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
2022
Muscle LIM Protein Force-Sensing Mediates Sarcomeric Biomechanical Signaling in Human Familial Hypertrophic Cardiomyopathy
Riaz M, Park J, Sewanan LR, Ren Y, Schwan J, Das SK, Pomianowski PT, Huang Y, Ellis MW, Luo J, Liu J, Song L, Chen IP, Qiu C, Yazawa M, Tellides G, Hwa J, Young LH, Yang L, Marboe CC, Jacoby DL, Campbell SG, Qyang Y. Muscle LIM Protein Force-Sensing Mediates Sarcomeric Biomechanical Signaling in Human Familial Hypertrophic Cardiomyopathy. Circulation 2022, 145: 1238-1253. PMID: 35384713, PMCID: PMC9109819, DOI: 10.1161/circulationaha.121.056265.Peer-Reviewed Original ResearchConceptsHypertrophic cardiomyopathySarcomeric mutationsFamilial hypertrophic cardiomyopathySudden cardiac deathCardiac myosin heavy chainMechanism-based treatmentsDevelopment of hypertrophyActivated T cellsCalcineurin-nuclear factorForce productionPhenotypic expressionPluripotent stem cell-derived cardiomyocytesStem cell-derived cardiomyocytesHeart failureCardiac deathVentricular hypertrophyCell-derived cardiomyocytesCardiac contractilityPharmacological interventionsT cellsCardiac diseaseCardiac hypertrophyPatient-specific induced pluripotent stem cellsPharmacological meansTwitch relaxation
2021
Worldwide differences in primary prevention implantable cardioverter defibrillator utilization and outcomes in hypertrophic cardiomyopathy.
Nauffal V, Marstrand P, Han L, Parikh VN, Helms AS, Ingles J, Jacoby D, Lakdawala NK, Kapur S, Michels M, Owens AT, Ashley EA, Pereira AC, Rossano JW, Saberi S, Semsarian C, Ware JS, Wittekind SG, Day S, Olivotto I, Ho CY. Worldwide differences in primary prevention implantable cardioverter defibrillator utilization and outcomes in hypertrophic cardiomyopathy. European Heart Journal 2021, 42: 3932-3944. PMID: 36282238, DOI: 10.1093/eurheartj/ehab598.Peer-Reviewed Original ResearchConceptsSudden cardiac deathAppropriate ICD therapyNon-US sitesHypertrophic cardiomyopathyUS sitesICD therapySCD riskICD recipientsImplantation rateImplantable cardioverter-defibrillator (ICD) implantation ratesMultivariable Cox proportional hazards modelsImplantable cardioverter-defibrillator utilizationSarcomeric Human Cardiomyopathy RegistryCox proportional hazards modelHCM Risk-SCD scoreICD implantation ratesPrimary prevention ICDsSCD risk factorsRisk stratification algorithmLong-term outcomesLow-risk populationSignificant differencesSCD/Cardiac deathClinical outcomesWorldwide differences in primary prevention implantable cardioverter defibrillator utilization and outcomes in hypertrophic cardiomyopathy
Nauffal V, Marstrand P, Han L, Parikh VN, Helms AS, Ingles J, Jacoby D, Lakdawala NK, Kapur S, Michels M, Owens AT, Ashley EA, Pereira AC, Rossano JW, Saberi S, Semsarian C, Ware JS, Wittekind SG, Day S, Olivotto I, Ho CY. Worldwide differences in primary prevention implantable cardioverter defibrillator utilization and outcomes in hypertrophic cardiomyopathy. European Heart Journal 2021, 42: 3932-3944. PMID: 34491319, PMCID: PMC8497072, DOI: 10.1093/eurheartj/ehab598.Peer-Reviewed Original ResearchConceptsSudden cardiac deathAppropriate ICD therapyNon-US sitesHypertrophic cardiomyopathyUS sitesICD therapySCD riskICD recipientsImplantation rateImplantable cardioverter-defibrillator (ICD) implantation ratesMultivariable Cox proportional hazards modelsImplantable cardioverter-defibrillator utilizationSarcomeric Human Cardiomyopathy RegistryCox proportional hazards modelHCM Risk-SCD scoreICD implantation ratesPrimary prevention ICDsSCD risk factorsRisk stratification algorithmLong-term outcomesLow-risk populationSignificant differencesSCD/Cardiac deathClinical outcomes
2020
Appropriate and inappropriate shocks in hypertrophic cardiomyopathy patients with subcutaneous implantable cardioverter-defibrillators: An international multicenter study
Nazer B, Dale Z, Carrassa G, Reza N, Ustunkaya T, Papoutsidakis N, Gray A, Howell SJ, Elman MR, Pieragnoli P, Ricciardi G, Jacoby D, Frankel DS, Owens A, Olivotto I, Heitner SB. Appropriate and inappropriate shocks in hypertrophic cardiomyopathy patients with subcutaneous implantable cardioverter-defibrillators: An international multicenter study. Heart Rhythm 2020, 17: 1107-1114. PMID: 32084597, PMCID: PMC8052990, DOI: 10.1016/j.hrthm.2020.02.008.Peer-Reviewed Original ResearchConceptsSecondary prevention patientsAdjusted odds ratioT-wave inversionAppropriate shocksInappropriate shocksHCM patientsR-wave amplitudeHypertrophic cardiomyopathyS-ICDsCardiology/American Heart Association criteriaElectrocardiographic R-wave amplitudeAmerican Heart Association criteriaHCM Risk-SCD scorePrimary prevention patientsS-ICD shocksAbnormal T-wave inversionSustained ventricular arrhythmiasSudden cardiac deathS-ICD implantationHypertrophic cardiomyopathy patientsInternational multicenter studyT-wave oversensingYounger patientsCardiac deathIndependent predictors
2019
Participation in thrill-seeking activities by patients with hypertrophic cardiomyopathy: Individual preferences, adverse events and physician attitude
Papoutsidakis N, Heitner S, Ingles J, Semsarian C, Mannello M, Salberg L, Waldman C, Vaccaro B, Maurizi N, Olivotto I, Jacoby D. Participation in thrill-seeking activities by patients with hypertrophic cardiomyopathy: Individual preferences, adverse events and physician attitude. American Heart Journal 2019, 214: 28-35. PMID: 31152873, DOI: 10.1016/j.ahj.2019.04.001.Peer-Reviewed Original ResearchConceptsMinor adverse eventsMajor adverse eventsAdverse eventsHCM patientsHypertrophic cardiomyopathyThrill-seeking activitiesPhysician adviceSudden cardiac deathAdult HCM patientsShared-decision makingTime of surveyCardiac deathDefinitive recommendationsRisk factorsHCM diagnosisSafety dataPhysicians' attitudesPatientsAnonymous online surveyReporting participationSame dayCardiomyopathySpecific adviceParticipantsAdvice