他法米地斯可改善A97S转甲状腺素蛋白心脏淀粉样变性患者的心肌纵向应变。
Tafamidis improves myocardial longitudinal strain in A97S transthyretin cardiac amyloidosis.
作者信息
Wu Yuan-Kun Aden, Yu An-Li, Cheng Mei-Fang, Lin Lung-Chun, Lee Ming-Jen, Chou Chia-Hung, Shun Chia-Tung, Hsueh Hsueh-Wen, Juang Jimmy Jyh-Ming, Tseng Ping-Huei, Lin Siao-Ping, Su Mao-Yuan, Chao Chi-Chao, Hsieh Sung-Tsang, Tsai Cheng-Hsuan, Lin Yen-Hung
机构信息
Division of Cardiology, Department of Internal Medicine, National Taiwan University Hospital, Taipei, Taiwan.
Cardiovascular Centre, National Taiwan University Hospital, Taipei, Taiwan.
出版信息
Ther Adv Chronic Dis. 2024 Jan 11;15:20406223231222828. doi: 10.1177/20406223231222828. eCollection 2024.
BACKGROUND
Transthyretin cardiomyopathy (ATTR-CM) is a debilitating disease that has received much attention since the emergence of novel treatments. The Transthyretin Cardiomyopathy Clinical Trial showed that tafamidis, a transthyretin tetramer stabilizer, effectively reduced the declines in functional capacity and quality of life. However, Ala97Ser (A97S) hereditary ATTR-CM is underrepresented in major ATTR-CM tafamidis trials.
OBJECTIVES
We aim to investigate the change in global longitudinal strain (GLS) of A97S ATTR-CM patients after 12 months of tafamidis treatment.
METHODS
We retrospectively analysed a prospective cohort of patients with A97S ATTR-CM who received tafamidis meglumine (61 mg/day) at the National Taiwan University Hospital. Echocardiography with speckle tracking strain analysis was performed at baseline and 12 months after treatment.
RESULTS
In all, 20 patients were included in the cohort. The baseline left ventricular ejection fraction (LVEF) and interventricular septum (IVS) thickness were 59.20 ± 13.23% and 15.10 ± 3.43 mm, respectively. After 12 months of tafamidis treatment, the LVEF and IVS were 61.83 ± 15.60% ( = 0.244) and 14.59 ± 3.03 mm ( = 0.623), respectively. GLS significantly improved from -12.70 ± 3.31% to -13.72 ± 3.17% ( = 0.048), and longitudinal strain (LS) in apical and middle segments significantly improved from -16.05 ± 4.82% to -17.95 ± 3.48% ( = 0.039) and -11.89 ± 4.38% to -13.58 ± 3.12% ( = 0.039), respectively. Subgroup analysis showed that patients with LVEF < 50% had a better treatment response and improvement in GLS. The patients with an IVS ⩾ 13 mm had an improvement in two-chamber LS from -10.92 ± 4.25% to -13.15 ± 3.87% ( = 0.042) and an improvement in apical left ventricular LS from -15.30 ± 5.35% to -17.82 ± 3.99% ( = 0.031).
CONCLUSION
Tafamidis significantly improved GLS, and particularly apical and middle LS in A97S ATTR-CM patients.
背景
转甲状腺素蛋白心肌病（ATTR-CM）是一种使人衰弱的疾病，自新型治疗方法出现以来备受关注。转甲状腺素蛋白心肌病临床试验表明，转甲状腺素蛋白四聚体稳定剂tafamidis能有效减缓功能能力和生活质量的下降。然而，Ala97Ser（A97S）遗传性ATTR-CM在主要的ATTR-CM tafamidis试验中的代表性不足。
目的
我们旨在研究A97S ATTR-CM患者接受tafamidis治疗12个月后整体纵向应变（GLS）的变化。
方法
我们回顾性分析了在台湾大学医院接受甲磺酸tafamidis（61毫克/天）治疗的A97S ATTR-CM患者的前瞻性队列。在基线和治疗12个月后进行了带有斑点追踪应变分析的超声心动图检查。
结果
该队列共纳入20例患者。基线时左心室射血分数（LVEF）和室间隔（IVS）厚度分别为59.20±13.23%和15.10±3.43毫米。tafamidis治疗12个月后，LVEF和IVS分别为61.83±15.60%（P = 0.244）和14.59±3.03毫米（P = 0.623）。GLS从-12.70±3.31%显著改善至-13.72±3.17%（P = 0.048），心尖和中间节段的纵向应变（LS）分别从-16.05±4.82%显著改善至-17.95±3.48%（P = 0.039）和从-11.89±4.38%改善至-13.58±3.12%（P = 0.039）。亚组分析显示，LVEF < 50%的患者治疗反应更好，GLS改善更明显。IVS≥13毫米的患者双腔LS从-10.92±4.25%改善至-13.15±3.87%（P = 0.042），心尖左心室LS从-15.30±5.35%改善至-17.82±3.99%（P = 0.031）。
结论
tafamidis显著改善了A97S ATTR-CM患者的GLS，尤其是心尖和中间节段的LS。
Zotero 插件