John Libbey Eurotext



Developments in thrombotic thrombocytopenic purpura and ADAMTS13 Ahead of print


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1 Service d’hématologie biologique, hôpital Lariboisière, AP-HP, Nord, Université de Paris, Paris, France
Sous l’égide du Centre national de référence des microangiopathies thrombotiques, hôpital Saint-Antoine, AP-HP, Sorbonne Université, Paris, France
2 EA3518, Recherche clinique en hématologie, immunologie et transplantation, institut de recherche Saint-Louis, Université de Paris, Paris, France
3 Normandie Univ, UNIROUEN, U1096, CHU Rouen, service de médecine interne, Rouen, France
4 Service d’hématologie, hôpital Saint-Antoine, AP-HP, Sorbonne Université, Paris, France
* Correspondence

Thrombotic thrombocytopenic purpura (TTP) is a specific form of thrombotic microangiopathy arising from a severe functional deficiency of ADAMTS13, the specific Von Willebrand factor (VWF) cleaving protease. This defect leads to the accumulation of hyperadhesive VWF high-molecular-weight multimers and to the spontaneous formation of microthrombi in the microcirculation, accompanied by peripheral thrombocytopenia and mechanical haemolytic anaemia. TTP is mainly an acquired disease, most often autoimmune (iTTP) but can also be inherited (cTTP) when deleterious bi-allelic mutations are found in the ADAMTS13 gene (Upshaw-Schulman syndrome). TTP is a relapsing-remitting disease requiring long-term follow-up. TTP is either idiopathic (50%) or associated with a physiological or pathological context such as pregnancy, autoimmune disease, infection, neoplasia or transplantation. TTP is a disease, the diagnosis and management of which have changed dramatically over the last 30 years, mainly due to breakthroughs in the understanding of the relationship between VWF and ADAMTS13. TTP is a therapeutic emergency and can be detected by clinical prediction scores (French Score, PLASMIC Score). However, formal diagnosis relies on the measurement of ADAMTS13 activity, which is