{"doi":"10.3390/jcm14228181","title":"Long-Term Real-World Outcomes of Mavacamten in Symptomatic Obstructive Hypertrophic Cardiomyopathy up to 108 Weeks","abstract":"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 ± 36.7 mm Hg to 10.0 ± 11.0 mm Hg) and Valsalva left ventricular outflow tract gradients (baseline mean 79.7 ± 33.2 mm Hg to 16.6 ± 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 >1 NYHA class improvement by Week 12). Mean maximal left ventricular wall thickness significantly decreased (β = 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 < 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.","journal":"Journal of Clinical Medicine","year":2025,"id":527559,"datarank":0.0,"base_score":0.0,"endowment":0.0,"self_citation_contribution":0.0,"citation_network_contribution":0.0,"self_endowment_contribution":0.0,"citer_contribution":0.0,"corpus_percentile":null,"corpus_rank":null,"citation_count":3,"citer_count":0,"citers_with_citation_signal":0,"citers_with_endowment":0,"datacite_reuse_total":0,"is_dataset":false,"is_dataset_confidence":0.625,"is_data_producer":false,"deposit_databanks":null,"is_oa":true,"file_count":0,"downloads":0,"has_version_chain":false,"published_date":"2025-01-01","fair_score":null,"fair_percentile":null,"algorithm_id":"datarank_citation_only_1hop_v6","ranking_scope":"data_only","authors":[{"id":1236253,"name":"Anandkumar Dubey","orcid":null,"position":1,"is_corresponding":false},{"id":298804,"name":"Nadim Mahmud","orcid":"0000-0003-1889-3954","position":2,"is_corresponding":false},{"id":386130,"name":"Melissa A. Austin","orcid":"0000-0001-7876-3962","position":3,"is_corresponding":false},{"id":1404876,"name":"Estherland Duqueney","orcid":null,"position":4,"is_corresponding":false},{"id":233857,"name":"Parth Patel","orcid":"0000-0002-8528-1029","position":5,"is_corresponding":false},{"id":653012,"name":"Ellen Boakye","orcid":"0000-0002-7625-9487","position":6,"is_corresponding":false},{"id":933538,"name":"Amy Marzolf","orcid":null,"position":7,"is_corresponding":false},{"id":1236254,"name":"Nicole Hornsby","orcid":null,"position":8,"is_corresponding":false},{"id":888781,"name":"Alejandro de Feria","orcid":null,"position":9,"is_corresponding":false},{"id":1235801,"name":"Thomas Carattini","orcid":"0009-0009-3557-194X","position":10,"is_corresponding":false},{"id":1404317,"name":"P. K. Schuler","orcid":"0000-0002-9913-7714","position":11,"is_corresponding":false},{"id":465189,"name":"Anjali Owens","orcid":"0000-0002-9669-8495","position":12,"is_corresponding":false},{"id":225626,"name":"Nosheen Reza","orcid":"0000-0002-2942-3731","position":0,"is_corresponding":true}],"reference_count":29,"raw_metadata":null,"created_at":"2026-07-19T02:50:39.280101Z","pmid":"41303215","pmcid":null,"fwci":null,"citation_percentile":null,"influential_citations":0,"oa_status":null,"license":null,"views":0,"total_file_size_bytes":0,"version_count":0,"fair_f":null,"fair_a":null,"fair_i":null,"fair_r":null,"fair_zscore":null,"fair_rationale":null,"fair_model":null,"fair_agent_version":null,"fair_fulltext_source":null,"fair_has_llm":null,"fair_computed_at":null,"clinical_trials":[],"software_tools":[],"db_accessions":[],"linked_datasets":[],"topics":[]}