Hämophilie A und B

  • Wolfgang Muntean
  • Martin Köstenberger

Zusammenfassung

Die Hämophilie ist molekularbiologisch, biochemisch und in ihrer klinischen Manifestation ausgezeichnet erforscht; eine pränatale Diagnostik ist möglich. Für die Therapie stehen hochgereinigte Faktorenkonzentrate zur Verfügung. Faktor VIII- und IX sind die bisher größten gentechnologisch erzeugten Proteine zum therapeutischen Einsatz. Erste Gentransferstudien laufen. Die Therapie der Hämophilie bedarf aber eines sehr großen medizinischen, logistischen und finanziellen Aufwandes, und der Erfolg wurde in der Vergangenheit aufgehoben durch die Nebenwirkung der Übertragung transfusionsrelevanter Viren. Inhibitorentstehung und Inhibitorsuppressionstherapie bedürfen weiterer Studien.

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Copyright information

© Springer Medizin Verlag Heidelberg 2006

Authors and Affiliations

  • Wolfgang Muntean
    • 1
  • Martin Köstenberger
    • 2
  1. 1.Klinische Abteilung für Allgemeine PädiatrieUniversitätsklinik für Kinder- und JugendheilkundeGrazÖsterreich
  2. 2.Abt. Allgemeine PädiatrieLudwig Boltzmann Institut für pädiatrische Hämostaseologie Univ. Klinik für Kinder- und Jugendheilkunde, GrazGrazÖsterreich

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