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Digital Strategies for Recruitment to Improve Diversity and Inclusion in Prospective Cardiovascular Clinical Research. 提高前瞻性心血管临床研究多样性和包容性的招聘数字策略。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-16 DOI: 10.1007/s40119-025-00435-3
Sneha S Jain, Fatima Rodriguez, Marco V Perez, Jingzhi Yu, Sumana Shashidhar, Susan Swope, Kaylin Nguyen, Manisha Desai, Haley Hedlin, Svati H Shah, Adrian F Hernandez, Mintu P Turakhia, Kenneth W Mahaffey

Introduction: Cardiovascular trials often underrepresent non-White participants, women, and older individuals.

Methods: We evaluated the impact of digital recruitment on improving diversity in two large prospective cardiovascular studies: the Apple Heart Study (AHS) and the Project Baseline Health Study (PBHS). While both leveraged digital tools, their strategies differed-AHS used a passive, app-based approach followed by a direct-to-participant outreach effort, while PBHS implemented an enrollment model to reach a prespecified diversity goal.

Results: While both methods led to a cohort of participants who were comparably aligned with the US Census, we found that intentional, goal-driven digital outreach, and participant selection targeting demographic representation improved inclusion of historically underrepresented groups.

Conclusions: These findings suggest that digital tools, when paired with intentional strategy, may help support more inclusive, representative cardiovascular research.

心血管试验通常未充分代表非白人参与者、女性和老年人。方法:我们在两项大型前瞻性心血管研究中评估了数字招募对改善多样性的影响:苹果心脏研究(AHS)和项目基线健康研究(PBHS)。虽然两者都利用了数字工具,但它们的策略有所不同——ahs采用了被动的、基于应用程序的方法,然后是直接面向参与者的推广工作,而PBHS则采用了一种注册模式,以达到预先设定的多样性目标。结果:虽然这两种方法都产生了与美国人口普查相当一致的参与者队列,但我们发现,有意的、目标驱动的数字外展和针对人口代表性的参与者选择改善了历史上代表性不足的群体的纳入。结论:这些发现表明,当数字工具与有意策略相结合时,可能有助于支持更具包容性和代表性的心血管研究。
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引用次数: 0
Resistant Blood Pressure: New Frontiers in Interventional Hypertension Therapy. 顽固性血压:介入高血压治疗的新领域。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-10 DOI: 10.1007/s40119-025-00434-4
Jeffrey D Taylor, Chukwunonyelum Uche, Vanessa Rowe, Aleyah Hattab, Arvind Draffen, Vicki Groo, Adhir Shroff

Hypertension remains a leading global health challenge, affecting over 1.3 billion individuals worldwide and contributing significantly to cardiovascular morbidity and mortality. In recent years, interventional device-based therapies have emerged as promising adjuncts by targeting sympathetic overactivity and autonomic dysregulation, key mechanisms in the pathogenesis of hypertension. Currently, the use of these interventional therapies is primarily reserved for patients with resistant hypertension (RH) who remain uncontrolled despite optimal medical therapy. This review provides an overview of the evolving landscape of interventional approaches, including renal denervation (RDN), baroreceptor activation therapy (BAT), carotid body modulation, hepatic denervation, and cardiac neuromodulation. Among these, RDN has the most robust clinical trial evidence, while other neuromodulatory strategies are being evaluated in early-phase studies. Additionally, this review underscores the importance of systematically identifying and managing secondary causes of hypertension, such as primary aldosteronism, renovascular disease, and obstructive sleep apnea, before considering procedural interventions. As the field advances, these therapies may assume a more prominent role in precision-based hypertension management.

