Tafamidis improves outcomes in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) versus placebo. ATTR-CM has historically been considered a predominantly male disease, and women are underrepresented in prior studies. Sex-specific data in contemporary patients treated with tafamidis remain limited. Data from two cohorts were pooled: an early-access cohort (NCT02791230) and patients enrolled in the Transthyretin Amyloidosis Outcomes Survey (THAOS; NCT00628745) in 2019–2023. This analysis evaluated sex-related differences in clinical characteristics and all-cause mortality, adjusting for age, cohort, baseline New York Heart Association (NYHA) class, and wild-type (ATTRwt-CM) versus variant (ATTRv-CM) disease. Overall, 1924 patients treated with tafamidis were included (1476 from the early-access cohort and 448 from THAOS), of whom 214 (11.1
Atherosclerotic cardiovascular disease (ASCVD) and chronic kidney disease (CKD) represent major public health challenges that contribute substantially to morbidity, mortality, and healthcare expenditures worldwide. Systemic inflammation (SI) has been recognized as a key pathophysiological mechanism underlying disease progression in both conditions; however, real-world evidence regarding its prevalence and associated clinical and economic impact remains limited, particularly in China. A cross-sectional analysis estimated the prevalence of ASCVD, CKD, and SI, alongside a retrospective cohort study evaluating clinical and economic burden associated with SI among patients with coexisting ASCVD and CKD, using data from Tianjin Health and Medical Data Platform (THMDP). SI was defined as C-reactive protein (CRP) or high-sensitivity CRP (hsCRP) ≥ 2 mg/L after excluding values attributable to acute events. Cox proportional hazards models were used to estimate adjusted hazard ratios (HRs) for major adverse cardiovascular events (MACE) and all-cause mortality. Between 2020 and 2023, ASCVD prevalence increased from 16.7
Transthyretin amyloid cardiomyopathy (ATTR-CM) is an underdiagnosed cause of heart failure, resulting from the deposition of misfolded transthyretin protein in the myocardium. While historically considered to be a male-predominant disease, an increasing number of studies have shown that ATTR-CM may be under-recognized in women, likely due to differences in disease presentation compared with men. Notably, left ventricular wall thickness ≥ 12 mm is frequently used as a marker for ATTR-CM workup; however, women with ATTR-CM often have a lower left ventricular wall thickness than men and may not meet this threshold despite significant myocardial involvement. Moreover, hormonal differences between male and female patients may result in distinct cardiac phenotypes. Socioeconomic factors disproportionately impacting women, such as increased caregiving burden, symptom dismissal, and fewer financial resources, are likely additional contributing factors. As a result, diagnostic delays up to several years have been noted in women. Improving the diagnosis of ATTR-CM in women requires a multifactorial approach, including reforming screening guidelines to reflect sex-specific differences in disease presentation and increasing awareness of ATTR-CM in the community. Furthermore, given the historical underrepresentation of women in clinical studies on ATTR-CM, further investigation is necessary to better understand the prevalence, treatment efficacy, and outcomes of ATTR-CM in women.
The optimal strategy and timing for percutaneous cervicocerebral artery revascularization (PCAR) combined with coronary artery bypass grafting (CABG) remain uncertain in patients with severe carotid or vertebral artery stenosis. We retrospectively analyzed adults undergoing concomitant or staged PCAR–CABG at Beijing Anzhen Hospital from January 2011 to December 2025. The primary endpoint was perioperative death or stroke. Strategies were compared using Firth penalized logistic regression. In the staged cohort, restricted cubic splines and interval-based analyses assessed the association between interprocedural interval and outcomes. Among 801 patients, 57 (7.1
Coronary artery disease in children arises from a diverse group of non-atherosclerotic conditions that may result in myocardial ischemia, ventricular dysfunction, and sudden cardiac death. Important etiologies include post-inflammatory vasculopathy following Kawasaki disease, transplant-related cardiac allograft vasculopathy, congenital coronary anomalies such as anomalous aortic origin of a coronary artery, and complications after coronary reimplantation during congenital heart surgery. The detection of myocardial ischemia in children is uniquely challenging, as symptoms are frequently absent or atypical, and traditional screening tools such as resting electrocardiography and transthoracic echocardiography lack sufficient sensitivity. A narrative review was performed of the literature on non-invasive myocardial ischemia evaluation in pediatric patients with acquired and congenital coronary artery disease, focusing on Kawasaki disease, congenital coronary anomalies, cardiac allograft vasculopathy, and postoperative conditions involving coronary artery manipulation. PubMed and Google Scholar were searched and supplemented by reference lists and guideline documents. Original studies, reviews, consensus statements, and society guidelines were qualitatively synthesized by disease category and imaging modality, with emphasis on diagnostic performance, clinical applications, safety considerations, limitations, and evidence gaps. Multimodality imaging plays a central role in the evaluation of pediatric coronary pathology. Stress echocardiography provides a widely available, radiation-free method for detecting inducible regional wall motion abnormalities but requires specialized expertise. Stress perfusion cardiac magnetic resonance imaging offers comprehensive assessment of ventricular function, myocardial perfusion, and scar tissue without ionizing radiation and is increasingly incorporated into surveillance strategies. Nuclear perfusion imaging remains a well-established technique but is used less frequently due to radiation exposure and modest specificity in some populations. Coronary computed tomography angiography provides high-resolution anatomic characterization of coronary origin, course, and luminal patency and is particularly valuable in congenital coronary anomalies and postsurgical coronary assessment. Optimal evaluation of pediatric coronary disease requires the integration of anatomic and functional imaging tailored to the underlying pathology and patient characteristics. Despite advances in multimodality imaging, pediatric-specific data remain limited, and many diagnostic thresholds are extrapolated from adult populations. Further multicenter studies are needed to refine risk stratification and establish evidence-based surveillance strategies for children at risk of myocardial ischemia.
