Cardiology and Therapy最新文献

{"title":"Real-World Safety and Effectiveness of Evolocumab in Korean Patients with Atherosclerotic Cardiovascular Disease or Familial Hypercholesterolemia: A Post-Marketing Surveillance Study.","authors":"Donggyu Moon, Seung-Ho Hur, Sang-Rok Lee, Myung Ho Jeong, Kyung Hoon Cho, Osung Kwon, JinSeon Jeong, Ki Dong Yoo","doi":"10.1007/s40119-025-00430-8","DOIUrl":"10.1007/s40119-025-00430-8","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"619-633"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680816/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145249931","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Hitting the Sweet Spot: A Review of SGLT2i in Cardiovascular Medicine.","authors":"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","doi":"10.1007/s40119-025-00436-2","DOIUrl":"10.1007/s40119-025-00436-2","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"531-553"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680823/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145273897","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Reverse Takotsubo Cardiomyopathy After an Adverse Reaction to Alteplase Treated with Intravenous Epinephrine.","authors":"Charles G Kinzig, Matthew R Carey, Lauren P Waldman, Robert P Giugliano","doi":"10.1007/s40119-025-00432-6","DOIUrl":"10.1007/s40119-025-00432-6","url":null,"abstract":"<p><p>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.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"695-706"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680817/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145343688","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incomplete Right Bundle Branch Block: A Narrative Review of Clinical Relevance, Diagnostic Pitfalls, and Prognostic Implications.","authors":"Jozef Dodulík, Jiří Plášek, Jiří Vrtal, Jan Václavík","doi":"10.1007/s40119-025-00429-1","DOIUrl":"10.1007/s40119-025-00429-1","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"495-512"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680827/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144944026","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Real-World Adherence and Effectiveness of Inclisiran in Lowering LDL-C: Results from 1 Year of Follow-Up.","authors":"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","doi":"10.1007/s40119-025-00438-0","DOIUrl":"10.1007/s40119-025-00438-0","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusions: </strong>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.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"671-685"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680804/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145586129","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Survival in a Contemporary, Real-World Cohort of Patients with Mixed-Phenotype Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis: An Analysis from THAOS.","authors":"Jonas Wixner, Angela Dispenzieri, Leslie Amass, Martin Carlsson, Steve Riley, Evan Powers, Jeffery W Kelly","doi":"10.1007/s40119-025-00421-9","DOIUrl":"10.1007/s40119-025-00421-9","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifier NCT00628745.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"583-599"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680828/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144590545","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Incidence and Healthcare Resource Utilization Among Patients with Hypertrophic Cardiomyopathy Hospitalized for Heart Failure in Japan.","authors":"Yuika Ikeda, Bruno Casaes Teixeira, Thomas Laurent, Tsunehisa Yamamoto","doi":"10.1007/s40119-025-00427-3","DOIUrl":"10.1007/s40119-025-00427-3","url":null,"abstract":"<p><strong>Introduction: </strong>Heart failure (HF) is one of the most common complications in patients with hypertrophic cardiomyopathy (HCM); however, there are limited data on HCM burden in Japan. We evaluated the burden of HF hospitalization and factors that predispose patients with HCM to HF hospitalization.</p><p><strong>Methods: </strong>This retrospective observational database study used a hospital-based claims database from January 01, 2011, to December 31, 2023, provided by Medical Data Vision Co., Ltd. The primary objective of the study was to calculate the incidence of first HF hospitalization after HCM diagnosis. A nested case-control design compared patients with or without hospitalization to identify factors associated with HF hospitalization. Hospitalization costs and outcomes after discharge were also described.