高血压仍然是一个主要的全球健康挑战,影响着全世界超过13亿人,并显著导致心血管疾病发病率和死亡率。近年来,基于介入装置的治疗已经成为有希望的辅助手段,针对交感神经过度活跃和自主神经失调,这是高血压发病的关键机制。目前,这些介入治疗主要用于顽固性高血压(RH)患者,这些患者尽管接受了最佳药物治疗,但仍未得到控制。本文综述了介入治疗方法的发展概况,包括肾去神经支配(RDN)、压力受体激活疗法(BAT)、颈动脉体调节、肝去神经支配和心脏神经调节。其中,RDN具有最有力的临床试验证据,而其他神经调节策略正在早期研究中进行评估。此外,本综述强调了在考虑程序性干预之前,系统地识别和管理高血压继发原因的重要性,如原发性醛固酮增多症、肾血管疾病和阻塞性睡眠呼吸暂停。随着该领域的发展,这些疗法可能在基于精确的高血压管理中发挥更突出的作用。
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引用次数: 0
Treatment Patterns and Healthcare Resource Utilization Among Patients with Obstructive Hypertrophic Cardiomyopathy in Japan: A Claims Database Study. 日本阻塞性肥厚性心肌病患者的治疗模式和医疗资源利用:一项索赔数据库研究
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-11 DOI: 10.1007/s40119-025-00431-7
Yuika Ikeda, Bruno Casaes Teixeira, Aman Malhi, Thomas Laurent, Yukako Matsuo

Introduction: Obstructive hypertrophic cardiomyopathy (HCM) increases the risk of developing cardiac complications and can impact long-term outcomes, necessitating appropriate treatment. However, real-world data on how patients with obstructive HCM are currently managed, especially in Japan, remain limited. This study evaluated pharmacological treatment patterns, incidence of nonpharmacological interventions, and healthcare resource utilization (HCRU) associated with obstructive HCM management in Japan.

Methods: This retrospective longitudinal study analyzed claims data (N = 12,503,399) provided by insurers between April 1, 2014, and December 31, 2022. The primary objective was to describe the treatment pattern of patients with obstructive HCM. Any medication changes (beta-blockers [BBs], non-dihydropyridine [DHP] calcium channel blockers [CCBs], and sodium channel blockers [SCBs]), incidence of nonpharmacological interventions, and HCRU were assessed.

Results: Among 403 eligible patients, 72.5% were female, with a mean ± standard deviation age of 75.5 ± 11.7 years. The most common index medication was BB only (74.19%), followed by BB + SCB (10.42%), SCB (8.93%), BB + CCB (1.99%), CCB (3.72%), BB + CCB + SCB (0.5%), and CCB + SCB (0.25%). Overall, 123 (30.5%) patients experienced a treatment change within 12 months after the first prescription, of whom 5 (1.2%) switched to a different medication, 51 (12.7%) had an add-on to the index medication, and 67 (16.6%) discontinued their treatment. SCB was prescribed as initial treatment to 20.1% of patients. The discontinuation rate at 12 months was the highest for SCB (22%), followed by BB (16%) and CCB (12%). Dose reduction was the highest for SCB (22.2%), followed by BB (12%) and CCB (4%). The rate of nonpharmacological interventions (events/100 person-years) was similar for pacemaker implantation and septal ablation (2.0), followed by implantable cardioverter-defibrillators (1.2) and mitral valve replacement (1.1).

Conclusions: These findings will guide future research aimed at optimizing obstructive HCM management.

