Positive and negative impacts of elexacaftor/tezacaftor/ivacaftor: Healthcare providers' observations across US centers

Bathgate, Christina J.; Muther, Emily F.; Georgiopoulos, Anna M.; Smith, Beth A.; Tillman, Laura; Graziano, Sonia; Verkleij, Marieke; Lomas, Paula; Quittner, Alexandra L.

doi:10.1002/ppul.26527

Cited by 15 publications

(7 citation statements)

References 38 publications

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“…Our results support the validity and reliability of the child‐ and parent‐report versions of the Spanish CFQ‐R, both of which showed acceptable internal consistency and stability over time (test−retest reliability), as well as adequate construct validity due to their relationship with another HRQoL questionnaire (KINDL) and with the participants' health status. Our study establishes the MDC 90 for each subscale of both versions, which helps determine the efficacy and potential adverse/secondary effects of various interventions to improve the quality of life of children with CF, such as the recently approved ETI therapy 6,39 . Based on the above, these Spanish versions of the CFQ‐R could be applied during routine medical check‐ups to monitor the quality of life and respiratory symptoms of children with CF in Spain, allowing changes to be detected and for appropriate early treatment to be applied 21,23 …”

Section: Discussionmentioning

confidence: 83%

“…16,20 Our results support the validity and reliability of the child-and parent-report versions of the Spanish which helps determine the efficacy and potential adverse/secondary effects of various interventions to improve the quality of life of children with CF, such as the recently approved ETI therapy. 6,39 Based on the above, these Spanish versions of the CFQ-R could be applied during routine medical check-ups to monitor the quality of life and respiratory symptoms of children with CF in Spain, allowing changes to be detected and for appropriate early treatment to be applied. 21,23…”

Section: Discussionmentioning

confidence: 99%

“…In fact, the only subscales in which a significant number of participants did not achieve the maximum score were those related to emotional/social aspects and respiratory symptoms, which could be the domains least likely to be affected by early CF detection 42 . With the use of new modulator therapy that virtually eliminates the consequences of CF, 6 however, this hypothesis could be tested in future studies.…”

Section: Discussionmentioning

confidence: 99%

“…5 To monitor CF progression, clinicians have numerous tools at their disposal, such as pulmonary function, exercise tolerance, and health-related quality-of-life (HRQoL) tests. Recent advances in the development of drugs and therapies for CF have improved the prognosis and quality of life for children with CF 6 ; standardized assessment tools are therefore essential for evaluating these changes. 7 Given that generic questionnaires designed to evaluate HRQoL are insufficiently sensitive in discriminating specific aspects of CF, such as the potential benefits of treatments and the impact of exacerbations or disease progression, there is a need for specific HRQoL questionnaires designed for patients with CF.…”

Section: Introductionmentioning

confidence: 99%

“…To monitor CF progression, clinicians have numerous tools at their disposal, such as pulmonary function, exercise tolerance, and health‐related quality‐of‐life (HRQoL) tests. Recent advances in the development of drugs and therapies for CF have improved the prognosis and quality of life for children with CF 6 ; standardized assessment tools are therefore essential for evaluating these changes 7 …”

Section: Introductionmentioning

confidence: 99%

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Spanish adaptation and validation of the child‐ and parent‐report cystic fibrosis questionnaire‐revised (CFQ‐R)

Blanco‐Orive,

del Corral,

Martín‐Casas

et al. 2023

Pediatric Pulmonology

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ObjectiveTo evaluate the psychometric properties of the Spanish versions of the child‐ and parent‐report cystic fibrosis questionnaire‐revised (CFQ‐R).MethodsA Spanish adaptation of the CFQ‐R was performed; 68 children with CF (6−13 years) and their parents completed the child‐ and parent‐report CFQ‐R, respectively, and the Revidierter KINDer Lebensqualitätsfragebogen (KINDL) questionnaire. The CFQ‐R was completed twice, 7−10 days apart, and its psychometric properties were analyzed.ResultsThe internal consistency of both CFQ‐R versions was adequate (child‐report version, Cronbach's α >.60 for all domains except “Treatment Burden” [α = .42] and “Social Functioning” [α = .57]; parent‐report version, α > .60 for all domains except “Social Functioning” [α = .58]). For the child‐report version, the lowest measurement error was for “Emotional Functioning” (standard error of measurement [SEM]: 8.3%; minimal detectable change [MDC90]: 19.3%), and the highest was for “Body Image” (SEM: 15%; MDC90: 35%). For the parent‐report version, the lowest measurement error was for “Physical Functioning” (SEM: 7.1%; MDC90: 16.5%), and the highest was for “Weight” (SEM: 17.2%; MDC90; 40.1%). The correlation between the versions showed higher agreement for the domains related to observable signs (“Physical Functioning”) and lower agreement for “Emotional Functioning.” There was a significant correlation between the CFQ‐R and KINDL.ConclusionBoth the child‐ and parent‐report versions of the Spanish CFQ‐R have adequate reliability and validity for clinical and research purposes. These versions can be administered before and after starting modulator therapy to assess its effect on daily functioning. The MDC90 can help identify, with a high probability, whether real changes have occurred in the quality‐of‐life subscales in children with CF.

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Section: Discussionmentioning

confidence: 83%

Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 3 more Smart Citations

Spanish adaptation and validation of the child‐ and parent‐report cystic fibrosis questionnaire‐revised (CFQ‐R)

Blanco‐Orive,

del Corral,

Martín‐Casas

et al. 2023

Pediatric Pulmonology

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Cystic fibrosis year in review 2023

Swetland,

Savant

2024

Pediatric Pulmonology

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This past year, there were many important advances for patients with cystic fibrosis (CF). Of the many publications related to CF in 2023, there was further evaluation of highly effective modulator therapy, new assessments and guidelines for clinical manifestations and therapies for CF, advances in newborn screening and diagnosis, and evaluation of outcomes for people with CF transmembrane conductance regulator‐related metabolic syndrome/CF screen positive, inconclusive diagnosis. The aim of this review article is not to provide a full assessment of the wide range of articles published in 2023, but to provide a brief review of publication that may lead to changes in clinical care.

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