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利妥昔单抗长期重复治疗儿童激素依赖型肾病综合征

Long-term repeated rituximab treatment for childhood steroid-dependent nephrotic syndrome.

作者信息

Kim Ji Hyun, Park Eujin, Hyun Hye Sun, Cho Myung Hyun, Ahn Yo Han, Choi Hyun Jin, Kang Hee Gyung, Ha Il-Soo, Cheong Hae Il

机构信息

Department of Pediatrics, Seoul National University Children's Hospital, Seoul, Korea.

Department of Pediatrics, Hallym University Kangnam Sacred Heart Hospital, Seoul, Korea.

出版信息

Kidney Res Clin Pract. 2017 Sep;36(3):257-263. doi: 10.23876/j.krcp.2017.36.3.257. Epub 2017 Sep 30.

Abstract

BACKGROUND

Rituximab (RTX) can be used as a rescue therapy for steroid-dependent nephrotic syndrome (SDNS). However, the efficacy and safety of long-term, repeated use of RTX are not established. This study was conducted to assess the efficacy and safety of long-term, repeated RTX treatment in children.

METHODS

Eighteen consecutive child patients with SDNS who were treated with three or more cycles of RTX for one year or longer were recruited, and their medical records were retrospectively reviewed.

RESULTS

The patients were followed for 4.7 ± 1.9 years and received 5.2 ± 2.3 cycles of RTX over 2.8 ± 1.1 years. Approximately 70% of the additional RTX cycles were administered due to recovery of B-cells without relapse. The relapse rate decreased from 3.4 ± 2.0 per year initially to 0.4 ± 0.8 per year at the third year after RTX treatment. Approximately 10% of the RTX infusions were accompanied by mild infusion reactions. Eight patients showed sustained remission without any oral medication after the last cycle of RTX, while 10 patients had one or more episodes of relapse after the last cycle of RTX. The relapse rate in the latter group decreased from 2.8 ± 1.5 per year before RTX treatment to 1.3 ± 0.8 per year after cessation of RTX treatment. No significant differences in clinical parameters were found between the two groups.

CONCLUSION

This retrospective study showed that pre-emptive and long-term, repeated RTX treatment is relatively effective and safe in children with SDNS. However, well-designed prospective studies are needed to confirm these findings.

摘要

背景

利妥昔单抗(RTX)可作为激素依赖型肾病综合征(SDNS)的挽救治疗药物。然而,长期、重复使用RTX的疗效和安全性尚未明确。本研究旨在评估长期、重复使用RTX治疗儿童SDNS的疗效和安全性。

方法

招募18例连续接受三个或更多周期RTX治疗一年或更长时间的儿童SDNS患者,并对其病历进行回顾性分析。

结果

患者随访4.7±1.9年,在2.8±1.1年内接受了5.2±2.3个周期的RTX治疗。约70%的额外RTX周期是由于B细胞恢复且无复发而进行的。复发率从最初的每年3.4±2.0次降至RTX治疗后第三年的每年0.4±0.8次。约10%的RTX输注伴有轻度输注反应。8例患者在最后一个RTX周期后无需任何口服药物即可持续缓解,而10例患者在最后一个RTX周期后有一次或多次复发。后一组的复发率从RTX治疗前的每年2.8±1.5次降至RTX治疗停止后的每年1.3±0.8次。两组临床参数无显著差异。

结论

这项回顾性研究表明,对于儿童SDNS患者,抢先性、长期、重复使用RTX治疗相对有效且安全。然而,需要设计良好的前瞻性研究来证实这些发现。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/e71b/5592893/fc2f673f2b2c/krcp-36-257f1.jpg

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