Cardiology and Therapy最新文献

{"title":"VERINA: A Phase IV Prospective Multicenter Study of Vericiguat in Indian Patients with Worsening Heart Failure and Reduced Ejection Fraction.","authors":"Sandeep Seth, Atul Abhyankar, Jabir Abdullakutty, Suvro Banerjee, Kamal Sharma, Vinod Vijan, Nirav Chandulal Bhalani, Vijay Kumar Chopra, Rakesh Kumar Aggarwal, Abraham Oomman, Sandeep Bansal, Rajendra Kumar Jain, Pravin Kahale, Priyanka Sodhi, Disha Gupta, Ashish Gawde","doi":"10.1007/s40119-026-00482-4","DOIUrl":"https://doi.org/10.1007/s40119-026-00482-4","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with heart failure (HF) with reduced ejection fraction (HFrEF) remain at high risk of cardiovascular (CV) death and hospitalization following a worsening heart-failure event. Data on vericiguat, a prescription oral medication used to treat symptomatic chronic HF, in adult Indian patients are limited.</p><p><strong>Methods: </strong>Adults with chronic HFrEF and a worsening heart-failure event were enrolled in a prospective, multicenter, open-label, single-arm phase IV study design. Participants received vericiguat in addition to foundational medical therapy (FMT) and were followed for approximately 48 weeks. The primary endpoint was CV death or first hospitalization for HF. Secondary endpoints included CV death, first hospitalization for HF, all-cause death or first hospitalization for HF, all-cause death and safety outcomes.</p><p><strong>Results: </strong>Of 231 patients who underwent screening, 205 received vericiguat and comprised the safety analysis set; of these, 203 patients constituted the full analysis set. The mean age of participants was 58 years. Baseline use of angiotensin receptor-neprilysin inhibitors and sodium-glucose cotransporter-2 inhibitors was 52.7% and 75.6%, respectively. At week 48, Kaplan-Meier (KM) estimates were 10.1% for the primary endpoint, 3.3% for CV death, 9.1% for first hospitalization for HF, 11.5% for all-cause death or first hospitalization for HF, and 5.9% for all-cause death. Attainment of the target dose was 91.6%, with 69.5% of participants reaching and maintaining 10 mg by week 4. Study drug-related adverse events (AEs) were reported in 3.4% of participants, and treatment-emergent AEs requiring discontinuation in 2.0% of participants.</p><p><strong>Conclusion: </strong>In Indian patients with worsening HFrEF, high target-dose attainment and no new safety signals were observed during 48 weeks of vericiguat treatment.</p><p><strong>Trial registration: </strong>Clinical Trials Registry-India: CTRI/2022/11/047636.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-10-20","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149227328","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Anticoagulation in Mechanical Circulatory Support: Contemporary Challenges and Future Directions in Cardiovascular Pharmacotherapy.","authors":"Lama Alfehaid, Mansour Alomran, Ahlam Althobaiti, Abdulmajeed Alshehri, Reem Bahmaid, Arif Albulushi","doi":"10.1007/s40119-026-00480-6","DOIUrl":"https://doi.org/10.1007/s40119-026-00480-6","url":null,"abstract":"<p><p>Mechanical circulatory support (MCS) is increasingly used in patients with cardiogenic shock, advanced heart failure, and severe respiratory failure; however, anticoagulation management remains challenging due to the competing risks of thrombosis, bleeding, hemolysis, and device dysfunction. In this review, we synthesize contemporary evidence on anticoagulant selection, monitoring strategies, and device-specific management across major MCS platforms, including extracorporeal membrane oxygenation, temporary ventricular assist devices, Impella devices, and durable left ventricular assist devices. Unfractionated heparin remains the cornerstone anticoagulant across most MCS platforms, while anti-factor Xa-guided monitoring is increasingly incorporated into clinical practice in many centers alongside activated partial thromboplastin time and other monitoring modalities. Direct thrombin inhibitors represent important alternatives, particularly for patients with heparin-induced thrombocytopenia or heparin resistance. Recent advances include bicarbonate-based purge strategies for Impella support and evolving antithrombotic approaches in HeartMate 3 recipients, introduced by the ARIES-HM3 trial. Emerging investigational strategies, including direct oral anticoagulants, factor XI/XII inhibition, and precision anticoagulation approaches, show promise for improving hemocompatibility; however, current evidence remains limited, and further prospective studies are required before these strategies can be routinely adopted. Overall, contemporary evidence supports an individualized approach to anticoagulation based on device characteristics, patient-specific risk profiles, and current guideline recommendations, while highlighting important knowledge gaps that warrant further high-quality prospective investigation.