Challenges in the use of highly effective modulator treatment for cystic fibrosis

Ramos, Kathleen J.; Pilewski, Joseph M.; Taylor‐Cousar, Jennifer L.

doi:10.1016/j.jcf.2021.01.007

Cited by 25 publications

(25 citation statements)

References 57 publications

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“…We evaluated a series of potential risk factors for severe COVID-19 (defined by hospital admission) collected at baseline visit, including sex, age, pancreatic insufficiency, severely impaired respiratory function (FEV 1 as percent of predicted, FEVp < 40%), chronic P. aeruginosa infection, underweight, organ transplant, CF-related diabetes and liver disease, current medical treatment (oxygen therapy, CFTR modulators [ 8 ], inhaled steroids, dornase alfa and azithromycin).…”

Section: Methodsmentioning

confidence: 99%

Clinical course and risk factors for severe COVID-19 among Italian patients with cystic fibrosis: a study within the Italian Cystic Fibrosis Society

et al. 2021

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Purpose To describe the clinical course of COVID-19 in patients with cystic fibrosis (CF) and to identify risk factors for severe COVID-19. Methods We conducted a prospective study within the Italian CF Society. CF centers collected baseline and follow-up data of patients with virologically confirmed SARS-CoV-2 infection between March 2020 and June 2021. Odds ratios (ORs) for severe SARS-CoV-2 (as defined by hospital admission) were estimated by logistic regression models. Results The study included 236 patients with positive molecular test for SARS-CoV-2. Six patients died, 43 patients were admitted to hospital, 4 admitted to intensive care unit. Pancreatic insufficiency was associated with increased risk of severe COVID-19 (OR 4.04, 95% CI 1.52; 10.8). After adjusting for age and pancreatic insufficiency, forced expiratory volume in one second (FEVp) < 40% (OR 4.54, 95% CI 1.56; 13.2), oxygen therapy (OR 12.3, 95% CI 2.91–51.7), underweight (OR 2.92, 95% CI 1.12; 7.57), organ transplantation (OR 7.31, 95% CI 2.59; 20.7), diabetes (OR 2.67, 95% CI 1.23; 5.80) and liver disease (OR 3.67, 95% CI 1.77; 7.59) were associated with increased risk of severe COVID-19, while use of dornase alfa was associated with a reduced risk (OR 0.34, 95% CI 0.13–0.88). No significant changes were observed in FEVp from baseline to a median follow-up of 2 months (median difference: 0, interquartile range: − 4; 5, P = 0.62). Conclusion Clinical features indicative of severe form of CF are associated with increased risk of COVID-19 hospitalization. SARS-CoV-2 infected patients do not experience a deterioration of respiratory function. Supplementary Information The online version contains supplementary material available at 10.1007/s15010-021-01737-z.

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Section: Methodsmentioning

confidence: 99%

Clinical course and risk factors for severe COVID-19 among Italian patients with cystic fibrosis: a study within the Italian Cystic Fibrosis Society

et al. 2021

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“…The authors point out the safe usage of CFTR modulators in their small patient cohort and the extrapulmonary benefits seen; however, Hayes et al also highlight disadvantages of the CFTR modulator usage post lung transplantation including drug–drug interactions, the need for drug dosage adjustments, and treatment costs [ 94 ]. Ramos et al, in a recently published review article, discuss additional potential benefits of CFTR modulator therapy following lung transplantation [ 95 ]. The authors focus on the multifactorial development of CLAD post lung transplant and the extrapulmonary manifestations of CF that potentially contribute to CLAD development such as gastroesophageal reflux disease and CF-associated chronic sinus disease [ 95 ].…”

Section: The Potential Role Of Cftr Modulators Post Lung Transplantationmentioning

confidence: 99%

“…Ramos et al, in a recently published review article, discuss additional potential benefits of CFTR modulator therapy following lung transplantation [ 95 ]. The authors focus on the multifactorial development of CLAD post lung transplant and the extrapulmonary manifestations of CF that potentially contribute to CLAD development such as gastroesophageal reflux disease and CF-associated chronic sinus disease [ 95 ]. Further, as there is evidence that the CFTR protein works as a tumor suppressor, improvement in CFTR function could potentially reduce the risk of malignancies following lung transplantation [ 95 ].…”

Section: The Potential Role Of Cftr Modulators Post Lung Transplantationmentioning

confidence: 99%

“…The authors focus on the multifactorial development of CLAD post lung transplant and the extrapulmonary manifestations of CF that potentially contribute to CLAD development such as gastroesophageal reflux disease and CF-associated chronic sinus disease [ 95 ]. Further, as there is evidence that the CFTR protein works as a tumor suppressor, improvement in CFTR function could potentially reduce the risk of malignancies following lung transplantation [ 95 ]. Based on the current published literature, a recent CF Foundation consensus statement found a lack of evidence to recommend for or against the use of CFTR modulators in patients after lung transplantation [ 78 ].…”

Section: The Potential Role Of Cftr Modulators Post Lung Transplantationmentioning

confidence: 99%

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CFTR Modulator Therapy and Its Impact on Lung Transplantation in Cystic Fibrosis

Benden

Schwarz²

2021

Pulm Ther

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Cystic fibrosis (CF) is the most common autosomal recessive disorder in Caucasian people and is caused by mutations in the gene encoding for the CF transmembrane conductance regulator (CFTR) protein. It is a multisystem disorder; however, CF lung disease causes most of its morbidity and mortality. Although survival for CF has improved over time due to a multifaceted symptomatic management approach, CF remains a life-limiting disease. For individuals with progressive advanced CF lung disease (ACFLD), lung transplantation is considered the ultimate treatment option if compatible with goals of care. Since 2012, newer drugs, called CFTR modulators, have gradually become available, revolutionizing CF care, as these small-molecule drugs target the underlying defect in CF that causes decreased CFTR protein synthesis, function, or stability. Because of their extremely high efficacy and overall respectable tolerability, CFTR modulator drugs have already proven to have a substantial

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“…In recent years targeted therapy in the form of CFTR modulators has revolutionized the treatment of patients with CF. Oral, small molecules were developed that target the CFTR protein and have proven to be clinically successful in correcting the defect of the CFTR protein in vivo [29]. They are extremely efficacious and are transforming the care of patients living with CF -a chronic, progressive, multiorgan disease-in an unprecedented way.…”

Section: Cftr Modulators -Targeted Treatment In Cf Diseasementioning

confidence: 99%

Introductory Chapter: Basics of Cystic Fibrosis

Mohite¹,

Gerovasili²

2021

Cystic Fibrosis - Facts, Management and Advances

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Challenges in the use of highly effective modulator treatment for cystic fibrosis

Cited by 25 publications

References 57 publications

Clinical course and risk factors for severe COVID-19 among Italian patients with cystic fibrosis: a study within the Italian Cystic Fibrosis Society

Clinical course and risk factors for severe COVID-19 among Italian patients with cystic fibrosis: a study within the Italian Cystic Fibrosis Society

CFTR Modulator Therapy and Its Impact on Lung Transplantation in Cystic Fibrosis

Introductory Chapter: Basics of Cystic Fibrosis

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