小样本应用贝利尤单抗治疗儿童系统性红斑狼疮的临床疗效观察

Clinical efficacy of beribizumab in a small sample of children with systemic lupus erythematosus

目的研究贝利尤单抗治疗儿童系统性红斑狼疮(SLE)的临床疗效。方法回顾性、真实世界临床研究分析2021年1月至2022年8月在河南省人民医院明确诊断并应用贝利尤单抗16周治疗的儿童SLE临床资料。结果共纳入11例患儿,发病年龄(13.91±1.70)岁;男1例,女10例;应用贝利尤单抗治疗时中位病程为(8.91±10.41)个月。治疗后患儿全身症状、皮肤黏膜、各系统受累比例下降,补体C3、C4恢复正常,抗ds-DNA定量下降,狼疮肾炎患儿24 h尿蛋白定量下降。治疗前后泼尼松剂量差异有统计学意义(P<0.01)。贝利尤单抗治疗的不同时间点(0、2、4、8、12、16周)的儿童SLE疾病活动指数(SLEDAI-2000)和医师整体评估(PGA)差异有统计学意义(P<0.05),全部病例在第16周获得SLE应答者指数(SRI-4)反应。患儿接受贝利尤单抗治疗期间,2例(18.18%)出现不良事件,1例治疗2周后出现血红蛋白及血小板下降,治疗12周时恢复正常;1例出现上呼吸道感染。结论贝利尤单抗治疗儿童SLE临床有效,减少糖皮质激素使用剂量,不良事件发生率低。

Objective To study the clinical effect of belimumab on children with systemic lupus erythematosus.Methods A retrospective,real-world clinical study was conducted to analyze the clinical data of CSLE patients who were diagnosed and treated with belimumab for 16 weeks in Henan Province People's Hospital from January 2021 to August 2022.Results A total of 11 children(1 boy and 10 girls)were enrolled,and the average age was(13.91±1.70)years.The median course of disease on receiving belimumab was(8.91±10.41)months.After treatment,the proportion of systemic symptoms,skin and mucous membrane,and other system involvement decreased;C3 and C4 returned to normal,the anti-double-stranded DNA(dsDNA)quantitation decreased,and the urinary protein quantitation of 24 hours decreased in children with lupus nephritis.The dosage of prednisone before and after treatment was statistically different(P<0.01).The difference in systemic lupus erythematosus disease activity index(SLEDAI-2000)and physician's global assessment(PGA)were statistically significant at different time points(0,2,4,8,12,16 weeks)after belimumab treatment(P<0.05).All patients achieved SRI-4 response at 16 weeks.Adverse events occurred in 2 patients(18.18%)during belimumab treatment;hemoglobin and platelet decreased in 1 case after 2 weeks of treatment;and returned to normal after 12 weeks of treatment;1 case had upper respiratory infection.Conclusion Belimumab is effective in the treatment of CSLE with low incidence of adverse events,and the dose of glucocorticoid is reduced.