CirculationPub Date : 2025-03-11Epub Date: 2025-02-05DOI: 10.1161/CIRCULATIONAHA.124.072849
Xavier Jeunemaitre, Elie Mousseaux, Michael Frank, Salma Adham, Francesca Pitocco, Clarisse Billon, Molka Ben Yakhlef, Mohamed El Hachmi, Alessandra Bura-Rivière, François-Xavier Lapébie, Claire Le Hello, Damien Laneelle, Christophe Seinturier, Klaus Dieterich, Marc Lambert, Sophie Dupuis-Girod, Stéphane Zuily, Laurence Bal-Theoleyre, Carine Boulon, Pierrick Henneton, Estelle Lu, Nicolas Denarié, Pierre Boutouyrie, Tristan Mirault, Gilles Chatellier, Michel Azizi
{"title":"Efficacy of Irbesartan in Celiprolol-Treated Patients With Vascular Ehlers-Danlos Syndrome.","authors":"Xavier Jeunemaitre, Elie Mousseaux, Michael Frank, Salma Adham, Francesca Pitocco, Clarisse Billon, Molka Ben Yakhlef, Mohamed El Hachmi, Alessandra Bura-Rivière, François-Xavier Lapébie, Claire Le Hello, Damien Laneelle, Christophe Seinturier, Klaus Dieterich, Marc Lambert, Sophie Dupuis-Girod, Stéphane Zuily, Laurence Bal-Theoleyre, Carine Boulon, Pierrick Henneton, Estelle Lu, Nicolas Denarié, Pierre Boutouyrie, Tristan Mirault, Gilles Chatellier, Michel Azizi","doi":"10.1161/CIRCULATIONAHA.124.072849","DOIUrl":"10.1161/CIRCULATIONAHA.124.072849","url":null,"abstract":"<p><strong>Background: </strong>Vascular Ehlers-Danlos syndrome is a rare genetic disorder characterized by defective type III collagen and a high risk of arterial morbidity and mortality. Several cardiovascular drugs are used for treatment, including celiprolol, but no controlled trial in this condition has been conducted to date. We hypothesized the benefit of the addition of an angiotensin II receptor blocker.</p><p><strong>Methods: </strong>A multicenter, randomized, placebo-controlled trial was conducted to assess the efficacy and safety of the angiotensin II receptor blocker irbesartan in adults with vascular Ehlers-Danlos syndrome on stable background celiprolol therapy. Patients were randomized 1:1 to receive irbesartan (150 mg/day titrated to 300 mg/day) or placebo for 2 years. The composite primary outcome was defined as any vascular Ehlers-Danlos syndrome-related fatal or nonfatal arterial event or any new or worsening arterial lesions detected by systematic head-to-pelvis computed tomography angiography or peripheral arterial duplex ultrasound at different time points, using a time-to-first-event analysis.</p><p><strong>Results: </strong>Twenty-nine participants (62% female; 40.3±11.3 years of age) were randomized to irbesartan, and 28 (64% female; 40.7±11.0 years of age) were randomized to placebo. The composite primary outcome occurred in 8 of 29 patients (27.6%) receiving irbesartan versus 15 of 28 patients (53.6%) receiving placebo (hazard ratio, 0.42 [95% CI, 0.17, 0.99]; <i>P</i><0.05). The risk of recurrent symptomatic or nonsymptomatic arterial events was lower with irbesartan than with placebo (risk ratio, 0.37 [95% CI, 0.19, 0.68]; <i>P</i>=0.002). A reduction of progression of arterial lesions was observed at all sites. Irbesartan significantly reduced systolic blood pressure compared with placebo (baseline-adjusted difference of 5.4 mm Hg [<i>P</i><0.001]), but no relation was observed with the reduction of the primary composite outcome. Eleven episodes of irbesartan-related hypotension were recorded, leading to a downtitration in 4 patients.</p><p><strong>Conclusions: </strong>Compared with placebo, irbesartan reduced the risk of severe symptomatic and asymptomatic arterial events in patients with vascular Ehlers-Danlos syndrome on background celiprolol therapy.</p><p><strong>Registration: </strong>URL: https://www.clinicaltrials.gov; Unique identifier: NCT02597361.</p>","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":"686-695"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143188583","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}
CirculationPub Date : 2025-03-11DOI: 10.1161/CIR.0000000000001310
Esther S H Kim, Shipra Arya, Yolanda Bryce, Heather L Gornik, Chandler A Long, Mary M McDermott, Amy West Pollak, Vincent Lopez Rowe, Alexander E Sullivan, Mary O Whipple