梗阻性肥厚性心肌病(HCM)增加发生心脏并发症的风险,并可能影响长期预后,需要适当的治疗。然而,关于目前如何管理阻塞性HCM患者的实际数据,特别是在日本,仍然有限。本研究评估了日本与阻塞性HCM管理相关的药物治疗模式、非药物干预发生率和医疗资源利用率(HCRU)。方法:回顾性纵向研究分析了2014年4月1日至2022年12月31日期间保险公司提供的索赔数据(N = 12,503,399)。主要目的是描述梗阻性HCM患者的治疗模式。评估任何药物变化(β受体阻滞剂[BBs],非二氢吡啶[DHP]钙通道阻滞剂[CCBs]和钠通道阻滞剂[SCBs]),非药物干预的发生率和HCRU。结果:403例符合条件的患者中,女性占72.5%,平均±标准差年龄75.5±11.7岁。指标用药最多的是BB(74.19%),其次是BB + SCB(10.42%)、SCB(8.93%)、BB + CCB(1.99%)、CCB(3.72%)、BB + CCB + SCB(0.5%)、CCB + SCB(0.25%)。总体而言,123例(30.5%)患者在第一次处方后的12个月内经历了治疗变化,其中5例(1.2%)切换到不同的药物,51例(12.7%)在指标药物的基础上增加了治疗,67例(16.6%)停止了治疗。20.1%的患者将SCB作为初始治疗。12个月停药率最高的是SCB(22%),其次是BB(16%)和CCB(12%)。剂量减少最多的是SCB(22.2%),其次是BB(12%)和CCB(4%)。起搏器植入和室间隔消融术的非药物干预率(事件/100人年)相似(2.0),其次是植入式心律转复除颤器(1.2)和二尖瓣置换术(1.1)。结论:这些发现将指导未来旨在优化阻塞性HCM管理的研究。
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引用次数: 0
Correction: Impella Versus VA-ECMO for Patients with Cardiogenic Shock: Preliminary Cost-Effectiveness Analysis in the Italian Context. 更正:Impella与VA-ECMO治疗心源性休克患者:意大利背景下的初步成本-效果分析。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 DOI: 10.1007/s40119-025-00433-5
Carla Rognoni, Vittoria Ardito, Dario La Fauci, Marina Pieri, Anna Mara Scandroglio, Rosanna Tarricone
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引用次数: 0
Real-World Safety and Effectiveness of Evolocumab in Korean Patients with Atherosclerotic Cardiovascular Disease or Familial Hypercholesterolemia: A Post-Marketing Surveillance Study. Evolocumab在韩国动脉粥样硬化性心血管疾病或家族性高胆固醇血症患者中的安全性和有效性:上市后监测研究
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-08 DOI: 10.1007/s40119-025-00430-8
Donggyu Moon, Seung-Ho Hur, Sang-Rok Lee, Myung Ho Jeong, Kyung Hoon Cho, Osung Kwon, JinSeon Jeong, Ki Dong Yoo

Introduction: The efficacy and safety of evolocumab have been established in clinical trials. This post-marketing surveillance (PMS) study investigated the real-world safety and effectiveness of evolocumab in patients with atherosclerotic cardiovascular disease (ASCVD) or familial hypercholesterolemia (FH) in Korea.

Methods: Patients who received evolocumab for approved indications in Korea between April 2017 and April 2023 across 43 centers were enrolled in this prospective PMS study. Safety (adverse events [AEs] and serious adverse events [SAEs]) and effectiveness (low-density lipoprotein cholesterol [LDL-C] lowering) were assessed up to 52 weeks. AEs and SAEs were categorized by LDL-C levels at 12 weeks: < 20, 20-40, and ≥ 40 mg/dL.

Results: Overall, 539 patients (mean age 58.8 ± 11.1 years, 78.7%/21.3% male/female) were included in the safety analysis set, while 361 patients assessed for LDL-C at least once during follow-up were included in the effectiveness analysis set. The majority of patients had ASCVD (myocardial infarction [55.1%], angina [33.5%], cerebral infarction [13.2%], peripheral artery disease [4.5%]). AEs were reported by 23.9% of patients, with SAEs occurring in 7.1%. When stratified by LDL-C levels, there was no difference in the frequencies of AEs and SAEs. Among all adverse drug reactions (ADRs; 2.4%), the most common were myalgia (0.9%) and headache (0.4%), with no reports of hemorrhagic stroke or nasopharyngitis. Evolocumab significantly reduced LDL-C levels from a baseline median of 100.2 mg/dL by 70.6% at week 12 and 69.0% at 52 weeks, achieving a target LDL-C goal (< 55 mg/dL and > 50% reduction) in 68.1% and 69.2% of patients, respectively.

Conclusions: Evolocumab effectively lowered LDL-C levels and had a favorable safety profile in Korean patients with ASCVD or FH. The incidence of AEs was lower than in clinical trials, and no ADRs were observed among the SAEs. These results support the use of evolocumab as a viable therapeutic option.

