Promising therapy for inherited form of kidney disease identified
Mayo Clinic researchers have found a potential drug therapy for treating an inherited form of kidney disease called autosomal dominant polycystic kidney disease (ADPKD).
The medication, tolvaptan, slowed the pace of kidney cyst growth over the three years of the study.
The multicenter study found tolvaptan demonstrated a nearly 50 percent 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.
"ADPKD is the most common inherited and the fourth most common overall cause of kidney failure worldwide," said lead author Vicente Torres, M.D., Ph.D., 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.
"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," Dr. Torres added.
While the trial findings are encouraging, tolvaptan has not yet been approved for this indication, Dr. Torres noted.
The phase three clinical trial results were being presented today at the American Society of Nephrology annual meeting and published online in the New England Journal of Medicine.