Titre
Impact of Elexacaftor-Tezacaftor-Ivacaftor on quality of life in children with cystic fibrosis
Type
mémoire de master/maîtrise/licence
Institution
UNIL/CHUV/Unisanté + institutions partenaires
Auteur(s)
KUMMERLI, S.
Auteure/Auteur
Directrices/directeurs
ROCHAT, I.
Directeur⸱rice
BLANCHON, S.
Codirecteur⸱rice
Liens vers les personnes
Liens vers les unités
Faculté
Université de Lausanne, Faculté de biologie et médecine
Statut éditorial
Accepté
Date de publication
2023
Nombre de pages
37
Langue
anglais
Résumé
Objectives: CFTR modulators have revolutionized the management of cystic fibrosis (CF) by targeting the defective protein rather than its consequences. The impact of CFTR modulators on quality of life
(QoL) has been studied in numerous trials using the CFQ-R (Cystic Fibrosis Questionnaire Revised), but very few data are available on QoL in children treated with Elexacaftor-Tezacaftor-Ivacaftor (ETI) given its recent authorization. We aimed to assess the impact of ETI on children's QoL.
Methods: This prospective observational study included children with CF (6-17 years) starting ETI treatment. QoL was assessed using the CFQ-R before (baseline) and 3 months after (M3) starting ETI, and its association with the patient's demographic, clinical, functional, microbiological, biological, and radiological characteristics was evaluated.
Results: We included 23 children (median IQR: 10.2 (5.9-17.2) years), including 13 (57%) with homozygous F508del genotype. At baseline, the low total score showed the clear impact of the
disease, notably regarding treatment burden in children’s questionnaires, whereas on weight/digestive/eating domains in caregivers’ questionnaires. At M3, patients showed a significant increase in QoL (p=0.0001), particularly regarding physical domains, when emotional/social/school domains showed hardly any improvement. Although QoL mean scores were comparable between children and parents groups, scores were poorly correlated within the same family. No studied covariates at baseline were correlated with the impact of ETI at M3, except homozygous F508del genotype associated with significantly better QoL improvement (p<0.001).
Conclusion: We reported a very significant impact of ETI on children's QoL, particularly in domains related to physical health. We stressed the importance of exploring other domains of QoL, that are
not as improved by ETI, in particular the psycho-social components. Finally, our results show the importance to consider independently the views of children and parents to get a holistic picture of their QoL.
(QoL) has been studied in numerous trials using the CFQ-R (Cystic Fibrosis Questionnaire Revised), but very few data are available on QoL in children treated with Elexacaftor-Tezacaftor-Ivacaftor (ETI) given its recent authorization. We aimed to assess the impact of ETI on children's QoL.
Methods: This prospective observational study included children with CF (6-17 years) starting ETI treatment. QoL was assessed using the CFQ-R before (baseline) and 3 months after (M3) starting ETI, and its association with the patient's demographic, clinical, functional, microbiological, biological, and radiological characteristics was evaluated.
Results: We included 23 children (median IQR: 10.2 (5.9-17.2) years), including 13 (57%) with homozygous F508del genotype. At baseline, the low total score showed the clear impact of the
disease, notably regarding treatment burden in children’s questionnaires, whereas on weight/digestive/eating domains in caregivers’ questionnaires. At M3, patients showed a significant increase in QoL (p=0.0001), particularly regarding physical domains, when emotional/social/school domains showed hardly any improvement. Although QoL mean scores were comparable between children and parents groups, scores were poorly correlated within the same family. No studied covariates at baseline were correlated with the impact of ETI at M3, except homozygous F508del genotype associated with significantly better QoL improvement (p<0.001).
Conclusion: We reported a very significant impact of ETI on children's QoL, particularly in domains related to physical health. We stressed the importance of exploring other domains of QoL, that are
not as improved by ETI, in particular the psycho-social components. Finally, our results show the importance to consider independently the views of children and parents to get a holistic picture of their QoL.
PID Serval
serval:BIB_8670D22A31A2
Date de création
2024-08-14T13:13:20.421Z
Date de création dans IRIS
2025-05-21T00:00:41Z
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Nom
Mémoire no 11094 Mme Kümmerli.pdf
Version du manuscrit
imprimatur
Taille
3.71 MB
Format
Adobe PDF
PID Serval
serval:BIB_8670D22A31A2.P001
Somme de contrôle
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