Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy
Itzler R, Buckner T, Leebeek F, Miller J, Recht M, Drelich D, Monahan P, Pipe S. Effect of etranacogene dezaparvovec on quality of life for severe and moderately severe haemophilia B participants: Results from the phase III HOPE‐B trial 2 years after gene therapy. Haemophilia 2024, 30: 709-719. PMID: 38462823, DOI: 10.1111/hae.14977.Peer-Reviewed Original ResearchConceptsEQ-5D-5LPercentage of participantsQuality of lifeEtranacogene dezaparvovecNegatively affecting health-related quality of lifeHealth-related quality of lifeMinimal clinically important improvementGene therapyDisease-specific patientMeasures linear mixed modelsClinically important improvementEQ-5D-5L index valuesFactor IXGene transfer productsHRQoLLinear mixed modelsImportant improvementTrial participantsLS mean scoresMean scoreBleed protectionFamily planningB participantsTotal scoreHemophilia B
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