摘要
目的观察持续性/慢性原发性免疫性血小板减少症(immune thrombocytopenia,ITP)患儿应用艾曲泊帕二线治疗的效果和安全性,探讨其对患儿及患儿父母生活质量的改善情况。方法持续性/慢性ITP患儿24例,均应用艾曲泊帕二线治疗,疗程>12周。艾曲泊帕起始剂量根据患儿年龄、体质量制订,治疗期间定期检测血常规,根据血小板计数调整艾曲泊帕剂量,维持血小板计数≥30×10^(9)/L。若治疗期间血小板计数下降至<10×10^(9)/L和/或发生活动性出血,给予地塞米松和/或丙种球蛋白对症处理。治疗1、4、12周评估疗效;比较治疗前及治疗1、4、12周血小板计数;比较治疗前及治疗4、12周出血评分;治疗前及治疗12周应用KIT量表评估患儿及父母的健康相关生活质量(health-related quality of life,HRQoL);记录治疗期间不良反应发生情况。随访至2022年10月,记录患儿药物应用情况,评价治疗效果。结果24例患儿治疗1周时有效率为50.0%,治疗4周时有效率为75.0%,治疗12周时有效率为79.2%;2例分别于治疗8、10周发生肝功能异常,均经保肝处理后恢复,无因不良反应停药者。治疗1、4、12周血小板计数[41.5(29.0,60.5)×10^(9)/L、69.0(38.0,105.5)×10^(9)/L、86.0(63.5,176.5)×10^(9)/L]均高于治疗前[18.5(7.5,25.0)×10^(9)/L](P<0.05);治疗12周血小板计数高于治疗1周(Z=-2.683,P=0.044),治疗4周血小板计数与治疗1、12周比较差异均无统计学意义(Z=-2.460,P=0.083;Z=-0.224,P>0.999)。治疗4、12周时出血症状评分[0(0,2)、0(0,2)分]均低于治疗前[2(1,3)分](Z=157.5,P=0.005;Z=159.5,P=0.006)。治疗12周患儿HRQoL总分[(85.1±10.1)分]与治疗前[(78.9±12.5)分]比较差异均无统计学意义(t=-1.867,P=0.068),父母HRQoL总分[(53.6±14.6)分]高于治疗前[(34.8±15.2)分](t=-4.354,P<0.001)。中位随访时间59.5(38.7,72.0)个月,16例未停药者中9例完全反应,7例有效,持续减量中;8例停药者中停药13、50周各1例持续完全�
Objective To observe the efficacy and safety of the second-line treatment of eltrombopag in children with persistent/chronic primary immune thrombocytopenia(ITP),and to investigate its role in improving the quality of life of ITP children and their parents.Methods Twenty-four patients with persistent/chronic ITP were administrated with eltrombopag for 12 weeks,and the initial dose was based on the age and body mass of the children.During the treatment,the blood routine examination was done regularly,and the dose of eltrombopag was adjusted till the platelet count was≥30×10^(9)/L.If the platelet count was 10×10^(9)/L and/or active bleeding occurred during treatment,the symptomatic management with dexamethasone and/or gamma globulin was given.The treatment response was assessed after 1-,4-and 12-week treatment,and the platelet counts were compared before treatment,and after 1-,4-and 12-week treatment.The bleeding scores were compared before treatment and after 4-and 12-week treatment,respectively.The health-related quality of life(HRQoL)of the children and their parents were assessed by KIT assessment scale before treatment and after 12-week treatment.The incidence of adverse reactions during treatment was recorded.The patients were followed up till October 2022to record the application of eltrombopag and to evaluate the treatment response.Results The effective rate was 50.0%after 1-week treatment,75.0%after 4-week treatment,and 79.2%after 12-week treatment,respectively.Hepatic dysfunction occurred after 8-week treatment in 1patient and after 10-week treatment in 1 patient,and both two children recovered after hepatic protection treatment,with no drug withdrawal due to adverse reactions.The platelet count was higher after 1-,4-and 12-week treatment[41.5(29.0,60.5)×10^(9)/L,69.0(38.0,105.5)×10^(9)/L,86.0(63.5,176.5)×10^(9)/L]than that before treatment[18.5(7.5,25.0)×10^(9)/L](P0.05),was higher after 12-week treatment than that after 1-week treatment(Z=-2.683,P=0.044),and showed no significant difference afte
作者
徐岩
王春美
李白
苏淑芳
魏林林
刘莹
刘玉峰
XU Yan;WANG Chun-mei;LI Bai;SU Shu-fang;WEI Lin-lin;LIU Ying;LIU Yu-feng(Department of Pediatric Hematology and Oncology,the First Affiliated Hospital of Zhengzhou University,Zhengzhou,Henan 450052,China)
出处
《中华实用诊断与治疗杂志》
2023年第3期313-317,共5页
Journal of Chinese Practical Diagnosis and Therapy
基金
河南省自然科学基金青年项目(202300410403)。
