Objective: To investigate the short-term efficacy and safety of rituximab (RTX) in children with calcineurin inhibitor (CNI) resistant steroid resistant nephrotic syndrome (SRNS). Methods: A retrospective case analysis was conducted. Thirteen children with CNI resistant SRNS who were regularly treated with RTX (375 mg/m2 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 t test and Wilcoxon signed-rank test. Results: 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) vs. 1.44 (0.76, 4.11) mg/mg, Z=-2.34, -2.34, both P<0.05), and serum albumin was significantly higher than baseline ((35±8), (34±7) vs. (30±6) g/L, t=2.30, 2.60, both P<0.05). The eGFR at 6 months after RTX treatment was not significantly different from the baseline ((110±32) vs. (113±35) ml/(min·1.73 m2),t=-0.76,P>0.05)). No serious adverse reactions occurred in this study. Conclusion: RTX could reduce urinary protein and increase serum albumin in short-term treatment in children with CNI resistant SRNS without significant side effects.
目的： 探讨利妥昔单抗（RTX）治疗儿童钙调磷酸酶抑制剂（CNI）抵抗型激素耐药型肾病综合征（SRNS）的短期疗效及安全性。 方法： 回顾性病例分析。以2016年1月至2023年12月在复旦大学附属儿科医院肾脏科规律应用RTX治疗［每剂375 mg/m2（最大剂量500 mg），每周1剂，共4剂］的13例CNI抵抗型SRNS患儿作为研究对象。收集患儿一般情况、临床表现、RTX治疗前尿蛋白/肌酐、血清白蛋白、血肌酐、RTX治疗后6个月内免疫抑制剂使用、不良反应和每个月尿蛋白/肌酐、血清白蛋白、血肌酐指标。通过配对样本t检验和Wilcoxon秩和检验分析RTX治疗前和治疗后3、6个月尿蛋白/肌酐、血清白蛋白和估算肾小球滤过率（eGFR）的变化情况。 结果： 13例患儿中男8例、女5例，起病年龄4.0（2.9，6.8）岁、RTX治疗年龄9.8（5.9，13.6）岁。局灶节段肾小球硬化8例、微小病变3例、系膜增生性肾小球肾炎2例。12例未检出有临床意义的基因变异，1例未行基因检测。RTX治疗前慢性肾脏病G1期11例，G2和G3期各1例。10例患儿完成4剂RTX治疗，1例完成3剂RTX，2例完成2剂RTX。13例患儿在RTX治疗后3、6个月尿蛋白/肌酐均较基线显著降低［0.60（0.13，2.04）、0.49（0.28，1.10）比 1.44（0.76，4.11）mg/mg，Z=-2.34、-2.34，均P<0.05］；3、6个月血清白蛋白均较基线显著升高［（35±8）、（34±7）比（30±6）g/L，t=2.30、2.60，均P<0.05］。RTX治疗后6个月eGFR较基线差异无统计学意义［（110±32）比（113±35）ml/（min·1.73 m2），t=-0.76，P>0.05］。未见严重不良反应发生。 结论： RTX可短期内降低CNI抵抗型SRNS患儿尿蛋白水平，提高血清白蛋白水平，药物总体耐受性良好。.