{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T16:45:12Z","timestamp":1787589912238,"version":"build-2736575974"},"reference-count":31,"publisher":"MDPI AG","issue":"22","license":[{"start":{"date-parts":[[2025,11,18]],"date-time":"2025-11-18T00:00:00Z","timestamp":1763424000000},"content-version":"vor","delay-in-days":0,"URL":"https:\/\/creativecommons.org\/licenses\/by\/4.0\/"}],"funder":[{"name":"National Heart, Lung, And Blood Institute of the National Institutes of Health","award":["K23HL166961"],"award-info":[{"award-number":["K23HL166961"]}]},{"name":"National Institute of Diabetes and Digestive and Kidney Diseases of the National Institutes of Health","award":["K08DK124577"],"award-info":[{"award-number":["K08DK124577"]}]},{"DOI":"10.13039\/100000002","name":"National Institutes of Health","doi-asserted-by":"crossref","id":[{"id":"10.13039\/100000002","id-type":"DOI","asserted-by":"crossref"}]},{"name":"Winkelman Family Fund"},{"name":"Bristol Myers Squibb"}],"content-domain":{"domain":[],"crossmark-restriction":false},"short-container-title":["JCM"],"abstract":"<jats:p>Background\/Objectives: Mavacamten is a first-in-class cardiac myosin inhibitor approved for the treatment of symptomatic obstructive hypertrophic cardiomyopathy (HCM). Long-term data regarding its real-world safety and effectiveness are limited. We aimed to describe the real-world experience of mavacamten in a large obstructive HCM cohort at a high-volume HCM center in the United States. Methods: Adult patients initiated on mavacamten between 29 April 2022 and 19 January 2025 at a single HCM center (n = 163) were retrospectively identified. Clinical effectiveness and safety data were collected through 108 weeks of treatment. Results: Rapid and sustained reductions in resting (baseline mean 53.0 \u00b1 36.7 mm Hg to 10.0 \u00b1 11.0 mm Hg) and Valsalva left ventricular outflow tract gradients (baseline mean 79.7 \u00b1 33.2 mm Hg to 16.6 \u00b1 15.4 mm Hg) were observed during treatment throughout the study period along with substantial improvements in New York Heart Association (NYHA) class (75% with &gt;1 NYHA class improvement by Week 12). Mean maximal left ventricular wall thickness significantly decreased (\u03b2 = 0.01 mm per week). Ten patients (6.1%) required temporary drug interruption due to decrement in left ventricular ejection fraction, and mavacamten was discontinued in six patients (3.7%). Doses of background beta blocker and nondihydropyridine calcium channel blocker were significantly reduced during the study period (p &lt; 0.001). Conclusions: In this large single-center real-world experience of mavacamten therapy, mavacamten was highly effective and maintained an acceptable safety profile, comparable to the clinical trial long-term extension experience.<\/jats:p>","DOI":"10.3390\/jcm14228181","type":"journal-article","created":{"date-parts":[[2025,11,19]],"date-time":"2025-11-19T12:55:31Z","timestamp":1763556931000},"page":"8181","update-policy":"https:\/\/doi.org\/10.3390\/mdpi_crossmark_policy","source":"Crossref","is-referenced-by-count":5,"title":["Long-Term Real-World Outcomes of Mavacamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy up to 108 Weeks"],"prefix":"10.3390","volume":"14","author":[{"ORCID":"https:\/\/orcid.org\/0000-0002-2942-3731","authenticated-orcid":false,"given":"Nosheen","family":"Reza","sequence":"first","affiliation":[{"name":"Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anandkumar","family":"Dubey","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb, Princeton, NJ 08540, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nadim","family":"Mahmud","sequence":"additional","affiliation":[{"name":"Division of Gastroenterology, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0001-7876-3962","authenticated-orcid":false,"given":"Melissa A.","family":"Austin","sequence":"additional","affiliation":[{"name":"NYU Langone Health, New York, NY 10016, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Estherland","family":"Duqueney","sequence":"additional","affiliation":[{"name":"Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Parth","family":"Patel","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, University of Southern California Keck School of Medicine, Los Angeles, CA 90033, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Ellen","family":"Boakye","sequence":"additional","affiliation":[{"name":"Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Amy","family":"Marzolf","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Nicole","family":"Hornsby","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Alejandro","family":"de Feria","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0009-0009-3557-194X","authenticated-orcid":false,"given":"Thomas","family":"Carattini","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb, Princeton, NJ 08540, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-9913-7714","authenticated-orcid":false,"given":"Patricia","family":"Schuler","sequence":"additional","affiliation":[{"name":"Bristol Myers Squibb, Princeton, NJ 08540, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Anjali Tiku","family":"Owens","sequence":"additional","affiliation":[{"name":"Division of Cardiovascular Medicine, Department of Medicine, Perelman School of Medicine at the University of Pennsylvania, Philadelphia, PA 19104, USA"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"1968","published-online":{"date-parts":[[2025,11,18]]},"reference":[{"key":"ref_1","doi-asserted-by":"crossref","first-page":"2324","DOI":"10.1016\/j.jacc.2024.02.014","article-title":"2024 AHA\/ACC\/AMSSM\/HRS\/PACES\/SCMR Guideline for the Management of Hypertrophic Cardiomyopathy","volume":"83","author":"Ommen","year":"2024","journal-title":"J. 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