老年心脏病学杂志（英文版）Issue(4)：254-257,4.DOI:10.3724/SP.J.1263.2011.00254
Novel pharmacological strategies to prevent aortic complications in Marfan syndrome
Novel pharmacological strategies to prevent aortic complications in Marfan syndrome
Peter Matt 1Friedrich Eckstein1
作者信息
- 1. Division of Cardiac Surgery, Basel University Hospital, Spitalstrasse 21, CH-4031 Basel, Switzerland
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摘要
Abstract
The Marfan syndrome (MFS) is a systemic connective tissue disorder caused by mutations in the FBN1 gene.Recent molecular studies,most performed in mouse models,revealed that the MFS is more a developmental abnormality with broad and complex effects on the morphogenesis and function of multiple organ systems.FBN1 haploinsufficiency and dysregulated transforming growth factor-beta (TGF-β)signaling seem to be critical for clinical manifestations in MFS including aortic root dilatation.Aortic root aneurysm and aortic dissection represent the main causes of morbidity and mortality in MFS.Most importantly,TGF-β antagonism through angiotensin Ⅱ type 1 receptor blockers (ARBs),for example losartan,has been shown to prevent and possibly reverse aortic root dilatation in a mouse model of MFS.A first human study on a small pediatric cohort confirmed those promising results in reducing the aortic root growth over a follow-up period of 12 to 47 months.So,a large multicenter trial has been set up and results should be available soon.Other therapeutic strategies which might be combined with losartan include traditional β-blockade,doxycyclin and statins.Such management could offer the first potential for primary prevention of clinical manifestations in MFS.关键词
Marfan syndrome/ aneurysm/ angiotensin receptor blockerKey words
Marfan syndrome/ aneurysm/ angiotensin receptor blocker引用本文复制引用
Peter Matt,Friedrich Eckstein..Novel pharmacological strategies to prevent aortic complications in Marfan syndrome[J].老年心脏病学杂志（英文版）,2011,(4):254-257,4.
