Hamostaseologie
DOI: 10.1055/a-1332-3066
Review Article

Thrombotic Thrombocytopenic Purpura: When Basic Science Meets Clinical Research

Gaëlle Bécel*
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
2  Service d'hématologie, Hôpital Saint-Antoine, AP-HP – Sorbonne Université, Paris, France
,
Sylvia Faict*
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
2  Service d'hématologie, Hôpital Saint-Antoine, AP-HP – Sorbonne Université, Paris, France
,
Adrien Picod
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
2  Service d'hématologie, Hôpital Saint-Antoine, AP-HP – Sorbonne Université, Paris, France
,
Raïda Bouzid
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
,
Agnès Veyradier
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
3  Service d'Hématologie Biologique, Groupe Hospitalier Saint-Louis-Lariboisière, AP-HP, Paris, France
4  Université Paris-Diderot, Paris, France
,
Paul Coppo
1  Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France
2  Service d'hématologie, Hôpital Saint-Antoine, AP-HP – Sorbonne Université, Paris, France
5  Centre de Recherche des Cordeliers, INSERM, Sorbonne Université, USPC, Université Paris Descartes, Paris, France
› Author Affiliations

Abstract

The 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.

* Both the authors equally contributed to this work.




Publication History

Received: 18 June 2020

Accepted: 07 December 2020

Publication Date:
19 February 2021 (online)

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