{"status":"ok","message-type":"work","message-version":"1.0.0","message":{"indexed":{"date-parts":[[2026,8,24]],"date-time":"2026-08-24T16:45:08Z","timestamp":1787589908914,"version":"build-2736575974"},"reference-count":28,"publisher":"BMJ","issue":"1","license":[{"start":{"date-parts":[[2025,4,16]],"date-time":"2025-04-16T00:00:00Z","timestamp":1744761600000},"content-version":"unspecified","delay-in-days":105,"URL":"http:\/\/creativecommons.org\/licenses\/by-nc\/4.0\/"}],"content-domain":{"domain":["bmj.com"],"crossmark-restriction":true},"short-container-title":["Open Heart"],"accepted":{"date-parts":[[2025,3,24]]},"published-print":{"date-parts":[[2025,1]]},"abstract":"<jats:sec>\n                  <jats:title>Background<\/jats:title>\n                  <jats:p>Hypertrophic cardiomyopathy (HCM) is characterised by abnormal thickening of ventricular myocardium. Left ventricular outflow tract (LVOT) obstruction occurs in up to 70% of patients, causing progressive symptoms, heart failure and mortality. Mavacamten, the first targeted therapy for obstructive HCM (oHCM), was approved for use in the UK in 2024. We present data from the early experience with mavacamten treatment for oHCM in three UK centres and describe different clinical pathways.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Methods<\/jats:title>\n                  <jats:p>All patients with symptomatic oHCM eligible for mavacamten therapy were included. Eligibility criteria included New York Heart Association (NYHA) class II\u2013III symptoms and LVOT gradients &gt;30\u2009mm Hg with normal left ventricular ejection function (LVEF &gt;55%), as per National Institute for Health and Care Excellence guidelines and product literature. Patients underwent CYP2C19 genotyping before treatment, and dosing was adjusted accordingly. Echocardiographic assessments, clinical reviews and biomarker analyses were conducted at weeks 0, 4, 8 and 12.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Results<\/jats:title>\n                  <jats:p>93 patients were initiated on mavacamten (mean age: 60\u00b113 years; 72% male). The Valsalva LVOT gradient significantly decreased during treatment, from 88.9\u00b131\u2009mm Hg at baseline to 43.8\u00b132.6\u2009mm Hg by week 12, and further to 27.7\u00b122.3\u2009mm Hg on the maintenance dose. NT-proBNP levels also improved markedly, from 689 ng\/L (IQR 343-1684 ng\/L) at baseline to 171 ng\/L (IQR 116-335 ng\/L) on the maintenance dose. By week 12, 74% of patients experienced an improvement of at least one NYHA class, increasing to 91% on the maintenance dose. Temporary treatment interruptions occurred in 13 patients; however, no patients required permanent discontinuation of treatment due to a reduced LVEF (&lt;50%). Outcomes were comparable across the three care pathways.<\/jats:p>\n               <\/jats:sec>\n               <jats:sec>\n                  <jats:title>Conclusions<\/jats:title>\n                  <jats:p>Mavacamten treatment was associated with significant symptomatic improvement, reduced LVOT gradients and improved biomarker profiles in patients with oHCM. The implementation of clinical services to deliver mavacamten in the UK should not follow a \u2018one-size-fits-all\u2019 approach but rather leverage the unique strengths of each specific centre.<\/jats:p>\n               <\/jats:sec>","DOI":"10.1136\/openhrt-2025-003218","type":"journal-article","created":{"date-parts":[[2025,4,17]],"date-time":"2025-04-17T20:10:51Z","timestamp":1744920651000},"page":"e003218","update-policy":"https:\/\/doi.org\/10.1136\/crossmarkpolicy","source":"Crossref","is-referenced-by-count":4,"title":["Tale of three services: early UK experience with mavacamten treatment for hypertrophic cardiomyopathy with left ventricular outflow tract obstruction"],"prefix":"10.1136","volume":"12","author":[{"given":"Edward","family":"Burford","sequence":"first","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"},{"name":"British Heart Foundation Centre of Research Excellence, King\u2019s College London School of Cardiovascular and Metabolic Medicine & Sciences, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-8951-2111","authenticated-orcid":false,"given":"Yande","family":"Kasolo","sequence":"additional","affiliation":[{"name":"Liverpool Heart and Chest Hospital NHS Foundation Trust, Liverpool, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Jane","family":"Draper","sequence":"additional","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-5905-8907","authenticated-orcid":false,"given":"Nabeel","family":"Sheikh","sequence":"additional","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"},{"name":"British