Hepatic arterial infusion of autologous CD34 cells for hepatitis C virus-related decompensated cirrhosis: A multicenter, open-label, exploratory randomized controlled trial.

INTRODUCTION

In this multicenter clinical study, we aimed to investigate the efficacy and safety of the transhepatic arterial administration of granulocyte-colony stimulating factor (G-CSF)-mobilized autologous peripheral blood (PB)-CD34 cells compared with standard therapy in patients with decompensated cirrhosis type C.

METHODS

Patients were randomly assigned (2:1) to the CD34 cell transplant (CD34 cell) or standard-of-care (SOC) group and followed up for 52 weeks. The primary endpoints were the non-progression rate of Child-Pugh (CP) scores at 24 weeks post-enrollment and the safety of the protocol treatment.

RESULTS

Fourteen patients (CD34 cell group: 10; SOC group: 4) were enrolled. CP scores at 24 weeks had a non-progression rate of 90% in the CD34 cell group and 100% in the SOC group, with no significant difference between groups. Importantly, 4 out of 10 patients in the CD34 cell group exhibited an improvement from decompensated to compensated cirrhosis, whereas all patients in the SOC group remained in decompensated cirrhosis. With regard to secondary endpoints, a trend toward increased serum albumin levels in the CD34 cell group was noted. Serious adverse events (SAEs) occurred in three patients in the CD34 cell group and in one patient in the SOC group. No causal relationship was observed between all SAEs and G-CSF, leukapheresis, or cell transplantation in the CD34 cell group. No patients died and no hepatocellular carcinoma occurred within the study period.

CONCLUSIONS

PB-CD34 cell infusion therapy may have the potential to circumvent the decompensated stage of cirrhosis, thus avoiding the need for liver transplantation.