Einfluss der ACE-Hemmer-Therapie auf das Fortschreiten der Niereninsuffizienz bei Patienten mit Alport-Syndrom

The Alport Syndrome (AS) is a hereditary progressive kidney disease. Genetic mutations lead to structural and functional defects of the Type-IV collagen. This collagen is an essential part of the glomerular basement membrane (GBM) of the kidney. The genetic mutations lead to an altered structure of the Type-IV collagen within the GBM. As a result, the actual filtration barrier for proteins is destroyed. The resulting chronic inflammation and following fibrosis of the kidney lead to a total loss of the nephritic function. The inevitable consequences for the patients are terminal kidney insufficiency, necessitating dialysis or kidney transplantation. Most of the affected patients get to this final stage before the age of 30. Cardinal symptoms of AS are hematuria and proteinuria, both of which are likely to start occurring during infancy. As of yet, there is no remedy to AS. Tests on animals have shown that an ACE-inhibitor therapy is quite effective in delaying the nephritic insufficiency of AS. In order to ascertain whether an ACE-inhibitor therapy can actually delay a kidney insufficiency a standardized questionnaire was sent to various infant kidney wards in Germany. 11 of those wards supported this project. The patients were interviewed according to the questionnaires by the attending physicians of the individual wards. In order to determine the course of the disease, information about clinical symptoms, kidney function parameters, probable necessity of dialysis or an already performed kidney transplant, and – first and foremost – the patient's age at the beginning of the terminal kidney insufficiency were gathered. Patients also gave detailed personal information, as well as data about their family's medical history and about their individual diagnosis. They also indicated their already applied therapies and treatments, and their age at the beginning of the respective treatment. Lastly, the patients were asked about any side effects of the therapy with ACE-inhibitors. The results of this investigation show that none of the 25 patients under treatment with ACE-inhibitors have reached the stage of terminal kidney failure, and that in average the proteinuria drops. It is to be presumed that an early treatment of AS with ACE-inhibitors at the patients' young age is effective. The patients did not experience any undesired side effects during the treatment. The positive tendency of this investigation about the effects of ACE-inhibitors on AS form a basis for a placebo-controlled randomized study.