P041 Correlation of improvements in sweat chloride and percent predicted FEV1 across twenty studies examining different corrector therapies for F508del
نویسندگان
چکیده
Objectives: Cystic fibrosis is caused by a variant in the cystic transmembrane conductance regulator protein, of which class II variant, F508del, commonest. A number therapies aiming to correct F508del defect have been assessed clinical trials using change FEV1 and sweat chloride as outcome measures. We aim establish if there correlation between changes studies included Cochrane systematic review evaluating corrector for people with variants. Methods: Clinical most recent update examining therapy variants were identified. selected that reported absolute from baseline percent predicted (ppFEV1) chloride. analysed data identify correlations across therapies. Results: Twenty different identified range interventions (1 monotherapy, 9 dual 10 triple trials). The ppFEV1 each trial ranged –1.64% 13.9% mean 8.2 mmol/L –51.4 mmol/L. There was clear relationship magnitude these outcomes, although individual patient not available better confirm relationship. Conclusion: These enrolled numbers it possible correlate parameters without data. Despite this, aggregate lung function therapy, contrast reports other modulators. Individual being sought characterise this relationship, but support validity measurement surrogate measure respect therapy.
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15 صفحه اولA chemical corrector modifies the channel function of F508del-CFTR.
The deletion of Phe-508 (F508del) constitutes the most prevalent cystic fibrosis-causing mutation. This mutation leads to cystic fibrosis transmembrane conductance regulator (CFTR) misfolding and retention in the endoplasmic reticulum and altered channel activity in mammalian cells. This folding defect can however be partially overcome by growing cells expressing this mutant protein at low (27 ...
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ژورنال
عنوان ژورنال: Journal of Cystic Fibrosis
سال: 2023
ISSN: ['1569-1993', '1873-5010']
DOI: https://doi.org/10.1016/s1569-1993(23)00416-2