Yu S S, Liu J L, Liu J J, Fang X Y, Chen J, Miao Q F, Tang X S, Zhang Z Q, Wang C Y, Dai R F, Han X L, Zhai Y H, Xu H, Shen Q
Department of Nephrology, Children's Hospital of Fudan University, National Children's Medical Center,Shanghai Kidney Development and Pediatric Kidney Disease Research Center State, Key Laboratory of Kidney Diseases, Shanghai 201102, China.
Zhonghua Er Ke Za Zhi. 2025 Feb 2;63(2):185-189. doi: 10.3760/cma.j.cn112140-20240714-00482.
To investigate the short-term efficacy and safety of rituximab (RTX) in children with calcineurin inhibitor (CNI) resistant steroid resistant nephrotic syndrome (SRNS). A retrospective case analysis was conducted. Thirteen children with CNI resistant SRNS who were regularly treated with RTX (375 mg/m per dose (maximum dose 500 mg), 1 dose per week, a total of 4 doses) in Department of Nephrology, Children's Hospital of Fudan University from January 2016 to December 2023 were enrolled. The general data, disease related information, urinary protein/creatinine, serum albumin, blood creatinine before RTX treatment, immunosuppressants, adverse events, and monthly urinary protein/creatinine, serum albumin, and blood creatinine indexes within 6 months after RTX treatment were collected. The changes of urinary protein/creatinine, serum albumin and estimated glomerular filtration rate (eGFR) before and after RTX at 3 and 6 months were analyzed by using paired sample test and Wilcoxon signed-rank test. Among the 13 patients, 8 were male and 5 were female. The age of disease onset was 4.0 (2.9, 6.8) years and the age of RTX treatment was 9.8 (5.9, 13.6) years. There were 8 cases of focal segmental glomerulosclerosis, 3 cases of minimal change disease and 2 cases of mesangial proliferative glomerulonephritis. No clinically significant gene variation was detected in 12 cases and the other one did not receive gene test. Before RTX treatment, 11 cases were in chronic kidney disease stage G1, and 1 case each was in stage G2 and stage G3. Ten children completed 4 doses of RTX treatment, 1 patient completed 3 doses, and 2 patients completed 2 doses. Urinary protein/creatinine in 13 children at 3 and 6 months after RTX treatment was significantly lower than baseline (0.60 (0.13, 2.04), 0.49 (0.28, 1.10) 1.44 (0.76, 4.11) mg/mg, =-2.34, -2.34, both <0.05), and serum albumin was significantly higher than baseline ((35±8), (34±7) (30±6) g/L, =2.30, 2.60, both <0.05). The eGFR at 6 months after RTX treatment was not significantly different from the baseline ((110±32) (113±35) ml/(min·1.73 m),=-0.76,>0.05)). No serious adverse reactions occurred in this study. RTX could reduce urinary protein and increase serum albumin in short-term treatment in children with CNI resistant SRNS without significant side effects.
探讨利妥昔单抗（RTX）治疗对钙调神经磷酸酶抑制剂（CNI）抵抗的激素抵抗型肾病综合征（SRNS）患儿的短期疗效及安全性。进行回顾性病例分析。纳入2016年1月至2023年12月在复旦大学附属儿科医院肾病科规律接受RTX治疗（375mg/m²/次（最大剂量500mg），每周1次，共4次）的13例CNI抵抗型SRNS患儿。收集患儿一般资料、疾病相关信息、RTX治疗前尿蛋白/肌酐、血清白蛋白、血肌酐、免疫抑制剂使用情况、不良事件以及RTX治疗后6个月内每月的尿蛋白/肌酐、血清白蛋白和血肌酐指标。采用配对样本t检验和Wilcoxon符号秩和检验分析RTX治疗3个月和6个月前后尿蛋白/肌酐、血清白蛋白及估计肾小球滤过率（eGFR）的变化。13例患者中，男8例，女5例。发病年龄为4.0（2.9，6.8）岁，RTX治疗年龄为9.8（5.9，13.6）岁。局灶节段性肾小球硬化8例，微小病变肾病3例，系膜增生性肾小球肾炎2例。12例未检测到具有临床意义的基因变异，另1例未接受基因检测。RTX治疗前，11例处于慢性肾脏病G1期，G2期和G3期各1例。10例患儿完成4剂RTX治疗，1例完成3剂，2例完成2剂。RTX治疗后3个月和6个月时,13例患儿的尿蛋白/肌酐均显著低于基线水平（0.60（0.13，2.04）、0.49（0.28，1.10）对比1.44（0.76，4.11）mg/mg，t=-2.34，-2.34，均P<0.05），血清白蛋白显著高于基线水平（（35±8）、（34±7）对比（30±6）g/L，t=2.30，2.60，均P<0.05）。RTX治疗后6个月时eGFR与基线水平无显著差异（（110±32）对比（113±35）ml/（min·1.73m²），t=-0.76，P>0.05）。本研究未发生严重不良反应。RTX可在短期治疗中降低CNI抵抗型SRNS患儿的尿蛋白，提高血清白蛋白，且无明显副作用。