evolocumab的有效性和安全性已在临床试验中得到证实。这项上市后监测(PMS)研究调查了evolocumab在韩国治疗动脉粥样硬化性心血管疾病(ASCVD)或家族性高胆固醇血症(FH)患者的安全性和有效性。方法:2017年4月至2023年4月期间在韩国43个中心接受evolocumab批准适应症的患者纳入了这项前瞻性经前综合征研究。安全性(不良事件[ae]和严重不良事件[sae])和有效性(降低低密度脂蛋白胆固醇[LDL-C])评估至52周。结果:总体而言,539例患者(平均年龄58.8±11.1岁,78.7%/21.3%男性/女性)被纳入安全性分析组,361例患者在随访期间至少评估一次LDL-C被纳入有效性分析组。大多数患者有ASCVD(心肌梗死[55.1%],心绞痛[33.5%],脑梗死[13.2%],外周动脉疾病[4.5%])。23.9%的患者报告了ae, 7.1%的患者报告了SAEs。当按LDL-C水平分层时,ae和sae的频率没有差异。在所有药物不良反应(adr; 2.4%)中,最常见的是肌痛(0.9%)和头痛(0.4%),无出血性中风或鼻咽炎的报告。Evolocumab在第12周显著降低LDL-C水平,从基线中位数100.2 mg/dL下降70.6%,在第52周显著降低69.0%,分别在68.1%和69.2%的患者中实现了LDL-C目标(降低50%)。结论:Evolocumab在韩国ASCVD或FH患者中有效降低LDL-C水平,并具有良好的安全性。ae的发生率低于临床试验,且未见不良反应。这些结果支持使用evolocumab作为一种可行的治疗选择。
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引用次数: 0
Hitting the Sweet Spot: A Review of SGLT2i in Cardiovascular Medicine. 击中甜蜜点:心血管医学中SGLT2i的综述。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-10 DOI: 10.1007/s40119-025-00436-2
Zaid A Zayyad, Neil Gupta, Aanya Roy, Swetha Kalagara, Atreya Mishra, Edward Salem, Suhas Rathna Seshadri, Deema Gichi, Bayan A Hammad, Adriana Ene, Kayla Torres, Stephanie Dwyer Kalzuna, Adhir R Shroff

Sodium-glucose cotransporter 2 inhibitors (SGLT2i) are a class of medications initially developed for glycemic control in type 2 diabetes mellitus (T2DM) but found to have broader cardiometabolic impacts. In this review, we discuss the proposed mechanisms of action of SGLT2i and review important trials that have demonstrated improvements in cardiovascular outcomes. SGLT2i have demonstrated benefits in the treatment of major categories of cardiovascular disease (CVD) and have been shown to reduce major adverse cardiovascular events (MACE) in patients with diabetes and CVD or renal disease. Findings have been translated into recommendations in clinical guidelines by the American College of Cardiology (ACC), American Heart Association (AHA), European Society of Cardiology (ESC), and American Diabetes Association (ADA). It is important to consider a patient's comorbid conditions, as well as potential medication side effects, prior to initiating SGT2i. While large trials have established the cardiovascular (CV) and renal benefits of SGLT2i, a number of studies are now exploring their role in acute care settings and novel patient populations.

钠-葡萄糖共转运蛋白2抑制剂(SGLT2i)是一类最初用于2型糖尿病(T2DM)血糖控制的药物,但发现具有更广泛的心脏代谢影响。在这篇综述中,我们讨论了SGLT2i的作用机制,并回顾了已经证明改善心血管预后的重要试验。SGLT2i在治疗主要类别心血管疾病(CVD)方面已被证明有益处,并已被证明可减少糖尿病和CVD或肾脏疾病患者的主要不良心血管事件(MACE)。研究结果已被美国心脏病学会(ACC)、美国心脏协会(AHA)、欧洲心脏病学会(ESC)和美国糖尿病协会(ADA)转化为临床指南的建议。在启动SGT2i之前,考虑患者的合并症以及潜在的药物副作用是很重要的。虽然大型试验已经确定了SGLT2i对心血管(CV)和肾脏的益处,但许多研究正在探索它们在急性护理环境和新患者群体中的作用。
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引用次数: 0
Reverse Takotsubo Cardiomyopathy After an Adverse Reaction to Alteplase Treated with Intravenous Epinephrine. 静脉肾上腺素治疗阿替普酶不良反应后的逆转Takotsubo心肌病。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-10-22 DOI: 10.1007/s40119-025-00432-6
Charles G Kinzig, Matthew R Carey, Lauren P Waldman, Robert P Giugliano

