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 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.

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

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

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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