Florence Wong, MD, Hepatologist at Toronto General Hospital discusses terlipressin, a potent vasopressin analogue being investigated for the treatment of hepatorenal syndrome type 1 (HRS-1).
In the first part of the interview, Dr. Wong describes the method of action of terlipressin. As she explains, patients with HRS-1 have systemic and splanchnic vasodilation. This causes low blood pressure which leads to vasoconstriction in the kidneys. Terlipressin works by causing vasoconstriction of splanchnic circulation which leads to vasoconstriction of the systemic circulation and vasodilation of the kidneys. This in turn allows the kidneys to return to proper functioning in terms of waste filtration and clearance.
In the second part of the interview, Dr. Wong discusses the results from the phase 3 CONFIRM study, assessing the efficacy and safety of terlipressin plus albumin in adults with HRS-1. The primary endpoint of the study was verified reversal of HRS, defined as two consecutive serum creatinine measurements of 1.5 mg/dL or less, at least 2 hours apart and survival without renal-replacement therapy for at least 10 days after the completion of treatment. The results of the trial were recently published in the New England Journal of Medicine (available here).
Verified reversal of HRS was reported in 32% of patients in the terlipressin group and 17% of patients in the placebo group. Adverse events, including abdominal pain, nausea, diarrhea, and respiratory failure, occurred more with terlipressin than with placebo. Death within 90 days due to respiratory disorders occurred in 11% of patients in the terlipressin group and 2% of patients in the placebo group. Overall, the results demonstrated that terlipressin was more effective than placebo in improving renal function but was associated with serious adverse events, including respiratory failure.
To learn more about HRS-1 and other rare kidney disorders, visit checkrare.com/diseases/kidney-and-urinary-diseases/