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Advancements in pharmacological therapy for transthyretin cardiac amyloidosis myocardiopathy and its comorbidities
- ZHONGGUO YAOFANG Vol. 34, Issue 21, Pages: 2665-2670(2023)
- 作者机构：
重庆医科大学附属第一医院心血管内科，重庆 400042
- 作者简介：
- 基金信息：
DOI：10.6039/j.issn.1001-0408.2023.21.17
CLC： R972;R542.2Published：15 November 2023，
Received：15 April 2023，
Revised：27 September 2023，
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李昀纾,罗素新,黄毕.转甲状腺素蛋白心脏淀粉样变心肌病及其合并症的药物治疗进展 Δ[J].中国药房,2023,34(21):2665-2670.
LI Yunshu,LUO Suxin,HUANG Bi.Advancements in pharmacological therapy for transthyretin cardiac amyloidosis myocardiopathy and its comorbidities[J].ZHONGGUO YAOFANG,2023,34(21):2665-2670.
李昀纾,罗素新,黄毕.转甲状腺素蛋白心脏淀粉样变心肌病及其合并症的药物治疗进展 Δ[J].中国药房,2023,34(21):2665-2670. DOI： 10.6039/j.issn.1001-0408.2023.21.17.
LI Yunshu,LUO Suxin,HUANG Bi.Advancements in pharmacological therapy for transthyretin cardiac amyloidosis myocardiopathy and its comorbidities[J].ZHONGGUO YAOFANG,2023,34(21):2665-2670. DOI： 10.6039/j.issn.1001-0408.2023.21.17.
摘要
转甲状腺素蛋白心脏淀粉样变心肌病（ATTR-CM）是由于不稳定的转甲状腺素蛋白（TTR）四聚体解离成单体后错误折叠为淀粉样物质沉积于心肌间质所致的一种浸润性心肌病。既往针对ATTR-CM无特异性的治疗药物，主要针对心力衰竭和心律失常对症治疗。近年来针对ATTR-CM的发病机制，学者们研发了两大类药物：一类是稳定TTR四聚体结构的药物（如氯苯唑酸和acoramidis），另一类是干扰TTR合成的药物（如patisiran）。其中氯苯唑酸已被研究证实是目前唯一能治疗ATTR-CM的药物，其他药物均尚处于临床试验阶段，临床证据有限。在ATTR-CM合并症的药物治疗方面，主要是针对最常见的心脏合并症（如心力衰竭和心律失常）的治疗；传统用于改善心力衰竭预后的药物（如β受体阻滞剂、肾素-血管紧张素受体拮抗剂）在ATTR-CM患者中未证实可改善预后，相反可能带来不良反应。对于合并房颤的ATTR-CM患者，推荐进行抗凝治疗以预防血栓形成，并可使用胺碘酮进行节律控制。尽管ATTR-CM在药物治疗方面取得了显著进展，但整体预后仍较差，需要在发病机制和靶点开发方面进一步研究，以进一步改善ATTR-CM患者的预后。
Abstract
Transthyretin cardiac amyloidosis myocardiopathy （ATTR-CM） is an infiltrative cardiomyopathy characterized by the deposition of amyloidogenic material in the myocardial interstitium due to the misfolding of monomers following the dissociation of unstable transthyretin （TTR） tetramers. Previous treatments for ATTR-CM lacked specificity，primarily targeting symptomatic management of heart failure and arrhythmias. In recent years，researchers have developed two major classes of drugs addressing the pathogenesis of ATTR-CM. The first class stabilizes TTR tetramer structure （such as tafamidis and acoramidis），while the second class interferes with TTR synthesis （such as patisiran）. Among these，tafamidis has been confirmed as the only currently effective treatment for ATTR-CM，while other drugs are still in clinical trial stages with limited clinical evidence. Concerning the management of comorbidities in ATTR-CM，treatment mainly focuses on common cardiac comorbidities （such as heart failure and arrhythmias）. Traditional drugs used to improve heart failure prognosis （such as β-blockers and renin-angiotensin-receptor blocker），have not demonstrated prognosis improvement in ATTR-CM patients and may even lead to adverse reactions. For ATTR-CM patients with concurrent atrial fibrillation，anticoagulation therapy is recommended to prevent thrombus formation，and amiodarone can be used for rhythm control. Despite significant advancements in pharmaceutical treatments for ATTR-CM，the overall prognosis remains poor，necessitating further research into the pathogenesis and target development to enhance the prognosis of ATTR-CM patients.
关键词
转甲状腺素蛋白心脏淀粉样变心肌病合并症心律失常心力衰竭药物治疗
Keywords
comorbiditiesarrhythmiasheart failurepharmacotherapy
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