Research priorities in Primary Ciliary Dyskinesia - patients' perspective

Lam, Yin Ting; Behan, L; Dexter, K; Dixon, L; Kuehni, C E; Pedersen, E S; Lucas, J; Goutaki, M

doi:10.1183/13993003.congress-2022.1069

Cited by 1 publication

(1 citation statement)

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“…Since paediatric pulmonologists represented only half of survey participants, it is noteworthy our study results include perspectives from other disciplines. Our study deliberately focused on healthcare professionals and researchers; we did not include people with PCD or parents of affected children-a separate, dedicated study focusing on patient and family perspectives regarding PCD research is ongoing [38]. Another limitation includes only one person coding all interviews.…”

Section: Discussionmentioning

confidence: 99%

Priorities and barriers for research related to primary ciliary dyskinesia

Goutaki,

Lam,

Rubbo

et al. 2024

Preprint

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BackgroundDespite advances in primary ciliary dyskinesia (PCD) research, many questions remain; diagnosis is complex and no disease specific therapies exist. Using a mixed-methods approach, we aimed to identify priorities for clinical and epidemiological research and explore barriers to research.MethodsTo obtain rich, relevant, diverse data, we performed in-depth semi-structured interviews with PCD specialists selected using purposive sampling. We transcribed, coded, and analysed interview data using thematic analysis. Based on interview themes we identified, we developed an anonymous survey and circulated it widely through the BEAT-PCD network.ResultsWe interviewed 28 participants from 15 countries across different disciplines and expertise levels. The main themes identified as priorities for PCD research were improving diagnosis, understanding prevalence, and disease course; phenotypic variability; disease monitoring; treatment strategies; clinical trial endpoints; and neglected research areas. In total, 136 participants (49% paediatric pulmonologists) from 36 countries completed the survey. Most commonly reported barriers for research were low awareness about PCD and difficulties securing funding—in more than one-third of cases, participants reported undertaking predominantly unfunded research. Research questions ranked highest included priorities related to further improving diagnosis, treating PCD, managing upper and lower airway problems, and studying clinical variability and disease prognosis.ConclusionWe need to overcome barriers of limited funding and low awareness and promote collaborations between centres, disciplines, experts, and patients to address PCD priorities effectively. Our results contribute to the ongoing efforts of guiding the use of existing limited research resources and setting up a roadmap for future research activities.Take home messageOur study defined PCD research priorities including improving diagnosis, treatments, managing upper and lower airway disease, and understanding prognosis. Key barriers identified include low disease awareness and limited funding opportunities.

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Section: Discussionmentioning

confidence: 99%