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
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