The efficacy and safety of rituximab in childhood steroid-resistant nephrotic syndrome (SRNS) remains unclear. Therefore, we conducted a retrospective cohort study at 28 pediatric nephrology centers from 19 countries in Asia, Europe, North America and Oceania to evaluate this. Children with SRNS treated with rituximab were analyzed according to the duration of calcineurin inhibitors (CNIs) treatment before rituximab [6 months or more (CNI-resistant) and under 6 months]. Primary outcome was complete/partial remission (CR/PR) as defined by IPNA/KDIGO guidelines. Secondary outcomes included kidney failure and adverse events. Two-hundred-forty-six children (mean age, 6.9 years; 136 boys; 57% focal segmental glomerulosclerosis, FSGS) were followed a median of 32.4 months after rituximab. All patients were in non-remission before rituximab. (146 and 100 children received CNIs for 6 month or more or under 6 months before rituximab, respectively). In patients with CNI-resistant SRNS, the remission rates (CR/PR) at 3-, 6-, 12- and 24-months were 26% (95% confidence interval 19.3-34.1), 35.6% (28.0-44.0), 35.1% (27.2-43.8) and 39.1% (29.2-49.9), respectively. Twenty-five patients were in PR at 12-months, of which 22 had over 50% reduction in proteinuria from baseline. The remission rates among children treated with CNIs under 6 months before rituximab were 42% (32.3-52.3), 52% (41.8-62.0), 54% (44.3-64.5) and 60% (47.6-71.3) at 3-, 6-, 12-, and 24-months. Upon Kaplan-Meier analysis, non-remission and PR at 12-months after rituximab, compared to CR, were associated with significantly worse kidney survival. Adverse events occurred in 30.5% and most were mild. Thus, rituximab enhances remission in a subset of children with SRNS, is generally safe and CR following rituximab is associated with favorable kidney outcome.

中文翻译：

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一项国际性多中心研究评估了利妥昔单抗治疗儿童类固醇耐药性肾病综合征


利妥昔单抗治疗儿童类固醇耐药肾病综合征 （SRNS） 的疗效和安全性尚不清楚。因此，我们在来自亚洲、欧洲、北美和大洋洲 28 个国家的 19 个儿科肾病中心进行了一项回顾性队列研究，以评估这一点。根据利妥昔单抗治疗前钙调磷酸酶抑制剂 （CNIs） 治疗的持续时间 [6 个月或更长时间 （CNI 耐药） 和 6 个月以下] 分析接受利妥昔单抗治疗的 SRNS 患儿。主要结局是 IPNA/KDIGO 指南定义的完全/部分缓解 （CR/PR）。次要结局包括肾衰竭和不良事件。对 246 名儿童 （平均年龄 6.9 岁;136 名男孩;57% 局灶节段性肾小球硬化，FSGS） 进行随访，中位时间为利妥昔单抗治疗后 32.4 个月。所有患者在利妥昔单抗治疗前均处于非缓解状态。（146 名和 100 名儿童分别在利妥昔单抗前接受了 6 个月或更长时间或 6 个月以下的 CNI）。在 CNI 抵抗性 SRNS 患者中，3 、 6 、 12 和 24 个月的缓解率 （CR/PR） 分别为 26% （95% 置信区间 19.3-34.1） 、35.6% （28.0-44.0） 、35.1% （27.2-43.8） 和 39.1% （29.2-49.9）。25 例患者在 12 个月时处于 PR 状态，其中 22 例蛋白尿较基线减少 50% 以上。利妥昔单抗治疗前 6 个月内接受 CNI 治疗的儿童缓解率为 42% （32.3-52.3） 、 52% （41.8-62.0） 、 54% （44.3-64.5） 和 60% （47.6-71.3） 在 3 、 6 、 12 和 24 个月。根据 Kaplan-Meier 分析，与 CR 相比，利妥昔单抗后 12 个月的未缓解和 PR 与肾脏生存率显著差相关。不良事件发生率为 30.5%，大多数为轻度。 因此，利妥昔单抗可增强 SRNS 儿童亚群的缓解，通常是安全的，并且利妥昔单抗后的 CR 与良好的肾脏结局相关。