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Hamostaseologie 2017; 37(03): 211-215
DOI: 10.5482/HAMO-16-07-0026
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Schattauer GmbH

New treatment options for thrombotic thrombocytopenic purpura

Neue Therapieoptionen bei thrombotisch thrombozytopenischer Purpura
Paul Knöbl
1   Department of Medicine 1, Division of Hematology and Hemostasis, Medical University of Vienna, Vienna, Austria
› Author Affiliations
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Publication History

received: 19 July 2016

accepted in revised form: 04 November 2016

Publication Date:
28 December 2017 (online)

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Summary

The thrombotic-thrombocytopenic purpura (TTP) is an acute, life-threatening disease, characterised by enhanced platelet aggregation, disturbed microcirculation and organ dysfunction. With the currently available treatment (plasma exchange, infusions, corticosteroids) mortality ist still as high as 10–15 %. Recent, pathophysiology-based developments may improve the outcome. The most promising candidates for future treatment of TTP are: rituximab for termination of the autoimmune process, caplacizumab for prevention of platelet-VWF-interaction, and recombinant ADAMTS13 for replacement of the inhibited or missing enzyme.

Zusammenfassung

Die thrombotisch-thrombozytopenische Purpura (TTP) ist eine akute, lebensbedrohliche Erkrankung, bei der durch eine vermehrte Thrombozytenaggregation die Mikrozirkulation und Organfunktion gestört ist. Mit den zur Zeit verfügbaren Behandlungsoptionen (Plasma-Austausch, Plasma-Infusionen, Steroide) liegt die Mortalität immer noch bei 10–15 %. Neuere Pathophysiologie-basierte Entwicklungen könnten die Überlebensrate verbessern. Die vielversprechendsten Kandidaten für die zukünftige Behandlung der TTP sind: Rituximab zur Beendigung des Autoimmunprozesses, Caplazizumab zur Verhinderung der Thrombozyten-VWF-Interaktion und rekombinantes ADAMTS13 zur Zufuhr des blockierten oder fehlenden Enzyms.

Keywords

Thrombotic-thrombocytopenic purpura - ADAMTS13 - von Willebrand factor - therapy

Schlüsselwörter

Thrombotisch-thrmbozytopenische Purpura - ADAMTS13 - von-Willebrand-Faktor - Therapie
 

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