2024
Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy Results From SEQUOIA-HCM
Sherrod C, Saberi S, Nassif M, Claggett B, Coats C, Garcia-Pavia P, Januzzi J, Lewis G, Ma C, Maron M, Miao Z, Olivotto I, Veselka J, Butzner M, Jacoby D, Heitner S, Kupfer S, Malik F, Meng L, Wohltman A, Spertus J. Effect of Aficamten on Health Status Outcomes in Obstructive Hypertrophic Cardiomyopathy Results From SEQUOIA-HCM. Journal Of The American College Of Cardiology 2024, 84: 1773-1785. PMID: 39217569, DOI: 10.1016/j.jacc.2024.08.014.Peer-Reviewed Original ResearchKCCQ overall summary scoresKansas City Cardiomyopathy QuestionnaireObstructive hypertrophic cardiomyopathyHealth statusQuality of lifePatient-reported health statusParticipants' health statusHealth status outcomesHypertrophic cardiomyopathyHealth status benefitsHealth status improvementPatients' health statusClinically important changeProportion of participantsSymptoms of fatigueSymptomatic obstructive hypertrophic cardiomyopathyStatus outcomesSummary scoreImprove healthCardiac myosin inhibitorProportion of patientsBaseline scoresImportant changesShortness of breathStatus improvement
2022
Pragmatic randomized trial assessing the impact of digital health technology on quality of life in patients with heart failure: Design, rationale and implementation
Victoria‐Castro A, Martin M, Yamamoto Y, Ahmad T, Arora T, Calderon F, Desai N, Gerber B, Lee KA, Jacoby D, Melchinger H, Nguyen A, Shaw M, Simonov M, Williams A, Weinstein J, Wilson FP. Pragmatic randomized trial assessing the impact of digital health technology on quality of life in patients with heart failure: Design, rationale and implementation. Clinical Cardiology 2022, 45: 839-849. PMID: 35822275, PMCID: PMC9346973, DOI: 10.1002/clc.23848.Peer-Reviewed Original ResearchConceptsQuality of lifeHF patientsYale New Haven Health SystemKansas City Cardiomyopathy QuestionnaireDigital health technologiesHeart failure careHealth technologiesDigital health interventionsDisease exacerbationHF clinicUsual carePrimary outcomeClinical outcomesHeart failurePatient engagementPatientsHealth interventionsDigital health toolsPatient empowermentHealth systemPatient usabilityHealth toolsEarly detectionFirst enrollmentHealthcare system
2021
Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial
Spertus JA, Fine JT, Elliott P, Ho CY, Olivotto I, Saberi S, Li W, Dolan C, Reaney M, Sehnert AJ, Jacoby D. Mavacamten for treatment of symptomatic obstructive hypertrophic cardiomyopathy (EXPLORER-HCM): health status analysis of a randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet 2021, 397: 2467-2475. PMID: 34004177, DOI: 10.1016/s0140-6736(21)00763-7.Peer-Reviewed Original ResearchConceptsKansas City Cardiomyopathy QuestionnaireSymptomatic obstructive hypertrophic cardiomyopathyObstructive hypertrophic cardiomyopathyHypertrophic cardiomyopathyPatients' health statusQuality of lifeHealth statusWeek 30KCCQ overall summary scoreAvailable pharmacological optionsKCCQ-OS scorePlacebo-controlled trialPhase 3 trialProportion of patientsPrimary treatment goalOverall summary scoreDisease-specific measuresEffect of mavacamtenHealth status analysisNew potential strategyEXPLORER-HCMPlacebo groupAdult patientsBristol-Myers Squibb CompanyPharmacological options
2020
Study Design and Rationale of EXPLORER-HCM
Ho CY, Olivotto I, Jacoby D, Lester SJ, Roe M, Wang A, Waldman CB, Zhang D, Sehnert AJ, Heitner SB. Study Design and Rationale of EXPLORER-HCM. Circulation Heart Failure 2020, 13: e006853. PMID: 32498620, DOI: 10.1161/circheartfailure.120.006853.Peer-Reviewed Original ResearchConceptsNew York Heart Association classLV outflow tract obstructionObstructive hypertrophic cardiomyopathyOutflow tract obstructionPeak oxygen consumptionEXPLORER-HCMTract obstructionAssociation classEnd pointExercise capacityNew York Heart Association functional class IIDynamic LV outflow tract obstructionKansas City Cardiomyopathy QuestionnaireLV outflow tract gradientSymptomatic obstructive hypertrophic cardiomyopathyUnexplained left ventricular hypertrophyOxygen consumptionExploratory end pointsFunctional class IIOutflow tract gradientPlacebo-controlled trialSecondary end pointsPhase 2 studyPhase 3 trialCurrent medical therapy