Abstract: PO1541
Global Real-World Evidence of Tolvaptan in Patients with Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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
- Maiese, Brett A., Xcenda LLC, Palm Harbor, Florida, United States
- Colby, Jennifer A., Xcenda LLC, Palm Harbor, Florida, United States
- Pareja, Kristin, Otsuka Pharmaceuticals Development & Commercialization, Princeton, New Jersey, United States
- Laplante, Annick, Otsuka Canada Pharmaceutical Inc, Saint Laurent, Quebec, Canada
- Wang, Xinyu, Otsuka Pharmaceutical Europe Ltd, Wexham, Buckinghamshire, United Kingdom
- Mccormick, Linda, Otsuka Pharmaceuticals Development & Commercialization, Princeton, New Jersey, United States
- Nourbakhsh, Ali, Otsuka Pharmaceuticals Development & Commercialization, Princeton, New Jersey, United States
- Wang, Tao, Otsuka Pharmaceuticals Development & Commercialization, Princeton, New Jersey, United States
- Sanon, Myrlene, Otsuka Pharmaceuticals Development & Commercialization, Princeton, New Jersey, United States
Background
ADPKD is a rare, hereditary, systemic kidney disease characterized by progressive renal damage. Patients frequently develop end stage kidney disease, requiring renal replacement therapy. Tolvaptan is the first and only treatment shown to slow kidney function decline in adults who are at risk of rapidly progressing ADPKD. The goal of this literature review was to understand real-world effectiveness and safety data currently available on tolvaptan treatment.
Methods
A review of the literature was conducted in January 2020 in Embase (including Medline) with no language, timeframe, or geography restrictions. Observational studies of ADPKD patients receiving tolvaptan were identified; outcomes of interest included clinical effectiveness and safety, healthcare resource utilization and costs, and quality of life (QoL).
Results
A total of 43 relevant publications were identified. Studies were conducted in Canada, Japan, and across Europe with sample sizes ranging from a single case report to registry analyses of more than 1,000 patients. Clinical results from 6 studies reported a slowing of total kidney volume (TKV) growth and no significant changes in annual decline of estimated glomerular filtration rate (eGFR) over a range of 3 months to 2 years following tolvaptan initiation. Commonly reported adverse events included polyuria (~10%) and liver function-related events (~9%). Reported in 6 studies, 15.6% of patients discontinued tolvaptan treatment, primarily for aquaretic symptoms. Two studies reported that tolvaptan treatment did not appear, over a 1-year period, to negatively impact QoL, with more than 75% of patients reporting little impact on daily activities. No eligible economic studies were identified.
Conclusion
Patients with ADPKD receiving tolvaptan in the real-world experienced improved clinical outcomes without negative impact on QoL. Additional studies assessing real-world evidence supporting tolvaptan treatment in this population are needed.
Funding
- Commercial Support –