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
2023
Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
Pipe S, Leebeek F, Recht M, Key N, Castaman G, Miesbach W, Lattimore S, Peerlinck K, Van der Valk P, Coppens M, Kampmann P, Meijer K, O’Connell N, Pasi K, Hart D, Kazmi R, Astermark J, Hermans C, Klamroth R, Lemons R, Visweshwar N, von Drygalski A, Young G, Crary S, Escobar M, Gomez E, Kruse-Jarres R, Quon D, Symington E, Wang M, Wheeler A, Gut R, Liu Y, Dolmetsch R, Cooper D, Li Y, Goldstein B, Monahan P. Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B. New England Journal Of Medicine 2023, 388: 706-718. PMID: 36812434, DOI: 10.1056/nejmoa2211644.Peer-Reviewed Original ResearchConceptsAnnualized bleeding rateLead-in periodAdeno-associated virus 5Factor IX prophylaxisGene therapyHemophilia BBleeding rateEtranacogene dezaparvovecFactor IX activityMonth 7Factor IX replacementPhase 3 studyIX activityNeutralizing antibody titersFactor IX concentrateOpen-labelRate ratiosSafety profileEfficacy measuresAdverse eventsPrevent bleedingNeutralizing antibodiesNoninferiority marginNoninferiority analysisNoninferiorityDurability of bleeding protection and Factor IX activity in individuals with and without adeno-associated virus serotype 5 neutralising antibodies (Titres <1:700) in the phase 3 HOPE-B trial of etranacogene dezaparvovec gene therapy for haemophilia B
Pipe W, Leebeek W, Recht M, Key S, Lattimore S, Castaman G, Cooper D, Verweij S, Dolmetsch R, Tarrant J, Li Y, Monahan E, Miesbach W. Durability of bleeding protection and Factor IX activity in individuals with and without adeno-associated virus serotype 5 neutralising antibodies (Titres <1:700) in the phase 3 HOPE-B trial of etranacogene dezaparvovec gene therapy for haemophilia B. Hämostaseologie 2023, 43: s45-s45. DOI: 10.1055/s-0042-1760524.Peer-Reviewed Original Research
This site is protected by hCaptcha and its Privacy Policy and Terms of Service apply