Efficacy of recombinant human thrombopoietin in patients with acute-on-chronic liver failure and thrombocytopenia: A prospective, open-label study

Abstract

BACKGROUND

Patients with acute-on-chronic liver failure (ACLF) have a high mortality rate, poor prognosis, and often experience concurrent thrombocytopenia and bleeding events.

AIM

To evaluate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in patients with ACLF with concomitant severe thrombocytopenia.

METHODS

This was a prospective, open-label study. We assigned 70 ACLF patients with severe thrombocytopenia into the rhTPO group and control group, with 35 patients in each group. Patients in the rhTPO group received subcutaneous injections of rhTPO at a dose of 15000 IU/day for 7 consecutive days, while patients in the control group did not receive rhTPO treatment. The primary endpoint was the proportion of patients with platelet count > 50 × 10⁹/L on day 14.

RESULTS

The proportion of patients with platelet count > 50 × 10⁹/L on day 14 was 60.7% in the rhTPO group, which was significantly higher than that (12.0%) in the control group (P < 0.001). The platelet count in the rhTPO group on day 14 was 64 × 10⁹/L, exceeding the baseline of 28 × 10⁹/L. Compared to the control group, the rhTPO group exhibited a significant increase in platelet count from baseline (P < 0.05). Model for end-stage liver disease score, albumin level and international normalized ratio improved significantly from baseline on day 14 after rhTPO injection. The concentrations of serum thrombopoietin and hepatocyte growth factor in the rhTPO group after 7 days were 143.7 and 195.4 pg/mL, respectively, showing a significant increase from baseline (P < 0.05). Eight (22.9%) patients had bleeding events in the control group compared with four (11.4%) in the rhTPO group. The incidence of 90-day mortality was also higher in the control group (6, 17.1%) than that in the rhTPO group (3, 8.6%).

CONCLUSION

rhTPO significantly increased the platelet count in ACLF patients with thrombocytopenia and reduce the occurrence of bleeding events, with a good safety profile.

Keywords: Recombinant human thrombopoietin; Acute-on-chronic liver failure; Thrombocytopenia; Hepatocyte growth factor; Prognosis

Core Tip: Patients with acute-on-chronic liver failure (ACLF) have a high mortality rate and poor prognosis. They often experience concurrent thrombocytopenia and bleeding events. This study aimed to evaluate the efficacy and safety of recombinant human thrombopoietin (rhTPO) in ACLF patients with concomitant severe thrombocytopenia. We assigned 70 ACLF patients with severe thrombocytopenia into the rhTPO group and the control group, with 35 patients in each group. Patients in the rhTPO group received subcutaneous injections of rhTPO for 7 consecutive days, while those in the control group did not receive rhTPO treatment. The platelet count in the rhTPO group increased significantly from the baseline. The model for end-stage liver disease score, albumin level, and international normalized ratio in patients of the rhTPO group improved significantly from the baseline on day 14 after rhTPO injection. In summary, rhTPO can significantly increase the platelet count in ACLF patients with thrombocytopenia, help improve liver function, and reduce the occurrence of bleeding events. It also has a good safety profile.