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Autosomal-dominante polyzystische Nierenerkrankung

Neue therapeutische Ansätze

Autosomal dominant polycystic kidney disease

Novel therapeutic options

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Zusammenfassung

Die autosomal-dominante polyzystische Nierenerkrankung („autosomal dominant polycystic kidney disease“, ADPKD) ist die häufigste genetische Nierenerkrankung, sie betrifft weltweit etwa 6 Mio. Menschen. Die Erkrankung ist durch eine progressive Entwicklung unzähliger Nierenzysten charakterisiert, die das normale Nierengewebe sukzessive verdrängen, was letztlich zum Verlust der Nierenfunktion ab der vierten Lebensdekade führen kann. Aktuell gibt es keine kausale Therapie, welche die Progression verlangsamen oder stoppen würde. In Tiermodellen wurde eine Vielzahl potenzieller Therapien getestet: Vasopressinrezeptor-2-Antagonisten (VPV2RA), mTOR („mammalian target of rapamycin“)-Inhibitoren, Somatostatin und -analoga sowie Inhibitoren von CFTR, B-Raf, MEK, Src, Tumornekrosefaktor-α und Cyclinen. In klinischen Studien wird derzeit die Wirksamkeit von VPV2RA, mTORi, Somatostatinanaloga und Renin-Angiotensin-Aldosteron-Inhibitoren getestet. Es besteht Hoffnung, dass diese schwere Erkrankung künftig kausal therapiert werden kann.

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most prevalent human genetic kidney disease, affecting about 6 million people worldwide. It is characterized by progressive development of innumerable renal cysts that gradually replace normal kidney tissue, leading ultimately to the loss of renal function starting from the fourth decade of life. There is currently no causal therapy that slows or stops ADPKD progression. In animal models of polycystic kidney disease, a variety of potential therapies have been tested: vasopressin receptor-2 antagonists (VPV2RA), mammalian target of rapamycin inhibitors (mTORi), somatostatin and its analogs, inhibitors of CFTR, B-Raf, MEK, Src, tumor necrosis factor alpha, and cyclins. Currently the efficacy of VPV2RA, mTORi, somatostatin analogs, and inhibitors of the renin angiotensin aldosterone system is being clinically tested. It is hoped that this serious disease can be treated causally in the coming years.

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Authors and Affiliations

  1. Klinik für Nephrologie, Universitätsspital, Rämistr. 100, 8091, Zürich, Schweiz

    A.L. Serra, D. Poster & R.P. Wüthrich

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  1. A.L. Serra
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  2. D. Poster
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  3. R.P. Wüthrich
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Correspondence to A.L. Serra.

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Serra, A., Poster, D. & Wüthrich, R. Autosomal-dominante polyzystische Nierenerkrankung. Nephrologe 5, 404–411 (2010). https://doi.org/10.1007/s11560-010-0421-6

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  • DOI: https://doi.org/10.1007/s11560-010-0421-6

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