Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease

Martin, Clémence; Burnet, Espérie; Ronayette-Preira, A.; Carli, P.; Martín, J A Portillo; Delmas, L.; Prieur, Benoît E.; Burgel, Pierre‐Régis

doi:10.1016/j.resmer.2021.100829

Cited by 21 publications

(30 citation statements)

References 11 publications

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“…The clinical trials of ETI in pwCF who were F/F or heterozygous for F and MF, RF, or G involved patients with mild-moderate lung disease, 1-3 but improvements have also been obtained in pwCF with advanced lung disease, and these have often obviated the need for lung transplantation. [4][5][6][7][8] Our patient had an F/G genotype and had been previously treated with ivacaftor but, after an initial improvement, experienced rapid disease progression (as has been previously described in ivacaftor-treated adults with G mutations), 9 possibly aggravated by Bcc colonization. 10 During her subsequent hospitalization, she did not improve significantly until ETI was administered, and, as all her other treatments remained unchanged, this suggests it played a role in her recovery.…”

Section: Discussionmentioning

confidence: 97%

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Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype F508DEL/G1244E

Salvatore

Colangelo

D’Andria

et al. 2021

Clinical Case Reports

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Section: Discussionmentioning

confidence: 97%

“…The clinical trials of ETI in pwCF who were F / F or heterozygous for F and MF , RF , or G involved patients with mild‐moderate lung disease, 1‐3 but improvements have also been obtained in pwCF with advanced lung disease, and these have often obviated the need for lung transplantation 4‐8 …”

Section: Discussionmentioning

confidence: 99%

Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype F508DEL/G1244E

Salvatore

Colangelo

D’Andria

et al. 2021

Clinical Case Reports

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“…For these conditions, low R2 values may limit the strength of the interpretations related to exploratory variables. Finally, our study was conducted before the large use of CFTR modulators such as elexacaftor/tezecaftor/ivacaftor, which are associated with a dramatic improvement in respiratory-related quality of life [ 50 , 51 ].…”

Section: Discussionmentioning

confidence: 99%

Health-Related Quality of Life in Adults with Cystic Fibrosis: Familial, Occupational, Social, and Mental Health Predictors

et al. 2022

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Background: Cystic Fibrosis (CF) adult patients experience daily physical symptoms and disabilities that may impact their quality of life and mental health. Methods: This prospective study aimed to evaluate the relative contribution of the familial, occupational, and social environment, besides that of the main physical and mental health factors, to the quality of life of CF adult patients using the Cystic Fibrosis Questionnaire-Revised (CFQ-R) in a multivariate model. Results: Fifty patients were analyzed (70% of men; median age of 25 years; median body mass index of 21 kg/m²; median FEV1 of 57%). Anxiety and depression scores were negatively associated with 9 of the 12 CFQ-R domains. When controlling for anxiety and depression, FEV1% and BMI were significant positive predictors of several domains of the CFQ-R. All the familial, occupational, and social components analyzed but one (professional training) were predictors of at least one domain of the CFQ-R. Conclusion: Anxiety and depression explained a greater proportion of the variance than physical variables (age, sex, BMI, FEV1%, and exacerbation in the last year) in CF HRQoL. Many familial, occupational, and social components were also specifically and independently predictors of some HRQoL domains. Their screening might help identifying CF patients eligible for specific interventions, focusing on the impaired QoL dimensions.

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“…Martin et al (2021) explored the patient’s perspectives in 101 patients with advanced CF aged 12 years and older who were treated with ELX/TEZ/IVA, using a 13-item online questionnaire that included 4 open questions [ 45 ]. Their goal was to gain a better understanding of how patients perceive the changes in respiratory symptoms, systemic manifestations, treatment burden, and overall quality of life.…”

Section: Elexacaftor/tezacaftor/ivacaftormentioning

confidence: 99%

“…The authors reported that ELX/TEZ/IVA was associated with a significant improvement in respiratory symptoms, sleep quality, and physical self-esteem and with a reduction in treatment burden (chest physiotherapy, IV antibiotic courses, hospitalizations, diabetes control, concurrent treatments, lung transplant criteria). Furthermore, initiation of ELX/TEZ/IVA was associated with positive physical, psychological, and social effects, which translated into improved quality of life [ 45 ].…”

Section: Elexacaftor/tezacaftor/ivacaftormentioning

confidence: 99%

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

Regard

Martin

Burnet

et al. 2022

Cells

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Cystic fibrosis (CF) is a rare genetic multisystemic disease, the manifestations of which are due to mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein and can lead to respiratory insufficiency and premature death. CFTR modulators, which were developed in the past decade, partially restore CFTR protein function. Their clinical efficacy has been demonstrated in phase 3 clinical trials, particularly in terms of lung function and pulmonary exacerbations, nutritional status, and quality of life in people with gating mutations (ivacaftor), homozygous for the F508del mutation (lumacaftor/ivacaftor and tezacaftor/ivacaftor), and in those with at least one F508del mutation (elexacaftor/tezacaftor/ivacaftor). However, many questions remain regarding their long-term safety and effectiveness, particularly in patients with advanced lung disease, liver disease, renal insufficiency, or problematic bacterial colonization. The impact of CFTR modulators on other important outcomes such as concurrent treatments, lung transplantation, chest imaging, or pregnancies also warrants further investigation. The French CF Reference Network includes 47 CF centers that contribute patient data to the comprehensive French CF Registry and have conducted nationwide real-world studies on CFTR modulators. This review seeks to summarize the results of these real-world studies and examine their findings against those of randomized control trials.

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Patient perspectives following initiation of elexacaftor-tezacaftor-ivacaftor in people with cystic fibrosis and advanced lung disease

Cited by 21 publications

References 11 publications

Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype F508DEL/G1244E

Elexacaftor/tezacaftor/ivacaftor as rescue therapy in a patient with the cystic fibrosis genotype F508DEL/G1244E

Health-Related Quality of Life in Adults with Cystic Fibrosis: Familial, Occupational, Social, and Mental Health Predictors

CFTR Modulators in People with Cystic Fibrosis: Real-World Evidence in France

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