Patients with atherosclerotic cardiovascular disease (ASCVD) and systemic inflammation (SI) or chronic kidney disease (CKD) have poorer outcomes than those without SI or CKD. This study evaluated the impact of both SI and CKD on major adverse cardiovascular events (MACE), healthcare costs, and healthcare resource utilization (HCRU) in patients with ASCVD. This retrospective cohort study examined the association between SI and revised MACE, healthcare costs, and HCRU among adult patients with ASCVD from the Komodo Healthcare Map®. CKD stage 3–4 was determined from claims or laboratory data. SI was defined as ≥ 1 high-sensitivity C-reactive protein (hsCRP) value of 2–10 mg/l (without SI, all hsCRP values < 2 mg/l). The primary endpoint, revised MACE, was defined as a composite of nonfatal myocardial infarction, nonfatal stroke, and all-cause mortality. Of 74,884 patients with ASCVD and ≥ 1 eligible hsCRP value, 8.5
In patients with Duchenne muscular dystrophy (DMD), the gene transfer therapy delandistrogene moxeparvovec delivers a functional form of dystrophin, which has been shown to stabilize or slow disease progression. We assessed cardiac safety of delandistrogene moxeparvovec in clinical trials with ≤ 5 years of follow-up. Data were collected from clinical trials 101 (NCT03375164, n = 4), 102 (NCT03769116, n = 41), ENDEAVOR (NCT04626674, n = 48), and EMBARK (NCT05096221, n = 125), which excluded patients with left ventricular ejection fraction (LVEF) < 40
Obstructions in the coronary arteries, arterial vasospasm, and microcirculation dysfunction may cause chronic coronary syndrome (CCS). Angina is a cardinal symptom of CCS and the journey of patients with angina is often filled with challenges and uncertainties. Diagnosis and treatment differences seem to vary between men and women with angina. This article is co-authored by a male and a female patient diagnosed with persistent stable angina and their treating cardiologist at the Heart Institute in São Paulo, Brazil. The patients share their personal experiences of more than 20 years of coping with pain and the related impact on their physical capacity, thus illustrating the challenging journey often faced by individuals with angina and how it differs between men and women. They describe the long pathway from the onset of symptoms to the correct diagnosis and list the various interventional procedures they underwent, focusing on the impact of angina on their daily lives. They also mention their participation in a clinical study. The cardiologist discusses the critical elements in the diagnostic process of angina, and reflects on how the misconception that heart disease primarily affects men can lead to delayed diagnosis and treatment for women suffering from this condition. Moreover, he emphasizes that many patients experience angina due to myocardial ischemia without obstructive coronary artery disease, highlighting the need to raise disease awareness among healthcare professionals, as well as in society. Finally, the physician underscores the importance of better patient assessment and medical treatment optimization to managing angina and meeting patients’ needs and expectations, while prioritizing patient quality of life. Chronic coronary syndrome is a long-lasting condition where the heart muscle receives insufficient blood supply. Patients with chronic coronary syndrome experience chest pain (angina) as the most common symptom, often described as a squeezing feeling or pressure in the chest. If not optimally controlled (persistent angina), this pain can negatively impact quality of life. In this article, two patients who have lived with angina for over 20 years share their stories, describing the first symptoms, the diagnostic process, and their active search for treatments that would give them some relief, including their participation in a clinical study. The patients recall their reaction to the angina diagnosis, describe the difficulties in managing the condition, and detail how angina has affected their daily lives. Their treating cardiologist, who is based at the Heart Institute in São Paulo, Brazil, also shares his perspectives by noting that these two patients are typical examples of the patients with angina he sees in the clinic. The physician explains why the journey of patients with angina is long, and why it differs between men and women. Finally, he encourages his colleagues to understand what the patients’ expectations are and to adjust treatment to control pain accordingly.
