Abstract: PO1539
Early Real-World Descriptive Findings on Tolvaptan-Treated Patients with Autosomal Dominant Polycystic Kidney Disease in the United States
Session Information
- Cystic Kidney Diseases: Mechanisms, Genetics, and Treatment
October 22, 2020 | Location: On-Demand
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1001 Genetic Diseases of the Kidneys: Cystic
Authors
- Sanon, Myrlene, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
- Tao, Sunning, United BioSource LLC, Blue Bell, Pennsylvania, United States
- Cosmatos, Irene, United BioSource LLC, Blue Bell, Pennsylvania, United States
- Dieck, Gretchen S., United BioSource LLC, Blue Bell, Pennsylvania, United States
- Julian, Michele L., United BioSource LLC, Blue Bell, Pennsylvania, United States
- Wilt, Timothy, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
- Pareja, Kristin, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
- Estilo, Alvin, Otsuka Pharmaceutical Development and Commercialization Inc, Princeton, New Jersey, United States
- Matthews, Carol I., United BioSource LLC, Blue Bell, Pennsylvania, United States
Background
Tolvaptan is the first and only treatment for autosomal dominant polycystic kidney disease (ADPKD) approved in the United States (US).
The Food and Drug Administration (FDA) required a Risk Evaluation and Mitigation Strategy (REMS) to mitigate the risk of severe liver injury potentially associated with tolvaptan. This study aims to characterize patient demographics and treatment patterns among patients enrolled in the REMS in the US.
Methods
A descriptive analysis of patients with ADPKD enrolled in the tolvaptan REMS from 14 May 2018 through 3 February 2020 was conducted. Only patients enrolled in the REMS with at least 1 tolvaptan prescription dispensed were included in the analysis. The observational period began from a patient’s first shipment date of tolvaptan (index date) and ended on 3 February 2020 or patient death, whichever occurred earlier. Descriptive analyses provided baseline patient characteristics and follow-up measures on a subset of patients eligible for treatment pattern assessment. Additionally, medication possession ratio (MPR) (number of days supplied over a 1-year period) and persistency (length of time taking tolvaptan allowing for ≤59 days between refills) were measured.
Results
A total of 5,366 patients who initiated tolvaptan in the REMS comprised the study population. Mean age at tolvaptan initiation was 46.8 years (standard deviation [SD]: 11.6); 2,751 (51.3%) were female. Of the patients with a known race (n=2,705, 50.4%), the majority were white (n=2,153, 79.6%). Of those with known ethnicity (n=2,682, 50.0%), 2,352 (87.7%) were non-Hispanic or non-Latino. Overall, 2,366 (44.1%) tolvaptan initiators had at least 1 year of follow-up after the index date and were included in the treatment pattern analysis. The most frequent dose of tolvaptan was 45/15 milligrams daily (47.5%). The mean MPR was 0.74 (SD: 0.32); mean persistency was 325.9 days (SD: 173.2).
Conclusion
This is the first descriptive demographic report of real-world ADPKD patients in the US initiating tolvaptan. Based on the data currently available, most patients were between 35-55 years, equally male or female, and were non-Hispanic or non-Latino whites. The patients included in the treatment pattern analysis remained on tolvaptan close to 1 year.
Funding
- Commercial Support – Otsuka Pharmaceutical Development & Commercialization Inc.