摘要
目的分析并总结沙利度胺治疗难治性全身型幼年特发性关节炎的有效性及安全性。方法回顾性分析2015年1月至2022年3月河北省儿童医院肾脏免疫科收治的10例难治性全身型幼年特发性关节炎患儿的病例资料,分析沙利度胺治疗难治性全身型幼年特发性关节炎的临床表现、疗效及安全性。采用全身型幼年关节炎疾病活动度评分评价治疗效果。采用一般线性模型重复测量进行统计分析。结果10例难治性全身型幼年特发性关节炎患儿中,男4例,女6例,发病年龄(7.5±3.3)岁。7例患儿病初合并巨噬细胞活化综合征。10例患儿平均病程(4.4±1.7)年,最长病程8.3年。10例患儿在应用沙利度胺前均有复发(2~10次不等)。10例患儿应用沙利度胺治疗第6、12个月与治疗前相比,外周血白细胞[(10.19±3.67)×10^(9)/L、(8.53±2.83)×10^(9)/L比(16.11±7.81)×10^(9)/L,F=7.918、11.084,P=0.020、0.009]、C反应蛋白[19.13(0.38,35.21)mg/L、8.05(0.10,18.00)mg/L比59.34(24.20,131.90)mg/L,F=7.030、12.731,P=0.026、0.006]、全身型幼年关节炎疾病活动度评分显著下降[6.00(1.50,12.50)分、3.00(0,12.50)分比20.00(11.50,28.00)分,F=14.710、17.870,P=0.004、0.002];泼尼松剂量明显减少[0.13(0,0.45)mg/(kg·d)、0.02(0,0.06)mg/(kg·d)比0.42(0.16,1.47)mg/(kg·d),F=5.890、7.632,P=0.041、0.022],差异均有统计学意义。7例成功停用泼尼松,3例逐渐脱离托珠单抗,1例可延长托珠单抗给药间隔时间。10例患儿均未发生严重不良反应。结论沙利度胺治疗难治性全身型幼年特发性关节炎临床有效,可以减少泼尼松所需剂量及延长托珠单抗给药间隔时间。
ObjectiveTo analyze and summarize the efficacy and safety of thalidomide in the treatment of refractory systemic juvenile idiopathic arthritis(sJIA).MethodsThe clinical data of ten patients with refractory sJIA admitted to Department of Nephrology and Immunology in Children's Hospital of Hebei Province from January 2015 to March 2022 were collected,and the clinical manifestations,efficacy and safety of thalidomide in the treatment of refractory sJIA were analyzed retrospectively.Systemic juvenile arthritis disease activity score(sJADAS)was used to evaluate the efficacy of the treatment.Statistical analysis was performed by repeated measurements using general linear models.ResultsAmong the 10 children(4 males and 6 females)with refractory sJIA,the average age of onset was(7.5±3.3)years.Seven patients were complicated with macrophage activation syndrome at an early stage of disease.The average course of disease was(4.4±1.7)years,and the longest course of disease was 8.3 years.Before the application of thalidomide,all the 10 children experienced relapses(ranging from 2 to 10 times).The indices of 10 children treated with thalidomide at 6 months and 12 months were compared with those before treatment.Peripheral blood leukocytes[(10.19±3.67)×10^(9)/L,(8.53±2.83)×10^(9)/L vs.(16.11±7.81)×10^(9)/L,F=7.918,11.084,P=0.020,0.009],C-reactive protein[19.13(0.38,35.21)mg/L,8.05(0.10,18.00)mg/L vs.59.34(24.20,131.90)mg/L,F=7.030,12.731,P=0.026,0.006],sJADAS scores[6.00(1.50,12.50)scores,3.00(0,12.50)scores vs.20.00(11.50,28.00)scores,F=14.710,17.870,P=0.004,0.002]were decreased significantly.The doses of prednisone[0.13(0,0.45)mg/(kg·d),0.02(0,0.06)mg/(kg·d)vs.0.42(0.16,1.47)mg/(kg·d),F=5.890,7.623,P=0.041,0.022]were significantly decreased.All the differences were statistically significant.Prednisone was successfully discontinued in 7 cases.Tocilizumab was gradually withdrawn in 3 cases,and tocilizumab administration interval was prolonged in 1 case.None of the 10 children had serious adverse reactions.ConclusionThalidomide is clinically effective in the treatment of sJIA,and can reduce the required dose of prednisone and prolong the tocilizumab free remission.
作者
陈新
房军臣
郭敬肖
葛兰兰
刘福娟
韩佩桐
刘玲
Chen Xin;Fang Junchen;Guo Jingxiao;Ge Lanan;Liu Fujuan;Han Peitong;Liu Ling(Department of Nephrology and Immunology,Children's Hospital of Hebei Province,Shijiazhuang 050000,China)
出处
《国际儿科学杂志》
2024年第2期132-137,共6页
International Journal of Pediatrics
基金
河北省卫生健康委员会青年科技课题(20211010)。