[Treatment of autosomal dominant polycystic kidney disease].
Autosomal dominant polycystic kidney disease is the most frequent hereditary kidney disease. However it lacks a specific treatment. Its prevalence is 1/800 and causes the need for renal replacement therapy in 8-10% of patients on dialysis or kidney transplant. It is caused by mutations in the PKD1 and PKD2 genes, which cause a series of alterations in the polycystic cells, which have become therapeutic targets. There are many molecules that are being tested to counteract the alterations of these therapeutic targets. There are studies in all phases of research, from phase i to phase iv. Some of the molecules being tested are tolvaptan, mTOR inhibitors and, among many other, somatostatin analogues. These drugs are extensively reviewed in this article. Based on the accumulated experience the primary objective of the trials is the slowing of the increase in renal volume. Yet other renal end points such as renal function and hypertension are necessary. It is expected that in the coming years we can have specific, well tolerated, effective and affordable drugs for the treatment of autosomal dominant polycystic kidney disease.
Enfermedades Renales Hereditarias, Fundación Puigvert, Institut d'Investigació Biomèdica Sant Pau, Universidad Autónoma de Barcelona, Barcelona, España. Electronic address: rtorra@fundacio-puigvert.es.
Roser
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Torra
D000328:Adult; D000368:Aged; D000818:Animals; D001552:Benzazepines / Q000627:therapeutic use; D004195:Disease Models, Animal; D005260:Female; D005919:Glomerular Filtration Rate; D006801:Humans; D006977:Hypertension, Renal / Q000209:etiology / Q000503:physiopathology; D007668:Kidney / Q000473:pathology; D008297:Male; D008875:Middle Aged; D008954:Models, Biological; D058990:Molecular Targeted Therapy; D009929:Organ Size; D016891:Polycystic Kidney, Autosomal Dominant / Q000453:epidemiology / Q000235:genetics / Q000628:therapy*; D017582:Renal Replacement Therapy; D012084:Renin-Angiotensin System / Q000187:drug effects / Q000502:physiology; D015398:Signal Transduction / Q000502:physiology; D013004:Somatostatin / Q000031:analogs & derivatives / Q000627:therapeutic use; D058570:TOR Serine-Threonine Kinases / Q000037:antagonists & inhibitors; D050396:TRPP Cation Channels / Q000235:genetics; D000077602:Tolvaptan; D055815:Young Adult
D001552:Benzazepines; D013004:Somatostatin; D058570:TOR Serine-Threonine Kinases; D050396:TRPP Cation Channels; D000077602:Tolvaptan; C546842:MTOR protein, human
Autosomal dominant polycystic kidney disease; Pathogenesis; Patogénesis; Poliquistosis renal autosómica dominante; Tratamiento; Treatment