共查询到20条相似文献,搜索用时 15 毫秒
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Riad Salem Margaret Gilbertsen Zeeshan Butt Khairuddin Memon Michael Vouche Ryan Hickey Talia Baker Michael M. Abecassis Rohi Atassi Ahsun Riaz David Cella James L. Burns Daniel Ganger Al B. Benson III Mary F. Mulcahy Laura Kulik Robert Lewandowski 《Clinical gastroenterology and hepatology》2013,11(10):1358-1365
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Rodolfo J. Oviedo 《Methodist DeBakey Cardiovascular Journal》2012,8(3):49-Sep;8(3):49
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《Archivos de bronconeumología》2023,59(9):556-565
IntroductionElexacaftor/tezacaftor/ivacaftor (ETI) was used through the early access programme in Spain from December 2019 in cystic fibrosis (CF) patients with homozygous or heterozygous F508del mutation with advanced lung disease.MethodologyMulticentre, ambispective, observational, study in which 114 patients in follow-up in 16 national CF units were recruited. Clinical data, functional tests, nutritional parameters, quality of life questionnaires, microbiological isolates, number of exacerbations, antibiotic treatments and side effects were collected. The study also compared patients with homozygous and heterozygous F508del mutations.ResultsOf the 114 patients, 85 (74.6%) were heterozygous for F508del mutation, and the mean age was 32.2 ± 9.96 years. After 30 months of treatment, lung function measured by FEV1% showed improvement from 37.5 to 48.6 (p < 0.001), BMI increased from 20.5 to 22.3 (p < 0.001), and all isolated microorganisms decreased significantly. The total number of exacerbations was also significantly reduced from 3.9 (±2.9) to 0.9 (±1.1) (p < 0.001). All items in the CFQ-R questionnaire showed improvement, except for the digestive domain. Oxygen therapy use decreased by 40%, and only 20% of patients referred for lung transplantation remained on the active transplant list. ETI was well-tolerated, with only 4 patients discontinuing treatment due to hypertransaminemia.ConclusionsETI decreases the number of exacerbations, increases lung function and nutritional parameters, decrease in all isolated microorganisms, for 30 months of treatment. There is an improvement in the CFQ-R questionnaire score except for the digestive item. It is a safe and well-tolerated drug. 相似文献
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Brett R. Cowan Alistair A. Young Craig Anderson Robert N. Doughty Rungroj Krittayaphong Eva Lonn Thomas H. Marwick Chris M. Reid John E. Sanderson Roland E. Schmieder Koon Teo Angela K. Wadham Stephen G. Worthley Cheuk-Man Yu Salim Yusuf Garry L. Jennings ONTARGET Investigators 《The American journal of cardiology》2009,104(11):1484-1575
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Adrian M. Di Bisceglie Ewa Janczweska–Kazek François Habersetzer Wlodzimierz Mazur Carol Stanciu Vicente Carreno Coman Tanasescu Robert Flisiak Manuel Romero–Gomez Alexander Fich Vincent Bataille Myew–Ling Toh Marie Hennequi Patricia Zerr Géraldine Honnet Geneviève Inchauspé Delphine Agathon Jean–Marc Limacher Heiner Wedemeyer 《Gastroenterology》2014
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