Background: People with cystic fibrosis (pwCF) are central in the development of patient-led assessment tools. Qualitative analysis of a frequently used CF-specific patient-reported outcome measure (PROM) sought patient recommendations for development of a new quality of life (QoL) tool. Methods: We performed an inventory of PROMs, symptom-report and QoL tools used in clinical trials within the European Cystic Fibrosis Society Clinical Trial Network (ECFS-CTN) and in routine clinical practice among Cystic Fibrosis Europe and ECFS members. A qualitative study using cognitive interviews with PWCF and their caregivers reviewed the Cystic Fibrosis Questionnaire (CFQ), the French initial form of the CFQ-R.Results: Survey results from 33 countries revealed over 70 tools used in routine clinical practice, utilized by clinical specialists (124), PWCF/parents/carers (49) and other allied health professionals (60). The Cystic Fibrosis Questionnaire-Revised (CFQ-R) was the main PROM used in clinical trials. The qualitative study enrolled 99 PWCF, 6 to 11 years (n=31); 12 to 18 years (n=38); >18 years (n=30) and 26 parents. Inductive thematic analysis based on the CFQ, revealed 19 key themes. Themes common across all cohorts included burden of treatment, impact of disease on day-to-day life, relationships/family, stress/mood, and nutrition. Themes unique to individual groups included, treatment when not symptomatic for the paediatric group; education/studies and planning for the future for adolescents, impact of anxiety and depression on day-to-day life for adults, and for parents, questions addressing anxiety and their role as carers.Conclusions: Patient-centeredness is paramount in development of an up-to-date PROM in the era of novel therapies.
|Number of pages||6|
|Journal||Journal of Cystic Fibrosis|
|Early online date||27 Feb 2021|
|Publication status||Early online date - 27 Feb 2021|