2024
Efficacy and Safety of Aficamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4
Masri A, Sherrid M, Abraham T, Choudhury L, Garcia-Pavia P, Kramer C, Barriales-Villa R, Owens A, Rader F, Nagueh S, Olivotto I, Saberi S, Tower-Rader A, Wong T, Coats C, Watkins H, Fifer M, Solomon S, Heitner S, Jacoby D, Kupfer S, Malik F, Meng L, Sohn R, Wohltman A, Maron M, Investigators R. Efficacy and Safety of Aficamten in Symptomatic Nonobstructive Hypertrophic Cardiomyopathy: Results From the REDWOOD-HCM Trial, Cohort 4. Journal Of Cardiac Failure 2024, 30: 1439-1448. PMID: 38493832, DOI: 10.1016/j.cardfail.2024.02.020.Peer-Reviewed Original ResearchNonobstructive hypertrophic cardiomyopathyHypertrophic cardiomyopathyKansas City Cardiomyopathy Questionnaire clinical summary scoreOpen-label phase 2 trialNew York Heart Association classHistory of aborted sudden cardiac deathNon-obstructive hypertrophic cardiomyopathyHigh-sensitivity cardiac troponin IAborted sudden cardiac deathBlood levels of biomarkersNT-proBNP levelsPhase 2 trialPlacebo-controlled studyHeart failure symptomsCardiac myosin inhibitorClinical summary scoreSudden cardiac deathClinically relevant improvementWeeks of washoutLevels of biomarkersCardiac troponin IHeart muscle cellsAsymptomatic reductionNT-proBNPAssociated with improvements
2020
Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy
Ho CY, Mealiffe ME, Bach RG, Bhattacharya M, Choudhury L, Edelberg JM, Hegde SM, Jacoby D, Lakdawala NK, Lester SJ, Ma Y, Marian AJ, Nagueh SF, Owens A, Rader F, Saberi S, Sehnert AJ, Sherrid MV, Solomon SD, Wang A, Wever-Pinzon O, Wong TC, Heitner SB. Evaluation of Mavacamten in Symptomatic Patients With Nonobstructive Hypertrophic Cardiomyopathy. Journal Of The American College Of Cardiology 2020, 75: 2649-2660. PMID: 32466879, DOI: 10.1016/j.jacc.2020.03.064.Peer-Reviewed Original ResearchConceptsNonobstructive hypertrophic cardiomyopathyGeometric mean differencePlacebo groupHypertrophic cardiomyopathyN-terminal pro-B-type natriuretic peptidePro-B-type natriuretic peptidePg/Mean differencePhase II studySerious adverse eventsVentricular ejection fractionMyocardial wall stressDose titrationNT-proBNPAdverse eventsII studySymptomatic patientsEjection fractionPharmacological therapyClinical parametersInitial doseNatriuretic peptideMean ageHigh burdenMavacamten
2019
Baseline Characteristics of the VANISH Cohort
Axelsson Raja A, Shi L, Day SM, Russell M, Zahka K, Lever H, Colan SD, Margossian R, Hall EK, Becker J, Jefferies JL, Patel AR, Choudhury L, Murphy AM, Canter C, Bach R, Taylor M, Mestroni L, Wheeler MT, Benson L, Owens AT, Rossano J, Lin KY, Pahl E, Pereira AC, Bundgaard H, Lewis GD, Vargas JD, Cirino AL, McMurray JJV, MacRae CA, Solomon SD, Orav EJ, Braunwald E, Ho CY. Baseline Characteristics of the VANISH Cohort. Circulation Heart Failure 2019, 12: e006231. PMID: 31813281, PMCID: PMC7219518, DOI: 10.1161/circheartfailure.119.006231.Peer-Reviewed Original ResearchMeSH KeywordsAdolescentAdultAngiotensin II Type 1 Receptor BlockersBrazilCanadaCardiomyopathy, HypertrophicChildDenmarkDisease ProgressionDouble-Blind MethodFemaleGenetic Predisposition to DiseaseHumansMaleMiddle AgedMutationPhenotypeRecovery of FunctionSarcomeresTime FactorsTreatment OutcomeUnited StatesValsartanYoung AdultConceptsAngiotensin receptor blockersHypertrophic cardiomyopathyReceptor blockersBaseline characteristicsImaging abnormalitiesPrimary cohortMutation carriersNew York Heart Association class IINew York Heart Association classFunctional class II symptomsOlder ageClass II symptomsNonobstructive hypertrophic cardiomyopathyNormal functional capacityPrevious trialsVentricular wall thicknessPeak oxygen consumptionPlacebo-controlled designCardiac magnetic resonanceGene mutation carriersLate gadolinium enhancementVANISH trialAdvanced diseaseAssociation classVentricular hypertrophy
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