Long-Term Follow-Up of Health-Related Quality of Life and Short-Term Intervention with CFTR Modulator Therapy in Adults with Cystic Fibrosis: Evaluation of Changes over Several Years with or without 33 Weeks of CFTR Modulator Therapy.

Autor:	Gruber W; Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.; Institute of Human Nutrition and Food Science, Christian-Albrechts-Universität zu Kiel, 24118 Kiel, Germany., Welsner M; Department of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany., Blosch C; Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany., Dillenhoefer S; Department of Pediatric Pulmonology, University Children's Hospital, Ruhr University Bochum, 44801 Bochum, Germany., Olivier M; Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany., Brinkmann F; Department of Pediatric Pulmonology, University Children's Hospital, Ruhr University Bochum, 44801 Bochum, Germany., Koerner-Rettberg C; Children's Hospital, Marienhospital Wesel, 46483 Wesel, Germany., Sutharsan S; Department of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany., Mellies U; Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany., Taube C; Department of Pulmonary Medicine, University Hospital Essen-Ruhrlandklinik, Adult Cystic Fibrosis Center, University of Duisburg-Essen, 45239 Essen, Germany., Stehling F; Pediatric Pulmonology and Sleep Medicine, Cystic Fibrosis Center, Children's Hospital, University of Duisburg-Essen, 45147 Essen, Germany.
Jazyk:	angličtina
Zdroj:	Healthcare (Basel, Switzerland) [Healthcare (Basel)] 2023 Oct 31; Vol. 11 (21). Date of Electronic Publication: 2023 Oct 31.
DOI:	10.3390/healthcare11212873
Abstrakt:	Background: Longitudinal data on changes in health-related quality of life (HRQoL) in adult people with cystic fibrosis (pwCF) and the longitudinal effects of Elexacaftor/Tezacaftor/Ivacaftor therapy (ETI) on HRQoL or HRQoL domains are currently scarce. This study aimed to investigate the effects of ETI on HRQoL and compare them with those of pwCF who did not receive highly effective CFTR modulators over a longer period. Methods: Baseline assessment and follow-up data for 5.6 years in pwCF with ( n = 21) and 6.5 years in pwCF without ( n = 6) ETI (≥18 years) were evaluated. The assessment of HRQoL and clinical parameters was identical at both time points. HRQoL was assessed using the CFQ-R, and clinical outcomes included BMI, ppFEV1, and FEV1 z-score. Results: ETI was found to improve all HRQoL domains at more than four points over time, and their increases were significant except for vitality, digestion, treatment burden, and social functioning ( p < 0.05). Without ETI, psychosocial domains remained almost constant, whereas most physical domains decreased over time. Conclusions: The results of the present study show that ETI therapy has a positive effect on HRQoL and clinical outcomes over time but not in pwCF without ETI treatment. Furthermore, our results suggest that disease progression over time affects the physical domains of HRQoL more than the psychosocial domains. Due to the small sample size and the heterogeneity of the study population (CFTR mutation genotype), the results should be interpreted with some caution. Competing Interests: The authors declare no conflict of interest.
Databáze:	MEDLINE
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