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利妥昔单抗作为新发特发性肾病综合征儿童的一线治疗药物。

Rituximab as a first-line therapy in children with new-onset idiopathic nephrotic syndrome.

作者信息

Zhang Xiaojing, Jin Yanyan, Liu Fei, Li Qiuyu, Xie Yi, Huang Guoping, Chen Junyi, He Xue, He Siyi, Fu Haidong, Wang Jingjing, Shen Huijun, Mao Jianhua

机构信息

Department of Nephrology, The Children's Hospital, Zhejiang University School of Medicine, National Clinical Research Center for Child Health, Hangzhou, Zhejiang, China.

Department of Pediatrics, Zhejiang University School of Medicine, Hangzhou, Zhejiang, China.

出版信息

Clin Kidney J. 2024 Nov 18;18(1):sfae348. doi: 10.1093/ckj/sfae348. eCollection 2025 Jan.

DOI:10.1093/ckj/sfae348
PMID:39811259
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC11730066/
Abstract

BACKGROUND

Idiopathic nephrotic syndrome (INS) in children, commonly treated with steroids, poses challenges due to associated side effects. Rituximab, known for its efficacy in reducing relapse frequency in difficult-to-treat cases, emerges a potential first-line therapy for pediatric new-onset INS.

METHOD

This is a single-center, retrospective, observational study to evaluate the efficacy and safety of rituximab as a first-line therapy for pediatric INS. The complete treatment strategy was weekly injections at a dose of 375 mg/m for four doses. Children with new-onset INS who received rituximab as a first-line monotherapy from 1 January 2022 to 31 December 2023 were included and followed until 31 May 2024.

RESULTS

Seventeen patients (median age at diagnosis 4.8 years) were included. Twelve patients achieved complete remission within a median time of 19 days. Over a follow-up period ranging from 41 to 112 weeks, 11 patients maintained remission even after B-cell reconstitution, with one patient experiencing a relapse at 85 weeks. Three patients, who presented with hematuria, hypocomplementemia or renal injury at initial diagnosis, exhibited resistance to rituximab. No severe adverse events were noted.

CONCLUSION

Rituximab may be an effective and safe option as a first-line therapy for inducing and maintaining remission in newly diagnosed INS.

摘要

背景

儿童特发性肾病综合征(INS)通常采用类固醇治疗,但因其相关副作用而面临挑战。利妥昔单抗以其在难治性病例中降低复发频率的疗效而闻名,成为小儿新发INS的潜在一线治疗方法。

方法

这是一项单中心、回顾性、观察性研究,旨在评估利妥昔单抗作为小儿INS一线治疗的疗效和安全性。完整的治疗方案是每周注射一次,剂量为375mg/m²,共注射四剂。纳入2022年1月1日至2023年12月31日期间接受利妥昔单抗作为一线单药治疗的新发INS患儿,并随访至2024年5月31日。

结果

纳入17例患者(诊断时中位年龄4.8岁)。12例患者在中位时间19天内实现完全缓解。在41至112周的随访期内,11例患者即使在B细胞重建后仍维持缓解,1例患者在85周时复发。3例在初诊时出现血尿、低补体血症或肾损伤的患者对利妥昔单抗耐药。未观察到严重不良事件。

结论

利妥昔单抗作为新诊断INS诱导和维持缓解的一线治疗可能是一种有效且安全的选择。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a33/11730066/93a8e01abeb3/sfae348fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a33/11730066/11d9b4dcdf15/sfae348fig1g.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a33/11730066/93a8e01abeb3/sfae348fig1.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a33/11730066/11d9b4dcdf15/sfae348fig1g.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/6a33/11730066/93a8e01abeb3/sfae348fig1.jpg

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本文引用的文献

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Combined Rituximab and Daratumumab Treatment in Difficult-to-Treat Nephrotic Syndrome Cases.利妥昔单抗与达雷妥尤单抗联合治疗难治性肾病综合征病例
Kidney Int Rep. 2024 Apr 4;9(6):1892-1896. doi: 10.1016/j.ekir.2024.04.006. eCollection 2024 Jun.
2
Autoantibodies Targeting Nephrin in Podocytopathies.足细胞病相关的 Nephrin 自身抗体
N Engl J Med. 2024 Aug 1;391(5):422-433. doi: 10.1056/NEJMoa2314471. Epub 2024 May 25.
3
Rituximab-induced hypogammaglobulinemia in nephrotic syndrome: what is the true burden?利妥昔单抗诱导的肾病综合征低丙种球蛋白血症:真正的负担是什么?
Pediatr Nephrol. 2024 Oct;39(10):3129. doi: 10.1007/s00467-024-06371-z. Epub 2024 Apr 11.
4
Successful Treatment of New-Onset Pediatric Nephrotic Syndrome With Rituximab as a First-Line Therapy.利妥昔单抗作为一线疗法成功治疗新发小儿肾病综合征
Kidney Int Rep. 2022 Oct 22;7(12):2750-2751. doi: 10.1016/j.ekir.2022.10.016. eCollection 2022 Dec.
5
IPNA clinical practice recommendations for the diagnosis and management of children with steroid-sensitive nephrotic syndrome.国际儿科肾病学会关于儿童激素敏感性肾病综合征诊断和治疗的临床实践建议。
Pediatr Nephrol. 2023 Mar;38(3):877-919. doi: 10.1007/s00467-022-05739-3. Epub 2022 Oct 21.
6
Long-Term Efficacy and Safety of Repeated Rituximab to Maintain Remission in Idiopathic Childhood Nephrotic Syndrome: An International Study.重复利妥昔单抗维持缓解治疗特发性儿童肾病综合征的长期疗效和安全性:一项国际研究。
J Am Soc Nephrol. 2022 Jun;33(6):1193-1207. doi: 10.1681/ASN.2021111472. Epub 2022 Mar 30.
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Significance of regulatory T cells in children with idiopathic nephrotic syndrome.调节性T细胞在儿童特发性肾病综合征中的意义
J Nephrol. 2022 Mar;35(2):711-713. doi: 10.1007/s40620-021-01201-z. Epub 2022 Jan 28.
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