JACC. Heart failure最新文献

{"title":"Mavacamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy","authors":"Milind Y. Desai MD, MBA ,&nbsp;Steve E. Nissen MD ,&nbsp;Theodore Abraham MD ,&nbsp;Iacopo Olivotto MD ,&nbsp;Pablo Garcia-Pavia MD, PhD ,&nbsp;Renato D. Lopes MD, PhD ,&nbsp;Nicolas Verheyen MD ,&nbsp;Omar Wever-Pinzon MD ,&nbsp;Kathy Wolski MPH ,&nbsp;Wael Jaber MD ,&nbsp;Lisa Mitchell RN ,&nbsp;Deborah Davey RN ,&nbsp;Jonathan Myers PhD ,&nbsp;Thomas Rano PhD ,&nbsp;Vandana Bhatia PhD ,&nbsp;Yue Zhong PhD ,&nbsp;Suzanne Carter-Bonanza RN ,&nbsp;Victoria Florea MD ,&nbsp;Ron Aronson MD ,&nbsp;Anjali T. Owens MD","doi":"10.1016/j.jchf.2024.11.013","DOIUrl":"10.1016/j.jchf.2024.11.013","url":null,"abstract":"<div><div>There are no approved therapies for patients with symptomatic nonobstructive hypertrophic cardiomyopathy (nHCM). The authors describe the baseline characteristics of ODYSSEY-HCM (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy), a phase 3, randomized, double-blind, placebo-controlled trial conducted worldwide at 201 sites evaluating mavacamten in symptomatic adult patients with nHCM. The 2 primary endpoints are the changes from baseline to week 48 in: 1) Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score; and 2) peak oxygen consumption (pVO₂) on cardiopulmonary exercise testing. Dose titrations are made on blinded core laboratory assessments. Of 1,088 patients screened, 580 are randomized (mean age 56 ± 15 years, 46% women, 43% with family histories). All patients are nonobstructive and symptomatic (70% in NYHA functional class II and 30% class III), with a mean Kansas City Cardiomyopathy Questionnaire 23-item Clinical Summary Score of 58 ± 20, and 77% are on beta-blockers. The mean left ventricular ejection fraction and pVO₂ are 66% ± 4% and 18 ± 6 mL/kg/min, respectively. ODYSSEY-HCM will report if mavacamten improves patient-reported health status and exercise capacity in patients with symptomatic nHCM. (A Study of Mavacamten in Non-Obstructive Hypertrophic Cardiomyopathy (ODYSSEY-HCM); <span><span>NCT05582395</span><svg><path></path></svg></span>)</div></div>","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 358-370"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143180876","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Endpoint Selection in Randomized Clinical Trials for Hypertrophic Cardiomyopathy","authors":"Alberto Aimo MD, PhD ,&nbsp;Iacopo Olivotto MD ,&nbsp;Giancarlo Todiere MD, PhD ,&nbsp;Andrea Barison MD, PhD ,&nbsp;Giorgia Panichella MD ,&nbsp;Mona Fiuzat PharmD ,&nbsp;Cecilia Linde MD, PhD ,&nbsp;Neal K. Lakdawala MD, MSc ,&nbsp;Milind Desai MD, MBA ,&nbsp;Faiez Zannad MD, PhD ,&nbsp;Martin S. Maron MD","doi":"10.1016/j.jchf.2024.10.016","DOIUrl":"10.1016/j.jchf.2024.10.016","url":null,"abstract":"<div><div>Randomized clinical trials (RCTs) for hypertrophic cardiomyopathy (HCM) have long been challenging caused by the condition’s rarity, low event rates, and diverse clinical presentations. However, recent advances in targeted therapies have sparked increased interest in HCM research. Despite this, designing effective RCTs remains complex, particularly in identifying clinically meaningful endpoints. HCM, often linked to sequence variation in sarcomeric protein genes like <em>MYH7</em> and <em>MYBPC3</em>, exhibits varied phenotypic expressions that influence disease progression and treatment responses. This genetic variability underscores the need for personalized approaches in clinical trials. Emerging gene therapies, such as CRISPR/Cas9, show promise in addressing these genetic factors. A major challenge in HCM RCTs is ensuring that endpoints are both statistically and clinically significant, given issues like test-retest variability and missing data. Primary endpoints often focus on symptom relief and functional improvement, while secondary and exploratory endpoints provide broader insights into treatment effects. Regulatory authorities are increasingly open to a wider range of endpoints, including patient-reported outcomes and functional measures, although the cost-risk balance is crucial, especially for high-risk interventions. Future HCM RCTs may incorporate hard clinical endpoints such as heart failure hospitalization, atrial fibrillation recurrence, and all-cause mortality, offering a more comprehensive evaluation of treatment efficacy. Integrating genetic insights and advanced technologies will be essential to improving trial design and enhancing patient outcomes in HCM.