背景:转甲状腺素蛋白心脏淀粉样变性(ATTR-CA)越来越被认可。随着诊断途径的变化和治疗方法的进步,临床结果随着时间的推移而发展。
目的:评估ATTR-CA患者随时间变化的临床结局。
方法:这是一项回顾性队列研究,对2001-2021年期间诊断为ATTR-CA的419例患者进行了研究,比较了不同时期的临床特征。主要终点是复合全因死亡率或原位心脏移植(OHT)。使用Cox比例风险模型控制队列之间的差异进行时间至事件分析。
结果:最近几年诊断的患者中位年龄较高(2017-2021年:78岁,2014-2016年:75年,2001-2013年:74年),更常见的是ATTRwt(81.9%vs82.5%vs.56.4%),但较不严重的表型,如哥伦比亚I期疾病患者更多(47.6%vs35.9%vs22.4%),由于较低的生物标志物,更多NYHAI-II级患者(68.9%vs47.6%vs.43.6%),环路利尿剂的使用率较低(67.0%vs78.6%vs89.1%)。随着时间的推移,患者接受Tafamidis治疗的频率更高(74%vs37%vs32%).在多变量分析中,哥伦比亚评分较高(HR1.42,95%CI1.30-1.54,p<0.001)可预测死亡或OHT,而Tafamidis(HR0.31,95%CI0.22-0.44,p<0.001)与更高的生存率和OHT的自由度相关。
结论:最近确诊的ATTR-CA患者有早期疾病,死亡率大大降低。Tafamidis与OHT的生存和自由显着改善有关。
BACKGROUND: Transthyretin cardiac amyloidosis (ATTR-CA) is increasingly recognized. Clinical outcomes have evolved over time amid changes in the diagnostic pathway and advances in therapeutics. We sought to evaluate clinical outcomes over time of patients with ATTR-CA with access to disease-modifying therapy.
RESULTS: This is a retrospective cohort study of 419 patients diagnosed with ATTR-CA during 2001-2021, comparing clinical characteristics across eras. The primary end point was composite all-cause mortality or orthotopic heart transplantation (OHT). Time-to-event analysis was performed using Cox proportional hazard modeling controlling for differences among cohorts. Patients diagnosed in the more recent years had higher median age (2017-2021, 78 years; 2014-2016, 75 years; 2001-2013, 74 years) and more often had wild-type ATTR (81.9% vs 82.5% vs 56.4%), but less severe phenotypes as evidenced by more individuals with Columbia stage I disease (47.6% vs 35.9% vs 22.4%), owing to lower biomarkers, more patients in New York Heart Association functional classes I and II (68.9% vs 47.6% vs 43.6%), and lower use of loop diuretics (67.0% vs 78.6% vs 89.1%). Over time, patients were treated more frequently with tafamidis (74% vs 37% vs 32%). On multivariable analysis, greater Columbia score (hazard ratio 1.42, 95% confidence interval 1.30-1.54, P < .001) was predictive of death or OHT, whereas tafamidis (hazard ratio 0.31, 95% confidence interval 0.22-0.44, P < .001) was associated with greater survival and freedom from OHT.
CONCLUSIONS: Patients recently diagnosed with ATTR-CA have earlier stage disease and substantially lower mortality. Tafamidis is associated with significantly improved survival and freedom from OHT.