Treatment of thrombotic microangiopathy with a focus on new treatment options

Summary

The thrombotic microangiopathies (TMA) are a heterogeneous group of disorders, characterized by microangiopathic haemolytic anaemia with red cell fragmentation, thrombocytopenia and signs of organ dysfunction due to disturbed microcirculation. Current laboratory methods can be used to better distinguish some of these entities. Organ dysfunction can be severe and life-threatening, and immediate start of sufficient therapy is necessary to avoid permanent damage or death. The therapeutic options, however, are often limited to symptomatic measures, and are not standardized or based on high scientific evidence. During the preceding years, not only considerable progress has been made in better diagnosis of TMA, but also new therapeutic strategies have been established. Initial treatment still is based on plasma exchange and symptomatic measures to protect organ function. New concepts (immunosuppression, targeted anti-von Willebrand factor or anti-complement therapy, replacement with recombinant enzymes) are discussed in this article.

Zusammenfassung

Die thrombotischen Mikroangiopathien sind eine heterogene Gruppe von Erkrankungen, die durch mikroangiopathische Hämolyse mit Erythrozytenfragmentation, Thrombozytopenie und Zeichen von Organschädigung durch Mikrozirkulationsstörungen charakterisiert sind. Moderne Labormethoden können die verschiedenen Entitäten besser unterscheiden. Die Organdysfunktion kann schwer und lebensbedrohlich sein, und die sofortige Einleitung einer suffizienten Therapie ist notwendig um permanente Schäden oder den Tod zu vermeiden. Die Behandlungsoptionen sind jedoch oft beschränkt auf symptomatische Maßnahmen, sind nicht standardisiert oder durch hohe wissenschaftliche Evidenz abgesichert. In den vergangenen Jahren konnten deutliche Fortschritte in der Diagnose und Behandlung der thrombotischen Mikroangiopathien erzielt werden. Etablierte Strategien, aber auch neue Behandlungsformen, die auf spezifische pathophysiologisch bedeutsame Mechanismen zielen, werden in diesem Artikel diskutiert.

Keywords

Thrombotic microangiopathy

Schlüsselwörter

Thrombotischen Mikroangiopathie

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