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-10-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149206548","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Neurologic Stability During Tafamidis Treatment in Transthyretin Amyloidosis with a Mixed Phenotype: A Multicenter Real-World Study.","authors":"Nowell M Fine, Nicholas S Streicher, Isabel Conceição, João Agostinho, Laura Obici, Giovanni Palladini, Martin Carlsson, Leslie Amass, Farooq H Sheikh","doi":"10.1007/s40119-026-00479-z","DOIUrl":"https://doi.org/10.1007/s40119-026-00479-z","url":null,"abstract":"<p><strong>Introduction: </strong>Real-world evidence on the effect of tafamidis in mixed-phenotype transthyretin (TTR) amyloidosis (ATTR amyloidosis) is limited. This study assessed changes in neurologic and other measures in patients with mixed-phenotype ATTR amyloidosis treated with tafamidis free acid 61 mg for ≥ 12 months in routine clinical practice.</p><p><strong>Methods: </strong>This observational, non-interventional, retrospective multicenter cohort study used anonymized data from four centers. Adults with variant or wild-type mixed-phenotype ATTR amyloidosis treated with tafamidis 61 mg for ≥ 12 months with pre- and post-treatment neurologic assessments were eligible. The primary objective was to assess changes in neurologic measures after ≥ 12 months of treatment; secondary and exploratory objectives were to assess changes in cardiac and frailty measures, modified body mass index (mBMI), and TTR concentration.</p><p><strong>Results: </strong>Overall, 67 patients were included (median age 77.0 years; 79.1% male patients; 55.2% with variant ATTR amyloidosis). Median duration of treatment with tafamidis 61 mg was 35.0 months. Neuropathy Impairment Score (NIS) motor subscale, NIS reflex subscale, and Medical Research Council Scale for Muscle Strength scores showed a median change of 0 from baseline after ≥ 12 months of treatment. Cardiac measures demonstrated minimal change, although interventricular septal thickness (median change, 0.1 cm; p = 0.017) and left atrial diameter (median change, 0.3 cm; p = 0.018) increased modestly. Median change in mBMI was 6.7 g/L × kg/m² (p = 0.925), and 90% of patients with data remained within the same Essential Frailty Toolset score range. TTR concentration increased significantly (median change, 3.0 mg/dL; p < 0.001).</p><p><strong>Conclusions: </strong>In this real-world study of patients with variant or wild-type mixed-phenotype ATTR amyloidosis, neurologic, cardiac, mBMI, and frailty measures were largely maintained after ≥ 12 months of treatment with tafamidis 61 mg. TTR concentration significantly increased. These findings provide real-world evidence of maintenance of neurologic function during treatment with tafamidis in mixed-phenotype ATTR amyloidosis.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov, NCT06393465.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126618","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A Comparative Evaluation of ChatGPT and Google Search Output for Caregiver Education in Tetralogy of Fallot.","authors":"Shreyas V Menon, Armaun D Rouhi, Jason J Han","doi":"10.1007/s40119-026-00481-5","DOIUrl":"https://doi.org/10.1007/s40119-026-00481-5","url":null,"abstract":"<p><strong>Introduction: </strong>Caregivers of children with tetralogy of Fallot (TOF) frequently seek medical information online to better understand their child's diagnosis, treatment, and long-term care. Recently, generative artificial intelligence (AI) tools, including Google AI Overview and ChatGPT, have emerged as widely accessible sources of health information. However, the quality, readability, and accuracy of these platforms for caregiver education in congenital heart disease remain poorly characterized.