Division of Nephrology, Department of Pediatrics, Indian Council of Medical Research Advanced Center for Research in Nephrology, India Institute of Medical Sciences, New Delhi, India.
Department of Biostatistics, All India Institute of Medical Sciences, New Delhi, India.
Kidney Int. 2019 Jan;95(1):210-218. doi: 10.1016/j.kint.2018.08.039. Epub 2018 Nov 26.
Both levamisole and mycophenolate mofetil (MMF) prevent relapses in patients with frequently relapsing nephrotic syndrome; however, their efficacy has not been compared prospectively. This single-center, randomized, open-label trial enrolled 149 children ages 6-18 years with frequently relapsing or steroid-dependent nephrotic syndrome. Participants were randomized in a 1:1 ratio to receive therapy with MMF (750-1000 mg/m daily) or levamisole (2-2.5 mg/kg on alternate days) for 1 year; prednisolone was discontinued by 2-3 months. In intention-to-treat analyses, the frequency of relapse was similar between participants treated with MMF and levamisole (mean difference -0.29 relapses/patient-year; 95% confidence interval -0.65, 0.08). Relapse rates declined to almost one-third of baseline for both treatment groups. Therapy with MMF was not superior to levamisole in terms of the proportions of participants with sustained remission (40.8% vs. 34.2%), frequent relapses (14.5% vs. 16.4%), or treatment failure, a composite outcome of frequent relapses, steroid resistance, or significant steroid toxicity (15.8% vs. 20.6%). These outcomes were also similar in time to event analyses. Changes in anthropometry and blood pressure were similar between the groups, and the rates of adverse effects were low in both groups. Flow cytometry in 32 participants demonstrated similar proportions of B cells and CD4+, CD8+, T helper (Th)1, Th2, Th17, and T regulatory (Treg) cells during follow-up. Therapy with MMF was not superior to levamisole in the frequency of relapses, likelihood of sustained remission or corticosteroid sparing in children with frequently relapsing or steroid-dependent nephrotic syndrome. Registration CTRI/2012/02/002394.
来氟米特和麦考酚酸酯（MMF）均可预防频繁复发型肾病综合征患者的复发；然而，其疗效尚未前瞻性比较。这项单中心、随机、开放标签试验纳入了 149 例年龄 6-18 岁的频繁复发或激素依赖型肾病综合征患儿。将患者按照 1:1 的比例随机分为 MMF 治疗组（750-1000mg/m 每日）和来氟米特治疗组（2-2.5mg/kg 隔日），疗程 1 年；2-3 个月内停用泼尼松。意向治疗分析中，MMF 治疗组和来氟米特治疗组的复发频率相似（平均差异-0.29 次/患者-年；95%置信区间-0.65，0.08）。两组的复发率均降至基线的近三分之一。MMF 治疗在持续缓解率（40.8%比 34.2%）、频繁复发率（14.5%比 16.4%）或治疗失败率（频繁复发、激素抵抗或显著激素毒性的复合结局）方面并不优于来氟米特（15.8%比 20.6%）。事件时间分析的结果也相似。两组的人体测量和血压变化相似，两组的不良反应发生率均较低。32 例患者的流式细胞术显示，在随访期间，B 细胞和 CD4+、CD8+、辅助性 T（Th）1、Th2、Th17 和调节性 T（Treg）细胞的比例相似。在频繁复发或激素依赖型肾病综合征患儿中，MMF 治疗的复发频率、持续缓解或皮质类固醇节省的可能性并不优于来氟米特。注册 CTRI/2012/02/002394。
