Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis

DiMango, Emily; Overdevest, Jonathan B.; Keating, Claire; Francis, Sarah Fracasso; Dansky, David; Gudis, David A.

doi:10.1016/j.jcf.2020.07.002

Cited by 69 publications

(72 citation statements)

References 18 publications

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“…This expands on one study reporting benefit of ELX/TEZ/IVA at three months, thus showing both early and durable response to therapy, with important implications for healthcare expenditures. 7 Overall, the cohort was representative of the CF population, with average pre-treatment SNOT-22 of 34.5, consistent with a diagnosis of CRS in the CF population. 9,10 By stratifying SNOT-22 scores into sub-scores, we highlight areas of greatest improvement (rhinologic, extra-rhinologic, and ear/face).…”

Section: Discussionmentioning

confidence: 54%

“…Limited data shows improved sinonasal outcomes with both ELX/TEZ/IVA and historic non-highly active CFTR modulators (eg, tezacaftor or lumacaftor [LUM] in combination with ivacaftor for DF508 homozygotes). 6,7 The aim of this study was to evaluate the effect of ELX/TEZ/IVA on sinonasal quality of life (QoL) as measured by the 22-item Sino-Nasal Outcome Test (SNOT-22) questionnaire 8 in a cohort of CF adults at the University of Pennsylvania. We hypothesized that initiation of ELX/TEZ/IVA would improve SNOT-22 scores, indicating enhanced sinonasal QoL.…”

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confidence: 99%

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Impact of novel CFTR modulator on sinonasal quality of life in adult patients with cystic fibrosis

Douglas

Civantos

Locke

et al. 2020

Int Forum Allergy Rhinol

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Section: Discussionmentioning

confidence: 54%

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confidence: 99%

Impact of novel CFTR modulator on sinonasal quality of life in adult patients with cystic fibrosis

Douglas

Civantos

Locke

et al. 2020

Int Forum Allergy Rhinol

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“…Although extensive research has been conducted to evaluate the benefit of highly effective CFTR modulators on pulmonary outcome metrics, there has been little evaluation of patient reported outcomes other than respiratory outcomes. Our previous work reported significant improvement in sinonasal quality of life as measured by the 22‐item Sino‐Nasal Outcome Test (SNOT‐22) for patients using this therapy 5 . Measures of health‐related quality of life (HRQoL) can identify benefits of new treatments that may not be reflected in objective measures such as pulmonary function, nasal endoscopy, and computer tomography (CT) imaging.…”

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confidence: 99%

Effect of highly effective modulator therapy on quality of life in adults with cystic fibrosis

DiMango

Spielman

Overdevest

et al. 2020

Int Forum Allergy Rhinol

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BackgroundElexacaftor/tezacaftor/ivacaftor is a highly effective modulator that improves function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, resulting in improved pulmonary function in patients with cystic fibrosis (CF). We hypothesize that improvements in lung function are associated with improvements in health‐related quality of life and sinonasal health. The aim of this study is to measure the effect of elexacaftor/tezacaftor/ivacaftor on patient‐reported sinonasal and overall quality of life, and to determine the relationship between changes in these 2 outcome measures.MethodsA prospective cohort study was conducted at an accredited adult CF care center. Participants completed the 22‐item Sino‐Nasal Outcome Test (SNOT‐22) and the Cystic Fibrosis Questionnaire–Revised (CFQ‐R), a validated patient‐reported outcome metric for CF patients, at baseline and at 3 months after initiation of elexacaftor/tezacaftor/ivacaftor.ResultsForty‐three individuals completed the study. There was significant improvement in nearly all domains of the SNOT‐22 and CFQ‐R after 3 months of therapy. SNOT‐22 improved from 34.8 to 24.4 (p = 0.000003). Mean baseline FEV‐1 improved from 65% to 76% predicted (p = 0.0000005). The greatest effect was seen in those participants previously taking modulator therapy. Linear regression between the change in SNOT‐22 individual domains and the CFQ‐R respiratory domain revealed the strongest correlation between the extranasal domain score and the respiratory domain of the CFQ‐R (R2 = 0.24).ConclusionCF patients taking elexacaftor/tezacaftor/ivacaftor experience a significant improvement in both sinonasal and health‐related quality of life.

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“…HRQoL has been also assessed by mean of the Sinonasal Outcome Test (SNOT-22) 11 a specialityspeci c questionnaire that covers a broad range of rhinologic and general health issues. This widely-used tool has is not speci c for the phenotype with NP and for its characteristics has been used to assess the presence and the severity of sino-nasal disorders in clinical conditions really different from CRSwNP: smell dysfunction 12 , sino-nasal symptoms in cystic brosis 13 , allergic rhinitis 14 , sleep apnea 15 , chronic obstructive pulmonary disease (COPD) 16 , hereditary haemorrhagic telangiectasia 17 , Wegener's granulomatosis 18 .…”

Section: Introductionmentioning

confidence: 99%

Development and validation of Nasal Polyposis Quality of Life Questionnaire (NPQ)

Baiardini

Paoletti

Mariani

et al. 2020

Preprint

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BACKGROUND: To date, no disease-speci c tool is available to assess the impact of Chronic Rhinosinusitis with Nasal Polyps (CRSwNP) on Health Related Quality of Life (HRQoL). Therefore, the purpose of this study was to develop and validate a questionnaire speci cally designed to this aim: the Nasal Polyposis Quality of Life questionnaire-NPQ. METHODS: According to the current guidelines, the development and validation of the NPQ occurred in two separate steps involving different groups of patients. RESULTS: In the development process of NPQ an initial list of items of 40 items was given to 60 patients with CRSwNP; the 27 most signi cant items were selected and converted into questions. The validation procedure involved 107 patients (mean age 52.9±12.4). NPQ revealed a ve-dimensional structure and high levels of internal consistency (Cronbach's alpha 0.95). Convergent validity (Spearman' coe cient r=0.75; p< 0.01), discriminant validity (sensitivity to VAS score), reliability in a sample of patients with a stable health status (Interclass Coe cient 0.882) were satisfactory. Responsiveness to clinical changes was accomplished. The minimal important difference was 7. CONCLUSIONS: NPQ is the rst questionnaire for the assessment of HRQoL in CRSwNP. Our results provide that the new tool is valid, reliable, and sensitive to individual changes.

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Effect of highly effective modulator treatment on sinonasal symptoms in cystic fibrosis

Cited by 69 publications

References 18 publications

Impact of novel CFTR modulator on sinonasal quality of life in adult patients with cystic fibrosis

Impact of novel CFTR modulator on sinonasal quality of life in adult patients with cystic fibrosis

Effect of highly effective modulator therapy on quality of life in adults with cystic fibrosis

Development and validation of Nasal Polyposis Quality of Life Questionnaire (NPQ)

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