Sodium–glucose cotransporter-2 inhibitors (SGLT2i) are recommended as foundational therapy for patients with heart failure (HF) across all ejection fractions; however, real-world adoption remains inconsistent, and data from Middle Eastern health systems are limited. This study aimed to assess real-world utilization of SGLT2i among patients with HF in Saudi Arabia and to identify clinical factors associated with prescribing and outcomes. We conducted a multicenter retrospective cohort study of adult patients with HF receiving longitudinal care at tertiary cardiac centers between January 2016 and December 2024 in Saudi Arabia. HF phenotypes included reduced, mildly reduced, preserved, and improved ejection fraction. The primary outcome was SGLT2i use during follow-up. Multivariable logistic regression identified factors associated with prescribing. Exploratory outcomes included HF hospitalization and all-cause mortality. Among 1081 patients with HF (median age 66 years [IQR 56–76]; 50.8
Duchenne muscular dystrophy (DMD) is a life-limiting disease characterized by progressive muscle wasting of skeletal and cardiac myocytes. Nowadays, heart failure is the principal cause of death. Current treatment is still unsatisfactory. Empagliflozin has shown excellent results in adults with heart failure, and is licensed for adolescents > 10 years of age with type 2 diabetes mellitus. In an effort to repurpose empagliflozin for DMD-associated cardiomyopathy, we aim to describe its pharmacokinetic behaviour in this population, assess ease-of-swallow, monitor safety, explore efficacy and screen efficacy markers. This is a single-arm, single-centre, open-label phase 2a trial in children and adolescents aged 6 to ≤ 18 years with DMD-associated cardiomyopathy. Participants (n = 12) will take empagliflozin 10 mg once daily for 6 months, with a whole-day stay at visit 1 for the evaluation of pharmacokinetics, and follow-up visits at 1 week, 6 weeks, 3 months and 6 months. On top of acceptability (ease-of-swallow), which will be assessed at visit 1, further secondary endpoints include safety and efficacy clinical (e.g. heart rate, blood pressure), biochemical (e.g. NT-proBNP, haemoglobin, uric acid, electrolytes, renal function), imaging (echocardiography, cardiac magnetic resonance) and bioimpedance (intra- and extracellular fluid volume) parameters. Characterization of primary and secondary pharmacokinetic parameters will allow one to define the dose range for children and adolescents with DMD-associated cardiomyopathy, informing both current compassionate care and the design of future efficacy trials. Additionally, this trial will enable the identification of efficacy markers to be used as endpoints in future efficacy trials and in clinical practice. NCT06643442, ISRCTN 12497973, IRAS number 1009946.
Timely diagnosis and treatment are essential for improving outcomes and quality of life in patients with transthyretin (ATTR) amyloidosis. Early multisystem manifestations are often unrecognized, leading to diagnostic delays and misdiagnosis. Large-scale, multicountry, observational studies are needed to better characterize the real-world trajectory of these patients. OverTTuRe, an ANTHOLOGY study, is a retrospective, observational, descriptive, longitudinal, multicountry study using secondary data from claims databases, electronic health records, and healthcare registries. The primary aim of this analysis was to characterize baseline characteristics, early clinical manifestations, and outcomes in patients with hereditary transthyretin (ATTRv) amyloidosis from the United States (US), United Kingdom (UK), Japan, Denmark, and Sweden. Of 1502 patients identified, the predominant phenotype across countries was ATTRv amyloidosis with polyneuropathy (ATTRv-PN 51.3–63.7
IntroductionTransthyretin amyloid cardiomyopathy (ATTR-CM), a progressive disease characterized by the accumulation of misfolded transthyretin amyloid proteins in the myocardium, often leads to heart failure. The experiences and treatment priorities of individuals living with ATTR-CM have not been evaluated in depth.MethodsThis multinational patient advisory board qualitatively explored patients' experiences with ATTR-CM and their preferences for the attributes of current and emerging treatments for heart failure caused by ATTR-CM. In a pre-advisory board survey, participants answered benefit-risk scaling questions to gauge the importance of treatment side effects, treatment benefits, and modes of administration. The results of the survey were then discussed during the advisory board.ResultsOf the 13 participants who completed the survey (two in Australia, two in Canada, one in Switzerland, four in the United Kingdom, and four in the United States), 11 participated in the advisory board discussion and two in follow-up discussions. The survey results and advisory board discussion revealed tiredness and fatigue to be the most bothersome and injection site pain to be the least bothersome of the side effects evaluated. Lowering the risk of death was the most important and being able to easily take a bath or a shower was the least important of the treatment benefits evaluated. On average, once-daily oral administration was the most preferred mode of administration tested, over twice-daily oral administration, subcutaneous injection, and infusion, although participants' preferences were heterogeneous. Although the length of life was important, quality of life was also important to participants: they especially valued spending time doing the activities they enjoyed and prioritized these activities over basic functioning and self-care routines.ConclusionsIndividuals living with ATTR-CM have heterogeneous preferences. As the ATTR-CM treatment landscape expands to include treatments with distinct attributes, patients and care teams should participate in shared decision-making to prioritize patients' preferences and goals.
