263 Comparison of FEV1 and BMI of the 4 common CF mutations in UAE

Redha, K.Y. Al; Shebani, Eyman; Nuaimi, Asma Al; Panickar, J.

doi:10.1016/s1569-1993(16)30501-x

Cited by 1 publication

(5 citation statements)

References 0 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…Five (9%) studies were multinational [ 28 , 32 , 33 , 39 , 40 , 89 ]. Thirteen (23%) studies had follow-up times of <12 months [ 38 , 69 , 72 , 78 , 79 , 82 , 84 , 86 , 87 , 88 , 89 , 94 , 102 ], 26 (46%) had follow-up times of 12–23 months [ 28 , 34 , 37 , 39 , 40 , 48 , 49 , 50 , 52 , 53 , 56 , 57 , 59 , 62 , 66 , 67 , 68 , 71 , 73 , 75 , 76 , 81 , 83 , 91 , 95 , 96 , 97 , 98 , 99 ], 14 (25%) had follow-up times of 24–59 months [ 31 , 33 , 51 , 54 , 55 , 61 , 70 , 74 ,…”

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%

“…Changes in BMI z -score with ivacaftor treatment in pwCF aged <20 years were reported in two multinational (i.e., VOCAL interim analysis [ 40 ], n = 71; US, Canada, Italy [ 72 ], n = 23), one national (BRIO interim analysis [ 53 ], n = 107), and one single-center study enrolling pediatric patients (United Arab Emirates [ 57 ], n = 12), with follow-up ranging from 3–12 months. A significant mean (SD) increase of 0.3 (0.4) points in BMI z -score from baseline to 3 months with ivacaftor treatment was reported in one study ( p < 0.01) [ 72 ]; a mean increase of 0.29–0.6 points from baseline to 12 months was reported in three studies, which was significant in two studies ( p < 0.0001 each), and significance was not reported in one study [ 40 , 53 , 57 ].…”

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

View full text Add to dashboard Cite

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.

show abstract