Department of Cardiovascular Medicine, Graduate School of Medical Sciences Kumamoto University Kumamoto Japan.
Department of Medical Information Science, Graduate School of Medical Sciences Kumamoto University Kumamoto Japan.
J Am Heart Assoc. 2024 May 21;13(10):e034518. doi: 10.1161/JAHA.124.034518. Epub 2024 May 18.
Although tafamidis treatment improves prognosis in patients with wild-type transthyretin amyloid cardiomyopathy, an optimal surrogate marker monitoring its therapeutic effect remains unclear. This study investigated the association between changes in cardiac biomarkers, high-sensitivity cardiac troponin T (hs-cTnT) and B-type natriuretic peptide (BNP) during the first year after tafamidis treatment and clinical outcomes.
In 101 patients with wild-type transthyretin amyloid cardiomyopathy receiving tafamidis at our institution, change in cardiac biomarkers from baseline to 1 year after tafamidis administration and its association with composite outcomes (composite of all-cause death and hospitalization attributable to heart failure) was assessed. During the follow-up period (median, 17 months), 16 (16%) patients experienced composite outcomes. The hs-cTnT level significantly decreased at 1 year after tafamidis treatment, unlike the BNP level. The frequencies of increased hs-cTnT and BNP levels were significantly higher in those with composite outcomes than in those without (44% versus 15%; =0.01). Kaplan-Meier survival analysis showed that patients in whom both hs-cTnT and BNP levels increased at 1 year after tafamidis had a higher probability of composite outcomes compared with those with decreased hs-cTnT and BNP levels (log-rank <0.01). Cox regression analysis identified increased hs-cTnT and BNP levels at 1 year after tafamidis administration as an independent predictor of higher cumulative risk of composite outcomes.
Deterioration in cardiac biomarkers during the first year after tafamidis treatment predicted a worse prognosis, suggesting the utility of serial assessment of cardiac biomarkers for monitoring the therapeutic response to tafamidis in patients with wild-type transthyretin amyloid cardiomyopathy.
尽管塔法米迪治疗可改善野生型转甲状腺素蛋白淀粉样心肌病患者的预后，但仍不清楚监测其治疗效果的最佳替代标志物。本研究旨在探讨塔法米迪治疗后第一年心脏生物标志物（高敏心肌肌钙蛋白 T[hs-cTnT]和 B 型利钠肽[BNP]）变化与临床结局之间的关系。
在本机构接受塔法米迪治疗的 101 例野生型转甲状腺素蛋白淀粉样心肌病患者中，评估了心脏生物标志物从基线到塔法米迪治疗后 1 年的变化及其与复合结局（全因死亡和心力衰竭住院的综合结局）之间的关系。在随访期间（中位数 17 个月），16 例（16%）患者发生了复合结局。与 BNP 水平不同，塔法米迪治疗 1 年后 hs-cTnT 水平显著降低。发生复合结局患者的 hs-cTnT 和 BNP 水平升高的频率显著高于未发生复合结局的患者（44%比 15%；=0.01）。Kaplan-Meier 生存分析显示，与 hs-cTnT 和 BNP 水平均降低的患者相比，塔法米迪治疗 1 年后 hs-cTnT 和 BNP 水平均升高的患者发生复合结局的可能性更高（对数秩检验<0.01）。Cox 回归分析确定，塔法米迪治疗 1 年后 hs-cTnT 和 BNP 水平升高是复合结局累积风险较高的独立预测因素。
塔法米迪治疗后第一年心脏生物标志物恶化预示着预后较差，表明连续评估心脏生物标志物可用于监测野生型转甲状腺素蛋白淀粉样心肌病患者对塔法米迪的治疗反应。
