This registry evaluates patient characteristics, real-world treatment patterns, and short- and long-term outcomes in a population of patients in the United States and Europe with symptomatic obstructive hypertrophic cardiomyopathy (HCM) who are receiving mavacamten, receiving other treatment for obstructive HCM, or not receiving treatment for obstructive HCM due to intolerance or failure of prior treatment.
United States Sub-Study: The purpose of this study is to evaluate the safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
Europe Sub-Study: The purpose of this study is to evaluate the effectiveness and safety of mavacamten in patients with symptomatic obstructive HCM in the real-world setting.
≥ 18 years of age at the time of informed consent.
Willing and able to provide written informed consent form (ICF) and any required privacy authorization prior to the initiation of study procedures (or in those situations where consent cannot be given by participants, consent provided by their legally acceptable representatives)
Diagnosis of obstructive HCM consistent with 2020 American Heart Association/American College of Cardiology (AHA/ACC) guidelines.
Obstructive HCM is defined clinically by the presence of increased LV wall thickness ≥ 15 mm (or ≥ 13 mm with positive family history of HCM) in a nondilated ventricular chamber that is not solely explained by abnormal loading conditions (eg, another cardiac or systemic disease) and peak LVOT gradient of ≥ 30 mmHg at rest or with provocation.
Known phenocopy disease (e.g., Fabry disease, amyloidosis) or LV hypertrophy associated with hypertension.
Documentation of any fixed obstruction of the outflow tract such as aortic valve stenosis or replacement.
Prior treatment of obstructive HCM with invasive septal reduction (surgical myectomy or percutaneous alcohol septal ablation [ASA]) within 6 months prior to enrollment; participants with an unsuccessful myectomy or percutaneous ASA performed > 6 months prior to enrollment may be enrolled.
Naïve to treatment for obstructive HCM (ie, never treated with BBs, nonDHP CCBs, or disopyramide).
The study population will include adult participants with symptomatic obstructive HCM who are receiving pharmacological treatment for obstructive HCM (for example, BBs, non-DHP CCBs, disopyramide, and/or mavacamten) as part of routine care in the United States or Europe.
Cohort
Prospective
Mavacamten
DrugAs per product label
Non-mavacamten symptomatic oHCM therapy
DrugIncluding Beta-blocker (BB), non-dihydropyridine (non-DHP) calcium channel blocker (CCB) and/or disopyramide as per product label
Incidence rate of new or worsening heart failure due to systolic dysfunction among participants with symptomatic obstructive HCM during periods of exposure to mavacamten and other oHCM treatment
Systolic dysfunction defined as symptomatic Left Ventricular Ejection Fraction (LVEF) \< 50%. United States participants only.
Change in resting left ventricular outflow tract (LVOT) gradient from baseline
European Participants only
Occurrence of arrhythmia
Incidence rates of participants with arrhythmia (new onset)
New or worsening heart failure due to systolic dysfunction
Symptomatic left ventricular ejection fraction (LVEF) \<50%. European Participants only
Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 50 mmHG
European participants only
Proportion of participants with a resting or provoked peak left ventricular outflow tract (LVOT) gradient below 30 mmHG
European participants only
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