Polycystic kidney disease, are there new treatment options? – WWN

I suffer from polycystic kidney disease: are there prospects for new therapies?

He replies Arrigo SchieppatiClinical Research Center for Rare Diseases, Mario Negri Institute, Bergamo (GO TO THE FORUM)

There autosomal dominant polycystic kidney diseasecommonly called polycystic kidney disease, the most frequent form of hereditary kidney disease. Over the course of life, the kidney is progressively deformed by the formation of cyst
. At the onset of the disease the cysts are few and small, but as time passes they increase in number and size and can end up occupying the entire kidney. These cysts occupy and destroy kidney tissuewhich gradually loses its function, to the point that it needs to be replaced with the dialysis. Experimental research and clinical studies have led to the development of new specific therapies for the disease. Between these tolvaptana vasopressin V2 receptor antagonist, e somatostatin analogues (octreotide-LAR and lanreotide acetate), drugs that inhibit a particular signal within the kidney cells, reducing or blocking the growth of cysts.

Studies

For now, tolvaptan is the only drug approved by international regulatory authorities in patients with polycystic kidney disease. Certainly one of the limits to the use of this drug lies in the marked increase in diuresis. Clinical studies conducted in our Institute (ALADIN and ALADIN 2) have documented that octreotide slows the deterioration of kidney function in patients with polycystic kidney disease in the early stages of the disease. Subsequent studies documented that three-year treatment with octreotide-LAR significantly slows cyst growth and progression tokidney failure at the end stage, in patients who had more severe renal failure at the start of treatment (the so-called stage 4).

The directions

The most frequent side effects of treatment with this drug (which is administered by subcutaneous injection) are diarrhea, formation of gallstones and gallstones. On the basis of these studies, the drug was admitted for reimbursement by the Italian Medicines Agency and can be prescribed to patients with polycystic kidney disease whose glomerular filtration rate is lower than 30 ml/min and higher than 15 ml/min, i.e. in patients with fairly advanced renal failure. Octreotide-LAR also appears to have a favorable effect on other organs: the drug reduces the growth of liver cysts and improves cardiac dysfunction.

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