Clinical research progress in the treatment of transthyretin amyloid with tafamidis
- VernacularTitle: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
- Author:
Panpan JIN
1
,
2
;
Yang LIU
1
,
2
;
Bo QIU
2
;
Huizhen WU
1
Author Information
1. School of Graduate，Hebei Medical University，Shijiazhuang 050017，China
2. Dept. of Pharmacy，Hebei Provincial People’s Hospital/Hebei Key Lab of Clinical Pharmacy，Shijiazhuang 050051，China
- Publication Type:Journal Article
- Keywords:
tafamidis;
transthyretin;
amyloid transthyretin;
clinical research
- From:
China Pharmacy
2024;35(17):2171-2175
- CountryChina
- Language:Chinese
-
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.
