Tolvaptan: A New Weapon for a Tough Kidney Disease

Bret Stetka, MD; Neil Turner


February 14, 2013

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Hello. I'm Bret Stetka, Editorial Director at Medscape. Welcome to the F1000 Practice-Changing Minute, where we report commentaries from the Faculty of 1000 on highly rated studies.

Our commentary today covers the study "Tolvaptan in Patients With Autosomal Dominant Polycystic Kidney Disease," from Dr. V. Torres and colleagues published in the New England Journal of Medicine.[1] The F1000 commentator has given this a ranking of Changes Clinical Practice, with the conclusion that, based on these results, tolvaptan should be prescribed to slow the progression of autosomal dominant polycystic kidney disease (ADPKD).

The following F1000 commentary on this study was written by Neil Turner, Professor of Nephrology, Department of Renal Medicine, University of Edinburgh, Edinburgh, United Kingdom.

In his commentary on this study, Professor Turner wrote:

"The vasopressin V2 antagonist tolvaptan is shown in this double-blind randomised controlled trial (RCT) to slow the progression of autosomal dominant polycystic kidney disease (ADPKD). This 3-year, multicentre, multinational study of 1445 patients showed a halving of the rate of kidney volume expansion and a 30% slowing of deterioration of glomerular filtration rate (GFR).

The chief side effect was polyuria, which a significant minority of patients found intolerable. The next most important questions become whether this effect can last over decades and who to target to achieve the most from treatment. The rate of deterioration of placebo-treated patients was somewhat lower than expected -- the authors speculate this may be because they were encouraged to drink, so lowering their antidiuretic hormone (ADH) production and producing a comparable effect that way. It seems certain that this result will change clinical practice, if not immediately. As PKD causes 10% of end stage renal disease (ESRD), the ability to delay or prevent ESRD in this group of patients is an important result."

This concludes today's commentary from Neil Turner for the F1000 Practice-Changing Minute. I am Bret Stetka. Thank you for listening.