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单剂量利妥昔单抗治疗激素依赖型微小病变肾病综合征患儿

Single dose of rituximab in children with steroid-dependent minimal change nephrotic syndrome.

作者信息

Niu Xiao-Ling, Hao Sheng, Wang Ping, Zhang Wei, Guo Gui-Mei, Wu Ying, Kuang Xin-Yu, Zhu Guang-Hua, Huang Wen-Yan

机构信息

Department of Nephrology and Rheumatology, Children's Hospital of Shanghai, Shanghai Jiaotong University, Shanghai 200062, P.R. China.

出版信息

Biomed Rep. 2016 Aug;5(2):237-242. doi: 10.3892/br.2016.711. Epub 2016 Jun 30.

Abstract

Rituximab (RTX) can be used in children with nephrotic syndrome, particularly in those with steroid-dependent nephrotic syndrome (SDNS). However, at present there is no unified standard of how to use RTX, with regard to the amount of doses and frequency, in children with nephrotic syndrome. The study aimed to investigate the therapeutic efficacy of a single dose of RTX in children with steroid-dependent minimal change nephrotic syndrome (SD-MCNS). The patients with biopsy-proven minimal change disease (MCD) and clinical features of SDNS received a single dose of RTX (375 mg/m). The toxicity and side effects of RTX were also observed. The study included 19 patients (10 males and 9 females). Follow-up of the patients was 1-50 months (28.1±16.6 months). B-cell depletion was achieved with RTX infusion (CD20<0.5%) and lasted 1-6 months (mean, 2.92±1.57 months). During follow-up, 10 patients remained in complete remission and did not relapse without administration of oral steroids or immunosuppressants for 4-50 months (mean, 30.1±12.6 months), despite recovery of the B-cell count. Nine patients relapsed in the process of reducing steroids, thus, treatment was maintained at a lower dosage (T=0, P<0.05) than prior to use of RTX. The number of relapses also decreased significantly (T=95, P<0.05). Five of the patients relapsed after stopping steroid for several months. At the end of follow-up, the efficacy of a single induction of RTX was 47.4% (9/19). There were no significant side effects associated with administration of RTX. In conclusion, RTX is a safe and effective alternative for children with SD-MCNS. RTX is an effective treatment for the rapid induction of remission and reduces relapse and steroid dependency. A single dose of RTX for children with SD-MCNS is recommended for rapid induction of remission, reduction of long-term steroid dosage, and decrease in the number of relapses, as it has few side effects.

摘要

利妥昔单抗(RTX)可用于治疗肾病综合征患儿,尤其是激素依赖型肾病综合征(SDNS)患儿。然而,目前对于肾病综合征患儿如何使用RTX,在剂量和频率方面尚无统一标准。本研究旨在探讨单剂量RTX治疗激素依赖型微小病变肾病综合征(SD-MCNS)患儿的疗效。经活检证实为微小病变病(MCD)且具有SDNS临床特征的患者接受单剂量RTX(375mg/m²)治疗。同时观察RTX的毒性和副作用。本研究纳入19例患者(10例男性,9例女性)。患者随访时间为1 - 50个月(平均28.1±16.6个月)。RTX输注后实现了B细胞耗竭(CD20<0.5%),且持续1 - 6个月(平均2.92±1.57个月)。随访期间,10例患者持续完全缓解,在未服用口服类固醇或免疫抑制剂的情况下4 - 50个月(平均30.1±12.6个月)未复发,尽管B细胞计数已恢复。9例患者在减停类固醇过程中复发,因此,维持治疗剂量低于使用RTX前(T = 0,P<0.05)。复发次数也显著减少(T = 95,P<0.05)。5例患者在停用类固醇数月后复发。随访结束时,单次诱导使用RTX的缓解率为47.4%(9/19)。RTX给药未出现明显副作用。总之,对于SD-MCNS患儿,RTX是一种安全有效的替代治疗方法。RTX是快速诱导缓解、减少复发和激素依赖的有效治疗手段。推荐对SD-MCNS患儿使用单剂量RTX进行快速诱导缓解、降低长期类固醇剂量并减少复发次数,因为其副作用较少。

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