摘要
目的分析15例应用贝利尤单抗治疗的儿童难治性系统性红斑狼疮(SLE)患儿的转归,初步评价该药的安全性和有效性。方法采用回顾性、真实世界临床研究方法,选取2020年4月1日至2022年3月31日北京儿童医院风湿科明确诊断并完整随访的难治性SLE患儿15例为研究对象。通过对比不同治疗时期(治疗前、治疗4周、8周、12周、6个月和12个月)患儿的临床症状、辅助检查结果、SLE疾病活动指数(SLEDAI-2000)、医师整体评估(PGA)评分的变化情况及不良事件发生情况,判断贝利尤单抗治疗的安全性和有效性。其中计数资料采用百分比表示,计量资料满足正态分布的采用±s表示,计量资料两样本比较采用t检验,P<0.05为差异有统计学意义。结果患儿男女比例为3∶2,起病年龄(7.93±4.99)岁;基础治疗时间4个月~5年1个月;以狼疮性肾炎(LN)为主要靶器官损害者8例,持续1年以上低补体血症为主者2例,中枢神经系统受累为主者2例;合并抗磷脂综合征2例;婴幼儿早发型SLE 1例。其中1例LN患者合并神经精神性狼疮及双膝关节股骨头远端梗死、3例合并腰椎压缩性骨折及髋关节梗死。患儿均经规范传统治疗诱导缓解3个月及以上,维持期存在疾病活动度轻-中度活动,糖皮质激素减药困难。应用贝利尤单抗基线时,SLEDAI-2000评分4~13分;PGA评分1~2.50分。基础治疗包括糖皮质激素联合免疫抑制剂(环孢素、吗替麦考酚酯、来氟米特片)和抗疟药,依据靶器官损伤情况不同,同时应用环磷酰胺和/或雷公藤治疗。接受静脉注射贝利尤单抗后的药物安全性显示,本研究患儿中有1例治疗后4周出现呼吸道感染症状;另外1例患儿于治疗后8周出现谷丙转氨酶轻度升高,分别给予对症治疗后恢复正常。余13例患儿未发现与药物相关的不良反应。治疗后4周,SLEDAI-2000与PGA评分较基线水平改善,差异有统计学意义(SLEDAI-2000P=0.002;PGAP=
Objective To analyze the outcome of 15 cases with refractory systemic lupus erythematosus(SLE)treated with Belimumab,and evaluate the safety and efficacy of the therapy.Methods A retrospective and real-world clinical research method was adopted.Fifteen children with confirmed refractory SLE and complete follow-up data were selected from the Department of Rheumatology,Beijing Children′s Hospital from April 1,2020 to March 31,2022.By comparing the changes of clinical symptoms,auxiliary examination results,SLE disease activity index(SLEDAI-2000)and Physician′s Global Assessment(PGA)scores as well as adverse events in different treatment periods(before treatment,4 weeks,8 weeks,12 weeks,6 months and 12 months after treatment),the safety and effectiveness of Belimumab treatment were all recorded.The counting data was expressed in percentage,the measurement data meeting the normal distribution was expressed in Mean±SD,and the two samples of measurement data were compared by t-test,P<0.05 means significant differences.Results The ratio of male to female was 3∶2,and the onset age was(7.93±4.99)years;The basic treatment time was 4 months to 5 years and 1 month.There were 8 cases with lupus nephritis(LN),2 cases suffering from hypocomplementemia for more than 1 year,2 cases with central nervous system involvements,2 cases complicated with antiphospholipid syndrome and 1 case with early-onset SLE.Of 8 LN cases,1 case was complicated with neuropsychiatric lupus(NPLE)and distal femoral head infarction of both knees,and 3 cases were complicated with lumbar compression fractures and hip infarction.All patients were treated with regular traditional therapy to induce remission.During the maintenance period,the disease activity maintained at light to moderate levels,and it was difficult to reduce glucocorticoid.At baseline,SLEDAI-2000 score was 4-13,and PGA score was 1-2.50.Basic treatment includes glucocorticoids combined with immunosuppressants(Cyclosporine,Mycophenolate Mofetil,Leflunomide tablets)and antimalarial drugs
作者
檀晓华
李彩凤
赵文甲
邝伟英
邓江红
张俊梅
Tan Xiaohua;Li Caifeng;Zhao Wenjia;Kuang Weiying;Deng Jianghong;Zhang Junmei(Department of Rheumatology,Beijing Children′s Hospital,Capital Medical University,National Center for Children′s Health,Beijing 100045,China)
出处
《中华实用儿科临床杂志》
CAS
CSCD
北大核心
2022年第13期983-987,共5页
Chinese Journal of Applied Clinical Pediatrics