{"title":"Sex Differences in Peripheral Vascular Disease: A Scientific Statement From the American Heart Association.","authors":"Esther S H Kim, Shipra Arya, Yolanda Bryce, Heather L Gornik, Chandler A Long, Mary M McDermott, Amy West Pollak, Vincent Lopez Rowe, Alexander E Sullivan, Mary O Whipple","doi":"10.1161/CIR.0000000000001310","DOIUrl":"https://doi.org/10.1161/CIR.0000000000001310","url":null,"abstract":"<p><p>Sex differences in the risk factors, diagnosis, treatment, and outcomes of patients with cardiovascular disease have been well described; however, the bulk of the literature has focused on heart disease in women. Data on sex differences in peripheral vascular disease are ill defined, and there is a need to report and understand those sex-related differences to mitigate adverse outcomes related to those disparities. Although peripheral vascular disease is a highly diverse group of disorders affecting the arteries, veins, and lymphatics, this scientific statement focuses on disorders affecting the peripheral arteries to include the aorta and its branch vessels. The purpose of this scientific statement is to report the current status of sex-based differences and disparities in peripheral vascular disease and to provide research priorities to achieve health equity for women with peripheral vascular disease.</p>","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":""},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143596447","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}
CirculationPub Date : 2025-03-11Epub Date: 2025-03-10DOI: 10.1161/CIR.0000000000001317
Andrew P Ambrosy, Daniel Bensimhon, Galina Bernstein, Brian Kolski, Joel Neutel, Anuradha Lala, Navin K Kapur, Benjamin Esque, Eric Adler
{"title":"Correction to: Randomized Study Comparing a Novel Intranasal Formulation of Bumetanide to Oral and Intravenous Formulations.","authors":"Andrew P Ambrosy, Daniel Bensimhon, Galina Bernstein, Brian Kolski, Joel Neutel, Anuradha Lala, Navin K Kapur, Benjamin Esque, Eric Adler","doi":"10.1161/CIR.0000000000001317","DOIUrl":"https://doi.org/10.1161/CIR.0000000000001317","url":null,"abstract":"","PeriodicalId":10331,"journal":{"name":"Circulation","volume":"151 10","pages":"e708"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143596456","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}
CirculationPub Date : 2025-03-11Epub Date: 2025-03-10DOI: 10.1161/CIRCULATIONAHA.124.072415
Wanpen Vongpatanasin, Adina F Turcu
{"title":"The Journey of Resistant Hypertension: From the Fifth Report of the Joint National Committee on Detection, Evaluation, and Treatment of High Blood Pressure to American Heart Association/American College of Cardiology Guidelines.","authors":"Wanpen Vongpatanasin, Adina F Turcu","doi":"10.1161/CIRCULATIONAHA.124.072415","DOIUrl":"https://doi.org/10.1161/CIRCULATIONAHA.124.072415","url":null,"abstract":"","PeriodicalId":10331,"journal":{"name":"Circulation","volume":"151 10","pages":"653-655"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143596480","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}
CirculationPub Date : 2025-03-11Epub Date: 2025-02-13DOI: 10.1161/CIR.0000000000001300
Shashank S Sinha, Bram J Geller, Jason N Katz, Cynthia Arslanian-Engoren, Christopher F Barnett, Erin A Bohula, Abdulla A Damluji, Venu Menon, Robert O Roswell, Saraschandra Vallabhajosyula, Amanda R Vest, Sean van Diepen, David A Morrow
{"title":"Evolution of Critical Care Cardiology: An Update on Structure, Care Delivery, Training, and Research Paradigms: A Scientific Statement From the American Heart Association.","authors":"Shashank S Sinha, Bram J Geller, Jason N Katz, Cynthia Arslanian-Engoren, Christopher F Barnett, Erin A Bohula, Abdulla A Damluji, Venu Menon, Robert O Roswell, Saraschandra Vallabhajosyula, Amanda R Vest, Sean van Diepen, David A Morrow","doi":"10.1161/CIR.0000000000001300","DOIUrl":"10.1161/CIR.0000000000001300","url":null,"abstract":"<p><p>Critical care cardiology refers to the practice focus of and subspecialty training for the comprehensive management of life-threatening cardiovascular diseases and comorbid conditions that require advanced critical