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

Autor: Coucke R; Service de Pneumologie et Allergologie Pédiatriques, Centre de Ressourcés et de Compétence de la Mucoviscidose, Hôpital Necker Enfants Malades 149 rue de Sévres, INSERM U1151, Institut Necker Enfants Malades, Université Paris Sorbonne, Paris 75743, France., Chansard A; CF Europe, Joseph Borlélaan 12, 1160 Brussels, Belgium., Bontemps V; Institut de Recherche Interdisciplinaire sur les Enjeux Sociaux, Campus Condorcet, Bâtiment Recherche Sud, 5 cours de Humanitiés, 93322 Aubervillers cedex, France., Grenet D; Hôpital Foch, 40 rue Worth, 92150 Suresnes, France., Hubert D; Hôpital Cochin, Assistance Publique Hôpitaux de Paris and Université de Paris, Institut Cochin, Inserm U1016, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France., Martin C; Hôpital Cochin, Assistance Publique Hôpitaux de Paris and Université de Paris, Institut Cochin, Inserm U1016, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France., Lammertyn E; CF Europe, Joseph Borlélaan 12, 1160 Brussels, Belgium., Bardin E; CF Europe, Joseph Borlélaan 12, 1160 Brussels, Belgium., Bulteel V; European Cystic Fibrosis Society, Karup, Denmark., Chedevergne F; Service de Pneumologie et Allergologie Pédiatriques, Centre de Ressourcés et de Compétence de la Mucoviscidose, Hôpital Necker Enfants Malades 149 rue de Sévres, INSERM U1151, Institut Necker Enfants Malades, Université Paris Sorbonne, Paris 75743, France., Bourgeois ML; Service de Pneumologie et Allergologie Pédiatriques, Centre de Ressourcés et de Compétence de la Mucoviscidose, Hôpital Necker Enfants Malades 149 rue de Sévres, INSERM U1151, Institut Necker Enfants Malades, Université Paris Sorbonne, Paris 75743, France., Burgel PR; Hôpital Cochin, Assistance Publique Hôpitaux de Paris and Université de Paris, Institut Cochin, Inserm U1016, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France., Honore I; Hôpital Cochin, Assistance Publique Hôpitaux de Paris and Université de Paris, Institut Cochin, Inserm U1016, 27 rue du Faubourg Saint-Jacques, 75014 Paris, France., de Keyser H; CF Europe, Joseph Borlélaan 12, 1160 Brussels, Belgium., Kirszenbaum M; Service de Pneumologie et Allergologie Pédiatriques, Centre de Ressourcés et de Compétence de la Mucoviscidose, Hôpital Necker Enfants Malades 149 rue de Sévres, INSERM U1151, Institut Necker Enfants Malades, Université Paris Sorbonne, Paris 75743, France., de Carli P; Association Vaincre la Mucoviscidose, 181 rue de Tolbiac, 75013 Paris, France., Sermet-Gaudelus I; Service de Pneumologie et Allergologie Pédiatriques, Centre de Ressourcés et de Compétence de la Mucoviscidose, Hôpital Necker Enfants Malades 149 rue de Sévres, INSERM U1151, Institut Necker Enfants Malades, Université Paris Sorbonne, Paris 75743, France., Hayes K; European Cystic Fibrosis Society, Karup, Denmark; Northern Ireland Clinical Research Facility, The Wellcome-Wolfson Centre for Experimental Medicine, Queen's University Belfast, 97 Lisburn Road, Belfast BT9 7BL, Ireland. Electronic address: k.hayes@qub.ac.uk.
Jazyk: angličtina
Zdroj: Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society [J Cyst Fibros] 2021 Nov; Vol. 20 (6), pp. e108-e113. Date of Electronic Publication: 2021 Feb 27.
DOI: 10.1016/j.jcf.2021.02.009
Abstrakt: 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.
Competing Interests: 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.
(Copyright © 2021. Published by Elsevier B.V.)
Databáze: MEDLINE
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