationale: Primary ciliary dyskinesia (PCD) is under diagnosed and underestimated. Most clinical research has used some form of questionnaires to capture data but none has been critically evaluated particularly with respect to its end-user feasibility and utility. Objective: To critically appraise a clinical data collection questionnaire for PCD used in a large national PCD consortium in order to apply conclusions in future PCD research. Methods: We describe the development, validation and revision process of a clinical questionnaire for PCD and its evaluation during a national clinical PCD study with respect to data collection and analysis, initial completion rates and user feedback. Results: 14 centers participating in the consortium successfully completed the revised version of the questionnaire for 173 patients with various completion rates for various items. While content and internal consistency analysis demonstrated validity, there were methodological deficiencies impacting completion rates and end-user utility. These deficiencies were addressed resulting in a more valid questionnaire. Conclusions: Our experience may be useful for future clinical research in PCD. Based on the feedback collected on the questionnaire through analysis of completion rates, judgmental analysis of the content, and feedback from experts and end users, we suggest a practicable framework for development of similar tools for various future PCD research.

scholarly journals Collecting clinical data in primary ciliary dyskinesia- challenges and opportunities

F1000Research ◽  
2016 ◽  
Vol 5 ◽  
pp. 2031
Author(s):  
Israel Amirav ◽  
Mary Roduta Roberts ◽  
Huda Mussaffi ◽  
Avigdor Mandelberg ◽  
Yehudah Roth ◽  
...  
Keyword(s):  
Data Collection ◽  
Clinical Research ◽  
Clinical Data ◽  
Primary Ciliary Dyskinesia ◽  
Completion Rates ◽  
End User ◽  
Revision Process ◽  
Data Collection And Analysis ◽  
Challenges And Opportunities ◽  
Ciliary Dyskinesia

Rationale: Primary ciliary dyskinesia (PCD) is under diagnosed and underestimated. Most clinical research has used some form of questionnaires to capture data but none has been critically evaluated particularly with respect to its end-user feasibility and utility. Objective: To critically appraise a clinical data collection questionnaire for PCD used in a large national PCD consortium in order to apply conclusions in future PCD research. Methods: We describe the development, validation and revision process of a clinical questionnaire for PCD and its evaluation during a national clinical PCD study with respect to data collection and analysis, initial completion rates and user feedback. Results: 14 centers participating in the consortium successfully completed the revised version of the questionnaire for 173 patients with various completion rates for various items. While content and internal consistency analysis demonstrated validity, there were methodological deficiencies impacting completion rates and end-user utility. These deficiencies were addressed resulting in a more valid questionnaire. Conclusions: Our experience may be useful for future clinical research in PCD. Based on the feedback collected on the questionnaire through analysis of completion rates, judgmental analysis of the content, and feedback from experts and end users, we suggest a practicable framework for development of similar tools for various future PCD research.

Lung clearance index predicts pulmonary exacerbations in individuals with primary ciliary dyskinesia: a multicentre cohort study

Thorax ◽  
2021 ◽  
pp. thoraxjnl-2020-215504
Author(s):  
Florian Singer ◽  
Anne Schlegtendal ◽  
Sylvia Nyilas ◽  
François Vermeulen ◽  
Mieke Boon ◽  
...  
Keyword(s):  
Cohort Study ◽  
Lung Function ◽  
Primary Ciliary Dyskinesia ◽  
Response To Treatment ◽  
Pulmonary Exacerbation ◽  
Lung Clearance ◽  
Lung Clearance Index ◽  
Pulmonary Exacerbations ◽  
The Impact ◽  
Ciliary Dyskinesia

BackgroundLung clearance index (LCI) is a promising lung function outcome in individuals with primary ciliary dyskinesia (PCD). The impact of events clinically important for individuals with PCD, such as pulmonary exacerbations, on LCI is unknown.MethodsWe conducted an international, multicentre, observational cohort study to assess the association of LCI and risk of pulmonary exacerbation, specific changes in LCI during pulmonary exacerbation and global variability of LCI across four visits every 4 months. Ninety individuals with PCD, aged 3–41 years, underwent nitrogen multiple-breath washout (MBW) and spirometry measurements. The association of LCI and pulmonary exacerbations was assessed by Cox proportional hazards and random-effects regression models.ResultsWe obtained 430 MBW and 427 spirometry measurements. In total, 379 person-years at risk contributed to the analysis. Per one unit increase (deterioration) in LCI, the risk of future pulmonary exacerbation increased by 13%: HR (95% CI), 1.13 (1.04 to 1.23). If LCI changed from a range of values considered normal to abnormal, the risk of future pulmonary exacerbations increased by 87%: 1.87 (1.08 to 3.23). During pulmonary exacerbations, LCI increased by 1.22 units (14.5%). After pulmonary exacerbations, LCI tended to decline. Estimates of variability in LCI suggested lower variation within individuals compared with variation between individuals. Findings were comparable for forced expiratory volume in 1 s.ConclusionOn a visit-to-visit basis, LCI measurement may add to the prediction of pulmonary exacerbations, the assessment of lung function decline and the potential lung function response to treatment of pulmonary exacerbations.

