Abstract
Background: Autosomal dominant polycystic disease (ADPKD) is characterized by the development of renal cysts. Objectives: 1) To assess the kidney and cyst volume growth in patients treated with Rapamycin compared to patients who receive standard ADPKD treatment. 2) Evaluate the occurrence of adverse effects related to the use of Rapamycin. 3) To assess the changes in blood pressure, proteinuria and estimated Glomerular Filtration rate (eGFR). Materials and Methods: During 24 months were randomized 12 patients with ADPKD in a Rapamycin group (6 patients, received rapamycin 2-3 mg/m2/day, max. 5mg/dia) and a Control group (standard treatment). Results: Of the 12 patients, 6 entered the Rapamycin group and had a total renal volume and cystic volume increase at study completion of 13% and 32% respectively. In the Control group (6 patients) the increases were 11% and 23% respectively. The eGFR was normal for both groups. The proteinuria for rapamycin group and control group was initially 7.3 mg/m²/hour and 6 mg/m2/hour respectively, at the end was normal for both groups. 3 patients had arterial hypertension, but at the 24th month, were normal. The adverse effects were: Anemia, diarrhea and oral sores. Conclusions: Rapamycin not decrease the kidney volume and cystic. There was no significant increase in proteinuria or eGFR decrease. Mean blood pressure remained normal.
Highlights
Autosomal dominant polycystic kidney disease is the most common hereditary kidney disorder, characterized by the progressive, bilateral development and enlargement of focal cysts
The proteinuria for rapamycin group and control group was initially 7.3 mg/m2/hour and 6 mg/m2/hour respectively, at the end was normal for both groups. 3 patients had arterial hypertension, but at the 24th month, were normal
There was no significant increase in proteinuria or eGFR decrease
Summary
Autosomal dominant polycystic kidney disease is the most common hereditary kidney disorder, characterized by the progressive, bilateral development and enlargement of focal cysts. Affected individuals usually present in the 3rd and 4th decade of life, progressing to end stage renal disease (ESRD) within 5–10 years after the development of renal insufficiency [1]. If not all, cases of ADPKD are caused by mutations in two genes, PKD1 (located on the short arm of chromosome 16) and PKD2 (located on the long arm of chromosome 4). 85% have mutations in PKD, which encodes for Polycystin, the rest have mutations in PKD2 which encodes for Polycystin 2. Autosomal dominant polycystic disease (ADPKD) is characterized by the development of renal cysts
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