BACKGROUND: The thrombopoietin receptor agonist Lusutrombopag was recently adapted for treatment of thrombocytopenia in chronic liver disease (CLD). However, no studies have compared the effects of Lusutorombopag and platelet transfusion. Therefore, we aimed to clarify the efficacy and proportion of responders of Lusutrombopag compared with platelet transfusion, by propensity score matching. METHODS: A total of 200 patients (Lusutrombopag, 90; platelet transfusion, 110) with thrombocytopenia were enrolled, and matched for age, liver function, renal function, platelet count, peripheral blood count, and spleen size, using the propensity score matching method. Finally, 52 patients (Lusutrombopag, 26; platelet transfusion, 26) were included. The primary endpoint was an increase in platelet count. Secondary endpoints were proportion of responders, duration of the sustained effect, incidence of adverse events, and predictors associated with an increase in platelet count. RESULTS: The median increase in platelets from baseline was 48 × 103 /μL vs. 9.5 × 103 /μL (Lusutrombopag vs. Transfusion, p < 0.0001). The proportion of responders (increase of > 10 × 103 /μL) was 100% vs. 50.0% (p < 0.0001). Median duration of the sustained effect (increase of > 50 × 103 /μL) was 10 vs. 2 days (p < 0.0001). Incidence of adverse events was 7.7% vs. 50.0% (p = 0.036). Predictors associated with an increase in platelets were hemoglobin and spleen size by multiple regression analysis. CONCLUSION: Lusutrombopag was more effective in CLD patients than platelet transfusion. The proportion of responders, effect duration, and non-incidence rate of adverse events were higher in the Lusutrombopag group. This article is protected by copyright. All rights reserved.
BACKGROUND: The thrombopoietin receptor agonist Lusutrombopag was recently adapted for treatment of thrombocytopenia in chronic liver disease (CLD). However, no studies have compared the effects of Lusutorombopag and platelet transfusion. Therefore, we aimed to clarify the efficacy and proportion of responders of Lusutrombopag compared with platelet transfusion, by propensity score matching. METHODS: A total of 200 patients (Lusutrombopag, 90; platelet transfusion, 110) with thrombocytopenia were enrolled, and matched for age, liver function, renal function, platelet count, peripheral blood count, and spleen size, using the propensity score matching method. Finally, 52 patients (Lusutrombopag, 26; platelet transfusion, 26) were included. The primary endpoint was an increase in platelet count. Secondary endpoints were proportion of responders, duration of the sustained effect, incidence of adverse events, and predictors associated with an increase in platelet count. RESULTS: The median increase in platelets from baseline was 48 × 103 /μL vs. 9.5 × 103 /μL (Lusutrombopag vs. Transfusion, p < 0.0001). The proportion of responders (increase of > 10 × 103 /μL) was 100% vs. 50.0% (p < 0.0001). Median duration of the sustained effect (increase of > 50 × 103 /μL) was 10 vs. 2 days (p < 0.0001). Incidence of adverse events was 7.7% vs. 50.0% (p = 0.036). Predictors associated with an increase in platelets were hemoglobin and spleen size by multiple regression analysis. CONCLUSION:Lusutrombopag was more effective in CLD patients than platelet transfusion. The proportion of responders, effect duration, and non-incidence rate of adverse events were higher in the Lusutrombopag group. This article is protected by copyright. All rights reserved.