Takotsubo (stress) cardiomyopathy (TTC) is an uncommon acute heart failure syndrome characterized by transient hypocontractility that usually affects a circumferential segment of the myocardium along the heart's apicobasal axis and spans multiple coronary artery territories. Classically, TTC occurs after an intense physical or emotional insult and is thought to be caused by catecholamine toxicity. The most frequent anatomic variant presents with apical hypokinesis and basal hyperkinesis, but the hypocontractility may also localize to the mid-ventricle or base, also known as "reverse TTC." Here, we describe a middle-aged woman who developed profound acute hypoxemic respiratory failure and mixed cardiogenic-distributive shock after an adverse reaction to alteplase treated with high-dose epinephrine. The patient was found to have a severely depressed left ventricular ejection fraction (10-15%) with apex-sparing hypokinesis and no evidence of obstructive coronary artery disease, consistent with reverse TTC. The patient's ejection fraction recovered to the normal range within days with supportive measures. This case highlights the distinctive echocardiographic features of this rare, potentially life-threatening form of TTC.Videos are available for this article.

应激性心肌病(TTC)是一种罕见的急性心力衰竭综合征,其特征是一过性收缩功能减退,通常影响沿心脏尖基轴的心肌周段,并跨越多个冠状动脉区域。通常,TTC发生在强烈的身体或情感侮辱之后,被认为是由儿茶酚胺毒性引起的。最常见的解剖变异表现为顶端运动不足和基底运动不足,但收缩不足也可能局限于中心室或基底,也被称为“反向TTC”。在这里,我们描述了一位中年妇女,她在对阿替普酶和大剂量肾上腺素治疗的不良反应后发生了严重的急性低氧性呼吸衰竭和混合性心源性休克。发现患者左心室射血分数严重下降(10-15%),伴有保留心尖的运动不足,无阻塞性冠状动脉疾病的证据,与反向TTC一致。患者的射血分数在几天内恢复到正常范围。本病例突出了这种罕见的、可能危及生命的TTC的独特超声心动图特征。本文提供视频。
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引用次数: 0
Incomplete Right Bundle Branch Block: A Narrative Review of Clinical Relevance, Diagnostic Pitfalls, and Prognostic Implications. 不完全右束分支阻滞:临床相关性、诊断缺陷和预后影响的叙述性回顾。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-08-27 DOI: 10.1007/s40119-025-00429-1
Jozef Dodulík, Jiří Plášek, Jiří Vrtal, Jan Václavík

Introduction: Incomplete right bundle branch block (iRBBB) is a frequent electrocardiographic (ECG) finding, often considered benign. However, recent evidence suggests it may be associated with underlying structural or electrical abnormalities, particularly in selected populations.

Methods: We conducted a narrative review of population-based cohorts, mechanistic studies, and clinical trials focused on the prevalence, pathophysiological mechanisms, differential diagnosis, and prognostic implications of iRBBB.

Results: iRBBB is common in athletes and individuals with pulmonary or structural heart diseases. Although frequently asymptomatic, it may reflect right ventricular strain, pulmonary hypertension, or a predisposition to arrhythmias such as atrial fibrillation. Specific ECG features, comorbidities, and clinical context help to differentiate benign from pathologic iRBBB.

Conclusions: iRBBB should not be routinely regarded as a harmless variant. In high-risk individuals, it may carry clinical and prognostic significance, warranting further evaluation. Standardized criteria and additional prospective studies are needed to better understand its implications. Infographic available for this article. INFOGRAPHIC.

不完全右束分支阻滞(iRBBB)是一种常见的心电图(ECG)发现,通常被认为是良性的。然而,最近的证据表明,它可能与潜在的结构或电异常有关,特别是在特定人群中。方法:我们对基于人群的队列、机制研究和临床试验进行了叙述性回顾,重点关注iRBBB的患病率、病理生理机制、鉴别诊断和预后意义。结果:iRBBB在运动员和患有肺部或结构性心脏病的个体中很常见。虽然经常无症状,它可能反映右心室紧张,肺动脉高压,或易患心律失常,如心房颤动。特定的心电图特征、合并症和临床背景有助于区分良性和病理性iRBBB。结论:iRBBB不应被常规视为一种无害的变异。在高危人群中,它可能具有临床和预后意义,值得进一步评估。需要标准化的标准和更多的前瞻性研究来更好地理解其影响。本文提供的信息图。信息图表。
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引用次数: 0
Real-World Adherence and Effectiveness of Inclisiran in Lowering LDL-C: Results from 1 Year of Follow-Up. 实际依从性和Inclisiran降低LDL-C的有效性:来自1年随访的结果
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-11-24 DOI: 10.1007/s40119-025-00438-0
Christie M Ballantyne, Timothy E Graham, Lucinda J Van Anglen, Bruce J Iteld, Harvey Serota, Xiaoli Niu, Sean McElligott, Kelly E Hanna, Tyler J Varisco

