Mayo Clinic identifies promising treatment for inherited form of kidney disease

A drug therapy shows promise for treating an inherited form of kidney disease called autosomal dominant polycystic kidney disease (ADPKD), Mayo Clinic researchers say. The medication, tolvaptan, slowed the pace of kidney cyst growth over the three years of the study. 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.

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

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

More information: The study was funded by Otsuka Pharmaceuticals.

Vicente E. Torres, Arlene B. Chapman, Olivier Devuyst, Ron T. Gansevoort, Jared J. Grantham, Eiji Higashihara, Ronald D. Perrone, Holly B. Krasa, John Ouyang, and Frank S. Czerwiec. Tolvaptan in patients with autosomal dominant polycystic kidney disease. New England Journal of Medicine. November 3, 2012.

add to favorites email to friend print save as pdf

Related Stories

Gene mutations predict early, severe form of kidney disease

Oct 24, 2011

The most common kidney disease passed down through families, autosomal dominant polycystic kidney disease (ADPKD) affects one in 400 to 1,000 individuals and is characterized by cysts on the kidneys. The condition slowly ...

New drug target for kidney disease discovered

Apr 26, 2011

Two discoveries at UC Santa Barbara point to potential new drug therapies for patients with kidney disease. The findings are published in this week's issue of the Proceedings of the National Academy of Sciences.

Recommended for you

Senegal monitors contacts of 1st Ebola patient

3 hours ago

Senegalese authorities on Monday were monitoring everyone who was in contact with a student infected with Ebola who crossed into the country, and who has lost three family members to the disease.

Cerebral palsy may be hereditary

9 hours ago

Cerebral palsy is a neurological developmental disorder which follows an injury to the immature brain before, during or after birth. The resulting condition affects the child's ability to move and in some ...

19 new dengue cases in Japan, linked to Tokyo park

15 hours ago

Japan is urging local authorities to be on the lookout for further outbreaks of dengue fever, after confirming another 19 cases that were contracted at a popular local park in downtown Tokyo.

User comments