Abstract
Deficiency of C1 Inhibitor leads to unopposed activation of complement, with localized,
unpredictable, and sometimes life-threatening attacks of angioedema. Treatment with
plasma-derived C1 Inhibitor rapidly aborts attacks, and may be lifesaving, but is
expensive, requires use of a pooled blood product, may need to be repeated and may
not be effective in autoantibody mediated angioedema. The antifibrinolytic agents
aprotinin, tranexamic acid, and ε-aminocaproic acid are useful for prophylaxis and
treatment of angioedema, likely by inhibiting plasmin. Specific drugs to replace the
deficient C1 Inh have not been reported. The kallikrein inhibitor DX-88® (Dyax) has received orphan drug status in Europe and is undergoing clinical trial
in Europe and the USA.
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