Huang Y T, Liu X F, Chen Y F, Fu R F, Liu W, Xue F, Zhang L, Yang R C
Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Tianjin 300020, China (Huang Yueting is working on the Department of Hematology, the First Affiliated Hospital of Xiamen University, Xianmen 361003, China).
Institute of Hematology & Blood Diseases Hospital, CAMS & PUMC, State Key Laboratory of Experimental Hematology, National Clinical Research Center for Blood Diseases, Tianjin 300020, China.
Zhonghua Xue Ye Xue Za Zhi. 2019 Dec 14;40(12):1031-1034. doi: 10.3760/cma.j.issn.0253-2727.2019.12.012.
To evaluate the efficacy and safety of eltrombopag in the treatment of pediatric primary immune thrombocytopenia (ITP) . 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. 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 (=-3.440, =0.001; =-1.964, =0.049; =-4.339, <0.001;=-5.794, <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 ((2)=7.558, =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. Eltrombopag is safe and effective in pediatric patients with primary ITP.
评估艾曲泊帕治疗儿童原发性免疫性血小板减少症（ITP）的疗效和安全性。回顾性分析2015年5月至2019年3月期间接受艾曲泊帕治疗的23例儿童ITP患者的临床特征。艾曲泊帕起始剂量为12.5 - 50.0mg/d，最大剂量为75.0mg/d。23例患儿中，男11例，女12例，中位年龄11.0（2.0 - 17.0）岁。新诊断ITP 4例，持续性ITP 8例，慢性ITP 11例。艾曲泊帕应用时间为4.5至95周（23例中有8例仍在用药）。治疗后2周、4周、3个月及6个月时血小板（PLT）计数中位数分别为40（4 - 170）×10⁹/L、20（4 - 130）×10⁹/L、60（4 - 110）×10⁹/L和70（18 - 160）×10⁹/L，均显著高于治疗前的14（2 - 82）×10⁹/L（分别为Z = - 3.440，P = 0.001；Z = - 1.964，P = 0.049；Z = - 4.339，P < 0.001；Z = - 5.794，P < 0.001）。总有效率为60.87%（14/23例）。PLT计数≥30×10⁹/L的中位时间为10.5（3 - 42）天。7例患者（30.43%）在第一周内有反应，10例患者（43.48%）在2周内PLT计数≥30×10⁹/L。所有患者按年龄分为三组（<6岁、6 - 12岁、13 - 17岁）。三组有效率相似，分别为33.33%、60.00%、85.71%。治疗前WHO出血评分为0、1、2分的患者分别为4例、12例和7例。治疗后出血评分为0、1、2分的患者人数分别变为13例、7例、3例（χ² = 7.558，P = 0.006）。艾曲泊帕耐受性良好，常见不良事件包括转氨酶升高（4例）和血清胆红素升高（4例）；轻度恶心（1例）、呕吐（1例）和头晕（1例）。无因不良事件停药情况。艾曲泊帕治疗儿童原发性ITP安全有效。
