Evaluation of the Efficacy of Lusutrombopag for Chronic Liver Disease Based on Pre-Treatment Platelet Counts: A Retrospective Multicenter Study.

Background: Oral thrombopoietin receptor agonists are used to treat thrombocytopenia in patients with chronic liver disease who are scheduled for invasive procedures. The efficacy of lusutrombopag based on the pretreatment platelet count was investigated.

Methods: Patients treated at nine hospitals from December 2015 to December 2023 were included. Efficacy was assessed by comparing the proportion of patients achieving a platelet count ≥ 50 000/μL and the change in platelet count.

Results: Seventy patients were eligible for evaluation. Patients with a pretreatment platelet count < 40 000/μL had a significantly lower rate of achieving a platelet count of ≥ 50 000/μL than those with a pretreatment count of 40 000-50 000/μL (62.5% vs. 84.2%,  = 0.038); however, there was no significant difference in the change in platelet count (25 700 vs. 24 400/μL,  = 0.972). Patients with viral-related cirrhosis showed a significantly greater change in platelet count than the others (29 100 vs. 19 200/μL,  = 0.012). For patients receiving multiple lusutrombopag treatments, the change in platelet count was significantly lower in the second treatment than in the first treatment (26 900 vs. 20 800/μL,  = 0.041). The main adverse event observed was thrombosis (2.9%).

Discussion: Lusutrombopag increases platelet count regardless of pretreatment levels, but efficacy, defined as achieving a platelet count of ≥ 50 000/μL, may be insufficient in patients with a pretreatment platelet count < 40 000/μL. Additionally, patients with non-viral liver disease responded less well to treatment compared to those with viral liver disease. Therefore, treatment strategies should be tailored based on pretreatment platelet counts and the etiology of liver disease.