Introduction: Atherosclerotic cardiovascular disease (ASCVD) is a global health concern. Reducing low-density lipoprotein cholesterol (LDL-C) is critical in ASCVD prevention and treatment. Inclisiran, a novel RNA therapeutic agent, offers a promising solution by lowering LDL-C with twice-yearly dosing after an initial and 3-month dose. The study objective was to evaluate adherence to inclisiran and its lipid-lowering effectiveness over a year of treatment.

Methods: This retrospective cohort study involved patients over 18 years old with ASCVD or hyperlipidemia who initiated inclisiran between January 1, 2022, and May 17, 2023, in US outpatient clinics. LDL-C reduction was evaluated in patients with ≥ 1 pre- and post-index LDL-C measurement by comparing baseline levels to the lowest value within 12 months after initiating inclisiran.

Results: Overall, 225 patients initiated inclisiran. The mean age was 69.9 years and 50.7% were female. Most patients (81.8%) had ASCVD. Most patients (91.6%) received a second dose, and 84.5% of these received a third dose. Overall, 202 patients had ≥ 2 LDL-C measurements, with a mean baseline LDL-C of 134.8 mg/dl. Mean absolute LDL-C reduction was 66.1 (standard deviation: 45.6) mg/dl, corresponding to a 46.8% (95% confidence interval: 42.7-51.0) relative reduction, and 46.8% achieved ≥ 50% reduction. Patients on concurrent statins and those without prior anti-proprotein convertase subtilisin/kexin type 9 monoclonal antibodies experienced the largest relative LDL-C reductions: 55.4% and 51.1%, respectively.

Conclusions: Inclisiran significantly reduced LDL-C. The dosing schedule promoted high adherence in a real-world setting, particularly among older adults with ASCVD. These findings indicate inclisiran may be a particularly valuable addition to lipid-lowering strategies.

动脉粥样硬化性心血管疾病(ASCVD)是一个全球性的健康问题。降低低密度脂蛋白胆固醇(LDL-C)是预防和治疗ASCVD的关键。Inclisiran是一种新型RNA治疗剂,它提供了一种很有希望的解决方案,即在初始剂量和3个月剂量后每年两次给药以降低LDL-C。该研究的目的是评估对inclisiran的依从性及其在一年治疗期间的降脂效果。方法:这项回顾性队列研究纳入了2022年1月1日至2023年5月17日期间在美国门诊诊所开始使用inclisiran的18岁以上ASCVD或高脂血症患者。通过将基线水平与开始使用inclisiran后12个月内的最低值进行比较,对≥1次指数前和指数后LDL-C测量的患者进行LDL-C降低评估。结果:总体而言,225例患者开始使用inclisiran。平均年龄69.9岁,女性占50.7%。大多数患者(81.8%)患有ASCVD。大多数患者(91.6%)接受了第二剂,其中84.5%接受了第三剂。总体而言,202例患者LDL-C≥2次,平均基线LDL-C为134.8 mg/dl。平均绝对LDL-C降低为66.1(标准差:45.6)mg/dl,相对降低46.8%(95%置信区间:42.7-51.0),46.8%达到≥50%的降低。同时服用他汀类药物的患者和先前没有抗蛋白转化酶枯草杆菌素/ keexin 9型单克隆抗体的患者LDL-C的相对降低幅度最大:分别为55.4%和51.1%。结论:Inclisiran显著降低LDL-C。该给药方案在现实环境中促进了高依从性,特别是在患有ASCVD的老年人中。这些发现表明,inclisiran可能是降脂策略中特别有价值的补充。
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引用次数: 0
Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS. 一项当代、现实世界的混合表现型转甲状腺蛋白淀粉样心肌病患者的生存率:来自THAOS的分析。
IF 2.7 Q2 CARDIAC & CARDIOVASCULAR SYSTEMS Pub Date : 2025-12-01 Epub Date: 2025-07-09 DOI: 10.1007/s40119-025-00421-9
Jonas Wixner, Angela Dispenzieri, Leslie Amass, Martin Carlsson, Steve Riley, Evan Powers, Jeffery W Kelly

Introduction: Tafamidis is approved to treat transthyretin amyloid cardiomyopathy (ATTR-CM). Many patients with ATTR-CM present with a mixed phenotype of both cardiac and neurologic symptoms, but real-world effectiveness studies of tafamidis in this population are lacking. This study assessed survival and other outcomes in a real-world, contemporary cohort of tafamidis-treated and untreated patients with mixed-phenotype ATTR-CM.

