New Treatment in Haemophilia: Challenges, Controversies and Uncertainties - PubMed
4 hours ago
- #nonfactor therapy
- #AAV gene therapy
- #haemophilia treatment
- Non-factor therapies (e.g., emicizumab) and AAV gene therapy are emerging for haemophilia, offering long-term prophylaxis but introducing clinical management complexities.
- Key challenges include acute bleed management protocols, surgical considerations, transitioning between treatments, and managing thrombotic risks and immunogenicity for non-factor therapies.
- AAV gene therapy faces immunogenicity issues, particularly T-cell-mediated hepatotoxicity, requiring tailored corticosteroid immunomodulation strategies for optimal transgene expression.