Contents
Therapies for Hemophilia: What's Available?
Published: Mar 22, 2024
Treating hemophilia effectively is essential for preventing complications and improving quality of life. This article explores available therapies for managing hemophilia.
Factor Replacement Therapy
Factor replacement therapy involves infusing the missing clotting factor into the bloodstream. This therapy is the cornerstone of hemophilia treatment and can be done on-demand in response to bleeding episodes or prophylactically to prevent bleeds. Regular infusions help maintain sufficient clotting factor levels and reduce the risk of complications.
Gene Therapy
Gene therapy is an emerging treatment that aims to provide a long-term solution by introducing a functional copy of the defective gene. This approach is still in clinical trials but shows promise in reducing or eliminating the need for regular factor infusions. Gene therapy represents a potential breakthrough in hemophilia management.
Managing Inhibitors
Some patients develop inhibitors, which are antibodies that neutralize the infused clotting factor. Managing inhibitors involves using bypassing agents or immune tolerance therapy to overcome this challenge. Early detection and treatment of inhibitors are crucial for maintaining effective hemophilia management.
Frequently Asked Questions
It involves infusing the missing clotting factor to prevent bleeding.
Gene therapy introduces a functional gene to reduce the need for infusions.
Inhibitors are antibodies that neutralize infused clotting factors.
Key Takeaways
Advancements in therapies offer hope for improved hemophilia management.
References
- Blanchette VS, et al. Definitions in hemophilia: communication from the SSC of the ISTH. J Thromb Haemost 2014; 12:1935.
- White GC 2nd, et al. Definitions in hemophilia. Thromb Haemost 2001; 85:560.
- Franchini M, et al. Mild hemophilia A. J Thromb Haemost 2010; 8:421.
This article has been reviewed for accuracy by one of the licensed medical doctors working for Doctronic. Always discuss health information with your healthcare provider.
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