Safety and preliminary efficacy of allogeneic umbilical cord-derived mesenchymal stem cells administered via the hepatic artery in patients with liver cirrhosis: A phase I open-label trial.

Abstract

Liver cirrhosis causes substantial morbidity, and options beyond transplantation are limited. Umbilical cord-derived mesenchymal stem cells (UC-MSCs) may support liver repair, but their delivery via the hepatic artery has not been evaluated. This study assessed the safety and exploratory efficacy of allogeneic UC-MSCs infusion in cirrhosis. Twenty patients with liver cirrhosis entered a single-center open-label pilot trial in Hanoi, Vietnam, between 2020 and 2023; 17 completed follow-up and were included in exploratory analyses. All received a single hepatic artery infusion of UC-MSCs at 1 × 10⁶ cells/kg and were assessed at baseline, 3, 6, and 12 months for liver function, Model for End-stage Liver Disease (MELD) and Child-Pugh scores, Chronic Liver Disease Questionnaire (CLDQ), and adverse events. No serious adverse events occurred; mild events were self-limited. Albumin increased at 3 and 6 months (P = 0.048; P = 0.027). Bilirubin, liver enzymes, coagulation, and MELD remained stable. Child-Pugh score decreased transiently at 3 months (P = 0.024). CLDQ increased across most domains, except systemic symptoms. Hepatic artery infusion of UC-MSCs was safe and well tolerated, with observed increases in albumin and in most CLDQ domains, while other parameters remained stable. Findings are exploratory because of the small sample size and lack of a control group and therefore require confirmation in larger, controlled studies.