贝利尤单抗治疗儿童系统性红斑狼疮的有效性及安全性分析

Efficacy and safety of belimumab treatment in childhood-onset systemic lupus erythematosus

目的:评估贝利尤单抗在儿童系统性红斑狼疮(cSLE)患儿中的有效性与安全性。方法:获取2018年8月至2021年12月在首都儿科研究所附属儿童医院风湿免疫科住院治疗的49例cSLE患儿临床资料,根据是否接受贝利尤单抗治疗将患儿分为贝利尤治疗组(18例)和传统治疗组(31例),传统治疗组患儿为在本院基线系统性红斑狼疮的病情活动程度(SLEDAI)评分相似、应用传统免疫抑制药物治疗的cSLE患儿。比较2组患儿治疗前后临床症状、改善情况以及随访过程中不良事件发生的差异。结果:18例贝利尤治疗组患儿年龄为(11.9±1.8)岁,病程 M( Q1, Q3)为7.5(2.0,16.8)个月,其中男3例,共有15例完成24周治疗;传统治疗组cSLE患儿31例,年龄为(11.3±2.4)岁,病程 M( Q1, Q3)为7.0(2.5,10.5)个月,男6例,共有25例完成24周治疗。基线时,贝利尤治疗组口服泼尼松剂量低于传统治疗组[(29.58±12.43)mg/d比(38.20±14.11)mg/d, P=0.037];治疗24周时,2组泼尼松剂量均减少(均 P<0.05),贝利尤治疗组泼尼松剂量为(14.12±5.86)mg/d,低于传统治疗组(23.51±9.79)mg/d( P=0.002)。治疗24周时,2组患儿补体C3、C4水平升高,抗双链DNA(dsDNA)水平和SLEDAI评分降低,但两组间差异均无统计学意义(均 P>0.05);贝利尤治疗组患儿不良事件发生率(3/15,3例)比传统治疗组患儿(32.0%,8/25)低( P>0.05)。 结论:贝利尤单抗治疗cSLE能够减少泼尼松初始剂量并利于其减量,明显改善临床症状及脏器受累情况,降低疾病活动度。贝利尤单抗治疗不良事件发生率低。

Objective To evaluate the efficacy and safety of belimumab in children with systemic lupus erythematosus(cSLE).Methods The clinical data of 49 cSLE patients admitted into the Department of Rheumatology and Immunology,Children′s Hospital affiliated to the Capital Institute of Pediatrics,from August 2018 to December 2021 were collected.They were divided into the belimumab treatment group(18 cases)and the traditional treatment group(31 cases)according to whether they received belimumab treatment.The cSLE patients in the traditional group had similar baseline SLEDAI scores with the belimumab group and were treated with traditional immunosuppressive drugs.The clinical symptoms and improvements before and after treatment,as well as the differences in adverse events during follow-up were compared between the two groups.Results In belimumab group,the age was(11.9±1.8)years old,the median(Q1,Q3)of disease duration was 7.5(2.0,16.8)months,3 patients were male,and 15 patients completed 24 weeks of treatment.In the traditional treatment group,31 patients with cSLE were enrolled,with an average age of(11.3±2.4)years,a median(Q1,Q3)of disease duration of 7.0(2.5,10.5)months,among whom 6 patients were male,and 25 patients completed 24 weeks of treatment.At baseline,the belimumab treatment group had a significantly lower oral prednisone dose than the conventional treatment group[(29.58±12.43)mg/d vs(38.20±14.11)mg/d,P=0.037].After 24 weeks of treatment,the dosage of prednisone in both groups was reduced,and the dosage of prednisone in the belimumab group was(14.12±5.86)mg/d,which was lower than that in the traditional treatment group[(23.51±9.79)mg/d](P=0.002).After 24 weeks of treatment,the levels of complement C3 and C4 increased,the dsDNA levels and SLEDAI score decreased in both groups(all P>0.05).The incidence of adverse events in belimumab group(3/15,3 cases)was lower than that in traditional treatment group(32.0%,8/25)(P>0.05).Conclusions Belimumab in the treatment of cSLE can reduce the initial dose of prednisone and facilitate the reduction of prednisone dose,significantly improve the clinical symptoms and organ involvement,and reduce the disease activity.The incidence of adverse events was low during belimumab treatment.