Rapamycin: eine neue Therapieoption bei autosomal dominanter polyzystischer Nierenerkrankung (ADPKD)?

Praxis ◽  
2009 ◽  
Vol 98 (25) ◽  
pp. 1511-1516
Author(s):  
Serra ◽  
Wüthrich
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
Mammalian Target Of Rapamycin ◽  
Mtor Inhibitors ◽  
In Utero ◽  
Polycystic Kidney ◽  
Kausale Therapie ◽  
Polyzystische Nierenerkrankung ◽  
Autosomal Dominant Polycystic Kidney

Die autosomal dominante polyzystische Nierenerkrankung (autosomal dominant polycystic kidney disease, ADPKD) ist charakterisiert durch eine massive Vergrösserung beider Nieren, bedingt durch unzählige Zysten. Die Zystenbildung beginnt bereits in utero und das kontinuierliche Zystenwachstum führt zur Kompression und Zerstörung des nicht-zystischen Nierenparenchyms, sodass schliesslich ein Nierenersatz in der 5. bis 6. Lebensdekade notwendig wird. Bisher gab es keine kausale Therapie, welche das Fortschreiten der Krankheiten aufhält. Tierexperimentelle Daten zeigen, dass die medikamentöse Inhibition eines zentralen Regulators der Zellproliferation, dem so genannten «mammalian target of rapamycin» (mTOR), den Kranheitsverlauf der ADPKD verlangsamen kann. Die vorliegende Übersicht vermittelt einen Einblick in die Erkrankung und in die neue therapeutische Möglichkeit des mTOR Inhibitors Sirolimus, welcher zurzeit in klinischen Studien getestet wird.

scholarly journals P04.03: In utero diagnosis of autosomal dominant polycystic kidney disease

2019 ◽  
Vol 54 (S1) ◽  
pp. 165-165
Author(s):  
M. Cal ◽  
I. Godinho ◽  
M. Soeiro e Sá ◽  
M. Cunha ◽  
R. Carvalho
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
In Utero ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney

scholarly journals Treatment of autosomal recessive and autosomal dominant polycystic kidney disease

2019 ◽  
Vol 64 (2) ◽  
pp. 22-29
Author(s):  
E. F. Andreeva ◽  
N. D. Savenkova
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Recessive ◽  
Autosomal Dominant ◽  
Kinase Inhibitor ◽  
Mtor Inhibitors ◽  
Somatostatin Analogues ◽  
Polycystic Kidney ◽  
V2 Receptor ◽  
Autosomal Dominant Polycystic Kidney

The article reflects the genetic variants of polycystic kidney disease, describes the modern strategy for the treatment of polycystic kidney disease in children and adults. The authors present the results of clinical trials of vasopressin V2 receptor antagonists (tolvaptan, liksivaptan), a multi-kinase inhibitor (tezevatinib), somatostatin analogues (lankreotide, octreotide), statins (pravastatin), mTOR inhibitors (everolimus, sirolimus), metformin in patients with autosomal recessive and autosomal polycystic kidney disease. The authors discuss the factors determining the prognosis and outcome of these diseases.

Pulse mTOR inhibitor treatment effectively controls cyst growth but leads to severe parenchymal and glomerular hypertrophy in rat polycystic kidney disease

2009 ◽  
Vol 297 (6) ◽  
pp. F1597-F1605 ◽  
Author(s):  
Ming Wu ◽  
Alexandre Arcaro ◽  
Zsuzsanna Varga ◽  
Alexander Vogetseder ◽  
Michel Le Hir ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Kidney Function ◽  
Mtor Inhibitor ◽  
Autosomal Dominant ◽  
Mtor Inhibitors ◽  
Glomerular Hypertrophy ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney ◽  
Inhibitor Treatment

The efficacy of mammalian target of rapamycin (mTOR) inhibitors is currently tested in patients affected by autosomal dominant polycystic kidney disease. Treatment with mTOR inhibitors has been associated with numerous side effects. However, the renal-specific effect of mTOR inhibitor treatment cessation in polycystic kidney disease is currently unknown. Therefore, we compared pulse and continuous everolimus treatment in Han:SPRD rats. Four-week-old male heterozygous polycystic and wild-type rats were administered everolimus or vehicle by gavage feeding for 5 wk, followed by 7 wk without treatment, or continuously for 12 wk. Cessation of everolimus did not result in the appearance of renal cysts up to 7 wk postwithdrawal despite the reemergence of S6 kinase activity coupled with an overall increase in cell proliferation. Pulse everolimus treatment resulted in striking noncystic renal parenchymal enlargement and glomerular hypertrophy that was not associated with compromised kidney function. Both treatment regimens ameliorated kidney function, preserved the glomerular-tubular connection, and reduced proteinuria. Pulse treatment at an early age delays cyst development but leads to striking glomerular and parenchymal hypertrophy. Our data might have an impact when long-term treatment using mTOR inhibitors in patients with autosomal dominant polycystic kidney disease is being considered.

scholarly journals A Rare Case of Autosomal Dominant Polycystic Kidney Disease Presenting in utero

2011 ◽  
Vol 3 (1) ◽  
pp. 44-45
Author(s):  
Hema Dhumale ◽  
Yeshita Pujar ◽  
Bhavana Sherigar ◽  
Babasaheb Raosaheb Desai ◽  
Geeta Durdi ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Rare Case ◽  
Autosomal Dominant ◽  
Mutant Gene ◽  
In Utero ◽  
Polycystic Kidney ◽  
Adult Polycystic Kidney Disease ◽  
Rare Presentation ◽  
Autosomal Dominant Polycystic Kidney

ABSTRACT Autosomal dominant polycystic kidney disease (ADPKD) is the most widespread cause of genetic nephropathy. Only 25% of patients are symptomatic.1 One in 1,000 people carries the autosomal dominant polycystic kidney disease mutant gene. Autosomal dominant polycystic kidney disease is usually asymptomatic until the third or fourth decade of life, although histological evidence of the disease is likely to be present from intrauterine life. Rarely, however, kidneys that are anatomically similar may cause death in infancy or early childhood, and the condition has been designated as “adult variety occurring in infancy”.2 In ADPKD, cysts develop only later in adulthood yet exceptionally cysts may be encountered in utero. We report a rare case of ADPKD, which was diagnosed by ultrasonography in utero as infantile polycystic kidney disease due to the presence of bilateral enlarged hyperechogenic kidneys with oligohydramnios. Pregnancy was terminated and autopsy revealed it to be an adult polycystic kidney disease. This case is reported due to its rare presentation in utero.

scholarly journals Autosomal dominant polycystic kidney disease diagnosed in utero. Review

2016 ◽  
Vol 87 (8) ◽  
pp. 605-608
Author(s):  
Magdalena Nowak ◽  
Hubert Huras ◽  
Marcin Wiecheć ◽  
Robert Jach ◽  
Małgorzata Radoń-Pokracka ◽  
...  
Keyword(s):  
Kidney Disease ◽  
Polycystic Kidney Disease ◽  
Autosomal Dominant ◽  
In Utero ◽  
Polycystic Kidney ◽  
Autosomal Dominant Polycystic Kidney
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