A Long Term Study of a Treatment for Autosomal Dominant Polycystic Kidney Disease Has Finished

According to a story from Medical Xpress, a long term study testing a treatment for autosomal dominant polycystic kidney disease (ADPKD) has recently concluded. The treatment in question is a vasopressin receptor antagonist called tolvaptan. ADPKD is considered the third leading cause of kidney failure worldwide. Overall, the results suggest that tolvaptan can slow the progression of ADPKD over the course of long term treatment with the medication.


ADPKD is a prevalent and potentially lethal monogenic disorder that may have a prevalence of as much as one in every 1,000 people worldwide. The disease is characterized by the development of cysts on the kidneys that can also appear in other parts of the body, such as the arachnoid membrane, the seminal vesicles, the pancreas, and the liver. It is also associated with hernias and heart defects. Often, problems do not appear until middle age. About half of patients eventually experience kidney failure. Symptoms include anemia, hypertension, acute loin pain, haematuria, and uremia. The development of cysts results in the expansion and enlargement of the kidneys. Patients who experience kidney failure will have to use dialysis or seek a kidney transplant. To learn more about ADPKD, click here.

The Study

The results of the study indicate that tolvaptan can delay the progression of ADPKD into end-stage kidney disease, and, ultimately, kidney failure. The drug can reduce the pace of the increase in kidney volume experienced with ADPKD, and also help alleviate pain associated with the disease. The research actually involved two studies, a one year long trial and a three year long trial. However, all of the 97 patients involved were permitted to continue using tolvaptan after these studies had concluded. Therefore, data has been gathered from a period of around 11 years.


While tolvaptan’s ability to slow kidney volume begins to disappear after an average of three years, the drug can still help delay disease progression beyond that time frame. Overall, the benefit of the medication appears to accumulate over time. The study was lead by Dr. Vicent Torres of the Mayo Clinic. Tolvaptan was first approved for use in Japan in 2014, followed by Europe the next year.

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