Click for an illustrated outline in Hemophilia Today, “The Rebalanced Patient: New Therapies and New Thrombotic Risks in Hemophilia.”
Introduction
For decades, the central therapeutic principle in hemophilia was straightforward: replace what was missing. In hemophilia A, this meant replacing FVIII; in hemophilia B, FIX. Prophylactic factor replacement transformed hemophilia from a condition dominated by recurrent spontaneous bleeding and progressive hemophilic arthropathy into a chronic disorder that could increasingly be managed preventively. Yet factor replacement has never completely solved the therapeutic problem. Intravenous administration, pharmacokinetic variability, frequent infusions, venous-access difficulties, inhibitor development, and breakthrough bleeding have continued to limit treatment for some patients. These limitations helped drive the development of a fundamentally different approach: rather than replacing the missing factor, could hemostasis be restored by modifying other components of the coagulation system?
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