Curative effect and follow-up analysis of 15 children with refractory systemic lupus erythematosus treated with Belimumab
10.3760/cma.j.cn101070-20220405-00363
- VernacularTitle:贝利尤单抗治疗儿童难治性系统性红斑狼疮15例的疗效及预后随访研究
- Author:
Xiaohua TAN
1
;
Caifeng LI
;
Wenjia ZHAO
;
Weiying KUANG
;
Jianghong DENG
;
Junmei ZHANG
Author Information
1. 国家儿童医学中心，首都医科大学附属北京儿童医院风湿科，北京 100045
- Keywords:
Childhood systemic lupus erythematosus;
Belimumab;
Safety;
Efficacy
- From:
Chinese Journal of Applied Clinical Pediatrics
2022;37(13):983-987
- CountryChina
- Language:Chinese
-
Abstract:
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, and Cyclophosphamide and/or Tripterygium Wilfordii were used at the same time according to the damage of target organs.The drug safety after intravenous injection of Belizumab showed that one patient in this group had respiratory tract infection symptoms 4 weeks after treatment; Another patient had a slight increase of alanine aminotransferase 8 weeks after treatment, and recovered to normal symptomatic treatment.No drug-related adverse reactions were found in the other 13 patients.After 4 weeks of treatment, the score of SLEDAI-2000 and PGA compared with the baseline level, and the difference was statistically significant (SLEDAI-2000 P=0.002; PGA P=0.006). There was no clinical recurrence.One patient with familial chilblain like lupus erythematosus showed significant improvement in rash 2 weeks after treatment, and low fever accompanied by increased rash 8 weeks after treatment; After 16 weeks of treatment, the body temperature was normal and the rash basically subsided. Conclusions:Belimumab is clinically effective in the treatment of refractory childhood SLE, with no serious adverse events reported.However, its long-term efficacy and safety need to be further studied by multi-center and long-term research with a large sample size.