</p><p><strong>Methods: </strong>Forty frequently asked caregiver questions regarding TOF were identified using Google's \"People Also Ask\" feature. Each question was submitted to ChatGPT-5.5 and entered as a Google search query, generating 80 total responses. Google search output comprised 37 AI Overviews and three organic search excerpts. Responses were evaluated for word count, Flesch Reading Ease (FRE), and Flesch-Kincaid grade level (FKGL). Two independent reviewers assessed response accuracy using a 5-point Likert scale, while one reviewer evaluated educational quality using a modified Ensuring Quality Information for Patients (EQIP) instrument.</p><p><strong>Results: </strong>Compared with Google search output (37 AI Overviews and three organic search excerpts), ChatGPT-5.5 generated significantly longer responses (327.9 ± 117.0 vs. 192.5 ± 85.9 words, p < 0.001), with a higher reading level (FKGL: 23.7 ± 8.5 vs. 15.4 ± 3.6, p < 0.001) and lower readability (FRE: 17.3 ± 14.9 vs. 27.4 ± 11.7, p < 0.001). ChatGPT-5.5 demonstrated higher reviewer-rated accuracy (4.95 ± 0.19 vs. 4.40 ± 0.44, p < 0.001) and higher modified EQIP scores (86.44% ± 7.01% vs. 59.62% ± 13.99%, p < 0.001).</p><p><strong>Conclusions: </strong>Under the study's prompting conditions, ChatGPT-5.5 responses received higher reviewer-rated accuracy and modified EQIP scores than Google search output but were longer and had higher calculated reading-grade levels. These findings do not establish improved caregiver comprehension, which was not directly assessed.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-10-07","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149126502","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cardiovascular Outcomes Among Adults with Type 2 Diabetes or Obesity Receiving Carbohydrate-Reduced Nutrition via Telemedicine: A Retrospective Claims-Based Cohort Study.","authors":"Caroline G P Roberts, Priya V Shanmugam, Shaminie J Athinarayanan, Adam J Wolfberg, Stephen Raskin, Francisco Lopez-Jimenez","doi":"10.1007/s40119-026-00478-0","DOIUrl":"https://doi.org/10.1007/s40119-026-00478-0","url":null,"abstract":"<p><strong>Introduction: </strong>Cardiovascular (CV) risk factors improve with carbohydrate-reduced nutrition, but the impact on CV outcomes is unknown. We assessed the incidence of CV outcomes associated with a carbohydrate-reduced telemedicine intervention in a retrospective claims-based cohort study.</p><p><strong>Methods: </strong>Adults with type 2 diabetes or obesity receiving individualized nutritional therapy (INT) between January 1, 2016 and June 1, 2025, were compared with propensity score-matched controls. Primary outcomes included (1) 3-point major adverse CV events (MACE), defined as nonfatal myocardial infarction, nonfatal stroke, or death from any cause (MACE-3); (2) 6-point MACE, adding percutaneous coronary intervention, hospitalization for heart failure or unstable angina (MACE-6); (3) all new-onset CV disease (CVD) events; and (4) new-onset hypertension.</p><p><strong>Results: </strong>In 4877 participants in each cohort (mean age, 51 [SD 9.5] years; 2939 [60.3%] female), INT was associated with lower risk of all primary outcomes. Incidence per 1000 person-years was 4.1 vs. 9.3 for MACE-3, 5.7 vs. 10.8 for MACE-6, 27.7 vs. 36.9 for all new-onset CVD, and 41.8 vs. 49.3 for new-onset hypertension. Hazard ratios were 0.44 (95% CI, 0.29-0.65; P < 0.001) for MACE-3, 0.52 (95% CI, 0.37-0.73; P < 0.001) for MACE-6, 0.70 (95% CI, 0.59-0.82; P < 0.001) for all CVD, and 0.81 (95% CI, 0.70-0.93; P < 0.001) for new-onset hypertension.</p><p><strong>Conclusions: </strong>Individualized telemedicine nutrition therapy was associated with lower CV event incidence compared to controls, suggesting the intervention may confer cardioprotection.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-10-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149104668","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Comparative Effectiveness of TTR Stabilizers for the Treatment of ATTR-CM Using Real-World Evidence.","authors":"Richard Wright, Trejeeve Martyn, Allison Keshishian, Elizabeth Nagelhout, Bret Zeldow, Margarita Udall, David Lanfear, Daniel P Judge","doi":"10.1007/s40119-026-00476-2","DOIUrl":"10.1007/s40119-026-00476-2","url":null,"abstract":"<p><strong>Introduction: </strong>Progression of transthyretin amyloid cardiomyopathy (ATTR-CM) can cause worsening congestion, requiring diuretic intensification (DI), heart failure (HF)-related hospitalizations (HFH), and death. Acoramidis and tafamidis are approved for ATTR-CM, but head-to-head trials are lacking. We evaluated the real-world comparative effectiveness of acoramidis versus tafamidis in newly treated patients with ATTR-CM.