Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation

Guimbellot, Jennifer S.; Baines, Arthur; Paynter, Alex; Heltshe, Sonya L.; VanDalfsen, Jill M; Jain, Manu; Rowe, Steven M.; Sagel, Scott D.

doi:10.1016/j.jcf.2020.11.008

Cited by 48 publications

(46 citation statements)

References 36 publications

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“…S. aureus and P. aeruginosa infections are associated with poor outcomes and increase treatment burden, often requiring repeated antibiotics courses [ 24 ]. Our findings are congruent with observations made in a recently published 5-year follow-up of a US cohort of pwCF treated with ivacaftor [ 25 ]; the current study enrolled a larger population, including children as young as 2 years of age, but had a shorter follow-up duration. Taken together, these results are highly clinically relevant and further support the benefits of ivacaftor and the potential to alter the clinical course of CF disease.…”

Section: Discussionsupporting

confidence: 92%

Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization

et al. 2021

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Introduction: Ivacaftor is a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator that has demonstrated clinical benefits in phase 3 trials. We report results from a real-world study (BRIO) to assess the effectiveness of ivacaftor in people with cystic fibrosis (pwCF) in France. Methods: BRIO was an observational study conducted at 35 centers in France. Both pwCF initiating ivacaftor treatment and those already taking ivacaftor were included and prospectively followed for 24 months. The primary objective was to evaluate the effect of ivacaftor on percent predicted forced expiratory volume in 1 s (ppFEV 1 ); secondary objectives were The members of the ''on behalf of the BRIO Study Group'' was processed under acknowledgements section.

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Section: Discussionsupporting

confidence: 92%

Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization

et al. 2021

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“…The strongest outcome was a 50% reduction in pulmonary exacerbations in both pre-and-post ivacaftor treatment [37]. Long-term data in a G551D population shows a sustainable effect on multiple outcome levels, including lung function, after 5.5 years of ivacaftor [38]. There is also evidence that treatment with ivacaftor has a positive effect on both insulin secretion in people with abnormal glucose tolerance and hepatic steatosis in people with CF-related liver disease [39,40].…”

Section: Symptomatic Treatmentmentioning

confidence: 99%

A new era for people with cystic fibrosis

et al. 2021

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Cystic fibrosis is the most prevalent inherited disease caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The impaired electrolyte homeostasis caused by the mutated or absent protein leads to symptoms in multiple organ systems. However, the pulmonary manifestation with chronic infections and eventually respiratory failure remains the most important threat. Until one decade ago, only symptomatic treatment was available. However, since 2012, different combinations of CFTR modulators are available for people with cystic fibrosis (pwCF) that carry different mutations. The advent of these drugs has impressively changed life expectancy and quality of life in people with cystic fibrosis and raised new challenges regarding long-term complications and tapering of conventional therapies.Conclusion: In this review, we provide an update on the latest developments around diagnostics, treatment, and prognosis of pwCF. What is Known:• Cystic fibrosis is an incurable and life-shortening disease asking for life-long symptomatic treatment.• Three combination CFTR modulating drugs has gained marked approval over the last 10 years. What is New:• The emerge of new (modulating) therapies contribute to the increasing life expectancy.• A high unmet need to develop new therapies for people with CF who cannot access or benefit from these drugs remains. This review gives an update on the current status.

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“…Life expectancy and clinical outcomes for patients with CF have significantly improved with widespread use of highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators, including the risk for undernutrition [6]. However, advancements in CF care have also led to significantly increased rates of overweight and obesity [13][14][15][16][17]. Additionally, the prevalence of non-pulmonary complications such as CFrelated diabetes (CFRD) continues to increase, particularly as the CF population ages [18,19].…”

Section: Introductionmentioning

confidence: 99%

The Relationship between Body Composition, Dietary Intake, Physical Activity, and Pulmonary Status in Adolescents and Adults with Cystic Fibrosis

et al. 2022

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Measures of body fat and lean mass may better predict important clinical outcomes in patients with cystic fibrosis (CF) than body mass index (BMI). Little is known about how diet quality and exercise may impact body composition in these patients. Dual X-ray absorptiometry (DXA) body composition, 24-h dietary recall, and physical activity were assessed in a cross-sectional analysis of 38 adolescents and adults with CF and 19 age-, race-, and gender-matched healthy volunteers. Compared with the healthy volunteers, participants with CF had a lower appendicular lean mass index (ALMI), despite no observed difference in BMI, and their diets consisted of higher glycemic index foods with a greater proportion of calories from fat and a lower proportion of calories from protein. In participants with CF, pulmonary function positively correlated with measures of lean mass, particularly ALMI, and negatively correlated with multiple measures of body fat after controlling for age, gender, and BMI. Higher physical activity levels were associated with greater ALMI and lower body fat. In conclusion, body composition measures, particularly ALMI, may better predict key clinical outcomes in individuals with CF than BMI. Future longitudinal studies analyzing the effect of dietary intake and exercise on body composition and CF-specific clinical outcomes are needed.

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Long term clinical effectiveness of ivacaftor in people with the G551D CFTR mutation

Cited by 48 publications

References 36 publications

Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization

Real-World Long-Term Ivacaftor for Cystic Fibrosis in France: Clinical Effectiveness and Healthcare Resource Utilization

A new era for people with cystic fibrosis

The Relationship between Body Composition, Dietary Intake, Physical Activity, and Pulmonary Status in Adolescents and Adults with Cystic Fibrosis

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