转甲状腺素淀粉样变性心肌病的肾脏预后

IF 14.8 1区医学 Q1 CARDIAC & CARDIOVASCULAR SYSTEMS JAMA cardiology Pub Date : 2024-11-17 DOI:10.1001/jamacardio.2024.4578

Adam Ioannou, Yousuf Razvi, Aldostefano Porcari, Muhammad U. Rauf, Ana Martinez-Naharro, Lucia Venneri, Salsabeel Kazi, Ali Pasyar, Carina M. Luxhøj, Aviva Petrie, William Moody, Richard P. Steeds, Brett W. Sperry, Ronald M. Witteles, Carol Whelan, Ashutosh Wechalekar, Helen Lachmann, Philip N. Hawkins, Scott D. Solomon, Julian D. Gillmore, Marianna Fontana

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引用次数: 0

摘要

重要性胰凝乳蛋白淀粉样变性心肌病（ATTR-CM）是一种进行性心肌病，通常伴有慢性肾脏疾病。慢性肾功能不全与较差的预后有关，但肾功能随时间变化的预后价值尚待确定。目的在一大群 ATTR-CM 患者中评估估计肾小球滤过率（eGFR）下降的预后重要性。这项回顾性、观察性、单中心队列研究对英国国家淀粉样变性中心（NAC）确诊的 ATTR-CM 患者进行了评估，这些患者在 2000 年 1 月至 2024 年 4 月期间接受了 eGFR 基线评估和 1 年随访评估。主要结果和测量指标主要结果是与肾功能下降（定义为 eGFR 下降&gt;20%）相关的全因死亡风险。结果2001 例患者中，平均（SD）年龄为 75.5（8.4）岁，263 例患者（13.1%）为女性。eGFR 变化的中位数（IQR）为-5 mlL/min/1.73 m2（-12 至 1），481 名患者（24.0%）出现肾功能下降。与肾功能稳定的患者相比，肾功能下降的患者更多具有 p.(V142I) 基因型（99 [20.6%] vs 202 [13.3%]；P &lt; .001），而且基线时心脏表型更严重，这体现在血清心脏生物标志物的中位数（IQR）浓度更高（N-端前 B 型钠尿肽 [NT-proBNP]: 2949 pg/mL [1759-5182] vs 2309 pg/mL [1146-4290]; P &lt; .001；肌钙蛋白 T：0.060 ng/mL [0.042-0.086] vs 0.052 ng/mL [0.033-0.074]; P &lt; .001），而基线中位数（IQR）肾功能在两组之间相似（eGFR：63 mL/min/1.73 m2 [51-77] vs 61 mL/min/1.73 m2 [49-77]; P = .41）。肾功能下降导致死亡风险增加 1.7 倍（危险比 [HR]，1.71；95% CI，1.43-2.04；P &lt; .001），3 种基因型的风险相似（野生型：HR，1.64；95% CI，1.43-2.04；P &lt; .001）：HR，1.64；95% CI，1.31-2.04；P.（V142I）：HR，1.70；95% CI，1.21-2.39；非（V142I）：HR，1.51；95% CI，0.87-2.61）（交互作用的 P = 0.93）和 3 个 NAC 疾病分期（1 期：HR，1.69；95% CI，1.22-2.32；2 期：HR，1.69；95% CI，1.30-2.18；3 期：HR，1.61；95% CI，1.11-2.35）（交互作用的 P = 0.97）。在调整了 NT-proBNP 的增加和门诊利尿剂的加强后，肾功能下降仍与死亡率独立相关（HR，1.48；95% CI，1.23-2.76；P &lt; .001）。结论与意义在这项回顾性队列研究中，ATTR-CM 患者的肾功能经常下降，即使在调整了 ATTR-CM 病情恶化的既定标志物后，肾功能下降仍与死亡风险增加相关。

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Kidney Outcomes in Transthyretin Amyloid Cardiomyopathy

