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

European Cystic Fibrosis Society-Clinical Trials Network Patient Advisory Group

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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

Bibliographical note

Funding Information:
The authors acknowledge the survey respondents and interview participants for sharing their responses and stories and the Paris-based CF Centres? personnel for their help in the recruitment process (H?pital Necker Enfants Malades, Centre Hospitalier Intercommunal de Cr?teil, H?pital Foch and H?pital Cochin); T Nguyen-Khoa; C-H Cottard and M. M Hautrive, H?pital Necker Enfants Malades. We would also like to thank the members of the ECFS CTN Patient Advisory Group for their expert advice and guidance throughout this study. This research did not receive any specific grant from funding agencies in the public, commercial, or not-for-profit sectors.

Funding Information:
RC, AC, VBontemps, EL, EB, VB, MK, FC, MB, HdeK, PdeC, KH and IH have no conflicts of interest. ISG has received grants and personal fees from Vertex Pharmaceuticals, personal fees from Eloxx, and non-financial support from PTC Therapeutics; P-RB has received personal fees from Astra Zeneca, Boehringer-Ingelheim, Chiesi, GSK, Insmed, Novartis and Pfizer and grant from GSK; DH has received personal fees from Vertex; CM personal fees from Chiesi and Zambon.

Publisher Copyright:
© 2021

Copyright 2021 Elsevier B.V., All rights reserved.


  • Cystic fibrosis
  • Patient reported outcome measures
  • Patient-led research

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Pulmonary and Respiratory Medicine


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