Background: The thrombopoietin (TPO) receptor agonist lusutrombopag was developed to treat thrombocytopenia in chronic liver disease (CLD). However, its effectiveness remains unclear. The purpose of this study was to assess the efficacy of lusutrombopag and identify predictors associated with increase in platelet count.
Methods: Eighty CLD patients with thrombocytopenia were enrolled. The primary endpoint was a satisfactory increase in platelets (greater than 1.0 × 104/μL from baseline) in the absence of platelet transfusion. The secondary endpoints were response rate (an increase of greater than 1.0 × 104/μL from baseline), independent predictors of increase in platelets, and the superiority of lusutrombopag over platelet transfusion.
Results: The primary endpoint was achieved in 93.8% (75 of 80) patients. The response rate was 96.2% (77 of 80). Renal function parameters (blood urea nitrogen, creatinine, eGFR) were significantly negatively associated with platelet count (p = 0.033, 0.049, and 0.0014, respectively) and were identified as independent predictors by multiple regression analysis (p = 0.049, 0.0023, and 0.0016, respectively). The median increase in platelet count was significantly higher after lusutrombopag than after platelet transfusion (41,000 vs. 12,000/μL, p = 0.015).
Conclusion: Lusutrombopag was more effective than platelet transfusion for CLD patients, and renal function independently predicted increase in platelet count. Renal function parameters were significantly associated with platelet count.
Keywords: chronic liver disease; lusutrombopag; platelet transfusion; thrombocytopenia; thrombopoietin receptor agonist.