Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B
| العنوان: | Gene Therapy with Etranacogene Dezaparvovec for Hemophilia B |
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| المؤلفون: | Steven W. Pipe, Frank W.G. Leebeek, Michael Recht, Nigel S. Key, Giancarlo Castaman, Wolfgang Miesbach, Susan Lattimore, Kathelijne Peerlinck, Paul Van der Valk, Michiel Coppens, Peter Kampmann, Karina Meijer, Niamh O’Connell, K. John Pasi, Daniel P. Hart, Rashid Kazmi, Jan Astermark, Cedric R.J.R. Hermans, Robert Klamroth, Richard Lemons, Nathan Visweshwar, Annette von Drygalski, Guy Young, Shelley E. Crary, Miguel Escobar, Esteban Gomez, Rebecca Kruse-Jarres, Doris V. Quon, Emily Symington, Michael Wang, Allison P. Wheeler, Robert Gut, Ying P. Liu, Ricardo E. Dolmetsch, David L. Cooper, Yanyan Li, Brahm Goldstein, Paul E. Monahan |
| المساهمون: | Vascular Medicine, ACS - Pulmonary hypertension & thrombosis, ACS - Amsterdam Cardiovascular Sciences, Real World Studies in PharmacoEpidemiology, -Genetics, -Economics and -Therapy (PEGET), Hematology |
| المصدر: | New England journal of medicine, 388(8), 706-718. Massachussetts Medical Society New England Journal of Medicine, 388(8), 706-718. MASSACHUSETTS MEDICAL SOC New England Journal of Medicine, 388(8), 706-718. Massachussetts Medical Society |
| سنة النشر: | 2023 |
| مصطلحات موضوعية: | Coagulation, Genetics, General Medicine, Genetics General, Childhood Diseases, Hematology/Oncology, Pediatrics |
| الوصف: | Background: Moderate-to-severe hemophilia B is treated with lifelong, continuous coagulation factor IX replacement to prevent bleeding. Gene therapy for hemophilia B aims to establish sustained factor IX activity, thereby protecting against bleeding without burdensome factor IX replacement. Methods: In this open-label, phase 3 study, after a lead-in period (≥6 months) of factor IX prophylaxis, we administered one infusion of adeno-associated virus 5 (AAV5) vector expressing the Padua factor IX variant (etranacogene dezaparvovec; 2×1013 genome copies per kilogram of body weight) to 54 men with hemophilia B (factor IX activity ≤2% of the normal value) regardless of preexisting AAV5 neutralizing antibodies. The primary end point was the annualized bleeding rate, evaluated in a noninferiority analysis comparing the rate during months 7 through 18 after etranacogene dezaparvovec treatment with the rate during the lead-in period. Noninferiority of etranacogene dezaparvovec was defined as an upper limit of the two-sided 95% Wald confidence interval of the annualized bleeding rate ratio that was less than the noninferiority margin of 1.8. Superiority, additional efficacy measures, and safety were also assessed. Results: The annualized bleeding rate decreased from 4.19 (95% confidence interval [CI], 3.22 to 5.45) during the lead-in period to 1.51 (95% CI, 0.81 to 2.82) during months 7 through 18 after treatment, for a rate ratio of 0.36 (95% Wald CI, 0.20 to 0.64; P |
| اللغة: | English |
| تدمد: | 0028-4793 |
| URL الوصول: | https://explore.openaire.eu/search/publication?articleId=doi_dedup___::18ec00c53efc81770ed02e6372363e8b https://hdl.handle.net/11370/bcd8e7fb-66c2-4b0f-b235-d2995c4d444f |
| حقوق: | OPEN |
| رقم الأكسشن: | edsair.doi.dedup.....18ec00c53efc81770ed02e6372363e8b |
| قاعدة البيانات: | OpenAIRE |
| تدمد: | 00284793 |
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