HTRS2025.P2.54 Corticosteroid Experience With Fidanacogene Elaparvovec in Adults With Moderately Severe or Severe Hemophilia B: Results From the Phase 3 BENEGENE-2 Gene Therapy Trial
Makaleler > Tam Makale · 2025
Yazarlar: Adam Cuker, Madison Snyder, Ramazan Kaan KAVAKLI, Robert Klamroth, Lisa J. Wilcox, Delphine Agathon, Pascal Klaus, Pengling Sun, Frank Plonski
Research and Practice in Thrombosis and Haemostasis, Uluslararası, Wos, 2025, cilt 9, ss 103074-103074
DOI: 10.1016/j.rpth.2025.103074
Özet
Background: Adeno-associated virus (AAV)-based gene therapy may stimulate a cellular immune response against capsid-presenting hepatocytes, which may cause a rise in transaminases and/or decrease in circulating factor IX (FIX:C). Corticosteroid administration generally controls the immune response. BENEGENE-2 is a pivotal phase 3 trial of fidanacogene elaparvovec, an AAV-based hemophilia B gene therapy.