International workshop complement system: rare diseases and targeted therapies second edition

The renin-angiotensin with its pro and anti-inflammatory effects, thecomplement system with c1 inhibitor that inhibits components of thekallikrein-kinin system (kks), such as activated fxii, fxi and plasmakallikrein, as well plasmin affecting the fibrinolytic system; thecoagulation and the kks, tightly interlinked with the contact system,which is integral to the intrinsic pathway of coagulation (fxi) andthrombus formation; each of them consist of a large number ofdistinct plasma and membrane bound proteins and receptors that canbe activated through proteolytic cascades.

They have potent pro-inflammatory and prothrombotic effects and theyincrease vascular permeability, leading to edema. Although thedysregulation of each protein system is involved in the pathogenesisof common disorders, only a few rare diseases such as hereditaryangioedema, paroxysmal nocturnal hemoglobinuria, atypical-uremichemolytic syndrome, c3 glomerulopathies, rare coagulopathies, areapproved for clinical treatment targeting renin-angiotensin,complement, coagulation and kks. the diagnosis and the treatment of these rare diseases are a challenge for health system because it is necessary to improve basic and clinical knowledge andmanagement skills in order to offer the best response to patient’s needs.

In our workshop we would love to discuss the role of these complex plasma protein systems in disease pathogenesis and then share future pharmacological strategies to treat rare and common diseases with plasma contact factors and complement targeted therapies.