2024
Impact of Aficamten on Echocardiographic Cardiac Structure and Function in Symptomatic Obstructive Hypertrophic Cardiomyopathy
Hegde S, Claggett B, Wang X, Jering K, Prasad N, Roshanali F, Masri A, Nassif M, Barriales-Villa R, Abraham T, Cardim N, Coats C, Kramer C, Maron M, Michels M, Olivotto I, Saberi S, Jacoby D, Heitner S, Kupfer S, Meng L, Wohltman A, Malik F, Solomon S, Investigators S. Impact of Aficamten on Echocardiographic Cardiac Structure and Function in Symptomatic Obstructive Hypertrophic Cardiomyopathy. Journal Of The American College Of Cardiology 2024, 84: 1789-1802. PMID: 39217556, DOI: 10.1016/j.jacc.2024.08.002.Peer-Reviewed Original ResearchLeft ventricular ejection fractionObstructive hypertrophic cardiomyopathySymptomatic obstructive hypertrophic cardiomyopathyLVOT gradientKCCQ-CSSNT-proBNPAssociated with improvementsHypertrophic cardiomyopathyKansas City Cardiomyopathy Questionnaire Clinical Summary ScoreCardiac structureMeasures of LV diastolic functionLV global circumferential strainN-terminal pro-B-type natriuretic peptideLV global longitudinal strainLV end-systolic volumePro-B-type natriuretic peptideEchocardiographic measures of cardiac structureMeasures of cardiac structureLateral e' velocityLV diastolic functionAtrial volume indexGlobal longitudinal strainLV systolic functionPlacebo-controlled studyGlobal circumferential strain
2021
Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy
Hegde SM, Lester SJ, Solomon SD, Michels M, Elliott PM, Nagueh SF, Choudhury L, Zemanek D, Zwas DR, Jacoby D, Wang A, Ho CY, Li W, Sehnert AJ, Olivotto I, Abraham TP. Effect of Mavacamten on Echocardiographic Features in Symptomatic Patients With Obstructive Hypertrophic Cardiomyopathy. Journal Of The American College Of Cardiology 2021, 78: 2518-2532. PMID: 34915982, DOI: 10.1016/j.jacc.2021.09.1381.Peer-Reviewed Original ResearchConceptsObstructive hypertrophic cardiomyopathySystolic anterior motionAnterior motionDiastolic functionHypertrophic cardiomyopathyN-terminal pro-B-type natriuretic peptideMitral valve systolic anterior motionPro-B-type natriuretic peptidePeak exercise oxygen consumptionLeft ventricular diastolic functionVentricular outflow tract obstructionKey echocardiographic parametersAtrial volume indexOutflow tract obstructionVentricular diastolic functionExercise oxygen consumptionEffect of mavacamtenMyocardial wall stressEXPLORER-HCMLVOT gradientEchocardiographic featuresEchocardiographic parametersLVOT obstructionTract obstructionExercise capacity
2020
Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial
Olivotto I, Oreziak A, Barriales-Villa R, Abraham TP, Masri A, Garcia-Pavia P, Saberi S, Lakdawala NK, Wheeler MT, Owens A, Kubanek M, Wojakowski W, Jensen MK, Gimeno-Blanes J, Afshar K, Myers J, Hegde SM, Solomon SD, Sehnert AJ, Zhang D, Li W, Bhattacharya M, Edelberg JM, Waldman CB, Lester SJ, Wang A, Ho CY, Jacoby D, investigators E, Bartunek J, Bondue A, Van Craenenbroeck E, Kubanek M, Zemanek D, Jensen M, Mogensen J, Thune J, Charron P, Hagege A, Lairez O, Trochu J, Axthelm C, Duengen H, Frey N, Mitrovic V, Preusch M, Schulz-Menger J, Seidler T, Arad M, Halabi M, Katz A, Monakier D, Paz O, Viskin S, Zwas D, Olivotto I, Rocca H, Michels M, Dudek D, Oko-Sarnowska Z, Oreziak A, Wojakowski W, Cardim N, Pereira H, Barriales-Villa R, Pavia P, Blanes J, Urbano R, Diaz L, Elliott P, Yousef Z, Abraham T, Afshar K, Alvarez P, Bach R, Becker R, Choudhury L, Fermin D, Jacoby D, Jefferies J, Kramer C, Lakdawala N, Lester S, Marian A, Masri A, Maurer M, Nagueh S, Owens A, Owens D, Rader F, Saberi S, Sherrid M, Shirani J, Symanski J, Turer A, Wang A, Wever-Pinzon O, Wheeler M, Wong T, Yamani M. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): a randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet 2020, 396: 759-769. PMID: 32871100, DOI: 10.1016/s0140-6736(20)31792-x.Peer-Reviewed Original ResearchConceptsObstructive hypertrophic cardiomyopathyKansas City Cardiomyopathy Questionnaire clinical summary scoreSymptomatic obstructive hypertrophic cardiomyopathyHypertrophic cardiomyopathyLVOT gradientNYHA classPrimary endpointDynamic left ventricular outflow tract obstructionNew York Heart Association class IILeft ventricular outflow tract obstructionTreatment-emergent adverse eventsVentricular outflow tract obstructionAvailable pharmacological optionsKey pathophysiological abnormalityNYHA class reductionNYHA functional classOutflow tract obstructionPlacebo-controlled trialPhase 3 trialML/Disease-specific treatmentPeak oxygen consumptionGreater increaseLVOT obstructionPlacebo group
2019
Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial.
Heitner SB, Jacoby D, Lester SJ, Owens A, Wang A, Zhang D, Lambing J, Lee J, Semigran M, Sehnert AJ. Mavacamten Treatment for Obstructive Hypertrophic Cardiomyopathy: A Clinical Trial. Annals Of Internal Medicine 2019, 170: 741-748. PMID: 31035291, DOI: 10.7326/m18-3016.Peer-Reviewed Original ResearchMeSH KeywordsAdministration, OralAdrenergic beta-AntagonistsAdultAgedBenzylaminesCardiomyopathy, HypertrophicCardiovascular AgentsDose-Response Relationship, DrugDrug Therapy, CombinationExercise ToleranceFemaleHumansMaleMiddle AgedOxygen ConsumptionProspective StudiesStroke VolumeUracilVentricular Function, LeftYoung AdultConceptsObstructive hypertrophic cardiomyopathyLVOT gradientDyspnea scoreAdverse eventsCohort BPeak VO2Hypertrophic cardiomyopathyLeft ventricular outflow tract gradientVentricular outflow tract gradientEnd pointCommon adverse eventsOutflow tract gradientUnrelated adverse eventsOpen-label designPrimary end pointSecondary end pointsPhase 2 trialVentricular ejection fractionML/Peak oxygen consumptionEffect of mavacamtenHigher plasma concentrationsMavacamten treatmentLVOT obstructionTract gradient