Methods: The Transthyretin Amyloidosis Outcomes Survey (THAOS) was a longitudinal, observational, phase 4 study of patients with transthyretin amyloidosis and asymptomatic carriers of pathogenic transthyretin gene variants and was completed in June 2023. This analysis included a contemporary cohort of patients enrolled in THAOS in 2019-2023 who were characterized as having mixed-phenotype ATTR-CM at enrollment. The tafamidis-treated cohort received the approved dose of tafamidis (meglumine 80 mg/free acid 61 mg) throughout the study, and the untreated cohort never received tafamidis.

Results: In tafamidis-treated (n = 116) and untreated patients (n = 223), respectively, median age at enrollment was 77.8 and 72.8 years, and 42.2% and 77.6% had variant ATTR-CM. Survival rates at 30 months were 81.5% (95% CI 66.7-90.2) in tafamidis-treated patients and 75.1% (95% CI 66.1-82.0) in untreated patients. Median yearly incidence of cardiovascular-related hospitalizations was 0.89 for tafamidis-treated and 1.70 for untreated patients, and median duration of cardiovascular-related hospitalizations was 7.0 and 11.5 days, respectively. There were 13 (11.2%) and 40 (17.9%) deaths in the respective groups.

Conclusion: Patients with mixed-phenotype ATTR-CM treated with the approved dose of tafamidis had numerically higher survival rates, a numerically lower rate of cardiovascular-related hospitalizations, and fewer deaths than untreated patients. These data parallel recent results for patients with predominantly cardiac ATTR-CM from THAOS and extend results of ATTR-ACT to a contemporary, real-world, mixed-phenotype population.

Trial registration: ClinicalTrials.gov identifier NCT00628745.

Tafamidis被批准用于治疗转甲状腺素淀粉样心肌病(atr - cm)。许多atr - cm患者表现为心脏和神经系统症状的混合表型,但缺乏他非他汀在这一人群中的实际有效性研究。该研究评估了现实世界中接受他非他汀治疗和未接受治疗的混合表型atr - cm患者的生存和其他结果。方法:转甲状腺素淀粉样变性结局调查(THAOS)是一项纵向、观察性的4期研究,研究对象是转甲状腺素淀粉样变性患者和致病性转甲状腺素基因变异的无症状携带者,研究于2023年6月完成。该分析纳入了2019-2023年纳入THAOS的当代患者队列,这些患者在入组时具有混合表型atr - cm。在整个研究过程中,接受他非他胺治疗的队列接受了批准剂量的他非他胺(meglumine 80 mg/free acid 61 mg),而未接受治疗的队列从未接受过他非他胺。结果:在接受他非他汀治疗(n = 116)和未接受他非他汀治疗(n = 223)的患者中,入组时的中位年龄分别为77.8岁和72.8岁,42.2%和77.6%的患者有变异型atr - cm。30个月时,接受他非他汀治疗的患者生存率为81.5% (95% CI 66.7-90.2),未接受他非他汀治疗的患者生存率为75.1% (95% CI 66.1-82.0)。他非他汀治疗组心血管相关住院的中位年发病率为0.89,未治疗组为1.70,心血管相关住院的中位时间分别为7.0天和11.5天。两组分别有13例(11.2%)和40例(17.9%)死亡。结论:与未经治疗的患者相比,经批准剂量的他非他汀治疗的混合表型atr - cm患者生存率更高,心血管相关住院率更低,死亡率更低。这些数据与最近在THAOS中主要为心脏atr - cm患者的结果相似,并将atr - act的结果扩展到当代,现实世界,混合表型人群。试验注册:ClinicalTrials.gov识别码NCT00628745。
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Cardiology and Therapy
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