Protease inhibitors in the treatment of hereditary angioedema.
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 epsilon-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.
Ritchie
Bruce C
BC
4-71 MSB, University of Alberta, AB, Edmonton, Canada. bruce.ritchie@ualberta.ca
D015119:Aminocaproic Acid / Q000627:therapeutic use; D000799:Angioedema / Q000188:drug therapy* / Q000235:genetics*; D000933:Antifibrinolytic Agents / Q000627:therapeutic use; D007611:Aprotinin / Q000627:therapeutic use; D002986:Clinical Trials as Topic; D003174:Complement C1 Inactivator Proteins / Q000627:therapeutic use; D006801:Humans; D011480:Protease Inhibitors / Q000627:therapeutic use*; D014148:Tranexamic Acid / Q000627:therapeutic use
D000933:Antifibrinolytic Agents; D003174:Complement C1 Inactivator Proteins; D011480:Protease Inhibitors; D014148:Tranexamic Acid; D007611:Aprotinin; D015119:Aminocaproic Acid