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低剂量利妥昔单抗联合霉酚酸酯治疗儿童激素依赖型/频繁复发型肾病综合征:病例系列

Low-dose rituximab followed by mycophenolate mofetil for steroid-dependent/frequently relapsing nephrotic syndrome in children: a case series.

作者信息

Song Jide, Chang Hong, Lin Yi, Shan Chunrong, Liu Jia, Zhang Ranran, Nie Nana, Bai Cui, Gao Shan, Zhang Qiuye, Wang Dahai

机构信息

Department of Pediatric Nephrology, Rheumatology, and Immunology, The Affiliated Hospital of Qingdao University, Qingdao, China.

Department of Pediatrics, Qingdao Women and Children's Hospital, Qingdao, China.

出版信息

Front Pharmacol. 2025 Aug 26;16:1646837. doi: 10.3389/fphar.2025.1646837. eCollection 2025.

DOI:10.3389/fphar.2025.1646837
PMID:40932863
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12417471/
Abstract

BACKGROUND

Rituximab (RTX) has gradually been accepted as a treatment for frequently relapsing nephrotic syndrome (FRNS) or steroid-dependent nephrotic syndrome (SDNS) in children, but no standardized recommendations for the dosage and combination therapy exist. Additionally, the efficacy and safety of low-dose RTX in FRNS/SDNS remain unclear, although it has been used to treat some autoimmune diseases.

METHODS

We report a case series of 24 children diagnosed with FRNS/SDNS treated with low-dose RTX followed by mycophenolate mofetil (MMF) for maintenance of remission of nephrotic syndrome between August 2021 and February 2023. These patients were followed up for at least 12 months.

RESULTS

The mean total dose for the initial four administrations of low-dose RTX was 470.83 ± 62.41 mg, which was significantly lower than the calculated values for one standard dose (525.62 ± 125.62 mg; = 0.006) and two standard doses (1051.2 ± 251.23 mg; < 0.001). After treatment initiation, the median follow-up was 24.6 (16.8, 28.5) months. At the 1-year follow-up, no child had experienced treatment failure, and the relapse-free rate was 83.3%. At the last follow-up, two children had experienced treatment failure, with both having frequent relapses, and the relapse-free rate was 75%. Compared with the calculated standard dose of RTX, low-dose RTX followed by MMF was less costly. No serious adverse reactions were observed during RTX use or follow-up, except for one death due to delayed treatment of severe infection.

CONCLUSION

Low-dose RTX followed by MMF can extend the remission duration of FRNS/SDNS in children, and decrease the economic burden on families, while offering good safety.

摘要

背景

利妥昔单抗(RTX)已逐渐被接受用于治疗儿童频繁复发的肾病综合征(FRNS)或激素依赖型肾病综合征(SDNS),但对于剂量和联合治疗尚无标准化建议。此外,低剂量RTX在FRNS/SDNS中的疗效和安全性仍不明确,尽管它已被用于治疗一些自身免疫性疾病。

方法

我们报告了一组病例系列,共24例诊断为FRNS/SDNS的儿童,于2021年8月至2023年2月接受低剂量RTX治疗,随后使用霉酚酸酯(MMF)维持肾病综合征缓解。这些患者至少随访了12个月。

结果

低剂量RTX最初四次给药的平均总剂量为470.83±62.41mg,显著低于一个标准剂量的计算值(525.62±125.62mg;P=0.006)和两个标准剂量的计算值(1051.2±251.23mg;P<0.001)。开始治疗后,中位随访时间为24.6(16.8,28.5)个月。在1年随访时,没有儿童出现治疗失败,无复发率为83.3%。在最后一次随访时,有两名儿童出现治疗失败,均频繁复发,无复发率为75%。与RTX的计算标准剂量相比,低剂量RTX联合MMF成本更低。在RTX使用或随访期间,除1例因严重感染延迟治疗死亡外,未观察到严重不良反应。

结论

低剂量RTX联合MMF可延长儿童FRNS/SDNS的缓解期,减轻家庭经济负担,且安全性良好。

https://cdn.ncbi.nlm.nih.gov/pmc/blobs/de72/12417471/fdb9932c604a/fphar-16-1646837-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/de72/12417471/fdb9932c604a/fphar-16-1646837-g002.jpg
https://cdn.ncbi.nlm.nih.gov/pmc/blobs/de72/12417471/fdb9932c604a/fphar-16-1646837-g002.jpg

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本文引用的文献

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Efficacy and safety of low- and ultralow-dose rituximab in neuromyelitis optica spectrum disorder.低剂量和超低剂量利妥昔单抗治疗视神经脊髓炎谱系障碍的疗效与安全性
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