IPD2.04 Long-term effects of ivacaftor in patients with G551D mutation and mild lung disease

Ellemunter, Helmut; Hindinger, Claudia; Steinkamp, G.

doi:10.1016/s1569-1993(18)30288-1

Cited by 1 publication

(5 citation statements)

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“…Fifteen (26%) studies were from the UK [ 29 , 30 , 31 , 55 , 58 , 59 , 60 , 69 , 70 , 74 , 78 , 82 , 83 , 87 , 90 , 91 , 93 , 100 ], 13 (23%) from the US [ 29 , 30 , 35 , 38 , 41 , 42 , 43 , 44 , 45 , 46 , 47 , 48 , 49 , 50 , 56 , 67 , 68 , 72 , 75 , 98 , 99 ], 17 (30%) from non-UK European countries [ 34 , 51 , 52 , 53 , 54 , 61 , 62 , 63 , 64 , 65 , 66 , 71 , 77 , 79 , 84 , 86 , 94 , 95 , …”

Section: Resultsmentioning

confidence: 99%

“…Thirty-one (54%) studies included both adult and pediatric populations [ 28 , 29 , 30 , 38 , 39 , 40 , 41 , 42 , 43 , 44 , 45 , 46 , 48 , 49 , 50 , 51 , 52 , 53 , 54 , 56 , 62 , 66 , 68 , 69 , 72 , 75 , 76 , 77 , 79 , 80 , 81 , 86 , 87 , 88 , 89 , 90 , 92 , 96 , 98 , 99 , 100 ], whereas 16 (28%) studies included only adult populations [ 32 , 33 , 36 , 37 , 58 , 60 , 61 , 70 , 71 , 74 , 82 , 83 , 84 , 85 , 94 , 95 ,…”

Section: Resultsmentioning

confidence: 99%

“…There were five (18%) studies that did not report the age of the included population [ 31 , 55 , 73 , 78 , 91 ]. Thirty-nine (68%) studies were restricted to pwCF who had a CFTR gating mutation [ 28 , 31 , 32 , 33 , 36 , 37 , 39 , 40 , 43 , 44 , 45 , 47 , 49 , 51 , 52 , 53 , 54 , 57 , 58 , 59 , 60 , 61 , 62 , 63 , 64 , 65 , 66 , 69 , 70 , 72 , 74 , 76 , 77 , 78 , 79 , 80 , 81 , 83 , 84 , 85 , 86 , 87 , 88 , 89 , 90 , 92 , 93 , 96 , 97 , 98 , 99 , …”

Section: Resultsmentioning

confidence: 99%

“…Improved lung function, as indicated by increases in ppFEV 1 , following ivacaftor treatment was reported in studies evaluating within-group change. Increases were observed as early as 1 month after initiation of ivacaftor compared to baseline, with ppFEV 1 generally remaining above baseline through all time points reported, regardless of study design and patient characteristics ( Figure 3 ) [ 30 , 32 , 34 , 37 , 40 , 41 , 42 , 44 , 45 , 47 , 49 , 51 , 53 , 54 , 55 , 57 , 58 , 61 , 62 , 64 , 66 , 67 , 71 , 72 , 73 , 74 , 75 , 76 , 77 , 78 , 79 , 80 , 82 , 83 , 84 , 85 , 86 , 87 , 88 , 91 , 92 , 93 , 94 , 95 , 96 , 97 , 98 , 99 , 102 ]. Specifically, in the majority of studies reporting 12 months of follow-up, the mean absolute change in ppFEV 1 from baseline with ivacaftor tr...…”

Section: Resultsmentioning

confidence: 99%

“… Change in mean ppFEV 1 with ivacaftor treatment (31 studies) [ 30 , 40 , 41 , 42 , 44 , 45 , 47 , 49 , 53 , 54 , 57 , 58 , 62 , 66 , 72 , 73 , 74 , 75 , 76 , 77 , 78 , 82 , 83 , 86 , 87 , 91 , 92 , 93 , 95 , 98 , 99 ]. Note: Figure only includes studies reporting absolute change from baseline within the ivacaftor-treated cohort in ppFEV 1 at 1, 3, 6, 12, 24, 36, 48, and/or 60 month time points following ivacaftor treatment.…”

Section: Figurementioning

confidence: 99%

See 4 more Smart Citations

Real-World Outcomes of Ivacaftor Treatment in People with Cystic Fibrosis: A Systematic Review

Duckers

Lesher

Thorat

et al. 2021

JCM

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Cystic fibrosis (CF) is a rare, progressive, multi-organ genetic disease. Ivacaftor, a small-molecule CF transmembrane conductance regulator modulator, was the first medication to treat the underlying cause of CF. Since its approval, real-world clinical experience on the use of ivacaftor has been documented in large registries and smaller studies. Here, we systematically review data from real-world observational studies of ivacaftor treatment in people with CF (pwCF). Searches of MEDLINE and Embase identified 368 publications reporting real-world studies that enrolled six or more pwCF treated with ivacaftor published between January 2012 and September 2019. Overall, 75 publications providing data from 57 unique studies met inclusion criteria and were reviewed. Studies reporting within-group change for pwCF treated with ivacaftor consistently showed improvements in lung function, nutritional parameters, and patient-reported respiratory and sino-nasal symptoms. Benefits were evident as early as 1 month following ivacaftor initiation and were sustained over long-term follow-up. Decreases in pulmonary exacerbations, Pseudomonas aeruginosa prevalence, and healthcare resource utilization also were reported for up to 66 months following ivacaftor initiation. In studies comparing ivacaftor treatment to modulator untreated comparator groups, clinical benefits similarly were reported as were decreases in mortality, organ-transplantation, and CF-related complications. The safety profile of ivacaftor observed in these real-world studies was consistent with the well-established safety profile based on clinical trial data. Our systematic review of real-world studies shows ivacaftor treatment in pwCF results in highly consistent and sustained clinical benefit in both pulmonary and non-pulmonary outcomes across various geographies, study designs, patient characteristics, and follow-up durations, confirming and expanding upon evidence from clinical trials.

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