</p><p><strong>Results: </strong>Of 12,145 patients with newly diagnosed HCM without HF hospitalization, 525 were hospitalized with HF during the follow-up period. The mean age ± standard deviation (SD) of the overall study population at cohort entry date was 71.4 ± 14.0 years, and 45.8% were female patients. The incidence of HF hospitalization was 17.2 events/1000 patient-years. Patients with HCM hospitalized for HF had higher rates of comorbidities, including HF (45.9%), diabetes mellitus (28.6%), hypertension (23.0%), atrial fibrillation (AF; 21.3%), myocardial infarction (MI; 17.5%), arrhythmia except AF (15.0%), and dyslipidemia (13.1%), than patients without HF hospitalization. Significant predictors of hospitalization among patients with HCM were AF (odds ratio [OR] 1.63; 95% confidence interval [CI] 1.18-2.25; p = 0.003), MI (OR 1.68; 95% CI 1.20-2.35; p = 0.003), HF (OR 1.82; 95% CI 1.39-2.39; p < 0.001), chronic obstructive pulmonary disease (OR 2.30; 95% CI 1.08-4.89; p = 0.031), and loop diuretics (OR 4.35; 95% CI 3.33-5.69; p < 0.001). The average costs, length of hospital stay, and overall mortality rate associated with HF hospitalization were 1035 kJPY (~ 156,750 USD), 20.0 days, and 8.8%, respectively.</p><p><strong>Conclusions: </strong>HF hospitalization in patients with HCM imposes a significant clinical and economic burden.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"601-618"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680822/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144667260","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Impact of Office and Ambulatory Blood Pressure on Mortality in Patients with Coronary Heart Disease.","authors":"Alejandro de la Sierra, Ernest Vinyoles, Manuel Gorostidi, Julián Segura, Luis M Ruilope","doi":"10.1007/s40119-025-00437-1","DOIUrl":"10.1007/s40119-025-00437-1","url":null,"abstract":"<p><strong>Introduction: </strong>There is scarce evidence on the prognostic impact of ambulatory blood pressure monitoring (ABPM) in patients with coronary heart disease (CHD). We aimed to investigate the risks of all-cause and cardiovascular mortality associated with office and ambulatory blood pressure (BP) in patients with CHD.</p><p><strong>Methods: </strong>Prospective, observational study with 2679 patients (mean age 67 years, 35% women) with CHD, selected from the Spanish ABPM Registry. Associations between BP indices, as well as BP phenotypes, with all-cause and cardiovascular mortality were assessed by means of Cox-survival models adjusted for clinical confounders and alternative BP measures.</p><p><strong>Results: </strong>During a median follow-up of 8.7 years, 740 patients (27.6%) died, 331 (12.4%) from cardiovascular causes. Office systolic BP was not associated with mortality, while 24-h (hazard ratio: 1.36; 95% confidence interval: 1.26-1.47), daytime (1.33; 1.23-1.44) and nighttime (1.33; 1.24-1.43) systolic BP were all associated with all-cause mortality, after adjustment for confounders and office BP. Masked hypertension (1.49; 1.10-2.01), sustained hypertension (1.46; 1.19-1.80), isolated daytime hypertension (1.80; 1.21-2.68) and isolated nighttime hypertension (1.33; 1.09-1.63), but not white-coat hypertension, were all associated with an increased risk of mortality compared to reference groups. A blunted nocturnal dipping (1.08; 1.01-1.16, for 1 standard deviation change in systolic night-to-day ratio) was also associated with an increased risk of death. Similar results were obtained for cardiovascular mortality.</p><p><strong>Conclusion: </strong>ABPM indices are more closely related with the risk of death than office BP in patients with CHD. These results emphasize the use of ABPM for a more accurate BP evaluation in this group of patients. Graphical abstract available for this article.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"635-649"},"PeriodicalIF":2.7,"publicationDate":"2025-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12680810/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145273800","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Rationale and Design of ANTHOLOGY: An ATTR Amyloidosis Real-World Evidence Program Aiming to Address Gaps in Amyloidosis Care.","authors":"Julian D Gillmore, Katrin Hahn, J Gustav Smith, Isabel Conceição, Zhuang Tian, Martha Grogan, Christina Pao, Eric Wittbrodt, Krister Järbrink, Mia A Papas, Margot K Davis","doi":"10.1007/s40119-025-00402-y","DOIUrl":"10.1007/s40119-025-00402-y","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with amyloid transthyretin (ATTR) amyloidosis typically experience rapid disease progression, poor treatment outcomes, irreversible loss of health-related quality of life (HRQoL), and premature mortality. Early diagnosis is vital. However, diagnostic delays and misdiagnosis are common due to under-recognition of early signs and symptoms.