--- title: "Real-World Evaluation of Mavacamten Safety and Effectiveness in Iranian Hypertrophic Obstructive C" id: "bmj-open-7-prospective-evaluation-of-real-world-safety-symptoms-improvement-and-adherence" canonical_url: "https://medichelpline.com/clinical-feed/bmj-open-7-prospective-evaluation-of-real-world-safety-symptoms-improvement-and-adherence" content_type: "clinical_feed_article" specialty: "Cardiology" source_name: "BMJ Open" source_url: "http://bmjopen.bmj.com/cgi/content/short/16/9/e124273?rss=1" published_at: "2026-09-03T12:23:28.000Z" evidence_level: "Journal Feed" license: "CC-BY-NC-4.0 / Informational Use" --- # Real-World Evaluation of Mavacamten Safety and Effectiveness in Iranian Hypertrophic Obstructive C ## Provenance & Clinical Metadata - **Canonical URL:** https://medichelpline.com/clinical-feed/bmj-open-7-prospective-evaluation-of-real-world-safety-symptoms-improvement-and-adherence - **Specialty:** [Cardiology](https://medichelpline.com/clinical-feed/cardiology.md) - **Primary Source:** BMJ Open - **Source URL:** [Original Journal Publication](http://bmjopen.bmj.com/cgi/content/short/16/9/e124273?rss=1) - **Published At:** 2026-09-03T12:23:28.000Z - **Evidence Rating:** Journal Feed ## Executive GIST (TL;DR) - Hypertrophic obstructive cardiomyopathy (HOCM) affects about 70% of individuals with hypertrophic cardiomyopathy, a genetic cardiovascular disorder. - Mavacamten, a cardiac myosin inhibitor, has shown efficacy in reducing left ventricular outflow tract (LVOT) gradients and improving symptoms in randomized trials for HOCM. - Prospective real-world safety and effectiveness data on mavacamten, especially outside Western populations, remain limited. - The PERSIA-HOCM study is a multicentre observational study in Iran enrolling symptomatic HOCM patients (NYHA class II-IV, LVOT gradient ≥50 mm Hg) prescribed mavacamten. - Exclusions include left ventricular ejection fraction under 55%, non-obstructive phenotypes, and lack of consent. - Participants are followed for 1 year at 4-week intervals with echocardiographic evaluations at multiple timepoints. - Data collected include symptoms, NYHA class, vital signs, echocardiographic parameters, dosing, concomitant therapies, and safety events such as mortality and hospitalizations. - Cardiac biomarkers and quality of life assessments occur at weeks 12 and 48. - Primary endpoints focus on safety, NYHA class changes, LVOT gradients, quality of life, biomarkers, and ejection fraction. - Secondary endpoints assess cardiac MRI measures, peak oxygen uptake, and genotype-related outcomes. - Statistical analysis will use R software with descriptive measures, paired t tests, and McNemar’s test. - Ethical approval was obtained; patient confidentiality and informed consent are ensured. - Findings will be reported biannually and shared at scientific forums and publications. ## Clinical Analysis & Structured Key Points Introduction Hypertrophic cardiomyopathy (HCM) is a genetic cardiovascular disorder affecting approximately 1 in 200 to 1 in 500 adults, with nearly 70% exhibiting the obstructive phenotype Hypertrophic Obstructive Cardiomyopathy (HOCM). Randomised trials have demonstrated the efficacy of mavacamten, a first-in-class cardiac myosin inhibitor which significantly reduced outflow tract gradients and improved symptoms, exercise capacity and quality of life in the HOCM cases. Despite these findings, prospective real-world data on its safety and effectiveness remain limited, particularly in non-Western populations and further evidence from routine clinical practice is needed. Methods and analysis PERSIA-HOCM is a prospective, multicentre, observational study to be conducted at 19 cardiovascular referral centres in Iran. Adults aged 18 years or older with symptomatic obstructive HCM, defined as New York Heart Association (NYHA) class II to IV and a left ventricular outflow tract (LVOT) gradient of at least 50 mm Hg at rest or with provocation, who are prescribed mavacamten, will be enrolled. Key exclusions include left ventricular ejection fraction below 55%, non-obstructive phenotype and lack of consent. Participants will be followed for 1 year at 4-week intervals. Data collection will include symptoms, NYHA class, vital signs, echocardiographic parameters (including outflow gradients and ejection fraction), mavacamten dosing and adjustments and concomitant therapies. Echocardiography will be performed at weeks 4, 8, 12 and 24. Safety will be assessed using a predefined adverse event framework that includes mortality, hospitalisations and major clinical events. At weeks 12 and 48, cardiac troponin, N-terminal pro B-type natriuretic peptide and quality of life will be assessed. Primary endpoints will be safety, change in the NYHA class, resting and provoked LVOT gradient, quality of life, cardiac biomarkers and left ventricular ejection fraction. Secondary endpoints will include cardiac magnetic resonance imaging parameters for cardiac fibrosis, peak oxygen intake and genotype-based outcome. Analyses will be performed using R V.4.5.1, employing descriptive statistics, paired sample t tests and McNemar's test. Ethics and dissemination Ethical approval was obtained from the Research Ethics Committee of Rajaie Cardiovascular, Medical and Research Institute/Iran National Committee for Ethics in Biomedical Research (Code: IR.RHC.REC.1404.210). Written informed consent will be required and filled by the patients. Considering full data confidentiality, analysis reports will be public every 6 months. 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