促血小板生成素受体激动剂芦曲泊帕对慢性肝病合并肾功能损害患者有效。

The Thrombopoietin Receptor Agonist Lusutrombopag Is Effective for Patients with Chronic Liver Disease and Impaired Renal Function.

机构信息

Department of Gastroenterology and Hepatology, Tokyo Medical University.

Department of Gastroenterology and Hepatology, Niizashiki Central General Hospital.

出版信息

J Nippon Med Sch. 2021 Jan 8;87(6):325-333. doi: 10.1272/jnms.JNMS.2020_87-603. Epub 2020 Mar 31.

DOI:10.1272/jnms.JNMS.2020_87-603

PMID:32238734

Abstract

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 × 10/μL from baseline) in the absence of platelet transfusion. The secondary endpoints were response rate (an increase of greater than 1.0 × 10/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.

摘要

背景

血小板生成素（TPO）受体激动剂芦曲泊帕被开发用于治疗慢性肝病（CLD）中的血小板减少症。然而，其疗效尚不清楚。本研究的目的是评估芦曲泊帕的疗效，并确定与血小板计数增加相关的预测因素。

方法

共纳入 80 例 CLD 合并血小板减少症患者。主要终点为无血小板输注情况下血小板（较基线增加>1.0×10/μL）显著增加。次要终点为反应率（较基线增加>1.0×10/μL）、血小板增加的独立预测因素以及芦曲泊帕优于血小板输注的优越性。

结果

93.8%（75/80）的患者达到了主要终点。反应率为 96.2%（77/80）。肾功能参数（血尿素氮、肌酐、eGFR）与血小板计数显著负相关（p=0.033、0.049 和 0.0014），多元回归分析显示其为血小板计数的独立预测因素（p=0.049、0.0023 和 0.0016）。与血小板输注相比，芦曲泊帕治疗后血小板计数的中位数增加更显著（41，000 比 12，000/μL，p=0.015）。