• Adrien Picod and Paul Coppo.
• Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France.
• Transfus Apher Sci. 2019 Jun 1; 58 (3): 273-277.

AbstractThrombotic thrombocytopenic purpura is a rare and severe disease that manifests as a thrombotic microangiopathy with severe thrombocytopenia and variable multiorgan failure. The disease relies on a severe deficiency in a disintegrin and metalloprotease with thrombospondin type-1 repeats, 13th member (ADAMTS13), the von Willebrand factor (vWF) cleaving protease which can be either inherited (congenital TTP) or immune-mediated (iTTP). In iTTP, the therapeutic strategy has long relied on therapeutic plasma exchange alone which still represents the only way to deliver large amounts of ADAMTS13 without risking fluid overload. Yet, several therapeutic strategies have been developed in recent years and are about to transform the standard of care of iTTP. The immunosuppressive regimen now increasingly encompasses the administration of frontline rituximab to all patients. Moreover, the impressive results of the anti-vWF nanobody caplacizumab in phase 2 and 3 studies have recently prompted its approval by health authorities for the initial treatment of the disease. The increasing use of these highly effective targeted therapies should translate in a reduced need for therapeutic plasma exchange and an improvement in the prognosis of the disease. Nevertheless, and until the development of a recombinant ADAMTS13, this cornerstone therapy remains irreplaceable.Copyright © 2019. Published by Elsevier Ltd.

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