Thrombotic Thrombocytopenic Purpura: When Basic Science Meets Clinical Research

Author:

Bécel Gaëlle12,Faict Sylvia12,Picod Adrien12,Bouzid Raïda1,Veyradier Agnès134,Coppo Paul125

Affiliation:

1. Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

2. Service d'hématologie, Hôpital Saint-Antoine, AP-HP – Sorbonne Université, Paris, France

3. Service d'Hématologie Biologique, Groupe Hospitalier Saint-Louis-Lariboisière, AP-HP, Paris, France

4. Université Paris-Diderot, Paris, France

5. Centre de Recherche des Cordeliers, INSERM, Sorbonne Université, USPC, Université Paris Descartes, Paris, France

Abstract

AbstractThe therapeutic landscape of thrombotic thrombocytopenic purpura (TTP) is rapidly changing with the recent availability of new targeted therapies. This progressive shift from empiricism to pathophysiology-based treatments reflects an intensive interaction between the continuous findings in the field of basic science and an efficient collaborative clinical research and represents a convincing example of the strength of translational medicine. Despite the rarity of TTP, national and international efforts could circumvent this limitation and shed light on the epidemiology, clinical presentation, prognosis, and long-term outcome of this disease. Importantly, they also provided high-quality results and practice changing studies for the benefit of patients. We report here the most recent therapeutic findings that allowed progressively improving the prognostic of TTP, both at the acute phase and through long-term outcome.

Publisher

Georg Thieme Verlag KG

Subject

Hematology

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