Heart Foundation Centre of Research Excellence, King\u2019s College London School of Cardiovascular and Metabolic Medicine & Sciences, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Gerry","family":"Carr-White","sequence":"additional","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"},{"name":"British Heart Foundation Centre of Research Excellence, King\u2019s College London School of Cardiovascular and Metabolic Medicine & Sciences, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Antonio","family":"de Marvao","sequence":"additional","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"},{"name":"British Heart Foundation Centre of Research Excellence, King\u2019s College London School of Cardiovascular and Metabolic Medicine & Sciences, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"ORCID":"https:\/\/orcid.org\/0000-0002-6086-6813","authenticated-orcid":false,"given":"Jason N","family":"Dungu","sequence":"additional","affiliation":[{"name":"Cardiology, Essex Cardiothoracic Centre, Basildon, UK"},{"name":"Anglia Ruskin University School of Medicine, Chelmsford, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Rachel","family":"Bastiaenen","sequence":"additional","affiliation":[{"name":"Inherited Cardiovascular Conditions Group, Guy's and St Thomas\u2019 NHS Foundation Trust, London, UK"},{"name":"British Heart Foundation Centre of Research Excellence, King\u2019s College London School of Cardiovascular and Metabolic Medicine & Sciences, London, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]},{"given":"Robert M","family":"Cooper","sequence":"additional","affiliation":[{"name":"Liverpool Heart and Chest Hospital NHS Foundation Trust, Liverpool, UK"},{"name":"Research Institute, Liverpool John Moores University School of Sport and Exercise Sciences, Liverpool, UK"}],"role":[{"vocabulary":"crossref","role":"author"}]}],"member":"239","published-online":{"date-parts":[[2025,4,17]]},"reference":[{"key":"2025081711073041000_12.1.e003218.1","doi-asserted-by":"publisher","DOI":"10.1161\/01.CIR.92.4.785"},{"key":"2025081711073041000_12.1.e003218.2","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2015.01.019"},{"key":"2025081711073041000_12.1.e003218.3","doi-asserted-by":"crossref","first-page":"74","DOI":"10.1016\/j.amjcard.2022.06.017","article-title":"What Causes Hypertrophic Cardiomyopathy?","volume":"179","author":"Maron","year":"2022","journal-title":"Am J 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Cardiol"},{"key":"2025081711073041000_12.1.e003218.10","doi-asserted-by":"publisher","DOI":"10.1002\/ejhf.541"},{"key":"2025081711073041000_12.1.e003218.11","doi-asserted-by":"crossref","first-page":"529","DOI":"10.1016\/j.acvd.2022.06.003","article-title":"Medical treatment of patients with hypertrophic cardiomyopathy: An overview of current and emerging therapy","volume":"115","author":"Iavarone","year":"2022","journal-title":"Arch Cardiovasc Dis"},{"key":"2025081711073041000_12.1.e003218.12","doi-asserted-by":"crossref","first-page":"324","DOI":"10.1001\/jamacardio.2016.0252","article-title":"Hospital Volume Outcomes After Septal Myectomy and Alcohol Septal Ablation for Treatment of Obstructive Hypertrophic Cardiomyopathy: US Nationwide Inpatient Database, 2003-2011","volume":"1","author":"Kim","year":"2016","journal-title":"JAMA Cardiol"},{"key":"2025081711073041000_12.1.e003218.13","doi-asserted-by":"crossref","first-page":"497","DOI":"10.1007\/s40256-022-00532-x","article-title":"The Impact of Mavacamten on the Pathophysiology of Hypertrophic Cardiomyopathy: A Narrative Review","volume":"22","author":"Edelberg","year":"2022","journal-title":"Am J Cardiovasc Drugs"},{"key":"2025081711073041000_12.1.e003218.14","first-page":"1127","article-title":"Mavacamten: First Approval","volume":"82","author":"Keam","year":"2022","journal-title":"Drugs (Abingdon Engl)"},{"key":"2025081711073041000_12.1.e003218.15","doi-asserted-by":"publisher","DOI":"10.1016\/S0140-6736(20)31792-X"},{"key":"2025081711073041000_12.1.e003218.16","doi-asserted-by":"publisher","DOI":"10.1016\/j.jacc.2022.04.048"},{"key":"2025081711073041000_12.1.e003218.17","doi-asserted-by":"crossref","unstructured":"Garcia-Pavia P , Oreziak A , Masri A , et al . Long-term safety and efficacy of mavacamten in obstructive hypertrophic cardiomyopathy: up to 3.5-year follow-up results of the EXPLORER cohort of MAVA-Long-Term Extension study. Eur Heart J 