Outcomes of a Multidisciplinary Approach to Management of Mavacamten in Obstructive Hypertrophic Cardiomyopathy

CIRCULATION(2023)

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摘要
Background: Traditional treatments for obstructive hypertrophic cardiomyopathy (oHCM) include beta-blockers, calcium channel blockers, and disopyramide. Mavacamten, a novel cardiac myosin inhibitor, is a promising oHCM therapy but has practical challenges limiting its use. This study aimed to describe a clinic workflow for mavacamten management in a real-world setting, addressing challenges such as cost, drug interactions, and monitoring requirements. The focus was on reducing patient-level costs while ensuring feasibility and efficiency. Methods: A retrospective analysis was conducted on 34 oHCM patients considered for mavacamten between May 2022 and May 2023. The clinic workflow involved cardiologist assessment, pharmacist evaluation of drug interactions, enrollment in the mavacamten REMS program, cost reduction measures, and initiation monitoring through scheduled echocardiograms. An algorithm detailing steps and tools used in this workflow is provided. Results: Of the 34 patients, 21 (62%) were initiated on mavacamten and followed for up to 1 year on therapy. Cost assessments indicated reduced out-of-pocket expenses with assistance programs. The median time from referral to first fill was 22 days. Patients demonstrated high adherence (99.1%) measured by proportion of days covered. Echocardiogram follow-ups showed significant improvements in left ventricular outflow tract parameters with no patients having a decrease in left ventricular ejection fraction to less than 50%. Conclusions: The described workflow effectively addressed challenges associated with mavacamten management, emphasizing roles for clinic personnel, cost reduction strategies, and structured patient monitoring. While the workflow's specifics may need adaptation in different settings, this report provides valuable insights for clinics implementing structured mavacamten management approaches. ### Competing Interest Statement AW serves as a clinical consultant for Bristol Myers Squibb and Cytokinetics. JSE reports receiving honoraria from Bristol Myers Squibb. ### Clinical Trial This is retrospective study. A clinical trial ID was not required. ### Funding Statement This study was unfunded. ### Author Declarations I confirm all relevant ethical guidelines have been followed, and any necessary IRB and/or ethics committee approvals have been obtained. Yes The details of the IRB/oversight body that provided approval or exemption for the research described are given below: This study was approved by the UC San Diego Health institutional review board. Informed consent was not required. I confirm that all necessary patient/participant consent has been obtained and the appropriate institutional forms have been archived, and that any patient/participant/sample identifiers included were not known to anyone (e.g., hospital staff, patients or participants themselves) outside the research group so cannot be used to identify individuals. Yes I understand that all clinical trials and any other prospective interventional studies must be registered with an ICMJE-approved registry, such as ClinicalTrials.gov. I confirm that any such study reported in the manuscript has been registered and the trial registration ID is provided (note: if posting a prospective study registered retrospectively, please provide a statement in the trial ID field explaining why the study was not registered in advance). Yes I have followed all appropriate research reporting guidelines, such as any relevant EQUATOR Network research reporting checklist(s) and other pertinent material, if applicable. Yes Data presented are not openly available due to institutional policy.
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Hypertrophic cardiomyopathy
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