is the light at the end of the tunnel nigh? a review of adpkd focusing on the burden of disease and tolvaptan as a new treatment

Rashid A Barnawi,1 Rahaf Z Attar,1 Sultan S Alfaer,1 Osama Y Safdar2 1Faculty of Medicine, King Abdulaziz University, Jeddah, Saudi Arabia; 2Pediatric Nephrology Center of Excellence, Faculty of Medicine, King Abdulaziz University, Jeddah, Saudi Arabia Abstract: Autosomal dominant polycystic kidney disease (ADPKD) causes pathological cystic changes to the kidney and is characterized by numerous renal and systemic manifestations. ADPKD is the fourth most common renal disease requiring renal replacement therapy. In this report, we present a detailed review of ADPKD, with a particular focus on its major economic, psychological, and social burden in affected patients. Treatment of this disease has been based on prophylactic and supportive measures. However, in recent years, new drugs have emerged as promising agents that may retard the progression of ADPKD, such as tolvaptan. In this report, we provide an in-depth discussion of tolvaptan, which has shown an effect in decreasing annual total kidney volume growth and renal function decline, thus slowing disease progression. The mechanism of action, side effects, and available data on cost-effectiveness are discussed together with the results of the first clinical trials and the most recent trials with regard to its efficacy and safety. Tolvaptan has recently received approval and been granted marketing authorization in Japan, Canada, Korea, Switzerland, and Europe. A demand for widely accepted guidelines for its use has emerged since its approval. The currently available series of recommendations and guidelines as to when to start treatment with tolvaptan, as well as which patients should be treated, are also reviewed in this report. We lastly offer some considerations for future trials, and raise unanswered questions. Keywords: autosomal dominant polycystic kidney disease, quality of life, economic burden, tolvaptan, tolvaptan cost-effectiveness, tolvaptan use recommendations