Clinical research progress in the treatment of transthyretin amyloid with tafamidis

JIN Panpan; LIU Yang; QIU Bo; WU Huizhen

doi:10.6039/j.issn.1001-0408.2024.17.20

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Clinical research progress in the treatment of transthyretin amyloid with tafamidis

更新时间：2024-09-09

- Clinical research progress in the treatment of transthyretin amyloid with tafamidis
- ZHONGGUO YAOFANG Vol. 35, Issue 17, Pages: 2171-2175(2024)
- 作者机构：
  
  1.河北医科大学研究生学院，石家庄　050017
  2.河北省人民医院药学部/河北省临床药学重点实验室，石家庄　050051
- 作者简介：
- 基金信息：
- DOI：10.6039/j.issn.1001-0408.2024.17.20
  CLC： R971;R972
- Published：15 September 2024，
  
  Received：06 March 2024，
  
  Revised：19 July 2024，
- 稿件说明：
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靳盼盼,刘洋,邱博等.氯苯唑酸治疗转甲状腺素蛋白淀粉样变性的临床研究进展 ^Δ[J].中国药房,2024,35(17):2171-2175.

JIN Panpan,LIU Yang,QIU Bo,et al.Clinical research progress in the treatment of transthyretin amyloid with tafamidis[J].ZHONGGUO YAOFANG,2024,35(17):2171-2175.
靳盼盼,刘洋,邱博等.氯苯唑酸治疗转甲状腺素蛋白淀粉样变性的临床研究进展 ^Δ[J].中国药房,2024,35(17):2171-2175. DOI： 10.6039/j.issn.1001-0408.2024.17.20.

JIN Panpan,LIU Yang,QIU Bo,et al.Clinical research progress in the treatment of transthyretin amyloid with tafamidis[J].ZHONGGUO YAOFANG,2024,35(17):2171-2175. DOI： 10.6039/j.issn.1001-0408.2024.17.20.

摘要

氯苯唑酸为转甲状腺素蛋白（TTR）的选择性稳定剂，用于治疗TTR淀粉样变性心肌病（ATTR-CM）和TTR淀粉样变性多发性神经病（ATTR-PN）。本文对氯苯唑酸的基本信息、有效性及安全性临床研究等进行综述，发现氯苯唑酸可通过抑制TTR四聚体的解离，减缓或阻止TTR淀粉样变性的进展。多项临床研究表明，氯苯唑酸具有良好的疗效和安全性，可以显著降低淀粉样变性患者全因死亡率和心血管相关住院率，延缓患者疾病进展。尽管氯苯唑酸治疗可能存在一定的局限性，但其仍然是用于治疗TTR淀粉样变性疾病的关键药物，也是第1种被批准用于治疗ATTR-CM的药物。

Abstract

Tafamidis is a selective stabilizer for transthyretin （TTR）， used for the treatment of transthyretin amyloidosis with cardiomyopathy （ATTR-CM） and transthyretin amyloidosis with polyneuropathy （ATTR-PN）. This article provides a review of the basic information and clinical studies on the efficacy and safety of tafamidis. It is found that tafamidis slows down or prevents the progression of TTR amyloidosis by inhibiting the dissociation of TTR tetramers. Multiple clinical studies have shown that tafamidis has good efficacy and safety， significantly reducing all-cause mortality and cardiovascular-related hospitalization rates in patients with amyloidosis， and delaying disease progression. Although tafamidis treatment may have certain limitations， it is still a key drug for the treatment of TTR amyloidosis， and the first drug approved for the treatment of ATTR-CM.

关键词

氯苯唑酸转甲状腺素蛋白淀粉样变性临床研究

Keywords

transthyretinamyloid transthyretinclinical research

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