Zusammenfassung
Familiäre zystische Nierenerkrankungen bilden eine heterogene Gruppe von seltenen Erkrankungen. Basierend auf molekularbiologischen Erkenntnissen wurden bereits zugelassene Medikamente kürzlich neu für die Indikationen autosomal-dominante polyzystische Nierenerkrankung (ADPKD) und tuberöse Sklerose geprüft. Da bei der ADPKD die Nierenfunktion über Jahrzehnte stabil ist, wird die Therapieeffizienz mittels Nierengröße als Surrogatmarker der Krankheitslast bestimmt. Tolvaptan, ein Aquaretikum, verminderte in einer 3-jährigen Studie mit 1445 ADPKD-Patienten das Größenwachstum der Nieren, den Nierenfunktionsverlust und die Nierenschmerzen. Die langwirksamen Somatostatine Octreotid und Lanreotid verminderten moderat die Leber- und die Nierengröße von Patienten mit ADPKD und polyzystischer Lebererkrankung. Die mTOR-Inhibitoren Sirolimus und Everolimus waren für die Indikation ADPKD nutzlos, während sie mit tuberöser Sklerose assoziierte renale Angiomyolipome schrumpfen ließen. Die Gesundheitsbehörden werden noch 2013 über die Zulassung von Tolvaptan für die Indikation ADPKD und über Everolimus für die Indikation tuberöse Sklerose entscheiden.
Abstract
Inherited cystic kidney diseases are a heterogeneous group of rare diseases. Based on molecular biological findings approved drugs have already been recently tested for the new indications for autosomal dominant polycystic kidney disease (ADPKD) and tuberous sclerosis complex. For ADPKD it is important to note that renal function remains stable for decades so the effectiveness of treatment is determined by renal size as a surrogate marker of disease burden. In a 3-year study of 1,445 ADPKD patients tolvaptan, an aquaretic agent, decreased the growth of kidneys, renal function loss and kidney pain. The long-acting somatostatins octreotide and lanreotide moderately decreased liver and kidney size in patients with ADPKD and polycystic liver disease. The mTOR inhibitors sirolimus and everolimus were ineffective for the indications of ADPKD while these drugs shrunk the renal angiomyolipomas associated with tuberous sclerosis. Health authorities will decide this year on the approval of tolvaptan for ADPKD and of everolimus for the indications for tuberous sclerosis complex.
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Einhaltung der ethischen Richtlinien
Interessenkonflikt. A.L. Serra ist als Berater für die Firma Otsuka und Novartis tätig. K. Petzold gibt an, dass kein Interessenkonflikt besteht. Dieser Beitrag beinhaltet keine Studien an Menschen oder Tieren.
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Serra, A., Petzold, K. Aktuelle Therapiestudien für die Indikation zystischer Nierenerkrankungen. Nephrologe 8, 396–405 (2013). https://doi.org/10.1007/s11560-012-0733-9
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DOI: https://doi.org/10.1007/s11560-012-0733-9
Schlüsselwörter
- Autosomal-dominante polyzystische Nierenerkrankung
- Tuberöse Sklerose
- Tolvaptan
- Mammalian target of rapamycin
- Somatostatin