</p><p><strong>Methods: </strong>Retrospective study using Komodo Healthcare Map® US claims data tokenized to Claritas. Patients aged ≥18 years newly initiating acoramidis or tafamidis between 12/11/2024 and 04/30/2025 with ≥1 prescription claim (first defined as index date), ≥6 months continuous enrollment preindex date, no multiple myeloma, light chain amyloidosis, or hematopoietic stem cell treatments, and no prior treatments for ATTR-CM were included and followed until disenrollment, death, treatment switch, or study end (07/31/2025). Outcomes included DI (initiation or dose-equivalent escalation of oral loop diuretics, parenteral loop diuretic use, or addition of thiazide-like diuretic) and composite of DI, HFH (inpatient admission with HF-related International Classification of Diseases, 10th Revision, Clinical Modification, diagnosis code in any position), and mortality. Propensity score weighting balanced baseline characteristics, disease severity, comorbidity burden, and baseline medication use. Time-to-event outcomes were assessed using weighted Cox proportional hazards models.</p><p><strong>Results: </strong>After weighting, patients treated with acoramidis (n = 170) and tafamidis (weighted sample size = 448) were comparable. Mean follow-up was 139 and 143 days, respectively. DI cumulative incidence curves separated early and remained divergent. Acoramidis significantly reduced the hazard of DI events by 43% versus tafamidis (11.8% vs. 20.5%; hazard ratio [HR], 0.57; 95% CI, 0.35-0.92; p = 0.021). Acoramidis had a significantly lower risk of composite events, with a 34% hazard reduction compared with tafamidis (17.6% vs. 26.4%; HR, 0.66; 95% CI, 0.44-0.99; p = 0.046).</p><p><strong>Conclusions: </strong>In this first real-world comparative effectiveness study of newly treated patients, acoramidis had a significantly lower risk of DI events and composite events of DI, HFH, and mortality than tafamidis, potentially supporting improved clinical stability with acoramidis initiation. Additional evaluation with longer follow-up, larger cohorts, and/or prospective clinical outcomes is warranted. Graphical abstract available for this article.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149029734","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Adverse Remodeling and Prognostic Significance of the Systemic Right Ventricle Across Congenital Heart Disease Subtypes.","authors":"Kernfan Puthikitakawiwong, Chodchanok Vijarnsorn, Teerapong Tocharoenchok, Jarupim Soongswang, Prakul Chanthong, Paweena Chungsomprasong, Thita Pacharapakornpong, Ploy Thammasate, Auttapol Srivichean, Kritvikrom Durongpisitkul, Kriangkrai Tantiwongkosri, Ekarat Nitiyarom, Thaworn Subtaweesin","doi":"10.1007/s40119-026-00477-1","DOIUrl":"https://doi.org/10.1007/s40119-026-00477-1","url":null,"abstract":"<p><strong>Introduction: </strong>Patients with congenitally corrected transposition of the great arteries (ccTGA), atrial-corrected transposition (acTGA), and right-ventricular-dominant univentricular heart (UVH) are predisposed to systemic right ventricular dysfunction (sRVd). However, lesion-specific differences in the incidence and timing of adverse outcomes remain unclear. This study aimed to compare the incidence of sRVd, tricuspid regurgitation (TR), and mortality across sRV lesions, and to identify their predictors.</p><p><strong>Methods: </strong>Retrospective 207 patients with an sRV diagnosed between 1975 and 2023 were categorized into acTGA (n = 53), UVH post-Fontan (n = 75), and ccTGA (n = 79). Incidence, survival analyses and Cox proportional hazards models were assessed for sRVd, TR and mortality.</p><p><strong>Results: </strong>The overall incidence of sRVd was 33.3% at a median age of 26.9 years. At the latest follow-up, sRVd was predominant in the acTGA group (37.7%), compared with UVH (16%) and ccTGA (16.5%). Remarkably, freedom of sRVd at 20 and 40 years were 65.9%, 13% in acTGA group; 30.5%, 5.8% in the UVH group and 43.5%, 26.6% in the ccTGA group, respectively (p < 0.001). Independent predictors included Ebsteinoid TV, pulmonary atresia, acTGA, and UVH. The 20- and 40-years survival was 62.3%, 14.7% (acTGA), 35.2%, 1.5% (UVH), and 46.8%, 22.7% (ccTGA) (p < 0.001). Mortality was associated with PA and NYHA functional class III-IV. Moderate-to-severe TR occurred in one-third of patients. Notably, TR interventions were predominant in ccTGA (p = 0.009).