care in an intensive care unit. The development of coronary care units is often credited for a dramatic decline in mortality rates after acute myocardial infarction throughout the 1960s. As the underlying patient population became progressively sicker, changes in organizational structure, staffing, care delivery, and training paradigms lagged. The coronary care unit gradually evolved from a focus on rapid resuscitation from ventricular arrhythmias in acute myocardial infarction into a comprehensive cardiac intensive care unit designed to care for the sickest patients with cardiovascular disease. Over the past decade, the cardiac intensive care unit has continued to transform with an aging population, increased clinical acuity, burgeoning cardiac and noncardiac comorbidities, technologic advances in cardiovascular interventions, and increased use of temporary mechanical circulatory support devices. Herein, we provide an update and contemporary expert perspective on the organizational structure, staffing, and care delivery in the cardiac intensive care unit; examine the challenges and opportunities present in the education and training of the next generation of physicians for critical care cardiology; and explore quality improvement initiatives and scientific investigation, including multicenter registry initiatives and randomized clinical trials, that may change clinical practice, care delivery, and the research landscape in this rapidly evolving discipline.</p>","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":"e687-e707"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143406120","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}
CirculationPub Date : 2025-03-11Epub Date: 2025-03-10DOI: 10.1161/CIR.0000000000001320
Efstathios Papatheodorou, Vincent L Aengevaeren, Thijs M H Eijsvogels, Khaled AlFakih, Rebecca Kathryn Hughes, Ahmed Merghani, Christine K Kissel, Saad Fyyaz, Athanasios Bakalakos, Mathew G Wilson, Damini Dey, Gherardo Finocchiaro, Gemma Parry-Williams, Camilla Torlasco, Michael Papadakis, James C Moon, Sanjay Sharma
{"title":"Correction to: Prevalence of Coronary Atherosclerosis in Female Masters Endurance Athletes.","authors":"Efstathios Papatheodorou, Vincent L Aengevaeren, Thijs M H Eijsvogels, Khaled AlFakih, Rebecca Kathryn Hughes, Ahmed Merghani, Christine K Kissel, Saad Fyyaz, Athanasios Bakalakos, Mathew G Wilson, Damini Dey, Gherardo Finocchiaro, Gemma Parry-Williams, Camilla Torlasco, Michael Papadakis, James C Moon, Sanjay Sharma","doi":"10.1161/CIR.0000000000001320","DOIUrl":"https://doi.org/10.1161/CIR.0000000000001320","url":null,"abstract":"","PeriodicalId":10331,"journal":{"name":"Circulation","volume":"151 10","pages":"e709"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143596376","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}
CirculationPub Date : 2025-03-11Epub Date: 2024-11-18DOI: 10.1161/CIRCULATIONAHA.124.072949
Andrew P Ambrosy, Daniel Bensimhon, Galina Bernstein, Brian Kolski, Joel Neutel, Anuradha Lala, Navin K Kapur, Benjamin Esque, Eric Adler
{"title":"Randomized Study Comparing a Novel Intranasal Formulation of Bumetanide With Oral and Intravenous Formulations.","authors":"Andrew P Ambrosy, Daniel Bensimhon, Galina Bernstein, Brian Kolski, Joel Neutel, Anuradha Lala, Navin K Kapur, Benjamin Esque, Eric Adler","doi":"10.1161/CIRCULATIONAHA.124.072949","DOIUrl":"10.1161/CIRCULATIONAHA.124.072949","url":null,"abstract":"","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":"737-740"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647065","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}
CirculationPub Date : 2025-03-11Epub Date: 2025-03-10DOI: 10.1161/CIRCULATIONAHA.124.072519
Hailong Dai, Xiangting Lu, Xu Zhou
