Yamada Toshihiro, Takashio Seiji, Arima Yuichiro, Nishi Masato, Morioka Mami, Hirakawa Kyoko, Hanatani Shinsuke, Fujisue Koichiro, Yamanaga Kenshi, Kanazawa Hisanori, Sueta Daisuke, Araki Satoshi, Usuku Hiroki, Nakamura Taishi, Suzuki Satoru, Yamamoto Eiichiro, Ueda Mitsuharu, Kaikita Koichi, Tsujita Kenichi
Department of Cardiovascular Medicine, Graduate School of Medical Sciences, Kumamoto University, 1-1-1 Honjo, Chou-ku, Kumamoto, 860-8556, Japan.
International Research Center for Medical Sciences (IRCMS), Kumamoto University, Kumamoto, Japan.
ESC Heart Fail. 2020 Oct;7(5):2829-2837. doi: 10.1002/ehf2.12884. Epub 2020 Jul 16.
The focus on wild-type transthyretin amyloid cardiomyopathy (ATTRwt-CM) is increasing because of novel treatment options. There is currently no report on a large number of Japanese patients with ATTRwt-CM. The study aimed to examine the characteristics and prognosis of ATTRwt-CM in Japan.
Consecutive patients (78.5 ± 6.4 years old at diagnosis) with ATTRwt-CM diagnosed at Kumamoto University Hospital between December 2002 and December 2019 were retrospectively reviewed. Data, including demographic characteristics, co-morbidities, clinical manifestations at diagnosis, laboratory results, electrocardiographic and echocardiographic data, imaging and pathological findings, and treatment were obtained. Of 129 patients included in this study, 110 patients (85%) were male. The median period from initial symptom onset to diagnosis was 15.5 (2-75) months. Heart failure was the most common clinical manifestation leading to diagnosis (61%) and initial manifestations (49%). Of 106 patients, carpal tunnel syndrome was observed in 57 patients (54%), and the median period from initial symptom onset to diagnosis was 96 (48-120) months. Histopathological confirmation of transthyretin amyloid was achieved in 94 patients (73%), including 66 (51%) and 28 cases (22%) with endomyocardial and extracardiac biopsies. During the observation period (median 15.0 [inter-quartile range, 5.4-33.2] months after diagnosis), 34 patients (26%) died. Of these, 27 patients (79%) had cardiovascular deaths (heart failure, 25; sudden death, two). The median survival duration was 58.9 months and the 5 years' survival rate was 48%. According to a multivariate Cox hazard analysis, age [hazard ratio (HR), 1.14; 95% confidence interval (CI), 1.05-1.23, P = 0.002] and low serum sodium levels (HR, 0.89; 95% CI, 0.79-0.996; P = 0.04) contributed to all-cause mortality, and low serum sodium levels contributed to hospitalization for heart failure (HR, 0.86; 95% CI, 0.77-0.96; P = 0.005).
Clinical characteristics and prognosis of ATTRwt-CM patients in Japan were examined. Carpal tunnel syndrome can be considered an indication for diagnosis of ATTRwt-CM. Age and low serum sodium level were significant predictive factors of all survival outcomes. The clinical features of ATTRwt-CM should be recognized to provide appropriate treatment.
由于有了新的治疗选择，野生型转甲状腺素蛋白淀粉样变心肌病（ATTRwt-CM）受到的关注日益增加。目前尚无关于大量日本ATTRwt-CM患者的报道。本研究旨在探讨日本ATTRwt-CM患者的特征及预后。
对2002年12月至2019年12月在熊本大学医院确诊的ATTRwt-CM连续患者（诊断时年龄78.5±6.4岁）进行回顾性分析。收集患者的人口统计学特征、合并症、诊断时的临床表现、实验室检查结果、心电图和超声心动图数据、影像学和病理学检查结果以及治疗情况等资料。本研究纳入的129例患者中，110例（85%）为男性。从最初症状出现到诊断的中位时间为15.5（2 - 75）个月。心力衰竭是导致诊断（61%）和最初表现（49%）的最常见临床表现。在106例患者中，57例（54%）观察到腕管综合征，从最初症状出现到诊断的中位时间为96（48 - 120）个月。94例患者（73%）经组织病理学证实为转甲状腺素蛋白淀粉样变，其中66例（51%）经心内膜活检、28例（22%）经心外组织活检证实。在观察期（诊断后中位时间15.0[四分位间距，5.4 - 33.2]个月）内，34例患者（26%）死亡。其中，27例（79%）死于心血管疾病（心力衰竭25例，猝死2例）。中位生存时间为58.9个月，5年生存率为48%。多因素Cox风险分析显示，年龄[风险比（HR），1.14；95%置信区间（CI），1.05 - 1.23，P = 0.002]和低血钠水平（HR，0.89；95%CI，0.79 - 0.996；P = 0.04）与全因死亡率相关，低血钠水平与因心力衰竭住院相关（HR，0.86；95%CI，0.77 - 0.96；P = 0.005）。
研究了日本ATTRwt-CM患者临床特征及预后。腕管综合征可作为ATTRwt-CM诊断的一个指标。年龄和低血钠水平是所有生存结局的重要预测因素。应认识到ATTRwt-CM的临床特征以提供恰当治疗。