</p><p><strong>Conclusions: </strong>SRV lesions exhibit distinct clinical phenotypes: acTGA carries the highest overall prevalence of dysfunction, UVH demonstrates the earliest onset, and ccTGA encounters the highest frequency of TR interventions. Despite these differing deterioration patterns, patients with biventricular configurations confer a survival advantage over single-ventricle physiology. These results underscore the importance of anatomy-specific monitoring and timely functional assessment, while also accounting for differences in age and follow-up duration when evaluating long-term outcomes. Graphical abstract available for this article.</p><p><strong>Clinical trial registry number: </strong>TCTR20250107006.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-18","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"149001372","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Phenotypic Considerations for Tafamidis Use in a Real-World Cohort of Older Patients with Transthyretin Amyloid Cardiomyopathy.","authors":"Carlo Fumagalli, Francesco Cappelli, Mathew S Maurer, Fabian Aus dem Siepen, Angela Dispenzieri, Pablo Garcia-Pavia, Mazen Hanna, Martin Carlsson, Thibaud Damy","doi":"10.1007/s40119-026-00474-4","DOIUrl":"https://doi.org/10.1007/s40119-026-00474-4","url":null,"abstract":"<p><strong>Introduction: </strong>Disease-modifying therapies for transthyretin amyloid cardiomyopathy (ATTR-CM) improve clinical outcomes, yet older patients are undertreated. In this analysis, we evaluated survival and cardiovascular-related hospitalizations (CVH) among patients treated with tafamidis (tafamidis free acid at 61 mg or bioequivalent tafamidis meglumine at 80 mg) aged < 80, 80-85, and > 85 years to assess effects of baseline clinical phenotype on outcomes.</p><p><strong>Methods: </strong>Data were pooled from an early access cohort (ClinicalTrials.gov ID: NCT02791230) and the Transthyretin Amyloidosis Outcomes Survey registry (NCT00628745; enrollment 2019-2023; cardiac/mixed phenotype). Outcomes included all-cause mortality (ACM), CVH, and ACM or CVH. Cox proportional hazards and negative binomial regression models were adjusted for age, sex, TTR genotype, phenotype, New York Heart Association (NYHA) class, and estimated glomerular filtration rate (eGFR).</p><p><strong>Results: </strong>Among the 1924 patients enrolled in the study, 1188, 505, and 231 were aged < 80, 80-85, and > 85 years, respectively. Patients aged > 85 versus 80-85 and < 80 years had lower baseline mean eGFR (39.7 vs. 49.8 and 64.1 mL/min/1.73 m², respectively) and lower modified body mass index (mBMI; 1047.1 vs. 1084.0 and 1134.3 kg g/[m² L]). Also, more patients aged > 85 years versus 80-85 and < 80 years were NYHA class IV (3.3% vs. 0.8% and 0.9%). Kaplan-Meier estimated survival rates were significantly lower in patients aged > 85 and 80-85 years versus those aged < 80 years at months 30 (56.3% and 66.2% vs. 77.0%) and 42 (42.9% and 57.2% vs. 70.9%). ACM risk was higher in patients aged > 85 years than in those aged < 80 years (hazard ratio 1.49, 95% confidence interval [CI] 1.11-2.01). Total annual CVH rates in patients aged > 85, 80-85, and < 80 years were 0.79, 0.65, and 0.59, respectively. CVH and ACM or CVH risks were similar across groups. Patients aged 80-85 and < 80 years with preserved mBMI, eGFR, or NYHA class I/II symptoms had similar survival rates. Across groups, patients with NYHA class III symptoms or eGFR < 45 mL/min/1.73 m² had similar survival rates.</p><p><strong>Conclusion: </strong>Patients aged ≥ 80 years who were treated with tafamidis had outcomes comparable to those of younger patients when clinical vulnerability markers were considered. These findings support individualized treatment decisions based on patient's overall clinical profile and multidimensional assessment rather than age alone.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifiers: NCT02791230, NCT00628745.