</p><p><strong>Methods: </strong>ANTHOLOGY is an ATTR amyloidosis program, evidence generation, and quality improvement opportunity comprised of two multi-country, longitudinal, observational, real-world evidence studies: OverTTuRe (ClinicalTrials.gov identifier, NCT06355934) and MaesTTRo (NCT06465810). OverTTuRe will retrospectively extract and analyze secondary data from a broad spectrum of sources, and MaesTTRo will prospectively collect and analyze data from patient-reported outcome questionnaires, electronic health records, and insurance claims.</p><p><strong>Planned outcomes: </strong>The primary objectives of OverTTuRe are to describe contemporary patient characteristics, treatment patterns and disease outcomes, and to characterize healthcare resource utilization (HCRU) and HRQoL in patients diagnosed with ATTR amyloidosis. Describing patient characteristics and HCRU before diagnosis is a secondary objective. The primary objectives of MaesTTRo are to describe patient characteristics, disease history and treatment patterns from diagnosis, and to prospectively define and assess the real-world effectiveness of current therapies. Secondary objectives are to compare the characteristics of patients according to the therapy received and compare the real-world effectiveness of current therapies. Exploratory objectives are to identify risk factors for disease progression and to describe healthcare costs.</p><p><strong>Conclusions: </strong>ANTHOLOGY aims to broaden understanding of the contemporary epidemiology of ATTR amyloidosis, identify opportunities to accelerate diagnosis, and assess real-world comparative effectiveness of treatments. This knowledge will be used to define world-class patient care, improve treatment outcomes and HRQoL, inform updates to clinical practice guidelines and treatment pathways, and transform ATTR amyloidosis management through evidence aimed at improving the quality of the current standard of care TRIAL REGISTRATION: ClinicalTrials.gov identifier, NCT06355934 (OverTTuRe) and NCT06465810 (MaesTTRo).</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"477-490"},"PeriodicalIF":2.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12379657/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143662568","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"An External Control Arm for the Oral Factor XIa Inhibitor Asundexian Phase 2 Trial in Atrial Fibrillation (PACIFIC-AF) Using Electronic Health Records.","authors":"Tatsiana Vaitsiakhovich, Alexander Hartenstein, Stephen Privitera, Manesh R Patel, Jonathan P Piccini, Craig I Coleman, Khaled Abdelgawwad, Gerlind Holberg, Igor Khorlo, Hardi Mundl, Bernhard Schaefer, Thomas Viethen, Kai Vogtländer, Alexander Vowinkel, Frank Kleinjung","doi":"10.1007/s40119-025-00411-x","DOIUrl":"10.1007/s40119-025-00411-x","url":null,"abstract":"<p><strong>Introduction: </strong>The aim of this study was to assess the applicability of an external control arm (ECA) approach in the clinical development of the oral factor XIa inhibitor asundexian for stroke prevention in patients with atrial fibrillation (AF), using prospectively collected data from the phase 2 PACIFIC-AF trial (NCT04218266) and real-world individual-level data from patients with AF treated with apixaban in the Optum^® de-identified Electronic Health Record data set (Optum^® EHR) 2013-2019.</p><p><strong>Methods: </strong>To build ECAs, real-world patients meeting trial eligibility criteria were matched to patients enrolled in PACIFIC-AF. The primary outcome was the composite of International Society on Thrombosis and Haemostasis-defined major bleeding or clinically relevant non-major bleeding. Event rates were compared between PACIFIC-AF and ECAs at 85 days of trial duration and projected up to 2 years.</p><p><strong>Results: </strong>Overall, 160,153 real-world patients met PACIFIC-AF eligibility criteria and were matched to patients from the PACIFIC-AF apixaban arm on 101 variables, with matching ratios of 1:10, 1:5, and 1:1. At day 85, the number of events for the primary outcome was 92 (3.68%) in the 1:10 ECA (2500 patients) and 6 (2.40%) in the PACIFIC-AF apixaban arm (250 patients), with incidence rates of 16.67 (90% confidence interval [CI] 13.92-19.63) and 11.10 (90% CI 4.83-19.45) per 100 person-years, respectively.</p><p><strong>Conclusions: </strong>ECAs matching the PACIFIC-AF apixaban arm could be built from EHRs with concordant event rates for key trial endpoints. The ECA approach enabled the determination of event rates for treatment duration up to 2 years, thereby informing the asundexian pivotal phase 3 trial in AF.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"403-421"},"PeriodicalIF":2.7,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12378266/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144076200","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}