OC 52.3 Efficacy and Safety of Fidanacogene Elaparvovec in Adults with Moderately Severe or Severe Hemophilia B: Results from the Phase 3 BENEGENE-2 Gene Therapy Trial
Makaleler > Tam Makale · 2023
Yazarlar: A. Cuker, H. Alzahrani, J. Astermark, L. Frenzel, O. Katsarou, K. Kavakli, R. Klamroth, D. Matino, A. Shapiro, J. Wang, J. Fuiman, J. McKay, P. Sun, J. Rupon
Research and Practice in Thrombosis and Haemostasis, Uluslararası, Wos, 2023, cilt 7, ss 100452-100452
DOI: 10.1016/j.rpth.2023.100452
Özet
insomnia and energy-mania in negative terms (4/6 and 3/6 respectively); others found them positive (2/6 and 3/6 respectively).7 described steroids as the most difficult part of having GT; 2 reported no adverse effects.Positively, 4 reported improved joint pain while taking steroids.Conclusion(s): Over half of PwH described steroids as the worst part of GT, impacting themselves and their families due to adverse side effects.Ensuring these impacts are understood, identified quickly, and dealt with appropriately and effectively is essential if PwH are to obtain the maximum benefit from GT.There remains a need for studies of less toxic approaches to immunosuppression.