Does Terlipressin work?
Yes, in large controlled trials: a 300-patient trial.
The CONFIRM trial randomized 300 patients with hepatorenal syndrome type 1 to terlipressin or placebo, both with albumin, and showed roughly double the rate of verified reversal of kidney failure. The label's efficacy section rests on CONFIRM, a double-blind randomized trial, so the approved use grades A, with the caveat that reversal of kidney injury did not translate into better 90-day survival. 12
CONFIRM was published in the New England Journal of Medicine in 2021. It randomized 300 patients with cirrhosis and hepatorenal syndrome type 1 at North American centers. They were assigned 2:1 to terlipressin (199) or placebo (101) for up to 14 days. Both arms also got albumin. Verified reversal of hepatorenal syndrome meant two creatinine readings in a row at or below the threshold. The patient also had to be alive and off dialysis for 10 days. This occurred in 32 percent of the terlipressin group and 17 percent of the placebo group. By day 30, reversal without dialysis or other kidney replacement therapy was also more common (34 versus 17 percent). Verified reversal without a relapse by day 30 (26 versus 17 percent) did not reach statistical significance. 1
Death by day 90 was 51 percent on terlipressin and 45 percent on placebo. This difference was not statistically significant. Serious or fatal respiratory failure occurred in 14 percent of terlipressin patients and 5 percent of placebo patients. Deaths from lung disorders within 90 days were 11 percent versus 2 percent. 12
Evidence checklist
- Randomised controlled trial CONFIRM, placebo-controlled with albumin
- Human study over 100 people CONFIRM 300 patients
- More than one research group North American trial plus earlier ones
- Clinical outcomes, not surrogate markers Kidney reversal; 90-day survival not improved
- Human safety data beyond case reports Boxed warning: respiratory failure
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