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中国循证儿科杂志

中国循证儿科杂志 ›› 2018, Vol. 13 ›› Issue (3): 161-165.

• 论著 •    下一篇

利妥昔单抗治疗儿童原发性难治性肾病综合征疗效及其影响因素的自身前后对照研究

张涛,沈茜,徐虹,方晓燕,翟亦晖,龚一女,缪千帆   

  1. 复旦大学附属儿科医院肾脏风湿科上海，201102
  • 收稿日期:2018-05-15 修回日期:2018-06-25 出版日期:2018-06-24 发布日期:2018-06-25
  • 通讯作者: 沈茜

The effect and multiple-factor analysis of rituximab treatment in children with primary refractory nephrotic syndrome

ZHANG Tao, SHEN Qian, XU Hong, FANG Xiao-yan, ZHAI Yi-hui, GONG Yi-nv, MIAO Qian-fan   

  1. Department of Nephrology and Rheumatology, Children's Hospital of Fudan University, Shanghai 201102, China
  • Received:2018-05-15 Revised:2018-06-25 Online:2018-06-24 Published:2018-06-25
  • Contact: SHEN Qian

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摘要： 目的：分析利妥昔单抗（RTX）治疗儿童原发性难治性肾病综合征的疗效和安全性，探讨影响RTX疗效的因素。方法：纳入2011年3月至2016年12月在复旦大学附属儿科医院（我院）接受RTX治疗且随访≥6个月的频复发-激素依赖或激素耐药-钙调磷酸酶抑制剂（CNI）敏感的原发性难治性肾病综合征患儿。予RTX每次375 mg·m-2（每次最大500 mg，每周1次，至多2次治疗），糖皮质激素在使用RTX治疗后3~5月内逐渐减停，CNI在RTX治疗后1月内减停。部分患儿加用霉酚酸酯(MMF)维持治疗1~2年。对可能影响RTX疗效的因素（性别、发病年龄、病程、临床分型、病理类型、治疗剂次、是否加用MMF等）行单因素及多因素分析。 结果：符合本文纳入和排除标准的55例患儿进入分析，频复发-激素依赖39例、激素耐药-CNI敏感16例；肾活检微小病变（MCD）40例、局灶节段肾小球硬化（FSGS）9例、6例未行肾活检；使用1次RTX 19例、2次RTX 36例；36例随访期间加用MMF治疗。使用RTX年龄（8.8±3.7）岁，RTX治疗前病程38（26.0，61.0）个月，RTX治疗后随访时间28.0（17.0，39.3）个月。RTX治疗前尿蛋白年复发次数2.0（1.0,3.0）次，RTX治疗后1.0（0,1.5）次,尿蛋白持续缓解时间为10.3（5.9,18.3）个月，RTX治疗后6个月和12个月尿蛋白持续缓解率分别为74.5%(41/55)和43.6%(24/55)。频复发-激素依赖组和激素耐药-CNI敏感组、使用1次和2次RTX治疗效果差异无统计学意义。预防性使用MMF可以提高RTX治疗后6个月和12个月尿蛋白持续缓解率，分别为94.4%和58.3%。使用RTX后随访期间未见严重不良反应发生。结论：RTX治疗儿童频复发-激素依赖和激素耐药-CNI敏感的原发性难治性肾病综合征有效且安全，RTX治疗后加用MMF维持治疗可以进一步延长尿蛋白持续缓解时间。

Abstract: Objective：We analyzed the effect and safety of rituximab in children with primary refractory nephrotic syndrome (NS)and explored the factors related to the prognosis. Methods：Frequent relapsing-steroid dependent NS and calcineurin inhibitor (CNI)-sensitive steroid resistant NS patients, who received rituximab and had been followed up for more than 6 months from March 2011 to December 2016, were enrolled in this study. Rituximab was given for 1 or 2 doses. Glucocorticoid was gradually reduced in 3-5 months after treatment of rituximab, CNI was stopped within 1 month after treatment of rituximab. Some of the patients were added mycophenolate mofetil for 1-2 years. The factors that may influence the effect of rituximab (gender, age of onset, pathogenesis, different clinical types, pathological types, therapeutic dosages and whether to add with mycophenolate mofetil) were analyzed. Results：A total of 55 cases were enrolled in this study. Frequent relapsing-steroid dependent NS accounted for 70.9% (39 cases) and minimal change disease accounted for 81.6% (40 cases). Nineteen cases received one dose of rituximab and 36 cases were added mycophenolate mofetiltherapy. The age of using rituximab was 8.8±3.7 years, the median course of disease before rituximab was 38(26.0，61.0) months. Patients were followed for a mean duration of 28.0(17.0, 19.3) months. The relapses of urine protein after rituximab were declined from 2.0(1.0, 3.0) to 1.0(0, 1.5) in one year. The median remission duration was 10.3(5.9, 18.3) months, sustained remission rate was 74.5% and 43.6% at 6 months and 12 months, respectively. There was no statistically significant difference in efficacy between different clinical groups or patients received different dosages of rituximab. Mycophenolate mofetil therapy could improve the sustained remission rate to 94.4% and 58.3% at 6 months and 12 months, respectively. No patients had severe complications. Conclusion：Rituximab is effective and safe to treat the primary refractory NS in children. Mycophenolate mofetil can further improve the sustained remission time of urine protein.

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