</div></div>","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 200-212"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142872112","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"EuroQol 5-Dimension Questionnaire in Heart Failure With Reduced, Mildly Reduced, and Preserved Ejection Fraction","authors":"Mingming Yang MD, PhD ,&nbsp;Toru Kondo MD, PhD ,&nbsp;Atefeh Talebi PhD ,&nbsp;Pardeep S. Jhund MBChB, MSc, PhD ,&nbsp;Kieran F. Docherty MBChB, MD ,&nbsp;Brian L. Claggett PhD ,&nbsp;Muthiah Vaduganathan MD, MPH ,&nbsp;Erasmus Bachus MD, PhD ,&nbsp;Adrian F. Hernandez MD ,&nbsp;Carolyn S.P. Lam MBBS, PhD ,&nbsp;Felipe A. Martinez MD ,&nbsp;Rudolf A. de Boer MD, PhD ,&nbsp;Mikhail N. Kosiborod MD ,&nbsp;Akshay S. Desai MD, MPH ,&nbsp;Lars Køber MD, DMSc ,&nbsp;Piotr Ponikowski MD, PhD ,&nbsp;Marc S. Sabatine MD, MPH ,&nbsp;Scott D. Solomon MD ,&nbsp;John J.V. McMurray MD","doi":"10.1016/j.jchf.2024.10.020","DOIUrl":"10.1016/j.jchf.2024.10.020","url":null,"abstract":"<div><h3>Background</h3><div>The value of generic quality of life (QoL) instruments in heart failure (HF) is uncertain.</div></div><div><h3>Objectives</h3><div>In this study, the authors sought to quantify individual dimension scores and the EuroQol 5-Dimension questionnaire (EQ-5D) Level Sum Score (LSS) in patients with HF with reduced, mildly reduced, or preserved ejection fraction, the association between those scores and outcomes, and the impact of treatment with dapagliflozin on the scores.</div></div><div><h3>Methods</h3><div>Analyses were conducted using patient-level data from DAPA-HF and DELIVER trials. Cox proportional hazards regression models were used to assess the association between EQ-5D scores (each dimension and LSS) and clinical outcomes. Sankey diagrams were used to illustrate changes in individual patient EQ-5D dimensions from baseline to 8 months’ follow-up.</div></div><div><h3>Results</h3><div>Of the 11,007 patients randomized in DAPA-HF and DELIVER, 10,135 (92.1%) completed the instrument at baseline. Scores varied markedly by question with 37%, 30%, and 33% of patients reporting no, slight, or moderate or greater problem, respectively for mobility; 67%, 20%, and 13% for self-care; 40%, 33%, and 27% for usual activities; 45%, 32%, and 23% for pain/discomfort; and 57%, 27%, and 16% for anxiety/depression. Patients with higher (worse) EQ-5D-LSS were more frequently female, had more comorbidities, and had worse HF status. Compared with patients free from any problem across all dimensions (ie, an EQ-5D-LSS of 5), the HRs for the composite outcome of time to first cardiovascular death or worsening HF were 1.27 (95% CI: 1.10-1.47), 1.70 (95% CI: 1.46-1.98), and 2.31 (95% CI: 1.88-2.85) in patients with EQ-5D-LSS of 6-10, 11-15, and 16-25 points, respectively. Dapagliflozin led to greater improvement and less worsening in mobility (OR: 1.13 [95% CI: 1.04-1.23]; <em>P =</em> 0.004), self-care (OR: 1.13 [95% CI: 1.02-1.24]; <em>P =</em> 0.016), usual activities (OR: 1.11 [95% CI: 1.02-1.21]; <em>P =</em> 0.015), and anxiety/depression (OR: 1.10 [95% CI: 1.01-1.21]; <em>P =</em> 0.034) after 8 months. The number needed to treat for 1 patient to report improvement in EQ-5D-LSS was 31 (95% CI: 20-72).</div></div><div><h3>Conclusions</h3><div>The EQ-5D revealed problems not often associated (eg, pain) with HF or commonly quantified in HF (eg, anxiety/depression). Dapagliflozin improved multiple QoL dimensions, and possibly anxiety/depression. (Study to Evaluate the Effect of Dapagliflozin on the Incidence of Worsening Heart Failure or Cardiovascular Death in Patients With Chronic Heart Failure [DAPA-HF]; <span><span>NCT03036124</span><svg><path></path></svg></span>; Dapagliflozin Evaluation to Improve the Lives of Patients With Preserved Ejection Fraction Heart Failure [DELIVER]; <span><span>NCT03619213</span><svg><path></path></svg></span>)</div></div>","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 277-292"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143179804","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Not All Hospitalized Heart Failure Patients Are the Same","authors":"Garrick C. Stewart MD, MPH","doi":"10.1016/j.jchf.2024.11.014","DOIUrl":"10.1016/j.jchf.2024.11.014","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 260-264"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143179837","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cardiac Cachexia","authors":"Elissa Driggin MD, MS,&nbsp;Kevin J. Clerkin MD, MS","doi":"10.1016/j.jchf.2024.12.002","DOIUrl":"10.1016/j.jchf.2024.12.002","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 