Kosin Med J.  2023 Jun;38(2):87-97. 10.7180/kmj.23.125.

Are you ready to accompany autosomal dominant polycystic kidney disease patients in their treatment journey? Real practice for selecting rapid progressors and treatment with tolvaptan

• Jung Y
• Oh YK

• KMID: 2543328
• DOI: http://doi.org/10.7180/kmj.23.125

Abstract

Tolvaptan treatment is costly, often accompanied by aquaresis-related adverse events, and requires careful monitoring by medical staff due to the possibility of hepatotoxicity. Nevertheless, it is the only disease-modifying drug to date that has been shown to successfully delay renal replacement therapy. For more patients to receive proper treatment, medical doctors, the rest of the medical team, and the patient must all work together. This paper reviews parameters that can help identify rapid autosomal dominant polycystic kidney disease progressors, who are the target of tolvaptan therapy. It is expected that these parameters will help nephrologists learn practical prescription methods and identify patients who can benefit from tolvaptan treatment. Although several strategies can be used to find rapid progressors, the present review focuses on a practical method to identify rapid progressors according to the presence or absence of evidence and the factors associated with rapid progression based on the Mayo image classification.

Keyword

Patient selection; Polycystic kidney, autosomal dominant; Tolvaptan

Figure

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  Fig. 1. Manual techniques available for estimating TKV in patients with ADPKD. (A) Ellipsoid formula applied to a coronal slice (a) and a sagittal slice (b); measurements of longitudinal length (L+L’/2), maximum width (W), and maximal depth (D) are used to calculate renal volume in the typical ADPKD calculator web-based application provided by the Mayo Polycystic Kidney Disease Center. (B) Stereology applied to a coronal slice using MRI; grid points covering both kidneys are defined. (C) Planimetry method is applied to a coronal slice on a contrast-enhanced CT image; all slices are manually traced with kidney. Coronal CT image from an expanded MIC 1B patient: a 24-year-old woman with PKD1 gene mutation whose baseline TKV decreased from 594 mL (MIC 1C) to 389 mL (MIC 1B) after the exclusion of exophytic cysts. (D) Coronal MRI from an expanded MIC 1C patient: a 38-year-old woman with PKD1 gene mutation whose baseline TKV decreased from 1,236 mL (MIC 1D) to 940 mL (MIC 1C) after the exclusion of exophytic cysts. TKV, total kidney volume; ADPKD, autosomal dominant polycystic kidney disease; MRI, magnetic resonance imaging; CT, computed tomography; MIC, Mayo imaging classification.

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  Fig. 2. STEP 1: Exclusion to treatment and assessment of prognostic biomarkers. Individuals having which proteinuria ≥1 g/day, vascular disease, uncontrolled arterial hypertension, and diabetes mellitus nephropathy that affect renal function deterioration are excluded. STEP 2: Assess the risk, evidence, and factors of rapid progression in an autosomal dominant polycystic kidney disease patient. Mayo imaging classification is the primary predictor of rapid progression. a)Patients aged 18–39 years are not excluded regardless of the estimated glomerular filtration rate (eGFR).

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  Fig. 3. Recommendation according to the presence or absence of evidence and factors of rapid progression according to the Mayo Image Classification in selecting patients with autosomal dominant polycystic kidney disease (ADPKD) who are at risk of rapidly progressive disease for treatment with tolvaptan. Special consideration can be considered depending on the individual patient. eGFR, estimated glomerular filtration rate; ERA, European Renal Association.

Reference

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