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148954818","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sex Differences in a Contemporary, Real-World Cohort of Patients with Transthyretin Amyloid Cardiomyopathy Treated with Tafamidis.","authors":"Marianna Fontana, Alberto Aimo, Martha Grogan, Maria Generosa Crespo-Leiro, Mathew S Maurer, Fabian Aus dem Siepen, Mazen Hanna, Diego Delgado, Martin Carlsson, Thibaud Damy, Margot K Davis","doi":"10.1007/s40119-026-00475-3","DOIUrl":"https://doi.org/10.1007/s40119-026-00475-3","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>Overall, 1924 patients treated with tafamidis were included (1476 from the early-access cohort and 448 from THAOS), of whom 214 (11.1%) were women. A smaller proportion of women than men had ATTRwt-CM (59.8% vs. 88.9%), whereas a greater proportion had ATTRv-CM (40.2% vs. 11.1%; p < 0.0001). Women were more frequently of African descent (23.9% vs. 7.4%; overall p < 0.001). Age at enrollment, age at symptom onset, and time from symptom onset to diagnosis were similar between sexes (p > 0.05). Women appeared more symptomatic at baseline, with a higher proportion in NYHA class III/IV (37.7% vs. 29.4%; overall p = 0.036) and classified as National Amyloidosis Centre stage II/III (68.1% vs. 47.7%; overall p = 0.023), despite similar N-terminal pro-B-type natriuretic peptide levels (p > 0.05). In adjusted analyses, women had a higher estimated glomerular filtration rate than men (β [men vs. women] = - 10.61; p < 0.001). Over a mean follow-up of 1.5 years, men had a higher risk of all-cause mortality than women (adjusted hazard ratio [men vs. women], 1.60, 95% CI 1.12-2.30).</p><p><strong>Conclusions: </strong>In this analysis of contemporary patients with ATTR-CM treated with tafamidis, women represented a small proportion of the overall population, and a greater proportion had ATTRv-CM compared with men. Although women appeared more symptomatic at presentation, men had a higher risk of all-cause mortality.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov identifiers: NCT02791230 and NCT00628745.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":""},"PeriodicalIF":3.2,"publicationDate":"2026-09-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148886629","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cardiac Safety Outcomes in Delandistrogene Moxeparvovec Clinical Trials for Duchenne Muscular Dystrophy with Up to 5 Years of Follow-up.","authors":"Aravindhan Veerapandiyan, John Bourke, Jonathan H Soslow, John W Day, Craig M McDonald, Jerry R Mendell, Craig M Zaidman, Stefanie Mason, Jianfeng Meng, Mark Vivien, Alexander P Murphy, Christoph Wandel, James Richardson","doi":"10.1007/s40119-026-00457-5","DOIUrl":"10.1007/s40119-026-00457-5","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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%. Adverse events and cardiac echocardiography were assessed regularly in all trials. Troponin I was assessed regularly in ENDEAVOR and EMBARK. Cardiac magnetic resonance imaging (MRI; without gadolinium enhancement) was assessed within an EMBARK substudy.</p><p><strong>Results: </strong>Of 218 patients (baseline mean age [range], 6.4 [3.2-20.2] years; mean LVEF [range], 63.8% [48.9-78.0%]), 210 (96%) were ambulatory; 216 received delandistrogene moxeparvovec treatment. Two myocarditis cases were reported within 4 days after delandistrogene moxeparvovec infusion; both resolved within 3 weeks. Except in the two myocarditis cases, troponin I fluctuations were asymptomatic. Thirteen patients with baseline and postbaseline echocardiography data had elevated troponin I at baseline; 1 year post infusion, only one of these patients had LVEF < 50%. LVEF in all four patients with 5-year follow-up remained > 50%. Although cardiac MRI without gadolinium revealed no relevant differences in heart function between patients 1 or 2 years after delandistrogene moxeparvovec versus patients 1 year after placebo infusion, subclinical fibrosis cannot be ruled out.</p><p><strong>Conclusion: </strong>Results from delandistrogene moxeparvovec trials with 1 to 5 years of follow-up suggest a manageable cardiac safety profile in this study population of predominantly younger, ambulatory patients with DMD who had no signs of persistent treatment-related cardiac injury.</p>","PeriodicalId":9561,"journal":{"name":"Cardiology and Therapy","volume":" ","pages":"301-317"},"PeriodicalIF":3.2,"publicationDate":"2026-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC13542014/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"148359672","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}