Real-World Evidence of Tolvaptan Treatment for Patients with ADPKD

Autosomal dominant polycystic kidney disease (ADPKD) is a hereditary systemic kidney disease characterized by progressive renal damage. Patients with ADPKD commonly progress to end-stage kidney disease and require renal replacement therapy. The first and only treatment to slow the progression of kidney function in adults with ADPKD at risk of rapid progression is tolvaptan.

Researchers, led by Brett A. Maiese, PhD, MHS, conducted a literature review to examine real-world effectiveness and safety data available on treatment with tolvaptan. Results of the review were reported during a virtual poster session at ASN Kidney Week 2020 in a poster titled Global Real-World Evidence of Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD).

The literature review was conducted in EMBASE (including MEDLINE) in January 2020 with no language, timeframe, or geography restrictions. The reviewers identified observational studies of patients with ADPKD receiving treatment with tolvaptan. Outcomes of interest were clinical effectiveness and safety, utilization of healthcare resources and costs, and quality of life.

The review identified 43 relevant publications. The studies were conducted in Canada, Japan, and Europe. Sample sizes ranged from a single case report to registry analyses of more than 1000 patients. In six studies reporting clinical results, treatment with tolvaptan slowed growth in total kidney volume and no significant changes in annual decline in estimated glomerular filtration rate over a range of 3 months to 2 years after initiation of tolvaptan. Polyuria (~10%) and liver function-related events (~9%) were commonly reported adverse events.

In six studies, 15.6% of patients discontinued tolvaptan treatment, primarily for aquaretic symptoms. In two studies, results suggested that there was no negative impact of treatment with tolvaptan on quality of life. More than 75% of patients reported little impact on daily activities.

In conclusion, the researchers said, “Patients with ADPKD receiving tolvaptan in the real world experienced improved clinical outcomes without negative impact on quality of life. Additional studies assessing real-world evidence supporting tolvaptan treatment in this population are needed.”

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Source: Maiese BA, Colby JA, Pareja K, et al. Global real-world evidence of tolvaptan in patients with autosomal dominant polycystic kidney disease (ADPKD). Abstract of a poster presented at the American Society of Nephrology virtual Kidney Week 2020 (PO1541), October 22, 2020. Funding for this poster was provided by Otsuka Pharmaceuticals Development & Commercialization, Inc.