Kuyama Naoto, Izumiya Yasuhiro, Takashio Seiji, Usuku Hiroki, Tabira Akihisa, Oguni Tetsuya, Yamamoto Masahiro, Hirakawa Kyoko, Ishii Masanobu, Tabata Noriaki, Hoshiyama Tadashi, Kanazawa Hisanori, Hanatani Shinsuke, Kidoh Masafumi, Oda Seitaro, Matsuzawa Yasushi, Yamamoto Eiichiro, Hirai Toshinori, Ueda Mitsuharu, Tsujita Kenichi
Department of Cardiovascular Medicine, Graduate School of Medical Sciences, Kumamoto University.
Department of Medical Information Science, Graduate School of Medical Sciences, Kumamoto University.
Circ J. 2025 Mar 25;89(4):421-431. doi: 10.1253/circj.CJ-24-0733. Epub 2025 Jan 9.
Accurate prediction of prognosis in transthyretin amyloid cardiomyopathy (ATTR-CM) is crucial for optimal treatment selection, including tafamidis, the only approved therapy for ATTR-CM. Although tafamidis has been proven to improve prognosis, the long-term serial changes in comprehensive parameters related to ATTR-CM, including cardiac biomarkers and imaging parameters, under tafamidis remain unknown.
In this study, we used Cox regression analysis on data from 258 consecutive patients diagnosed with ATTR-CM at Kumamoto University to determine prognostic factors. During clinical follow-up, the serial changes in parameters were compared between tafamidis-treated and tafamidis-naïve patients. An elevated high-sensitivity cardiac troponin T (hs-cTnT) level at baseline was identified as a stronger independent predictor of all-cause death compared with left ventricular ejection fraction (LVEF) and extracellular volume. During follow-up (median: 24.4 months), estimated glomerular filtration rate and LVEF declined significantly with time in both cohorts. Notably, serum hs-cTnT and B-type natriuretic peptide levels were significantly elevated in the tafamidis-naïve cohort compared to baseline, but this increase was prevented by tafamidis treatment.
Of the ATTR-CM-related parameters investigated, an increased hs-cTnT level at baseline was a promising determinant of poor prognosis. Long-term tafamidis treatment prevented a deterioration in cardiac biomarkers, and the measurement of these markers may enable appropriate monitoring of disease progression.
准确预测转甲状腺素蛋白淀粉样变心肌病（ATTR-CM）的预后对于选择最佳治疗方案至关重要，包括他法米地，这是唯一获批用于ATTR-CM的疗法。尽管已证明他法米地可改善预后，但在他法米地治疗下，与ATTR-CM相关的综合参数（包括心脏生物标志物和影像学参数）的长期系列变化仍不清楚。
在本研究中，我们对熊本大学连续诊断为ATTR-CM的258例患者的数据进行Cox回归分析以确定预后因素。在临床随访期间，比较了接受他法米地治疗的患者和未接受他法米地治疗的患者之间参数的系列变化。与左心室射血分数（LVEF）和细胞外容积相比，基线时高敏心肌肌钙蛋白T（hs-cTnT）水平升高被确定为全因死亡更强的独立预测因素。在随访期间（中位数：24.4个月），两个队列中的估计肾小球滤过率和LVEF均随时间显著下降。值得注意的是，与基线相比，未接受他法米地治疗的队列中血清hs-cTnT和B型利钠肽水平显著升高，但他法米地治疗可防止这种升高。
在所研究的与ATTR-CM相关的参数中，基线时hs-cTnT水平升高是预后不良的一个有前景的决定因素。长期他法米地治疗可防止心脏生物标志物恶化，对这些标志物的测量可能有助于对疾病进展进行适当监测。
