Sheng Ai-Qin, Liu Fei, Li Qiu-Yu, Dou Ya-Lan, Zhang Xiao-Jing, Zhao Jing-Li, Huang Ling-Fei, He Si-Yi, Lu Zhi-Hong, Feng Chun-Yue, Wang Jing-Jing, Shen Hui-Jun, Fu Hai-Dong, Yan Wei-Li, Mao Jian-Hua
Department of Nephrology, Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, China.
Department of Clinical Epidemiology & Clinical Trial Unit, Children's Hospital of Fudan University, National Children's Medical Center, Shanghai, China.
Ren Fail. 2025 Dec;47(1):2499902. doi: 10.1080/0886022X.2025.2499902. Epub 2025 May 6.
Nephrotic syndrome (NS) is a common form of glomerular disease in children, characterized by a high propensity for relapse. Prolonged use of glucocorticoids (GCs) can result in various side effects. Rituximab (RTX) may be considered as an initial treatment option for primary nephrotic syndrome in pediatric patients.
We conducted a prospective, single-center, randomized controlled trial (RCT) to evaluate whether the initial use of RTX monotherapy is superior to GC therapy in treating pediatric idiopathic NS and to assess its safety. The primary outcome and secondary outcomes were compared between the two groups.
A total of 24 pediatric patients were included in the study, comprising 19 males and 5 females. After six weeks of treatment, the complete remission (CR) rate in the GC group was significantly higher than that in the RTX group (100% vs 33.3%). Compared with the RTX group, the GC group had a shorter time to first remission (14.25 d vs. 9.5 d). During the follow-up period, none of the patients in the RTX group who achieved CR experienced relapse, with the longest relapse-free duration being 79 weeks. In the GC group, nine patients experienced relapse with the longest relapse-free period being 94 weeks. No serious adverse events occurred in either group. The cumulative steroid dosage was not statistically different between the 2 groups ( = 0.41).
Although the CR rate of children with idiopathic NS treated with RTX alone is significantly lower, the relapse rate among responders to RTX is also lower than that of the GC treatment.
肾病综合征（NS）是儿童常见的肾小球疾病形式，其特点是复发倾向高。长期使用糖皮质激素（GCs）会导致各种副作用。利妥昔单抗（RTX）可被视为小儿原发性肾病综合征的初始治疗选择。
我们进行了一项前瞻性、单中心、随机对照试验（RCT），以评估初始使用RTX单药治疗在治疗小儿特发性NS方面是否优于GC治疗，并评估其安全性。比较两组的主要结局和次要结局。
该研究共纳入24例儿科患者，其中男性19例，女性5例。治疗六周后，GC组的完全缓解（CR）率显著高于RTX组（100%对33.3%）。与RTX组相比，GC组首次缓解的时间更短（14.25天对9.5天）。在随访期间，RTX组中达到CR的患者均未复发，最长无复发持续时间为79周。在GC组中，9例患者复发，最长无复发期为94周。两组均未发生严重不良事件。两组间累积类固醇剂量无统计学差异（=0.41）。
尽管单独使用RTX治疗的特发性NS患儿的CR率显著较低，但RTX反应者的复发率也低于GC治疗。
