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特发性甲状腺素运载蛋白淀粉样变患者接受塔法米迪治疗后左心室肥厚的消退。

Regression of left ventricular hypertrophy after tafamidis therapy in a patient with transthyretin amyloidosis variant.

机构信息

Department of Cardiovascular Medicine, Tokushima University Graduate School of Biomedical Sciences, Tokushima, Japan.

Department of Community Medicine and Human Resource Development, Tokushima University Graduate School of Biomedical Sciences, Tokushima, Japan.

出版信息

J Med Invest. 2022;69(3.4):320-322. doi: 10.2152/jmi.69.320.

DOI:10.2152/jmi.69.320
PMID:36244789
Abstract

Transthyretin amyloidosis (ATTR) variant is a life-threatening hereditary disease predominantly affecting the peripheral nervous system and heart. Tafamidis, which prevents the deposition of amyloid by stabilizing transthyretin, is available for the treatment of neuropathy and cardiomyopathy of ATTR. However, whether tafamidis could eliminate established amyloid deposits and improve cardiac function remains unknown. We reported a case of regression of left ventricular hypertrophy after tafamidis therapy in a patient with an ATTR variant. J. Med. Invest. 69 : 320-322, August, 2022.

摘要

转甲状腺素蛋白淀粉样变性(ATTR)变异型是一种危及生命的遗传性疾病,主要影响周围神经系统和心脏。特立氟胺可通过稳定转甲状腺素蛋白来预防淀粉样物质的沉积,可用于治疗ATTR 的神经病和心肌病。然而,特立氟胺是否可以消除已形成的淀粉样沉积物并改善心脏功能尚不清楚。我们报告了一例 ATTR 变异型患者在接受特立氟胺治疗后左心室肥厚消退的病例。J. Med. Invest. 69 : 320-322, August, 2022.

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Tafamidis decreased cardiac amyloidosis deposition in patients with Ala97Ser hereditary transthyretin cardiomyopathy: a 12-month follow-up cohort study.他法米替尼可降低 Ala97Ser 遗传性转甲状腺素蛋白心肌病患者的心脏淀粉样变性沉积:一项为期 12 个月的随访队列研究。
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