The inaugural 2025 Cardiometabolic Summit in Saclay, France, aimed to (i) disseminate the latest research on cardiometabolic diseases (CMDs) from the Maghreb and/or Middle East and North Africa region; (ii) discuss CMD management and provide clinical practice suggestions for improving adherence and reducing clinical inertia; and (iii) suggest policy and clinical practice initiatives to improve outcomes in patients with CMDs. Cardiovascular disease (CVD) is the leading cause of death worldwide, with 80
Improvement of symptoms, physical function, and quality of life is one treatment goal for patients with obstructive hypertrophic cardiomyopathy (HCM). Mavacamten improved left-ventricular outflow tract gradients in Chinese patients with obstructive HCM in the EXPLORER-CN trial. We report here a detailed analysis of patient-reported health status per the 23-item Kansas City Cardiomyopathy Questionnaire (KCCQ-23) in Chinese patients after 78 weeks of mavacamten. Patients who completed the double-blind, placebo-controlled (DBPC) period of EXPLORER-CN with no active safety concerns could enter a long-term extension (LTE) period to receive mavacamten for 48 weeks at either the 30-week dose (mavacamten-mavacamten group) or a once-daily starting dose of 2.5 mg (subsequently adjusted via pharmacodynamics-based dose titration: placebo-mavacamten group). Health status endpoints included KCCQ-23 clinical summary score (CSS), overall symptom score (OSS), total symptom score (TSS), physical limitations score, and quality-of-life scores through week 78. Week 78 endpoint analyses were descriptive. In the DBPC period, 54 patients received mavacamten and 27 received placebo; of these, 54 and 25, respectively, entered the LTE period. KCCQ-23 CSS, OSS, and TSS improved with mavacamten and worsened with placebo during the DBPC period (mean change baseline to week 30: 5.7, 6.4, and 8.1, respectively [mavacamten-mavacamten group] and – 5.4, – 4.3, and – 4.8, respectively [placebo-mavacamten group]). CSS, OSS, and TSS continued to improve in the mavacamten-mavacamten group during the LTE period (mean change baseline to week 78: 7.1, 8.2, and 10.0, respectively). Scores improved in the placebo-mavacamten group after switching to mavacamten (mean change week 30 to week 78: 7.3, 9.5, and 5.7, respectively). Similar improvements in KCCQ-23 physical limitations and quality-of-life scores with mavacamten were observed throughout both study periods. Long-term mavacamten treatment for up to 78 weeks led to sustained improvements in patient-reported health status, supporting long-term treatment with mavacamten for Chinese patients with symptomatic obstructive HCM. ClinicalTrials.gov identifier NCT05174416.
The multidisciplinary heart team (HT) remains the cornerstone of decision-making for complex cardiovascular disease. Large language models (LLMs) and other generative artificial intelligence models have recently emerged as potential decision support tools across diverse clinical settings. We sought to synthesize current evidence and quantitatively estimate concordance between LLM recommendations and HT decisions. A literature search was performed using PubMed, Scopus, and Web of Science for primary studies published between November 2022 and February 2026 that evaluated recommendations by LLMs against multidisciplinary HT decisions. Studies reporting overall agreement were included for quantitative pooling. Random-effects meta-analysis was performed to determine proportion of agreement. Four retrospective concordance studies were included regarding decision-making in coronary revascularization and aortic valve intervention. LLM–HT concordance ranged from 65
Cardiogenic shock (CS) is a syndrome of low cardiac output associated with high inpatient morbidity and mortality. Studies have supported the early transfer of patients with CS from spoke sites to the nearest regional hub center, forming the basis for the "hub-and-spoke" model. Contemporary CS algorithms revolve around invasive hemodynamic monitoring and temporary mechanical circulatory support guided by a multidisciplinary CS team. However, these protocols have limited applicability to the majority of spoke sites due to the limitations of resources, equipment, and personnel, further contributing to inequities in CS care. In this review, we seek to provide a blueprint for a generalizable approach to CS, an algorithm for intrahospital care escalation based on CS severity, and an example protocol detailing stabilization and monitoring strategies, independent of multidisciplinary CS teams.
