Treatment for inherited form of kidney disease identified

Researchers from the Mayo Clinic researchers found that medication, tolvaptan, slowed the pace of kidney cyst growth over the three years of the study.

"ADPKD is the most common inherited and the fourth most common overall cause of kidney failure worldwide," said lead author Vicente Torres, Mayo Clinic nephrologist.

"In most patients with this disease, relentless cyst growth within the kidneys destroys the tissue, causes hypertension and painful complications, and negatively impacts the quality of life," Torres said.

The study found tolvaptan demonstrated a nearly 50 per cent reduction in the rate of increase in total kidney volume (a measurement of kidney cyst growth) in ADPKD patients over the study period, compared to placebo.

"The results of this study reveal a potential treatment that blunts kidney growth, lessens associated symptoms and slows kidney function decline when given over three years," Torres added.

While the trial findings are encouraging, tolvaptan has not yet been approved for this indication, Torres noted.

The phase three clinical trial results were presented at the American Society of Nephrology annual meeting and published in the New England Journal of Medicine.