CFTR biomarkers: time for promotion to surrogate end-point?
| Autor: | Margarida D. Amaral, Lisa Kent, K. De Boeck, Kevin W Southern, François Vermeulen, G Rault, Peter G. Middleton, Inez Bronsveld, T. Nguyen-Khoa, H. R. De Jonge, Isabelle Fajac, Isabelle Sermet-Gaudelus, A Bot, M J Hug, Paola Melotti, M. Proesmans, Y B de Rijke, Michael Wilschanski, S Boerner, Steven M. Rowe, R.A. de Nooijer, Teresinha Leal, E de Wachter, Celeste Barreto, I Danner-Boucher, Nico Derichs, Jane C. Davies |
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| Přispěvatelé: | Gastroenterology & Hepatology, Clinical Chemistry, Faculty of Medicine and Pharmacy, Physiotherapy, Human Physiology and Anatomy, Clinical sciences, Pediatrics |
| Rok vydání: | 2013 |
| Předmět: |
Pulmonary and Respiratory Medicine
Oncology CFTR Biomarkers medicine.medical_specialty medicine.diagnostic_test biology Surrogate endpoint business.industry medicine.disease Cystic fibrosis Cystic fibrosis transmembrane conductance regulator Surgery Ivacaftor Clinical trial Internal medicine medicine biology.protein Biomarker (medicine) business Sweat test Reliability (statistics) medicine.drug |
| Zdroj: | European Respiratory Journal, 41(1), 203-216. European Respiratory Society |
| ISSN: | 0903-1936 |
| Popis: | In patients with cystic fibrosis, cystic fibrosis transmembrane conductance regulator (CFTR) biomarkers, such as sweat chloride concentration and/or nasal potential difference, are used as end-points of efficacy in phase-III clinical trials with the disease modifying drugs ivacaftor (VX-770), VX809 and ataluren. The aim of this project was to review the literature on reliability, validity and responsiveness of nasal potential difference, sweat chloride and intestinal current measurement in patients with cystic fibrosis. Data on clinimetric properties were collected for each biomarker and reviewed by an international team of experts. Data on reliability, validity and responsiveness were tabulated. In addition, narrative answers to four key questions were discussed and agreed by the team of experts. The data collected demonstrated the reliability, validity and responsiveness of nasal potential difference. Fewer data were found on reliability of sweat chloride concentration; however, validity and responsiveness were demonstrated. Validity was demonstrated for intestinal current measurement, but further information is required on reliability and responsiveness. For all three end-points, normal values were collected and further research requirements were proposed. This body of work adds useful information to support the promotion of CFTR biomarkers to surrogate end-points and to guide further research in the area. |
| Databáze: | OpenAIRE |
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