Stress-test echocardiography with speckle tracking modality (STM) is sensitive to detect myocardial ischemia. Data on trimetazidine (TMZ) therapy’s effectiveness on myocardial ischemia and contractile function using STM is limited. This study aimed to assess the anti-ischemic effectiveness of trimetazidine 80 mg once daily (TMZ OD) in patients with symptomatic stable coronary artery disease (CAD) with a history of myocardial infarction (MI), in real-world practice. The METHOD study (NCT05210465) was a 6-month observational study evaluating TMZ OD’s anti-ischemic and antianginal effectiveness in CAD patients with MI history using stress echocardiography with dobutamine and STM. Adult outpatients starting TMZ OD alongside standard antianginal therapy were included. The primary outcome was the mean change in left ventricular global longitudinal strain (LV GLS) after 6 months. Secondary outcomes were changes in regional peak systolic deformation (RPSD) and global post-systolic index, along with traditional antianginal effectiveness indicators and quality of life (QoL) using the EQ-5D-5L questionnaire. Thirty-six patients were included. TMZ OD significantly improved LV GLS by 2
Hereditary transthyretin amyloidosis (hATTR) manifests as cardiomyopathy and/or polyneuropathy. Polyneuropathy may be suspected for a variety of reasons, but the clinical evidence supporting that impression varies. We used International Classification of Diseases, Tenth Revision (ICD-10) coding to broadly capture patients thought to have polyneuropathy in a V142I-predominant cohort and examined what clinical documentation actually supported the diagnosis and whether documentation level influences treatment selection. Retrospective chart review of 54 patients identified by co-occurring ICD-10 codes for transthyretin amyloidosis and polyneuropathy at a major academic medical center, with pathogenic TTR variant confirmation. Polyneuropathy codes were then classified by the level of clinical evidence supporting the diagnosis. Treatment with TTR stabilizers and gene silencers was assessed. Of 54 patients (90.7
Cardiovascular disease is the leading cause of mortality among individuals with kidney failure, yet evidence-based strategies to reduce cardiovascular risk in this population are limited. Kidney transplantation has been consistently shown to improve survival, including cardiovascular-specific outcomes among those with kidney failure. Substantial heterogeneity exists across transplant centers regarding the pre-transplant cardiovascular evaluation, and the evidence supporting specific testing strategies remains sparse. Current scientific guidelines and consensus statements on pre-kidney transplant cardiovascular assessment, which guide clinical practice, recommend asymptomatic testing in most individuals. However, emerging retrospective evidence suggests limited short-term benefit from routine asymptomatic screening. Ongoing randomized studies may clarify optimal strategies for repeat cardiovascular screening in waitlisted individuals who have undergone initial cardiovascular evaluation. In this review of kidney transplant candidates, we (i) examine contemporary data on cardiovascular screening (ii) highlight the diagnostic limitations of noninvasive testing in kidney failure and the lack of clear benefit from revascularization in the pre-kidney transplant setting, and (iii) outline the consequences of untimely and potentially unnecessary cardiovascular testing.
Among kidney transplant recipients (KTR), cardiovascular disease remains the leading cause of mortality, with heart failure (HF) being especially common in this population. Risk factors for HF in KTR can be categorized as traditional (risks relevant to the general population) versus nontraditional (unique to kidney disease and transplantation). Despite the substantial burden of cardiovascular disease, KTR have been excluded from large clinical trials in cardiovascular medicine and from landmark studies looking at the kidney-protective effects of novel cardiorenal metabolic agents, including sodium–glucose cotransporter 2 inhibitors (SGLT2i). Thus, there is uncertainty about the safety and efficacy of the use of guideline directed medical therapy (GDMT) for HF in this population. Long-term optimal use of GDMT in KTR is limited by various barriers, including the risk of hyperkalemia from concomitant use of renin–angiotensin inhibitors and mineralocorticoid receptor antagonists, fears about genitourinary infections from SGLT2i, and lack of evidence for kidney-protective effects from GDMT agents among KTR. Overcoming these barriers will require a multifaceted approach that involves evidence generation to understand the efficacy and safety of GDMT among KTR, implementation-based strategies to increase uptake of GDMT, and development of multispecialty combined clinical care approaches to care for KTR with HF.
