Caplacizumab therapy for relapsed immune thrombotic thrombocytopenic purpura

Le résumé fourni par la source

Thrombotic thrombocytopenic purpura (TTP), a thrombotic microangiopathy, is characterized by a triad of symptoms: non-immune hemolytic anemia, profound thrombocytopenia, and ischemic organ damage. TTP is caused by a deficiency of the plasma metalloproteinase ADAMTS13, which is responsible for the breakdown of ultra-large von Willebrand factor multimers (UL-VWF MM). In individuals with a deficiency of this enzyme, UL-VWF accumulates and binds to platelets, forming clots that block blood flow in capillaries and small arterioles. Recently, caplacizumab has been introduced in the treatment of immune thrombotic thrombocytopenic purpura (iTTP). It is a nanobody that binds to the A1 domain of von Willebrand factor (vWF) and blocks interactions with glycoprotein Ib-IX-V (GPIb-IX-V), thereby preventing platelet agglutination typical of TTP, which causes disease symptoms, and consequently improving the prognosis of patients undergoing this therapy.

Ce résumé expose les affirmations des auteurs. BNTIC ne l’interprète pas comme une validation indépendante des résultats.