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 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 optionsChanges in Use of Left Ventricular Assist Devices as Bridge to Transplantation With New Heart Allocation Policy
Mullan CW, Chouairi F, Sen S, Mori M, Clark KAA, Reinhardt SW, Miller PE, Fuery MA, Jacoby D, Maulion C, Anwer M, Geirsson A, Mulligan D, Formica R, Rogers JG, Desai NR, Ahmad T. Changes in Use of Left Ventricular Assist Devices as Bridge to Transplantation With New Heart Allocation Policy. JACC Heart Failure 2021, 9: 420-429. PMID: 33714748, DOI: 10.1016/j.jchf.2021.01.010.Peer-Reviewed Original ResearchConceptsDonor heart allocation systemNumber of patientsHeart allocation systemVentricular assist deviceWaitlist survivalAssist deviceNew heart allocation policyLeft ventricular assist deviceAllocation system changePost-transplant mortalityOrgan Sharing databaseOutcomes of patientsPost-transplantation outcomesProportion of patientsTime of transplantContinuous-flow LVADPost-transplantation survivalHeart allocation policyNew allocation systemBaseline recipientStatus 1AHeart transplantationIschemic timeLVAD implantationTransplant list