"Il faut continuer à poser des questions" patient reported outcome measures in cystic fibrosis: An anthropological perspective.
Cystic fibrosis
Patient reported outcome measures
Patient-led research
Journal
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society
ISSN: 1873-5010
Titre abrégé: J Cyst Fibros
Pays: Netherlands
ID NLM: 101128966
Informations de publication
Date de publication:
11 2021
11 2021
Historique:
received:
20
10
2020
revised:
11
01
2021
accepted:
17
02
2021
pubmed:
3
3
2021
medline:
10
2
2022
entrez:
2
3
2021
Statut:
ppublish
Résumé
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. 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 Cystic Fibrosis Questionnaire-Revised (CFQ-R). Survey results from 33 countries revealed over 70 tools used in routine clinical practice, utilized by clinical specialists (n=124), pwCF/parents/carers (n=49) and other allied health professionals (n=60). The 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. Patient-centeredness is paramount in development of an up-to-date PROM in the era of novel therapies.
Sections du résumé
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 Cystic Fibrosis Questionnaire-Revised (CFQ-R).
RESULTS
Survey results from 33 countries revealed over 70 tools used in routine clinical practice, utilized by clinical specialists (n=124), pwCF/parents/carers (n=49) and other allied health professionals (n=60). The 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.
Identifiants
pubmed: 33648900
pii: S1569-1993(21)00044-8
doi: 10.1016/j.jcf.2021.02.009
pii:
doi:
Types de publication
Journal Article
Langues
eng
Sous-ensembles de citation
IM
Pagination
e108-e113Investigateurs
Jutta Bend
(J)
Claire Bresnihan
(C)
Anne Calvert
(A)
Anna Fonts
(A)
Andreas Hager
(A)
Maxime Hautrive
(M)
Trudy Havermans
(T)
Diana Hofmann
(D)
Informations de copyright
Copyright © 2021. Published by Elsevier B.V.
Déclaration de conflit d'intérêts
Declaration of Competing Interest 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.