"Il faut continuer à poser des questions" patient reported outcome measures in cystic fibrosis: An anthropological perspective

Rosa Coucke, Audrey Chansard, Véronique Bontemps, Dominique Grenet, Dominique Hubert, Clémence Martin, Elise Lammertyn, Emmanuelle Bardin, Veerle Bulteel, Frédérique Chedevergne, Muriel Le Bourgeois, Pierre-Régis Burgel, Isabelle Honore, Hilde de Keyser, Maya Kirszenbaum, Paola de Carli, Isabelle Sermet-Gaudelus, Kate Hayes*

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review


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.
Original languageEnglish
Number of pages6
JournalJournal of Cystic Fibrosis
Early online date27 Feb 2021
Publication statusEarly online date - 27 Feb 2021

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