Abstract: TH-PO412
Multicenter Real-Life Experience With Tolvaptan Treatment in Patients With Autosomal Dominant Polycystic Kidney Disease
Session Information
- Genetic Diseases of the Kidneys: Cystic - I
November 03, 2022 | Location: Exhibit Hall, Orange County Convention Center‚ West Building
Abstract Time: 10:00 AM - 12:00 PM
Category: Genetic Diseases of the Kidneys
- 1101 Genetic Diseases of the Kidneys: Cystic
Authors
- Duriseti, Parikshit, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Wigerinck, Stijn, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Chedid, Maroun, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Radhakrishnan, Yeshwanter, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Mao, Michael A., Mayo Clinic in Florida, Jacksonville, Florida, United States
- Hommos, Musab S., Mayo Clinic Scottsdale, Scottsdale, Arizona, United States
- Garg, Arvind K., Mayo Clinic Health System, La Crosse, Wisconsin, United States
- Zoghby, Ziad, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Keddis, Mira T., Mayo Clinic Scottsdale, Scottsdale, Arizona, United States
- Trautman, Christopher L., Mayo Clinic in Florida, Jacksonville, Florida, United States
- Dahlen, Erin, Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Gay, Melissa K., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Mcfadden, Jennifer E., Mayo Clinic Scottsdale, Scottsdale, Arizona, United States
- Bhat, Samrat V., Mayo Clinic Health System, La Crosse, Wisconsin, United States
- Harris, Peter C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Erickson, Stephen B., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Hogan, Marie C., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Torres, Vicente E., Mayo Clinic Minnesota, Rochester, Minnesota, United States
- Chebib, Fouad T., Mayo Clinic in Florida, Jacksonville, Florida, United States
Background
Autosomal dominant polycystic kidney disease (ADPKD) is the most common monogenic cause of ESKD. Tolvaptan was approved for the treatment of ADPKD patients who are at risk of rapid progression. The goal of our study is to examine the real-life experience of tolvaptan in ADPKD following its FDA approval.
Methods
We retrospectively identified 95 patients initiated on tolvaptan for management of ADPKD at Mayo Clinic across Minnesota, Florida, Arizona, and Wisconsin. Patients started on tolvaptan while enrolled in clinical trials were excluded. Baseline data including age, Mayo imaging classification (MIC), height-adjusted total kidney volume (htTKV) at tolvaptan initiation, and eGFR were documented. Monthly or quarterly laboratory monitoring including serum LFTs, sodium, creatinine, and urine osmolality were collected. Patient registry was created in electronic medical record and nursing surveillance program was utilized in monitoring labs and side effects on tolvaptan.
Results
Ninety-two patients started on tolvaptan between June 2018 and May 2022 were included in the study. Of whom, 58% were female, 91% were white with mean (SD) age at initiation of 40.7 (9.6) years. Mean (SD) htTKV at baseline was 1234.9 (909.9) mL/m. Based on Mayo imaging classification, 2% had MIC1B, 43% MIC1C, 28% MIC1D and 22% MIC1E. At last follow-up, 4% were on 30mg, 9% on 45mg, 57% on 60mg,18% on 90 mg and 12% on 120mg. Tolvaptan was discontinued in 23 (25%) patients. Aquaresis-related side effect was the main reason for drop-out (10.8%). Other reasons included concern for hepatotoxicity (5.4%), non-compliance with lab monitoring (4.3%), nausea (1%), AKI (1%), and lack of coverage by insurance for prescription renewal (1%). Of 5 patients in whom tolvaptan was stopped with concern for hepatotoxicity, none (0%) reached Hy’s Law for drug-induced liver injury.
Conclusion
In this multicenter real-life experience, tolvaptan was well tolerated in treating patients with ADPKD. The main reasons for discontinuation of therapy included aquaresis affecting quality of life and concern for hepatoxicity. Our findings are concurrent with data from clinical trials for tolvaptan in ADPKD.