Expertenstellungnahme zur Substitutionstherapie bei Patienten mit Alpha-1 Antitrypsin-Mangel^[*]

 

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Zusammenfassung

Die Substitutionstherapie bei Alpha-1 Antitrypsin-Mangel soll das Voranschreiten eines Lungenemphysems verzögern, die Exazerbationshäufigkeit reduzieren und die Lebensqualität der Patienten verbessern. Dieses Expertenstatement fasst die wichtigsten Therapiestudien zur Substitutionstherapie bei Patienten mit schwerem Alpha-1 Antitrypsin-Mangel und Lungenemphysem kurz zusammen. Aus den verfügbaren Studienergebnissen werden Indikationen und Kontra­indikationen für eine dauerhafte intravenöse Substitutionstherapie mit humanem Alpha-1 Antitrypsin abgeleitet. Sicherheitsaspekte und die Kontroversen um das Thema werden ausführlich diskutiert.

Abstract

Augmentation therapy in Alpha-1 Antitrypsin Deficiency aims to reduce the progression of lung emphysema, to reduce exacerbation frequency, and to improve quality of life. This expert statement briefly summarizes the most important treatment studies performed in patients with Alpha-1 Antitrypsin Deficiency and severe lung emphysema. Indications and contraindications for long-term intravenous augmentation therapy with human Alpha-1 Antitrypsin are derived from the available study results. Safety issues and the controversies of this topic are discussed in detail.

^* Diese Expertenstellungnahme wurde auf einem Treffen von Vertretern der Alpha-1 Center aus Deutschland und Österreich am 7. 12. 2013 entwickelt. Die vollständige Liste der Mitwirkenden befindet sich am Ende der Expertenstellungnahme.

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