Aim: The thrombopoietin receptor agonist, lusutrombopag, was recently adapted for treatment of thrombocytopenia in chronic liver disease. 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 (90 lusutrombopag, 110 platelet transfusion) 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 (26 lusutrombopag, 26 platelet transfusion) were included. The primary end-point was an increase in platelet count. Secondary end-points were the 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 versus 9.5 × 103 /μL (lusutrombopag vs. transfusion, P < 0.0001). The proportion of responders (increase of >10 × 103 /μL) was 100% versus 50.0% (P < 0.0001). Median duration of the sustained effect (increase of >50 × 103 /μL) was 10 versus 2 days (P < 0.0001). The incidence of adverse events was 7.7% versus 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 chronic liver disease patients than platelet transfusion. The proportion of responders, effect duration, and non-incidence rate of adverse events were higher in the lusutrombopag group.
Keywords: chronic liver disease; lusutrombopag; platelet transfusion; thrombocytopenia; thrombopoietin receptor agonist.
© 2020 The Japan Society of Hepatology.