scholarly journals Recovery of baseline lung function after pulmonary exacerbation in children with primary ciliary dyskinesia

2016 ◽  
Vol 51 (12) ◽  
pp. 1362-1366 ◽  
Author(s):  
Meera Sunther ◽  
Andrew Bush ◽  
Claire Hogg ◽  
Lauren McCann ◽  
Siobhán B. Carr
Keyword(s):  
Lung Function ◽  
Primary Ciliary Dyskinesia ◽  
Pulmonary Exacerbation ◽  
Ciliary Dyskinesia

scholarly journals Changes in airway inflammation during pulmonary exacerbations in patients with cystic fibrosis and primary ciliary dyskinesia

2015 ◽  
Vol 47 (3) ◽  
pp. 829-836 ◽  
Author(s):  
Felix Ratjen ◽  
Valerie Waters ◽  
Michelle Klingel ◽  
Nancy McDonald ◽  
Sharon Dell ◽  
...  
Keyword(s):  
Cystic Fibrosis ◽  
Inflammatory Response ◽  
Neutrophil Elastase ◽  
Primary Ciliary Dyskinesia ◽  
Bacterial Pathogens ◽  
Pulmonary Exacerbation ◽  
Elastase Activity ◽  
Cell Counts ◽  
Pulmonary Exacerbations ◽  
Ciliary Dyskinesia

Lung disease in patients with both primary ciliary dyskinesia (PCD) or cystic fibrosis (CF) is associated with impaired mucociliary clearance; however, clinical outcomes are typically worse in CF patients. We assessed whether CF and PCD patients differ in inflammatory response in the airways during pulmonary exacerbation.We first studied clinically stable PCD patients with a spectrum of bacterial pathogens to assess inflammatory response to different pathogens. Subsequently, PCD and CF patients with similar bacterial pathogens were studied at the time of a pulmonary exacerbation and after 21 days of antibiotics treatment. Qualitative and quantitative microbiology, cell counts, interleukin-8 concentrations, and neutrophil elastase activity were assessed in sputum samples obtained before and after treatment.In stable PCD patients, no significant differences were found in sputum inflammatory markers between individuals colonised with different bacterial pathogens. Pulmonary exacerbation severity assessed by a pulmonary exacerbation score and lung function decline from their previous baseline did not differ between CF and PCD patients. Bacterial density for Staphylococcus aureus and Haemophilus influenzae was higher in CF versus PCD (p<0.05), but absolute neutrophil counts were higher in PCD patients (p=0.02). While sputum elastase activity was similar in PCD and CF at the time of exacerbation, it decreased with antibiotic therapy in PCD (p<0.05) but not CF patients.PCD patients differ from those with CF in their responses to treatment of pulmonary exacerbations, with higher neutrophil elastase activity persisting in the CF airways at the end of treatment.

scholarly journals Report of the 2021 Primary ciliary dyskinesia foundation annual meeting

2021 ◽  
pp. 1-4
Author(s):  
Thomas G. Saba ◽  
Steven L. Brody
Keyword(s):  
Clinical Research ◽  
Annual Meeting ◽  
Primary Ciliary Dyskinesia ◽  
Virtual Conference ◽  
Ciliary Dyskinesia

In August 2021, the Primary Ciliary Dyskinesia (PCD) Foundation hosted a 2-day international virtual conference designed to share discoveries in PCD genetics, cilia biology, and clinical research and care. The conference was organized around the theme of building a community for clinical research. This article provides a report of the proceedings.

scholarly journals The BEAT-PCD (Better Experimental Approaches to Treat Primary Ciliary Dyskinesia) Clinical Research Collaboration

2021 ◽  
Vol 57 (2) ◽  
pp. 2004601
Author(s):  
Myrofora Goutaki ◽  
Suzanne Crowley ◽  
Eleonora Dehlink ◽  
René Gaupmann ◽  
Katie L. Horton ◽  
...  
Keyword(s):  
Clinical Research ◽  
Primary Ciliary Dyskinesia ◽  
Research Collaboration ◽  
Experimental Approaches ◽  
Ciliary Dyskinesia

Pulmonary Infection Related to Mimivirus in Patient with Primary Ciliary Dyskinesia

2020 ◽  
Vol 26 (8) ◽  
Author(s):  
Fatemeh Sakhaee ◽  
Farzam Vaziri ◽  
Golnaz Bahramali ◽  
Seyed Davar Siadat ◽  
Abolfazl Fateh
Keyword(s):  
Primary Ciliary Dyskinesia ◽  
Pulmonary Infection ◽  
Ciliary Dyskinesia
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