摘要
目的:通过对国产重组人血小板生成素(rhTPO)治疗3岁以下婴幼儿重症原发免疫性血小板减少症(SITP)的临床资料进行分析,为该年龄段SITP患儿临床联合应用rhTPO的有效性及安全性提供依据。方法:采用回顾性方法收集3岁以下联合应用rhTPO治疗的SITP患儿治疗前后相关临床数据并进行比较,分析其安全性及有效性。结果:①共有55例3岁以下SITP患儿纳入本研究,其中男37例,女18例;中位月龄为19(1.37~34.50)个月;中位病程为0.67(0~12)个月;41例(74.5%)为新诊断的ITP,11例(20.0%)为持续性ITP,3例(5.5%)为慢性ITP。患儿基线血小板计数为6(1~9)×10^9/L。出血情况:Ⅱ级42例,Ⅲ级11例,Ⅳ级2例。②患儿均为联合用药,其中10例rhTPO单独联合丙种球蛋白,45例同时联合激素及丙种球蛋白。③给药后第1天、第3天、第7天、第14天及停药后第7天,血小板中位计数分别为17(0~121)×10^9/L、63(1~349)×10^9/L、91(4~996)×10^9/L、176(11~1687)×10^9/L及129(1~697)×10^9/L。在给药第7天、第14天及停药后第7天,患儿总有效率分别达76.0%、90.2%、73.9%。④在联合应用rhTPO第3天有6例患儿出血评分降为0级,第7天有11例患儿出血降为0级,其余患儿在停药7天出血降为0级。⑤未观察到发热、皮疹等与rhTPO相关不良反应发生。结论:rhTPO可协同免疫治疗,迅速有效提高婴幼儿SITP患儿血小板水平,帮助其度过危险期,有望提高疗效。
Objective:To explore the efficacy and safety of recombinant human thrombopoietin(rhTPO)-based combination treatment in children under 3 years old with severe immune thrombocytopenia(SITP).Method:A retrospective analysis was made.We analyzed the clinical data of children under 3 years old with SITP who received combination treatment of rhTPO and other drugs.Result:①A total of 55 cases were enrolled in this study,37 cases were male and 18 cases were female.The median age was 19(1.37-34.50)months,the median course was 0.67(0-12)months.Among these 55 patients,41 cases(74.5%)were newly diagnosed ITP,11 cases(20.0%)were persistent ITP,and 3 cases(5.5%)were chronic ITP.The median platelet count was 6(1-9)×10^9/L before treatment.For bleeding complication,42 cases gradedⅡ,11 cases gradedⅢ,2 cases gradedⅣ.②All of the 55 patients were treated with rhTPO combined with other drugs.Among them,10 cases were combined with intravenous immune globulin(IVIG),and 45 cases were combined with IVIG and glucocorticoids.(3)The median platelet count reached 17(0-121)×10^9/L,63(1-349)×10^9/L,91(4-996)×10^9/L,176(11-1687)×10^9/L and 129(1-697)×10^9/L on 1 days,3 days,7 days and 14 days after rhTPO treatment and 7 days after rhTPO withdrawal,respectively.There were significantly differences of platelet counts between before and after treatment(P<0.01).The total response rate was 76.0%,90.2%,73.9%on 7 days and 14 days after rhTPO treatment and 7 days after rhTPO withdrawal,respectively.(4)The bleeding grade of 6 patients declined to 0 on 3 days after rhTPO treatment,11 patients on 7 days after rhTPO treatment,and the rest patients on 7 days after rhTPO withdrawal.(5)No adverse events related to rhTPO were observed during the whole study.Conclusion:As for children under 3 years old with SITP,rhTPO combined with immunotherapy can be used to increase their platelet count effectively and rapidly,help them through the danger phase smoothly,and improve their curative efficacy.
作者
胡玉
马洁
马静瑶
张利强
张元元
马宏浩
姚佳峰
张蕊
吴润晖
HU Yu;MA Jie;MA Jingyao;ZHANG Liqiang;ZHANG Yuanyuan;MA Honghao;YAO Jiafeng;ZHANG Rui;WU Runhui(Beijing Key Laboratory of Pediatric Hematology Oncology,National Key Discipline of Pediatrics,Capital Medical University,Key Laboratory of Major Diseases in Children,Ministry of Education,Hematology Oncology Center,Beijing Children's Hospital,Capital Medical University,National Center for Children's Health,Beijing,100045,China)
出处
《临床血液学杂志》
CAS
2020年第2期166-169,175,共5页
Journal of Clinical Hematology
基金
北京市自然科学基金课题(No:7192064)
国家自然科学基金课题(No:81970111)
北京市医院管理局儿科学科协同发展中心“儿科专项”(No:XTZD20180205)
国家科技重大专项(No:2017ZX09304029004).
