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The Ghent Marfan Trial - A randomized, double-blind placebo controlled trial with losartan in Marfan patients treated with beta-blockers
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| Abstract |
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| Background: Aortic root dilation, dissection and rupture are major clinical problems in Marfan syndrome (MFS). Although beta-blockers remain the standard of preventive treatment, preliminary results from animal studies and a selected group of severely affected MFS children show significant benefit from treatment with losartan, an angiotensin II receptor blocker with TGF-beta inhibiting potential. Large-scale human trials are now needed to confirm these results. This trial aims to evaluate the combined effect of both drugs. Methods: We are conducting a prospective randomized placebo controlled double blind phase III study aiming to include 174 MFS patients (age >= 10 years and z-score >= 2). Patients already taking beta-blockers are randomized for weight-adjusted treatment with losartan versus placebo. The primary endpoint is decrease in aortic root growth rate. Secondary endpoints are aortic dissection/surgery, progression of aortic/mitral regurgitation, arterial stiffness, left ventricular systolic/diastolic function, quality of life and genetic modifiers. Echocardiography, vascular echo-Doppler and quality of life assessment will be performed at baseline and at 6-monthly follow-ups for 3 years. MRI evaluation will be performed at baseline and at the end of the trial. Conclusion: This trial will study new therapeutic strategies for the prevention of serious cardiovascular complications in MFS. The uniqueness in our trial is that the additive effect of losartan and beta-blocker will be evaluated in a large spectrum of disease severity. A combination of ultrasound and MRI will allow detailed evaluation of anatomic and functional properties of the aorta and left ventricle. (C) 2010 Elsevier Ireland Ltd. All rights reserved. |
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English
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International journal of cardiology. - Amsterdam, 1981, currens | |
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Amsterdam : 2012
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0167-5273
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157:3(2012), p. 354-358
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000304245900015
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