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The Latest on X

Kidney Week

Abstract: TH-PO420

Tolvaptan Use and Prescribing Patterns in Patients with Autosomal Dominant Polycystic Kidney Disease: A Multicenter Real-World Experience

Session Information

Category: Genetic Diseases of the Kidneys

  • 1201 Genetic Diseases of the Kidneys: Cystic

Authors

  • Rao, Vinamratha, University of Kansas School of Medicine, Kansas City, Kansas, United States
  • Alshorman, Abrar, University of Kansas School of Medicine, Kansas City, Kansas, United States
  • Ammar, Shahed, University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States
  • McGreal, Kerri A., University of Kansas School of Medicine, Kansas City, Kansas, United States
  • Winklhofer, Franz, University of Kansas School of Medicine, Kansas City, Kansas, United States
  • Noureddine, Lama A., University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States
  • Jalal, Diana I., University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States
  • Fravel, Michelle A., University of Iowa Hospitals and Clinics, Iowa City, Iowa, United States
  • Yu, Alan S.L., University of Kansas School of Medicine, Kansas City, Kansas, United States
  • Mustafa, Reem, University of Kansas School of Medicine, Kansas City, Kansas, United States
Background

Autosomal Dominant Polycystic Kidney Disease (ADPKD) is the most common genetic disease leading to End Stage Kidney Disease. Tolvaptan was approved and is being used to treat patients with rapidly progressing disease. In this study we describe the real-world experience of Tolvaptan use among patients with ADPKD.

Methods

We retrospectively identified adult patients with ADPKD enrolled in the Risk Evaluation and Mitigation Strategy (REMS) program and treated with Tolvaptan at the University of Kansas Medical Center (KUMC) and University of Iowa Hospitals and Clinics (UIHC) from 2018 to 2023. The investigators abstracted data and treating physicians reviewed and confirmed the data. We performed descriptive analysis of the patients’ demographics, baseline labs, Tolvaptan prescribing trends, reasons for stopping treatment, and frequency of imaging surveillance.

Results

The study includes 134 patients, 115 from KUMC and 19 from UIHC. The average age of start of Tolvaptan treatment was 42.67 and 36.17 years respectively for KUMC and UIHC. Male to female ratio was approximately 1:1 for KUMC and 1:2 for UIHC. In the KUMC cohort, 87% were Caucasian, and the remaining 13% comprised of African American and Hispanic ethnic groups. In the UIHC cohort, 95% were Caucasian. By March 2023, 63% and 58% remained on Tolvaptan and of the KUMC and UIHC cohorts respectively. The two major reasons for discontinuing Tolvaptan included intolerance of polyuria and polydipsia (26% in KUMC and 50% in UIHC) and renal transplantation (17% in KUMC and 13% in UIHC). The average fluid intake was 5.23 and 3.36 Liters respectively in the KUMC and UIHC cohorts. The starting dose combination of 45 mg (AM)/15 mg (PM) was prescribed to most patients at both KUMC (90%) and UIHC (89%). For many patients, prescribing physicians had to considerably titrate Tolvaptan doses to moderate side effects.

Conclusion

The findings of this descriptive study provide valuable insights into the real-world use of Tolvaptan in treating patients with ADPKD. A higher percentage of patients with ADPKD are discontinuing Tolvaptan compared to trials findings due to polyuria and polydipsia. The study also highlights the need for considerable titration of Tolvaptan dose to manage symptoms.