Objective: To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) . Methods: The clinical characteristics of 23 pediatric ITP patients who received eltrombopag from May 2015 to March 2019 were retrospectively analyzed. Eltrombopag started with an initial dose of 12.5-50.0 mg/d and the maximum dose was 75.0 mg/d. Results: Among 23 children, there were 11 boys and 12 girls with median age 11.0 (2.0-17.0) years. Four cases were newly diagnosed ITP, the other 8 of persistent ITP and 11 of chronic ITP. The duration of eltrombopag application ranged from 4.5 to 95 weeks (8/23 still ongoing) . The median platelet (PLT) counts at 2 weeks, 4 weeks, 3 months and the 6 months after treatment were 40 (4-170) ×10(9)/L, 20 (4-130) ×10(9)/L, 60 (4-110) ×10(9)/L, and 70 (18-160) ×10(9)/L, which were all significantly higher than that before treatment 14 (2-82) ×10(9)/L (z=-3.440, P=0.001; z=-1.964, P=0.049; z=-4.339, P<0.001;z=-5.794, P<0.001 respectively) . The overall response rate was 60.87% (14/23 cases) . The median time to PLT count ≥30×10(9)/L was 10.5 (3-42) days. Seven patients (30.43%) responded within the first week, and 10 cases (43.48%) achieved PLT counts ≥30×10(9)/L within 2 weeks. All patients were divided into three groups according to the age (<6 years old, 6-12 years old, 13-17 years old) . The response rates were similar in three groups, as 33.33%, 60.00%, 85.71%, respectively. WHO bleeding scores as 0, 1, 2 were corresponded to 4, 12 and 7 patients before treatment. Patient numbers changed to 13, 7, 3 with bleeding scores 0, 1, 2 respectively after treatment (χ(2)=7.558, P=0.006) . Eltrombopag was well tolerated, the common adverse events included elevated transaminase (4 cases) and serum bilirubin (4 cases) ; mild nausea (1 case) , vomiting (1 case) and dizziness (1 case) . No drug withdrawal occurred due to adverse events. Conclusion: Eltrombopag is safe and effective in pediatric patients with primary ITP.
目的： 评估艾曲泊帕治疗儿童原发免疫性血小板减少症（ITP）的疗效和不良反应。 方法： 回顾性分析2015年5月至2019年3月接受艾曲泊帕治疗的23例儿童ITP患者临床资料。 结果： 23例患者中，男11例，女12例，中位年龄11（2~17）岁。新诊断ITP 4例（17.39%），持续性ITP 8例（34.78%），慢性ITP 11例（47.83%）。艾曲泊帕中位起始剂量为25.0（12.5~50.0）mg/d。基线中位血小板计数为14（2~82）×10(9)/L，治疗后第2周（23例）、第4周（23例）、第3个月（16例）、第6个月（11例）的中位血小板计数分别为40（4~170）×10(9)/L、20（4~130）×10(9)/L、60（4~110）×10(9)/L、70（18~160）×10(9)/L，均高于基线水平（z=-3.440，P=0.001；z=-1.964，P=0.049；z=-4.339，P<0.001；z=-5.794，P<0.001）。7例（30.43%）患者的起效时间≤7 d，10例（43.48%）患者的起效时间≤2周，总有效率为60.87%（14/23）。<6岁（6例）、6~12岁（10例）、13~17岁（7例）组的总有效率分别为33.33%（2/6）、60.00%（6/10）、85.71%（6/7），组间比较差异均无统计学意义（P>0.05）。23例患者基线WHO出血评分0、1、2级分别有4、12、7例，治疗后分别为13、7、3例，出血症状得到明显改善（χ(2)=7.558，P=0.006）。治疗过程中发生转氨酶、胆红素增高各4例，轻度恶心、呕吐及头晕各1例，对治疗均未造成影响。 结论： 艾曲泊帕作为二线用药治疗儿童ITP疗效及耐受性均较好。.
Keywords: Child; Drug toxicity; Eltrombopag; Thrombocytopenia; Treatment outcome.