Thrombotic Thrombocytopenic Purpura: When Basic Science Meets Clinical Research

Gaëlle Bécel; Sylvia Faict; Adrien Picod; Raïda Bouzid; Agnès Veyradier; Paul Coppo

doi:10.1055/a-1332-3066

Hämostaseologie, Inhaltsverzeichnis

Hamostaseologie 2021; 41(04): 283-293
DOI: 10.1055/a-1332-3066

Review Article

Thrombotic Thrombocytopenic Purpura: When Basic Science Meets Clinical Research

Gaëlle Bécel‡^*

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

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

,

Sylvia Faict‡^*

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

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

,

Adrien Picod

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

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

,

Raïda Bouzid

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

,

Agnès Veyradier

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

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

⁴Université Paris-Diderot, Paris, France

,

Paul Coppo

¹Centre de Référence des MicroAngiopathies Thrombotiques, Paris, France

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

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

› Institutsangaben

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.

Keywords

plasma exchange - thrombotic thrombocytopenic purpura - thrombotic microangiopathy - rituximab - caplacizumab - ADAMTS13

Volltext

Referenzen

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