P204 An increase in weight and fat mass observed following five months of ivacaftor treatment plateaus at 24 months in adults with G551D-related cystic fibrosis

Tierney, Ann; King, Scott; Edgeworth, D.; Williams, E.; Finlayson, F.; Keating, D.; Clark, D.; Button, Brenda; Kotsimbos, Tom; Wilson, J.W.

doi:10.1016/s1569-1993(18)30499-5

Cited by 2 publications

(4 citation statements)

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“…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%

“…Across studies that evaluated within-group change, increases in weight [ 32 , 37 , 42 , 45 , 46 , 49 , 51 , 72 , 75 , 78 , 84 , 92 , 96 , 97 , 98 , 99 , 101 , 102 ] and BMI [ 30 , 32 , 34 , 37 , 40 , 41 , 42 , 44 , 45 , 46 , 47 , 53 , 58 , 64 , 66 , 69 , 71 , 72 , 73 , 75 , 79 , 83 , 86 , 92 , 94 , 95 , 96 , 98 , 99 , 101 ] were consistently noted following the initiation of ivacaftor therapy, starting as early as 1 month and improving over time as seen in studies with longer follow-up periods ( Figure 4 ). Weight increased from baseline by 0.8–2.8 kg at 1 month [ 42 , 45 , 46 , 96 ] and by 2.8–7.2 kg at 12 months following initiation of ivacaftor […”

Section: Resultsmentioning

confidence: 99%

“… Change in mean weight ( A ); 10 studies) and mean BMI ( B ); 17 studies) with ivacaftor treatment [ 30 , 34 , 37 , 40 , 42 , 44 , 45 , 46 , 49 , 51 , 53 , 58 , 66 , 69 , 71 , 72 , 73 , 75 , 78 , 83 , 86 , 92 , 96 , 98 , 99 , 101 ]. Note: Figure only includes studies reporting absolute change from baseline within the ivacaftor-treated cohort in BMI and/or weight at 1, 3, 6, 12, 24, 36, 48, and/or 60 month time points following ivacaftor treatment.…”

Section: Figurementioning

confidence: 99%

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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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Section: Resultsmentioning

confidence: 99%

Section: Resultsmentioning

confidence: 99%

Section: Figurementioning

confidence: 99%

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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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“…This prospective study assessed the outcome of IVA in G551D patients during five months and 24 months. They observed the consequences of gaining weight and BMI in five-months with a plateau level at 24 months of IVA treatment [ 30 ]. Another study, a clinical trial by Lacotuci et al in 2016, evaluated the IVA effects at six and 12 months of duration.…”

Section: Reviewmentioning

confidence: 99%

How Clinically Efficient Are Lumacaftor/Ivacaftor for Cystic Fibrosis Patients: An Updated Literature Review

Perveen¹,

Chaudhry²,

Albabtain³

et al. 2020

Cureus

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P204 An increase in weight and fat mass observed following five months of ivacaftor treatment plateaus at 24 months in adults with G551D-related cystic fibrosis

Cited by 2 publications

References 0 publications

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

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

How Clinically Efficient Are Lumacaftor/Ivacaftor for Cystic Fibrosis Patients: An Updated Literature Review

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