文献简介

出版社:Springer Science+Business Media New York

作  者:Mazen Hanna

编  号:10.1007/s11897-013-0182-4

关键字:Transthyretin .Amyloid .Amyloidosis .Cardiac . Cardiomyopathy . Neuropathy . Familial . Senile .Hereditary . Pharmacologic . Therapy . Drugs

年  份:2014   点击量:754

文献摘要

Abstract Transthyretin amyloidosis (ATTR) is either ahereditary disease related to a mutation in the transthyretin gene that leads to neuropathy and/or cardiomyopathy or an acquired disease of the elderly that leads to restrictive cardiomyopathy. The prevalence of this disease is higher than once thought and awareness is likely to increase amongst physicians and in particular cardiologists. Until recently there have been no treatment options for this disease except to treat the heart failure with diuretics and the neuropathy symptomatically. However, there are several emerging pharmacologic therapies designed to slowor stop the progression of ATTR. This article reviews novel therapeutic drugs that work at different points in the pathogenesis of this disease attempting to change its natural history and improve outcomes.

 

摘要:转甲状腺素蛋白淀粉样变病(ATTR)要么是与转甲状腺素蛋白基因的突变有关的导致神经病和/或心肌病的一种遗传性疾病,要么是导致限制性心肌病的一种老年人后天性疾病。这种疾病的患病率比以前认为的高,并且医生之间对它的关注可能会增加,尤其是心脏科医生。直到最近还没有这种疾病的治疗方法,除了用利尿剂治疗心脏衰竭和神经病变症状。但是,已有一些旨在减缓或停止ATTR的进展的新兴药物疗法。本文综述了新的治疗药物,这些药物在该病的发病机制的不同方面起效,试图改变其自然病程和改善预后。