ImportanceTransthyretin amyloid cardiomyopathy (ATTR-CM) is a progressive cardiomyopathy that commonly presents with concomitant chronic kidney disease. Chronic kidney dysfunction is associated with worse outcomes, but the prognostic value of changes in kidney function over time has yet to be defined.ObjectiveTo assess the prognostic importance of a decline in estimated glomerular filtration rate (eGFR) in a large cohort of patients with ATTR-CM.Design, Setting, and ParticipantsThis retrospective, observational, single-center cohort study evaluated patients diagnosed with ATTR-CM at the National Amyloidosis Centre (NAC) in the UK who underwent an eGFR baseline assessment and a follow-up assessment at 1 year between January 2000 and April 2024. Data analysis was performed in June 2024.Main Outcomes and MeasuresThe primary outcome was the risk of all-cause mortality associated with decline in kidney function (defined as a decrease in eGFR &gt;20%).ResultsAmong 2001 patients, mean (SD) age was 75.5 (8.4) years, and 263 patients (13.1%) were female. The median (IQR) change in eGFR was −5 mlL/min/1.73 m2 (−12 to 1), and 481 patients (24.0%) experienced decline in kidney function. Patients who experienced decline in kidney function more often had the p.(V142I) genotype than patients with stable kidney function (99 [20.6%] vs 202 [13.3%]; P &lt; .001) and had a more severe cardiac phenotype at baseline, as evidenced by higher median (IQR) concentrations of serum cardiac biomarkers (N-terminal pro-B-type natriuretic peptide [NT-proBNP]: 2949 pg/mL [1759-5182] vs 2309 pg/mL [1146-4290]; P &lt; .001; troponin T: 0.060 ng/mL [0.042-0.086] vs 0.052 ng/mL [0.033-0.074]; P &lt; .001), while baseline median (IQR) kidney function was similar between the 2 groups (eGFR: 63 mL/min/1.73 m2 [51-77] vs 61 mL/min/1.73 m2 [49-77]; P = .41). Decline in kidney function was associated with a 1.7-fold higher risk of mortality (hazard ratio [HR], 1.71; 95% CI, 1.43-2.04; P &lt; .001), with a similar risk across the 3 genotypes (wild type: HR, 1.64; 95% CI, 1.31-2.04; p.(V142I): HR, 1.70; 95% CI, 1.21-2.39; non-p.(V142I): HR, 1.51; 95% CI, 0.87-2.61) (P for interaction = .93) and the 3 NAC disease stages (stage 1: HR, 1.69; 95% CI, 1.22-2.32; stage 2: HR, 1.69; 95% CI, 1.30-2.18; stage 3: HR, 1.61; 95% CI, 1.11-2.35) (P for interaction = .97). Decline in kidney function remained independently associated with mortality after adjusting for increases in NT-proBNP and outpatient diuretic intensification (HR, 1.48; 95% CI, 1.23-2.76; P &lt; .001).Conclusions and RelevanceIn this retrospective cohort study, decline in kidney function was frequent in patients with ATTR-CM and was consistently associated with an increased risk of mortality, even after adjusting for established markers of worsening ATTR-CM. eGFR decline represents an independent marker of ATTR-CM disease progression that could guide treatment optimization in clinical practice.

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来源期刊

JAMA cardiology Medicine-Cardiology and Cardiovascular Medicine

CiteScore

45.80

自引率

1.70%

发文量

264

期刊介绍： JAMA Cardiology, an international peer-reviewed journal, serves as the premier publication for clinical investigators, clinicians, and trainees in cardiovascular medicine worldwide. As a member of the JAMA Network, it aligns with a consortium of peer-reviewed general medical and specialty publications. Published online weekly, every Wednesday, and in 12 print/online issues annually, JAMA Cardiology attracts over 4.3 million annual article views and downloads. Research articles become freely accessible online 12 months post-publication without any author fees. Moreover, the online version is readily accessible to institutions in developing countries through the World Health Organization's HINARI program. Positioned at the intersection of clinical investigation, actionable clinical science, and clinical practice, JAMA Cardiology prioritizes traditional and evolving cardiovascular medicine, alongside evidence-based health policy. It places particular emphasis on health equity, especially when grounded in original science, as a top editorial priority.