{"title":"Letter by Dai et al Regarding Article, \"Tadalafil for Treatment of Combined Postcapillary and Precapillary Pulmonary Hypertension in Patients With Heart Failure and Preserved Ejection Fraction\".","authors":"Hailong Dai, Xiangting Lu, Xu Zhou","doi":"10.1161/CIRCULATIONAHA.124.072519","DOIUrl":"https://doi.org/10.1161/CIRCULATIONAHA.124.072519","url":null,"abstract":"","PeriodicalId":10331,"journal":{"name":"Circulation","volume":"151 10","pages":"e686"},"PeriodicalIF":35.5,"publicationDate":"2025-03-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143596475","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":"Rivaroxaban for 18 Months Versus 6 Months in Patients With Cancer and Acute Low-Risk Pulmonary Embolism: An Open-Label, Multicenter, Randomized Clinical Trial (ONCO PE Trial).","authors":"Yugo Yamashita, Takeshi Morimoto, Nao Muraoka, Wataru Shioyama, Ryuki Chatani, Tatsuhiro Shibata, Yuji Nishimoto, Yoshito Ogihara, Kosuke Doi, Maki Oi, Taro Shiga, Daisuke Sueta, Kitae Kim, Yasuhiro Tanabe, Norimichi Koitabashi, Takuma Takada, Satoshi Ikeda, Hitoshi Nakagawa, Kengo Tsukahara, Masaaki Shoji, Jiro Sakamoto, Shinji Hisatake, Yutaka Ogino, Masashi Fujita, Naohiko Nakanishi, Tomohiro Dohke, Seiichi Hiramori, Ryuzo Nawada, Kazuhisa Kaneda, Koh Ono, Takeshi Kimura","doi":"10.1161/CIRCULATIONAHA.124.072758","DOIUrl":"10.1161/CIRCULATIONAHA.124.072758","url":null,"abstract":"<p><strong>Background: </strong>The optimal duration of anticoagulation therapy for patients with cancer and acute low-risk pulmonary embolism (PE) is clinically relevant, but evidence is lacking. Prolonged anticoagulation therapy could have a potential benefit for prevention of thrombotic events; however, it could also increase the risk of bleeding.</p><p><strong>Methods: </strong>In a multicenter, open-label, adjudicator-blinded, randomized clinical trial at 32 institutions in Japan, we randomly assigned patients with cancer and acute low-risk PE of the simplified version of the Pulmonary Embolism Severity Index score of 1, in a 1:1 ratio, to receive either an 18-month or a 6-month rivaroxaban treatment. The primary end point was recurrent venous thromboembolism (VTE) at 18 months. The major secondary end point was major bleeding at 18 months according to the criteria of the International Society on Thrombosis and Hemostasis. The primary hypothesis was that an 18-month treatment was superior to a 6-month treatment in terms of the primary end point.</p><p><strong>Results: </strong>From February 2021 to March 2023, 179 patients were randomized, and after the exclusion of one patient who withdrew consent, 178 were included in the intention-to-treat population: 89 patients in the 18-month rivaroxaban group and 89 in the 6-month rivaroxaban group. The mean age was 65.7 years; 47% of the patients were men, and 12% had symptoms of PE at baseline. The primary end point of recurrent VTE occurred in 5 of the 89 patients (5.6%) in the 18-month rivaroxaban group and in 17 of the 89 (19.1%) in the 6-month rivaroxaban group (odds ratio, 0.25 [95% CI, 0.09-0.72]; <i>P</i>=0.01). Among 22 recurrent VTE, 5 patients presented with a symptomatic recurrent VTE; recurrent PE occurred in 11 patients, including 2 with main and 4 with lobar PEs; and recurrent deep vein thrombosis was seen in 11 patients, including 3 with proximal deep vein thromboses. The major secondary end point of major bleeding occurred in 7 of the 89 patients (7.8%) in the 18-month rivaroxaban group and in 5 of the 89 patients (5.6%) in the 6-month rivaroxaban group (odds ratio, 1.43 [95% CI, 0.44-4.70]; <i>P</i>=0.55).</p><p><strong>Conclusions: </strong>In patients with cancer and acute low-risk PE of the simplified version of the Pulmonary Embolism Severity Index score of 1, the 18-month rivaroxaban treatment was superior to the 6-month rivaroxaban treatment with respect to recurrent VTE events.</p><p><strong>Registration: </strong>URL: https://www.clinicaltrials.gov; Unique identifier: NCT04724460.</p>","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":"589-600"},"PeriodicalIF":35.5,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11875411/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647068","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}
CirculationPub Date : 2025-03-04Epub Date: 2024-11-18DOI: 10.1161/CIRCULATIONAHA.124.072771
