Early Experience of Tafamidis Treatment in Japanese Patients With Wild-Type Transthyretin Cardiac Amyloidosis From the Kochi Amyloidosis Cohort
Early Experience of Tafamidis Treatment in Japanese Patients With Wild-Type Transthyretin Cardiac Amyloidosis From the Kochi Amyloidosis Cohort
复制标题
DOI：
10.1253/circj.cj-21-0965
复制
发表时间：
2022-06-01
影响因子：
3.3
通讯作者：
Kitaoka, Hiroaki
中科院分区：
文献类型：
--
作者：
Ochi, Yuri;Kubo, Toru;Kitaoka, Hiroaki
背景：Tafamidis已成为野生型甲状腺素运载蛋白心脏淀粉样变性（ATTRwt CA）患者的有效治疗方法。本文报告了日本ATTRwt CA患者使用他伐他汀治疗的早期经验。方法和结果：在过去2年中，在82例ATTRwt CA患者（平均年龄为81.7 ± 6.0岁）中，38例患者开始接受他伐他汀治疗。其余44例患者未接受他法莫司治疗。未给予他法莫司的最常见原因是晚期心力衰竭，第二大原因是患者虚弱。在接受他伐他汀治疗的患者中，没有因不良事件而停用他伐他汀，每年心血管相关住院率为0.19，1年生存率为92%。在继续他伐他汀治疗12-18个月的患者中，高敏心肌肌钙蛋白T水平、血浆B型利钠肽水平、左心室射血分数、室间隔壁厚度或左心室纵向应变值较基线无显著恶化。结论：在实际实践中，大约一半的ATTRwt CA研究患者接受了Tafamidis治疗。在选定的日本ATTRwt CA患者中，Tafamidis可能是安全的，并可能在短期内维持疾病严重程度的状态。需要进一步的研究来确定合适的患者选择tafamlutamine治疗和tafamlutamine的长期疗效。
Background: Tafamidis has emerged as an effective treatment for patients with wild-type transthyretin cardiac amyloidosis (ATTRwt CA). The early experience of tafamidis treatment for Japanese patients with ATTRwt CA is reported here. Methods and Results: Over the past 2 years, in 82 patients with ATTRwt CA (mean age of 81.7 +/- 6.0 years), tafamidis treatment was initiated for 38 patients. The remaining 44 patients were not administered tafamidis. The most frequent reason for non -administration of tafamidis was advanced heart failure and the second most reason was the patient's frailty. In patients who received tafamidis treatment, there was no discontinuation of tafamidis due to adverse events, the rate of cardiovascular-related hospitalizations per year was 0.19, and the 1-year survival rate was 92%. In the patients who continued tafamidis for 12-18 months, there was no significant deterioration from baseline for high-sensitivity cardiac troponin T level, plasma B-type natriuretic peptide level, left ventricular ejection fraction, inter-ventricular septum wall thickness, or value of left ventricular longitudinal strain. Conclusions: Tafamidis treatment was introduced for approximately half of the study patients with ATTRwt CA in real-world practice. Tafamidis is likely to be safe and may maintain the status of disease severity in the short-term in selected Japanese patients with ATTRwt CA. Further research is needed to determine appropriate patient selection for tafamidis treatment and efficacy of tafamidis in the long term.