A NEW study has shown that recombinant human thrombopoietin (rhTPO) may be a promising treatment for patients with acute-on-chronic liver failure (ACLF) complicated by severe thrombocytopenia. ACLF is a serious condition marked by sudden liver deterioration in patients with pre-existing liver disease, often accompanied by low platelet counts, coagulopathy, and high short-term mortality. Bleeding events are a major complication, emphasising the need for effective platelet-boosting therapies.
In this prospective, open-label study, patients with ACLF received daily rhTPO injections for seven days. Results revealed a significant rise in platelet counts, from a baseline of 28 × 10⁹/L to 64 × 10⁹/L. This increase was accompanied by improved liver function, including lower MELD scores and better INR levels. Importantly, rhTPO also appeared to stimulate hepatocyte growth factor (HGF) levels, which play a crucial role in liver regeneration.
Compared to controls, the rhTPO group experienced fewer bleeding events and a lower mortality rate (8.6% versus 17.1%). No thrombotic events were observed, and only mild, transient side effects such as fever were reported, suggesting rhTPO has a favourable safety profile.
rhTPO works by mimicking endogenous thrombopoietin, binding to receptors on megakaryocytes and platelets to stimulate platelet production. Beyond managing thrombocytopenia, it may promote liver regeneration by increasing levels of HGF and other growth factors. This aligns with findings from animal models showing improved liver recovery with elevated platelet counts.
Although the findings are encouraging, the authors note limitations including the study’s single-centre design, small sample size, and short follow-up duration. Further large-scale studies with stratified patient groups are needed to confirm rhTPO’s long-term efficacy and safety. Nevertheless, these results provide valuable insight into a potential new therapeutic option for managing bleeding risk and supporting liver regeneration in patients with ACLF.
Reference
Liu G, et al. Efficacy of recombinant human thrombopoietin in patients with acute-on-chronic liver failure and thrombocytopenia: a prospective, open-label study. World J Gastroenterol. 2025;31(14):105004.
