BACKGROUND & AIMS: There has been great interest in recent years to take advantage of bone marrow stem cells to treat cirrhosis. Our uncontrolled trial showed promising results for bone marrow mesenchymal stem cell (MSC) transplantation in cirrhosis. Therefore, we conducted a randomized, placebo-controlled trial to evaluate the efficacy of autologous MSC transplantation in cirrhosis. METHODS: The enrolled patients with decompensated cirrhosis were randomly assigned to receive MSC or placebo infusions. A median of 195 million (range: 120-295 million) cultured MSCs were infused through a peripheral vein. The primary outcome was absolute changes in MELD score. Secondary outcomes were absolute changes in Child score, liver function tests and liver volumes between the MSC and placebo group 12 months after infusion. RESULTS: A total of 27 patients were enrolled. Of these, 15 patients received MSC and 12 patients received placebo. One patient in the MSC group and one patient in the placebo group were lost to follow-up. Three patients in the MSC group died of liver failure 3 months (one patient), or 5 months (two patients) after cellular infusion. The baseline MELD scores of the deceased patients were significantly higher than those who remained alive in either group (20.0 vs. 15.1; P = 0.02). The absolute changes in Child scores, MELD scores, serum albumin, INR, serum transaminases and liver volumes did not differ significantly between the MSC and placebo groups at 12 months of follow-up. CONCLUSION: Based on this randomized controlled trial, autologous bone marrow MSC transplantation through peripheral vein probably has no beneficial effect in cirrhotic patients. Further studies with higher number of patients are warranted to better clarify the impact of MSC infusion through peripheral vein or portal vein in cirrhosis.
RCT Entities:
BACKGROUND & AIMS: There has been great interest in recent years to take advantage of bone marrow stem cells to treat cirrhosis. Our uncontrolled trial showed promising results for bone marrow mesenchymal stem cell (MSC) transplantation in cirrhosis. Therefore, we conducted a randomized, placebo-controlled trial to evaluate the efficacy of autologous MSC transplantation in cirrhosis. METHODS: The enrolled patients with decompensated cirrhosis were randomly assigned to receive MSC or placebo infusions. A median of 195 million (range: 120-295 million) cultured MSCs were infused through a peripheral vein. The primary outcome was absolute changes in MELD score. Secondary outcomes were absolute changes in Child score, liver function tests and liver volumes between the MSC and placebo group 12 months after infusion. RESULTS: A total of 27 patients were enrolled. Of these, 15 patients received MSC and 12 patients received placebo. One patient in the MSC group and one patient in the placebo group were lost to follow-up. Three patients in the MSC group died of liver failure 3 months (one patient), or 5 months (two patients) after cellular infusion. The baseline MELD scores of the deceased patients were significantly higher than those who remained alive in either group (20.0 vs. 15.1; P = 0.02). The absolute changes in Child scores, MELD scores, serum albumin, INR, serum transaminases and liver volumes did not differ significantly between the MSC and placebo groups at 12 months of follow-up. CONCLUSION: Based on this randomized controlled trial, autologous bone marrow MSC transplantation through peripheral vein probably has no beneficial effect in cirrhotic patients. Further studies with higher number of patients are warranted to better clarify the impact of MSC infusion through peripheral vein or portal vein in cirrhosis.