Characteristics of Patients with ADPKD Treated with Tolvaptan in the United States

Kidney Week 2020

The most common inherited kidney disease is autosomal dominant polycystic kidney disease (ADPKD). Tolvaptan is the first and only FDA-approved treatment for ADPKD in the United States. Results of clinical trials of tolvaptan have shown tolvaptan slows decline in kidney function in patients with ADPKD. To date, there are few data available on the characteristics of real-world patients initiating tolvaptan in the United States.

Irene Cosmatos, MS, and colleagues conducted an observational, retrospective analysis to examine baseline measures in patients with ADPKD who initiated treatment with tolvaptan from May 14, 2018, through January 9, 2020, in the United States. Results of the analysis were reported during a virtual poster session at ASN Kidney Week 2020 in a poster titled Early Findings of Patients with Autosomal Dominant Polycystic Kidney Disease Initiating Tolvaptan in the United States: A Claims-Based Analysis.

Researchers linked data from a nationally representative billing database, Symphony Health Integrated Dataverse (IDV), with Specialty Pharmacy data from the tolvaptan risk evaluation and mitigation strategy, a mandatory program for patients with ADPKD prescribed tolvaptan. The study index date was the date of the first shipment of tolvaptan.

Analyses were performed on baseline measures including demographics, comorbidities, and disease characteristics. All measures were identified within the 6-month period prior to the index date in the Symphony Health IDV. The chronic kidney disease (CKD) stage closest to the index date was captured for patients with more than one CKD stage diagnosis.

The analysis included 4355 patients. Mean age at the time of initiation of tolvaptan was 48.8 years, and 51.7% of patients were female. The most commonly observed comorbidity was hypertension (80.8%, n=3520), followed by diabetes (6.3%, n=273). Of the 1566 patients with available data on CKD stage, 6.2% (n=97) had CKD stage 1, 13.4% (n=210) had CKD stage 2, 55.2% (n=864) had CKD stage 3, 22.9% (n=359) had CKD stage 4, and 2.3% (n=36) had CKD stage 5.

“This is one of the first real-world studies to describe comorbidities and disease characteristics in patients with ADPKD initiating tolvaptan in the United States. Stage 3 was the most commonly reported CKD stage among patients with a known CKD stage during their baseline period. Additional analyses evaluating the real-life impact of tolvaptan on clinical outcomes, healthcare utilization, and quality of life are needed,” the researchers said.

Source: Cosmatos I, Tao S, Dieck GS, et al. Early findings of patients with autosomal dominant polycystic kidney disease initiating tolvaptan in the United States: A claims-based analysis. Abstract of a poster presented at the American Society of Nephrology virtual Kidney Week 2020 (PO1538), October 22, 2020. Funding for this poster was provided by Otsuka Pharmaceutical Development & Commercialization, Inc.