Recent Advances in the Management of Autosomal Dominant Polycystic Kidney Disease

Abstract

Autosomal dominant polycystic kidney disease (ADPKD), the most common monogenic cause of ESKD, is characterized by relentless development of kidney cysts, hypertension, and destruction of the kidney parenchyma. Over the past few years, major advancements in diagnosing, prognosticating, and understanding the pathogenesis and natural course of the disease have been made. Currently, no kidney disease is more suitable for nephron-protective strategies. Early nephrology referral and implementation of these strategies may have a substantial effect. Total kidney volume is a good prognostication marker and allows stratification of patients into slow or rapid progressing disease, with implications for their management. Measurement of total kidney volume, disease stratification, and prognostication are possible using readily available tools. Although some patients require only monitoring and basic optimized kidney protective measures, such as rigorous BP control and various lifestyle and dietary changes, others will benefit from disease-modifying treatments. Vasopressin V2 receptor antagonists, a likely disease-modifying treatment, has been approved in several countries and recently by the US Food and Drug Administration; other therapies, such as somatostatin analogs and other novel agents, are currently in clinical trials. The purpose of this article is to present our views on the optimal management to delay kidney disease progression in ADPKD.

A Practical Guide for Treatment of Rapidly Progressive ADPKD with Tolvaptan

“…Physicians prescribing tolvaptan should not overlook other simpler interventions that, in combination, can have a substantial effect on the long-term outcome of ADPKD ( Figure 4). These are discussed in a recent review 36 and consist of specific BP target goals, treatment with preferred antihypertensive agents (i.e., angiotensin-converting enzyme inhibitors or angiotensin II receptor blockers), and lifestyle modifications. Their inclusion in the tolvaptan protocol presents an opportunity to optimize the management of ADPKD.…”

The societal economic burden of autosomal dominant polycystic kidney disease in the United States

“…Moreover, effective management of increasing comorbidities with advancing disease stage, including heart failure and hyperkalemia, may offer further potential for cost reductions. Novel treatments for ADPKD are currently the subject of several clinical trials, and, in 2018, one treatment received regulatory approval in the US for the treatment of ADPKD in patients at risk of rapidly progressing disease [52][53][54][55][56][57]. These treatments have the potential help alleviate the burden of ADPKD for both individuals living with this disease, and their caregivers.…”

Erbliche Zystennierenerkrankungen: Autosomal-dominante und autosomal-rezessive polyzystische Nierenerkrankung (ADPKD und ARPKD)

“…Prognostisch lässt sich festhalten, dass es bei 50 % der betroffenen Patienten bis zur 6. Lebensdekade zum Erreichen einer terminalen Niereninsuffizienz kommt [10,11].…”

“…In der TEMPO 3:4-Studie wurde gezeigt, dass eine Behandlung mit dem Vasopressin-2-Rezeptor-Antagonist Tolvaptan die Zunahme des Nierengesamtvolumens verlangsamen ließ. Ebenso ließ sich eine Reduktion des eGFR-Verlustes nachweisen [11]. Es sind jedoch relevante Nebenwirkungen, wie eine Polyurie oder eine mögliche Hepatotoxizität zu beachten.…”

“…konsequente Blutdruckeinstellung mittels ACE-Hemmer zu einer Verlangsamung des Anstieges des Nierengesamtvolumens als Surrogatparameter für den Krankheitsverlauf führt [10,11].…”