330-331"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143180874","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Asymptomatic But Obstructive","authors":"Ozlem Bilen MD,&nbsp;Michael A. Burke MD","doi":"10.1016/j.jchf.2024.10.017","DOIUrl":"10.1016/j.jchf.2024.10.017","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 344-345"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142965048","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Antiobesity Medications in Patients With Heart Failure","authors":"Sureya F. Hussani MD ,&nbsp;Kelly Nguyen PharmD ,&nbsp;Ka man Yu PharmD ,&nbsp;Darius Sinha ,&nbsp;Amanda R. Vest MBBS, MPH","doi":"10.1016/j.jchf.2024.10.018","DOIUrl":"10.1016/j.jchf.2024.10.018","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 375-380"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142988722","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pacing-Induced Cardiomyopathy","authors":"Kiran Sidhu MD ,&nbsp;Andrea M. Russo MD","doi":"10.1016/j.jchf.2024.11.012","DOIUrl":"10.1016/j.jchf.2024.11.012","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 274-276"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143180873","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Context for Deciding Therapy in Mitral Regurgitation","authors":"Paul Sorajja MD","doi":"10.1016/j.jchf.2024.09.025","DOIUrl":"10.1016/j.jchf.2024.09.025","url":null,"abstract":"","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":"13 2","pages":"Pages 226-228"},"PeriodicalIF":10.3,"publicationDate":"2025-02-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143179808","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Beta-Blockers and Long-Term Mortality in Takotsubo Syndrome: Results of the Multicenter GEIST Registry.","authors":"Sergio Raposeiras-Roubin, Francesco Santoro, Luca Arcari, Ravi Vazirani, Giuseppina Novo, Aitor Uribarri, Mariano Enrica, Javier Lopez-Pais, Federico Guerra, Fernando Alfonso, Toni Pätz, Clara Fernandez-Cordon, Roberta Montisci, Miguel Corbi-Pascual, Maria Francesca Marchetti, Manuel Almendro, Luca Cacciotti, Oscar Vedia, Ibrahim El-Battrawy, Emilia Blanco-Ponce, Natale Daniele Brunetti, Ibrahim Akin, Manuel Martinez-Sellés, Holger Thiele, Thomas Stiermaier, Ingo Eitel, Ivan J Nuñez-Gil","doi":"10.1016/j.jchf.2024.11.015","DOIUrl":"https://doi.org/10.1016/j.jchf.2024.11.015","url":null,"abstract":"<p><strong>Background: </strong>Beta-blockers are considered a reasonable therapy for patients with Takotsubo syndrome (TTS), commonly used despite the absence of consistent evidence about its prognosis impact.</p><p><strong>Objectives: </strong>This study aimed to assess the impact of beta-blocker therapy on long-term mortality and TTS recurrence.</p><p><strong>Methods: </strong>The authors analyzed 2,853 patients discharged with a confirmed TTS diagnosis, enrolled in the international multicenter GEIST (The GErman Italian Spanish Takotsubo Registry). They performed a propensity score matching analysis to draw up 2 groups of 697 patients paired according to whether or not they received medical therapy with beta-blockers at hospital discharge. The prognostic value of beta-blockers at discharge to predict mortality and TTS recurrence during follow-up was analyzed using Cox regression.</p><p><strong>Results: </strong>During a mean follow-up of 2.6 years, 485 patients (17.0%) died and 97 (3.4%) have had TTS recurrence. Patients treated with beta-blockers at discharge (n = 2,125) (74.5%) had a lower mortality rate (6.0 vs 8.1 per 100 patients/year). After propensity score matching, the authors found that mortality during follow-up was lower in the beta-blocker group (HR: 0.71; 95% CI: 0.55-0.90). Differences in mortality were especially at the expense of mortality in the first year. No differences were found by subgroups. Moreover, beta-blocker therapy was not associated with lower TTS recurrence during the follow-up (HR: 0.74, 95% CI: 0.61-1.89). No association between the use of beta-blockers at discharge and left ventricle ejection fraction recovery has also been observed.</p><p><strong>Conclusions: </strong>Beta-blocker therapy in patients with TTS is associated with lower follow-up mortality, but not with lower TTS recurrence. (The GErman Italian Spanish Takotsubo Registry [GEIST]; NCT04361994).</p>","PeriodicalId":14687,"journal":{"name":"JACC. Heart failure","volume":" ","pages":""},"PeriodicalIF":10.3,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143364879","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}