2024;45. doi:10.1093\/eurheartj\/ehae666.2025","DOI":"10.1093\/eurheartj\/ehae666.2025"},{"key":"2025081711073041000_12.1.e003218.18","first-page":"164","article-title":"Mavacamten Treatment for Symptomatic Obstructive Hypertrophic Cardiomyopathy","volume":"12","author":"Rader","year":"2024","journal-title":"JACC: Heart Failure"},{"key":"2025081711073041000_12.1.e003218.19","doi-asserted-by":"crossref","first-page":"850","DOI":"10.1161\/CIRCULATIONAHA.122.062534","article-title":"Dose-Blinded Myosin Inhibition in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy: Outcomes Through 32 Weeks","volume":"147","author":"Desai","year":"2023","journal-title":"Circulation"},{"key":"2025081711073041000_12.1.e003218.20","doi-asserted-by":"crossref","DOI":"10.1161\/CIRCIMAGING.122.014986","article-title":"Myosin Inhibition and Left Ventricular Diastolic Function in Patients With Obstructive Hypertrophic Cardiomyopathy Referred for Septal Reduction Therapy: Insights From the VALOR-HCM Study","volume":"15","author":"Cremer","year":"2022","journal-title":"Circ Cardiovasc Imaging"},{"key":"2025081711073041000_12.1.e003218.21","doi-asserted-by":"crossref","first-page":"606","DOI":"10.1161\/CIRCULATIONAHA.120.052359","article-title":"Mavacamten Favorably Impacts Cardiac Structure in Obstructive Hypertrophic Cardiomyopathy: EXPLORER-HCM Cardiac Magnetic Resonance Substudy Analysis","volume":"143","author":"Saberi","year":"2021","journal-title":"Circulation"},{"key":"2025081711073041000_12.1.e003218.22","unstructured":"NICE . Mavacamten for treating symptomatic obstructive hypertrophic cardiomyopathy. 2023. Available: www.nice.org.uk\/guidance\/ta913"},{"key":"2025081711073041000_12.1.e003218.23","unstructured":"Mavacamten Summary of Product Characteristics (SmPC) . Bristol Myers Squibb, PLGB 15105\/0180. 2023. Available: https:\/\/www.medicines.org.uk\/emc\/product\/15030\/smpc#gref [Accessed 28 Jul 2023]."},{"key":"2025081711073041000_12.1.e003218.24","doi-asserted-by":"crossref","first-page":"1397","DOI":"10.1002\/phar.2874","article-title":"Transitioning disopyramide to mavacamten in obstructive hypertrophic cardiomyopathy: A case series and clinical guide","volume":"43","author":"Willeford","year":"2023","journal-title":"Pharmacotherapy"},{"key":"2025081711073041000_12.1.e003218.25","doi-asserted-by":"crossref","DOI":"10.1161\/CIRCHEARTFAILURE.120.006853","article-title":"Study Design and Rationale of EXPLORER-HCM: Evaluation of Mavacamten in Adults With Symptomatic Obstructive Hypertrophic Cardiomyopathy","volume":"13","author":"Ho","year":"2020","journal-title":"Circ Heart Fail"},{"key":"2025081711073041000_12.1.e003218.26","doi-asserted-by":"crossref","first-page":"1275","DOI":"10.1002\/jcph.2298","article-title":"Drug-Drug Interaction Potential of Mavacamten with Oral Contraceptives: Results from a Clinical Pharmacokinetic Study and a Physiologically Based Pharmacokinetic Model","volume":"63","author":"Chiang","year":"2023","journal-title":"J Clin Pharmacol"},{"key":"2025081711073041000_12.1.e003218.27","doi-asserted-by":"crossref","first-page":"11","DOI":"10.1093\/bmb\/ldae003","article-title":"The development of inherited cardiac conditions services: current position and future perspectives","volume":"150","author":"Alway","year":"2024","journal-title":"Br Med Bull"},{"key":"2025081711073041000_12.1.e003218.28","doi-asserted-by":"crossref","first-page":"100004","DOI":"10.1016\/j.clinme.2023.100004","article-title":"Extending the reach of expert amyloidosis care: A feasibility study exploring the staged implementation of a UK amyloidosis network","volume":"24","author":"Choy","year":"2024","journal-title":"Clin Med (Northfield)"}],"container-title":["Open Heart"],"original-title":[],"language":"en","link":[{"URL":"https:\/\/syndication.highwire.org\/content\/doi\/10.1136\/openhrt-2025-003218","content-type":"unspecified","content-version":"vor","intended-application":"similarity-checking"}],"deposited":{"date-parts":[[2025,8,17]],"date-time":"2025-08-17T18:07:42Z","timestamp":1755454062000},"score":1,"resource":{"primary":{"URL":"https:\/\/openheart.bmj.com\/lookup\/doi\/10.1136\/openhrt-2025-003218"}},"subtitle":[],"short-title":[],"issued":{"date-parts":[[2025,1]]},"references-count":28,"journal-issue":{"issue":"1","published-online":{"date-parts":[[2025,4,17]]},"published-print":{"date-parts":[[2025,1]]}},"alternative-id":["10.1136\/openhrt-2025-003218"],"URL":"https:\/\/doi.org\/10.1136\/openhrt-2025-003218","relation":{},"ISSN":["2053-3624"],"issn-type":[{"value":"2053-3624","type":"electronic"}],"subject":[],"published":{"date-parts":[[2025,1]]}}}