Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial
Coppens M, Pipe S, Miesbach W, Astermark J, Recht M, van der Valk P, Ewenstein B, Pinachyan K, Galante N, Le Quellec S, Monahan P, Leebeek F, Investigators H, Castaman G, Crary S, Escobar M, Gomez E, Haley K, Hermans C, Kampmann P, Kazmi R, Key N, Klamroth R, Konkle B, Kruse-Jarres R, Lattimore S, Lemons R, Meijer K, O'Connell N, Quon D, Raheja P, Symington E, Verhamme P, Visweshwar N, von Drygalski A, Wang M, Wheeler A, White S, Young G. Etranacogene dezaparvovec gene therapy for haemophilia B (HOPE-B): 24-month post-hoc efficacy and safety data from a single-arm, multicentre, phase 3 trial. The Lancet Haematology 2024, 11: e265-e275. PMID: 38437857, DOI: 10.1016/s2352-3026(24)00006-1.Peer-Reviewed Original ResearchConceptsAnnualised bleeding rateLead-in periodFactor IX prophylaxisEtranacogene dezaparvovecPhase 3 trialGene therapyFactor IX activityAdverse eventsIX activityHemophilia BHemophilia B.Safety dataTreatment-related adverse eventsPost hoc efficacy analysisPost-hocTreatment-related deathsFactor IX expressionFull analysis setHemophilia B treatmentEffective therapeutic optionInfluenza-like illnessBleed protectionData cutoffBleeding phenotypePrimary endpoint
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