WS20.6 The use of LCI as an effective tool for monitoring clinical response to ivacaftor therapy in CF patients with at least one G551D-allele

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

“…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 , …”

“…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 ,…”

“…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 ,…”

“…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 , …”

“…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...…”