Daniel P Judge, Julian D Gillmore, Kevin M Alexander, Amrut V Ambardekar, Francesco Cappelli, Marianna Fontana, Pablo García-Pavía, Justin L Grodin, Martha Grogan, Mazen Hanna, Ahmad Masri, Jose Nativi-Nicolau, Laura Obici, Steen Hvitfeldt Poulsen, Nitasha Sarswat, Keyur Shah, Prem Soman, Ted Lystig, Xiaofan Cao, Kevin Wang, Maria Lucia Pecoraro, Jean-François Tamby, Leonid Katz, Uma Sinha, Jonathan C Fox, Mathew S Maurer
{"title":"Long-Term Efficacy and Safety of Acoramidis in ATTR-CM: Initial Report From the Open-Label Extension of the ATTRibute-CM Trial.","authors":"Daniel P Judge, Julian D Gillmore, Kevin M Alexander, Amrut V Ambardekar, Francesco Cappelli, Marianna Fontana, Pablo García-Pavía, Justin L Grodin, Martha Grogan, Mazen Hanna, Ahmad Masri, Jose Nativi-Nicolau, Laura Obici, Steen Hvitfeldt Poulsen, Nitasha Sarswat, Keyur Shah, Prem Soman, Ted Lystig, Xiaofan Cao, Kevin Wang, Maria Lucia Pecoraro, Jean-François Tamby, Leonid Katz, Uma Sinha, Jonathan C Fox, Mathew S Maurer","doi":"10.1161/CIRCULATIONAHA.124.072771","DOIUrl":"10.1161/CIRCULATIONAHA.124.072771","url":null,"abstract":"<p><strong>Background: </strong>In the phase 3 randomized controlled study ATTRibute-CM (Efficacy and Safety of AG10 in Subjects With Transthyretin Amyloid Cardiomyopathy), acoramidis, a transthyretin stabilizer, demonstrated significant efficacy on the primary end point. Participants with transthyretin amyloid cardiomyopathy who completed ATTRibute-CM were invited to enroll in an open-label extension study (OLE). We report the efficacy and safety data of acoramidis in participants who completed ATTRibute-CM and enrolled in the ongoing OLE.</p><p><strong>Methods: </strong>Participants who previously received acoramidis through month 30 in ATTRibute-CM continued to receive it (continuous acoramidis), and those who received placebo through month 30 were switched to acoramidis (placebo to acoramidis). Participants who received concomitant tafamidis in ATTRibute-CM were required to discontinue it to be eligible to enroll in the OLE. Clinical efficacy outcomes analyzed through month 42 included time to event for all-cause mortality (ACM) or first cardiovascular-related hospitalization (CVH), ACM alone, first CVH alone, ACM or recurrent CVH, change from baseline in NT-proBNP (N-terminal pro-B-type natriuretic peptide), 6-minute walk distance, serum transthyretin, and Kansas City Cardiomyopathy Questionnaire Overall Summary score. Safety outcomes were analyzed through month 42.</p><p><strong>Results: </strong>Overall, 438 of 632 participants in ATTRibute-CM completed treatment, and 389 enrolled in the ongoing OLE (263 continuous acoramidis and 126 placebo to acoramidis). The hazard ratio for ACM or first CVH was 0.57 (95% CI, 0.46-0.72) at month 42 based on a stratified Cox proportional hazards model (<i>P</i><0.0001) favoring continuous acoramidis. Similar analyses were performed on ACM alone and first CVH alone, with hazard ratios of 0.64 (95% CI, 0.47-0.88) and 0.53 (95% CI, 0.41-0.69), respectively, at month 42. Treatment effects for NT-proBNP and 6-minute walk distance also favored continuous acoramidis. On initiation of open-label acoramidis in the placebo-to-acoramidis arm, there was a prompt increase in serum transthyretin. Quality of life assessed by Kansas City Cardiomyopathy Questionnaire Overall Summary score was well preserved in continuous-acoramidis participants compared with the placebo-to-acoramidis participants. No new clinically important safety issues were identified in this long-term evaluation.</p><p><strong>Conclusions: </strong>Early initiation and continuous use of acoramidis in the ATTRibute-CM study through month 42 of the ongoing OLE study were associated with sustained clinical benefits in a contemporary transthyretin amyloid cardiomyopathy cohort, with no clinically important safety issues newly identified.</p><p><strong>Registration: </strong>URL: https://www.clinicaltrials.gov; Unique identifier: NCT04988386.</p>","PeriodicalId":10331,"journal":{"name":"Circulation","volume":" ","pages":"601-611"},"PeriodicalIF":35.5,"publicationDate":"2025-03-04","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11875408/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142647061","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}