Settembre Ottobre 2021

Slowing progression of chronic kidney disease in polycistic kidney disease patients with tolvaptan: from guidelines to clinical practice

Abstract

Autosomal dominant polycystic kidney disease (ADPKD) is the most common hereditary kidney disease and accounts for∼10% of patients on renal replacement therapy. In the last decade, no specific treatment was available and only preventive measures could be put in place to delay the onset of ESRD. Following the results of the TEMPO 3:4 study, tolvaptan was approved in many countries, for the purpose of slowing the progression of renal insufficiency.

In Italy tolvaptan is available since 2016 for patients with chronic kidney disease (CKD) stage 1-3, and since 2020 for patients with CKD stage 4, who fulfil the criteria of “rapid disease progression”, according to the European recommendations.

After this approval, Italian nephrology units have had to change their organization to be able to identify the patients eligible for the drug and to guarantee frequent patient monitoring.

In this paper, we present our three-year experiences with tolvaptan, focusing on its safety profile and tolerability, but also on the high burden of care that such therapy represents not only for doctors, but also for patients. Strategies to implement remote monitoring may be useful to reduce the burden of assistance on one side, and the medicalization of ADPKD patients in the early stage of the disease, on the other.

Keywords: ADPKD, tolvaptan, remote monitoring, burden of care

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Introduzione

La malattia del rene policistico autosomica dominante (ADPKD) è la più comune malattia ereditaria renale, ed è presente in circa il 10% dei pazienti in terapia sostitutiva renale [1]. È una patologia che esordisce tipicamente in età adulta e all’incirca il 70% dei pazienti raggiunge lo stadio terminale dell’insufficienza renale all’età di 58 anni [2]. La malattia presenta tuttavia un’estrema variabilità fenotipica sia inter che intra familiare, legata alla complessità genetica, motivo per cui alcuni pazienti hanno un decorso della malattia più aggressivo con necessità di trattamento sostitutivo anche prima dei 40 anni, mentre altri presentano un decorso più lento, e non raggiungono mai lo stadio terminale dell’insufficienza renale cronica (IRC), neanche in età avanzata [3].

 

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