Longitudinal Trends in Real-World Outcomes after Initiation of Ivacaftor. A Cohort Study from the Cystic Fibrosis Registry of Ireland

Kirwan, Laura; Fletcher, G.; Harrington, Mary; Jeleniewska, Paulina; Zhou, Shijun; Casserly, Brian; Gallagher, Charles G.; Greally, Peter; Gunaratnam, Cedric; Herzig, M.; Linnane, Barry; McElvaney, Noel G.; McKone, Edward F.; McNally, Paul; Mullane, D.; Chróinín, Muireann Ní; O’Mahony, Michael; Plant, Barry J.; Jackson, Abaigeal D.

doi:10.1513/annalsats.201802-149oc

Cited by 53 publications

(31 citation statements)

References 24 publications

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“…In Irish adults, mean ppFEV 1 % increased initially by 7.4% after ivacaftor initiation, but had a more rapid lung function decline than before initiation, with an absolute reduction in mean FEV 1 % predicted of −1.74% per annum thereafter. Slowing the decline in lung function may be difficult where lung disease is more advanced …”

Section: Discussionmentioning

confidence: 99%

Effectivenesss of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations

Salvatore

Carnovale²,

Iacotucci³

et al. 2019

Pediatric Pulmonology

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Background Ivacaftor is a significant innovation in the treatment of cystic fibrosis (CF) with gating mutations. A substantial percentage of patients with CF have severe lung involvement, but these patients are usually excluded from phase III clinical trials. Thus, the effectiveness of ivacaftor in this population has not been fully determined. Methods Data were collected from Italian CF centers with patients enrolled in an ivacaftor compassionate use programme (percent predicted [pp] forced expiratory volume in 1 second [FEV1] < 40%, or on lung transplant waiting list, or with a fast worsening trend of lung function). Data were collected for 1 year before and 1 year after ivacaftor commencement. Results Thirteen patients received ivacaftor for a median of 320 days. Mean (SD) ppFEV1 increased from 35.1% (14.3%) before treatment to 46.6% (18.8%) after 12 months of treatment (absolute increase 11.5%, relative increase 32.8%). Mean distance of the 6‐minute walking test improved significantly, from 535.1 m before to 611.6 m after 12 months of treatment (P = .002). The number of pulmonary exacerbations decreased significantly, from 57 during the year before ivacaftor to 28 in the year following ivacaftor (P = .0048). Five of the 13 patients (38.5%) had no exacerbations during the 12 months after starting ivacaftor. Median weight increased significantly, from 52.7 kg to 55.6 kg (P = .0031). Mean (SD) sweat chloride concentration decreased significantly, from 99.5 (22.8) mmol/L to 39.3 (15.8) mmol/L (P < .0001). No safety concerns were registered. Conclusions Ivacaftor was safe and effective in patients with CF with severe lung disease and non‐G551D gating mutations.

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Section: Discussionmentioning

confidence: 99%

Effectivenesss of ivacaftor in severe cystic fibrosis patients and non‐G551D gating mutations

Salvatore

Carnovale²,

Iacotucci³

et al. 2019

Pediatric Pulmonology

View full text Add to dashboard Cite

show abstract

“…Analyses of long‐term outcomes from registries around the globe gives further insight into modulator effects. Data from 80 patients in the CF Registry of Ireland, 1256 patients in the US CFFPR combined with 411 patients in the UK CF Registry, 57 patients in the French CF Registry, and a single center evaluation in 33 patients from Cork, Ireland shared outcomes for 1 to 3 years post ivacaftor approval in patients with a G551D mutation. Only the study using the CFFPR and UK CF Registry also included patients with other gating mutations in their analysis.…”

Section: Cftr Modulatorsmentioning

confidence: 99%

“…Only the study using the CFFPR and UK CF Registry also included patients with other gating mutations in their analysis. In Ireland, 14.8% of patients have a G551D mutation, and in Cork, 23% of patients have a G551D mutation. Methodology differed; the Cork study was prospective, and the others retrospective analyses of prospectively collected data.…”

Section: Cftr Modulatorsmentioning

confidence: 99%

“…Methodology differed; the Cork study was prospective, and the others retrospective analyses of prospectively collected data. Both the French and Irish CF Registry Studies compared pre and post ivacaftor use in patients, while the CFF PR and UK CFR compared those on ivacaftor to matched comparators for age, sex, and CFTR genotype severity, not on ivacaftor. The studies showed consistently favorable outcomes in FEV1pp, BMI, sweat chloride values, use of IV and oral antibiotics, and hospitalizations for pulmonary exacerbations.…”

Section: Cftr Modulatorsmentioning

confidence: 99%

“…Only the study using the CFFPR and UK CF Registry also included patients with other gating mutations in their analysis. In Ireland, 14.8% of patients have a G551D mutation, 26 and in Cork, 23% of patients 29 27 but not in the French CF Registry. 28 The US CFFPR/UK CFR demonstrated reductions in hepatobiliary complications but not gastrointestinal or pulmonary complications.…”

Section: Modulator Short and Long-term Effects On Outcomesmentioning

confidence: 99%

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