Patients' research priorities and participation in primary ciliary dyskinesia research.
Publisher DOI
PubMed ID
41397810
Abstract
Introduction
People living with chronic diseases can provide a unique perspective for research that often differs from that of healthcare professionals. This is particularly important in rare diseases like primary ciliary dyskinesia (PCD), with many knowledge gaps and limited research resources. We aimed to assess participation of patients and caregivers in PCD research and identify their research priorities in a mixed-method study.Methods
We conducted in-depth, semistructured interviews with adults and adolescents with PCD, and caregivers of children with PCD. After verbatim transcription and thematic analysis, we developed an anonymous online survey, translated it into eight languages and circulated it widely in collaboration with PCD support groups worldwide and the European Lung Foundation.Results
The findings from the interviews identified key areas to be explored further through the survey including: developing treatments for PCD and increasing knowledge about different topics such as mental health, fertility, upper airway problems, treatment burden and impact of environment and lifestyle. 399 participants completed the online survey from 29 countries with median age 41 (IQR 33-49), 74% were female. 180 participants (45%) had participated in research before. For the remaining, the main reason for no participation was not being informed about studies (65%). 172 (43%) preferred regular research updates during a study. The top three ranked research priorities were (1) finding a cure to restore ciliary function; (2) developing treatments to improve lung function and reduce infections and mucus production; (3) finding the best way to manage the disease using existing medication. Other priorities were: involving more doctors and people with PCD in research, raising awareness of the condition and increasing knowledge about mental health and fertility.Conclusion
We found that people with PCD are motivated to participate in research when they are informed appropriately and invited. Their main research priorities relate to developing new treatments or improving the evidence base for existing treatments. Our findings will help the PCD research community to improve patient engagement in research and to draw common priorities together with the people who live with PCD and their families.
People living with chronic diseases can provide a unique perspective for research that often differs from that of healthcare professionals. This is particularly important in rare diseases like primary ciliary dyskinesia (PCD), with many knowledge gaps and limited research resources. We aimed to assess participation of patients and caregivers in PCD research and identify their research priorities in a mixed-method study.Methods
We conducted in-depth, semistructured interviews with adults and adolescents with PCD, and caregivers of children with PCD. After verbatim transcription and thematic analysis, we developed an anonymous online survey, translated it into eight languages and circulated it widely in collaboration with PCD support groups worldwide and the European Lung Foundation.Results
The findings from the interviews identified key areas to be explored further through the survey including: developing treatments for PCD and increasing knowledge about different topics such as mental health, fertility, upper airway problems, treatment burden and impact of environment and lifestyle. 399 participants completed the online survey from 29 countries with median age 41 (IQR 33-49), 74% were female. 180 participants (45%) had participated in research before. For the remaining, the main reason for no participation was not being informed about studies (65%). 172 (43%) preferred regular research updates during a study. The top three ranked research priorities were (1) finding a cure to restore ciliary function; (2) developing treatments to improve lung function and reduce infections and mucus production; (3) finding the best way to manage the disease using existing medication. Other priorities were: involving more doctors and people with PCD in research, raising awareness of the condition and increasing knowledge about mental health and fertility.Conclusion
We found that people with PCD are motivated to participate in research when they are informed appropriately and invited. Their main research priorities relate to developing new treatments or improving the evidence base for existing treatments. Our findings will help the PCD research community to improve patient engagement in research and to draw common priorities together with the people who live with PCD and their families.
Date Issued
2025-12-14
Publication Type
Article
Subject(s)
Subjects
Patient Outcome Assessment
•
Rare lung diseases
•
Surveys and Questionnaires
•
Systemic disease and lungs
Language(s)
en
Author(s)
Behan, Laura | |
Dexter, Katie | |
Dixon, Lucy | |
Lucas, Jane S | |
Additional Credits
Journal
BMJ Open Respiratory Research
Publisher
BMJ Publishing Group
ISSN
2052-4439
Access(Rights)
open.access