2024
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
2021
Reduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study
Journeycake J, Cheng D, Chrisentery-Singleton T, Desai V, von Drygalski A, Fedor C, Hirsh N, Patel B, Raffini L, Recht M, Sidinio R, Wang M, Zhang X, Neufeld E. Reduced Dosing Frequency Following a Switch to Rix-FP for the Treatment of Hemophilia B: Results from the Athn 2 Study. Blood 2021, 138: 1039. DOI: 10.1182/blood-2021-144617.Peer-Reviewed Original ResearchEntity's Board of DirectorsExtended dosing intervalRIX-FPDosing intervalHemophilia treatment centersDosing frequencyCSL BehringProspective armRetrospective armOnce-weeklyHemophilia BHemophilia B.Half-life recombinant factorTreatment switchUS hemophilia treatment centersRange of therapeutic optionsFrequent dosing regimenPrescribed dosing frequencyTreatment of hemophilia BFactor replacement productsWeeks prior to enrollmentAmerican ThrombosisSevere hemophilia BBaseline demographic dataReduced dosing frequency
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