Department of Nephrology, People's Hospital of Guizhou Province, Guiyang, China.
Division of Nephrology, West China Hospital of Sichuan University, Chengdu, China.
Medicine (Baltimore). 2023 Aug 25;102(34):e34800. doi: 10.1097/MD.0000000000034800.
Therapy of childhood-onset systemic lupus erythematosus (cSLE) with drugs is unsatisfactory. Some new drugs such as belimumab and rituximab may improve the course of severe cSLE, although there are few reports on treatment efficiency for these new drugs, especially belimumab.
Here we report on a 16-year-old girl who was diagnosed with cSLE at the age of 13. After several immunosuppressive treatments, which included high-dose steroids, hydroxychloroquine sulfate, cyclophosphamide, etc for blood system damage, she showed little clinical improvement and developed severe pericarditis. Induction treatment with a combination of intravenous high-dose steroids, methylprednisolone, and cyclophosphamide was started, but, after 55 days, the patient developed lupus encephalopathy, lung infection, and lupus nephritis. After using high-dose steroids, cyclophosphamide, plasma exchange, gamma globulin, and appropriate anti-pulmonary inflammation drugs, treatment with tacrolimus was attempted but poorly tolerated by the patient and withdrawn. Eventually, in December 2019, belimumab was initiated on an off-label basis as a last resource to treat lupus nephritis. Belimumab was well tolerated by the patient and resulted in a rapid and marked improvement in clinical symptoms and reduction in proteinuria, serum complement levels and anti-double strand DNA antibodies titer; of note, the patient developed no infectious complications.
Treatment with belimumab could result in prompt remission of severe cSLE with multiple organ damage without the pulmonary infection side effects for children deemed intolerant to conventional and second-line induction therapies. Belimumab should be considered as a potentially efficacious treatment in patients in severe childhood-onset systemic lupus erythematosus.
儿童发病的系统性红斑狼疮（cSLE）的药物治疗效果并不令人满意。一些新药，如贝利尤单抗和利妥昔单抗，可能改善严重 cSLE 的病程，尽管这些新药的治疗效果报告很少，尤其是贝利尤单抗。
我们在此报告一例 16 岁女孩，她在 13 岁时被诊断为 cSLE。在接受了包括大剂量类固醇、硫酸羟氯喹、环磷酰胺等多种免疫抑制治疗后，她的血液系统损伤有所改善，但临床改善甚微，并出现严重心包炎。随后开始采用静脉注射大剂量类固醇、甲泼尼龙和环磷酰胺联合诱导治疗，但在 55 天后，患者出现狼疮性脑病、肺部感染和狼疮性肾炎。在使用大剂量类固醇、环磷酰胺、血浆置换、丙种球蛋白和适当的抗炎药物治疗后，尝试使用他克莫司治疗，但患者耐受性差并停药。最终，在 2019 年 12 月，作为治疗狼疮性肾炎的最后手段，基于超适应证使用贝利尤单抗。患者对贝利尤单抗耐受良好，导致临床症状迅速显著改善，蛋白尿、血清补体水平和抗双链 DNA 抗体滴度降低；值得注意的是，患者没有发生感染并发症。
对于不耐受传统和二线诱导治疗的儿童，使用贝利尤单抗治疗可能会迅速缓解多器官损伤的严重儿童发病的系统性红斑狼疮，且不会产生肺部感染副作用。贝利尤单抗应被视为治疗严重儿童发病的系统性红斑狼疮的潜在有效治疗方法。
