Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST)

McKone, Edward F.; Borowitz, Drucy; Dřevı́nek, Pavel; Griese, Matthias; Konstan, Michael W.; Wainwright, Claire; Ratjen, Félix; Sermet‐Gaudelus, Isabelle; Plant, Barry J.; Munck, À.; Jiang, Ying; Gilmartin, Geoffrey; Davies, Jane C.

doi:10.1016/s2213-2600(14)70218-8

Cited by 198 publications

(163 citation statements)

References 23 publications

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“…In clinical studies, ivacaftor administration led to improvement in pulmonary function and nutritional status and to lower sweat chloride concentrations. Fewer hospital stays, pulmonary exacerbations and antibiotic use were also observed 6,7 . However, this revolutionary drug faces one very serious drawback.…”

Section: Introductionmentioning

confidence: 91%

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Fila¹,

Bartakova²,

Grandcourtova³

et al. 2016

BIOMED PAP

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Aims. Ivacaftor is a revolutionary treatment option for cystic fibrosis (CF) patients with G551D and other gating mutations. The aim of this study was to evaluate the clinical status of patients on ivacaftor who were followed for up to 6 years together with an evaluation of ivacaftor therapy in one patient with an initial FEV 1 less than 40% of predicted value. Methods. Data on development of clinical status and sinopulmonary-related therapies were obtained from patient health records during ivacaftor treatment lasting for up to six years and were compared with an equivalent period before ivacaftor administration. Results. Five CF adults with a median age 28.6 years (range 21.4−35.6 years) with median FEV 1 45% pred. (range 16−85% pred.) were included in the study. Four subjects were also participants in the STRIVE and PERSIST studies. Altogether, twenty-four patient-years of ivacaftor treatment were analyzed. The median FEV 1 decline per year decreased from -4.5 to -0.9% pred. (P = 0.043). Reduction in number of days on antibiotic treatment and hospital stays was 21% (P < 0.001) and 75% (P = 0.003), respectively. Improvement and stabilization of lung function was observed for up to six years of treatment. In a patient with severe airway obstruction, an increase in the FEV 1 value (30.4% from baseline) was documented during the first twelve months of treatment. Conclusion. Ivacaftor therapy resulted in improved and stabilized lung function in up to six years of treatment with a reduction in number of days on antibiotic treatment and hospital stays. Its efficiency was also displayed in a patient with severe airway obstruction.

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Section: Introductionmentioning

confidence: 91%

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Fila¹,

Bartakova²,

Grandcourtova³

et al. 2016

BIOMED PAP

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“…In our analysis, comparison with patients in the placebo group and the use of z-scores in children to normalize gains relative to what is expected for age enables us to demonstrate a true BMI body mass index, ppFEV 1 percent predicted forced expiratory volume in 1 s, SD standard deviation Dig Dis Sci effect of ivacaftor on growth in children and teens across various pubertal stages. Subsequent observation of the long-term effects of ivacaftor on weight indicates that children continued to gain weight during a 144-week observation period, while adults do not continue to gain after the initial improvement, as would be expected in healthy individuals who have reached optimal nutritional status [12]. Prepubertal children and teens would also be expected to gain height, which was not seen during this study.…”

Section: Discussionmentioning

confidence: 57%

Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor

et al. 2015

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“…It is still unclear when the improvements in survival will plateau. The very encouraging results from corrector and corrector/potentiator combination therapy indicates that cystic fibrosis transmembrane conductance regulator (CFTR) is a "druggable" target [11][12][13]. If started early in life, these treatments could prevent the development of severe lung disease and postpone morbidity and result in further improvements in survival in the future [14].…”

Section: Forecasting Of Numbers Of Adults With Cfmentioning

confidence: 99%

Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis

et al. 2015

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The improved survival in people with cystic fibrosis has led to an increasing number of patients reaching adulthood. This trend is likely to be maintained over the next decades, suggesting a need to increase the number of centres with expertise in the management of adult patients with cystic fibrosis. These centres should be capable of delivering multidisciplinary care addressing the complexity of the disease, in addition to addressing the psychological burden on patients and their families. Further issues that require attention are organ transplantation and end of life management.Lung disease in adults with cystic fibrosis drives most of the clinical care requirements, and major lifethreatening complications, such as respiratory infection, respiratory failure, pneumothorax and haemoptysis, and the management of lung transplantation require expertise from trained respiratory physicians. The taskforce therefore strongly reccommends that medical leadership in multidisciplinary adult teams should be attributed to a respiratory physician adequately trained in cystic fibrosis management.The task force suggests the implementation of a core curriculum for trainees in adult respiratory medicine and the selection and accreditation of training centres that deliver postgraduate training to the standards of the HERMES programme. @ERSpublications Respiratory physicians, trained in adult CF management, should lead multidisciplinary teams taking care of patients http://ow.ly/SgRgj

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Long-term safety and efficacy of ivacaftor in patients with cystic fibrosis who have the Gly551Asp-CFTR mutation: a phase 3, open-label extension study (PERSIST)

Cited by 198 publications

References 23 publications

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Ivacaftor in cystic fibrosis adults: Czech experience with six years of follow-up

Nutritional Status Improved in Cystic Fibrosis Patients with the G551D Mutation After Treatment with Ivacaftor

Report of the European Respiratory Society/European Cystic Fibrosis Society task force on the care of adults with cystic fibrosis

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