Abstract
BACKGROUND: Treatments for hepatorenal syndrome with acute kidney injury (HRS-AKI) that are not FDA-approved have been widely used in the United States (US) with variable outcomes. This study describes the practice patterns, outcomes, and healthcare utilization around vasopressor use before terlipressin approval in 2022. METHODS: A retrospective chart review study was conducted at 10 US medical centers, assessing adult patients diagnosed with HRS-AKI between 2016 and 2019. The primary outcome was treatment response (change in serum creatinine [SCr] from the day of vasopressor treatment initiation to Day 14/vasopressor discontinuation). Secondary outcomes included overall and transplant-free survival, treatment patterns, and healthcare resource use. RESULTS: Of the 198 eligible patients, 129 and 69 had mild/moderate (SCr < 5 mg/dL, acute-on-chronic liver failure [ACLF] ≤ 2) and severe disease (SCr ≥ 5 mg/dL, ACLF > 2), respectively. The mean age was 57 years; 52.5% were males, and 71.2% were White. Alcohol-associated cirrhosis (53.5%) was the most common cause of cirrhosis. All 198 patients had a physician-diagnosed HRS-AKI, and only 30.3% met all International Club of Ascites (ICA)-HRS criteria. Most patients (85.4%) initiated treatment with midodrine and octreotide for a median of 7 days. The overall response rate (n = 157) was 20.3%. Median (95% CI) overall and transplant-free survival from vasopressor initiation was 48 (32-81) and 28 (19-36) days. Notably, 33.8% of patients died during hospitalization, and 31.3% required renal replacement therapy. CONCLUSION: Before 2022, hospitalized HRS-AKI patients experienced suboptimal treatment response with off-label treatments and poor survival. There remains an unmet need for safe and effective non-transplant treatments for hospitalized HRS-AKI patients in the United States.