Karunamoorthy Saravanakumar, Thanigachalam Dineshkumar, Jeyachandran Dhanapriya, Ramanathan Sakthirajan, Natarajan Gopalakrishnan, Thoppalan Balasubramaniam
Institute of Nephrology, Madras Medical College, Chennai, India.
Clin Kidney J. 2019 Jun 6;13(2):179-183. doi: 10.1093/ckj/sfz061. eCollection 2020 Apr.
Steroid-dependent nephrotic syndrome (SDNS) patients experience frequent relapse or adverse effects on long-term treatment with steroids or cyclophosphamide. This study assessed the efficacy and side effect profile of mycophenolate mofetil (MMF) therapy in children with nephrotic syndrome in our population.
A retrospective study was performed on children with SDNS who were on MMF therapy for a minimum period of 1 year, and were on regular follow-up in the Department of Nephrology at the Institute of Child Health and hospital for children attached to Madras Medical College.
The study included 87 patients, with a male:female ratio of 2:1. The median age at diagnosis of nephrotic syndrome was 3 years [95% confidence interval (CI): 1-8 years], which was found to be a statistically significant risk factor for MMF failure. The median duration of follow-up after initiation of MMF therapy was 3 years and 3 months (95% CI: 1 year and 3 months to 6 years and 6 months). At initial evaluation, 31 (36%) patients presented with SDNS while the remaining had frequently relapsing nephrotic syndrome progressing to SDNS. Intravenous cyclophosphamide was used as first-line therapy in 82 patients, of whom 24 patients had persistent proteinuria while the remaining 58 had attained remission for a median duration of 6 months. The median duration of treatment with MMF was 2 years and 6 months (95% CI: 1 year and 3 months to 4 years and 6 months). MMF was used at a mean dose of 28.5 mg/kg. Seventy-two (83%) patients were MMF-sensitive, and these patients had a reduction in mean prednisolone dose from 1.28 to 0.35 mg/kg (P < 0.05). Among the MMF-sensitive patients, 31 had stopped MMF after a minimum period of 2 years, following which they had a median remission period of 5 months (95% CI: 1-8 months). MMF failure occurred in 15 (17%) patients. Adverse events were documented in 19 (22%) patients.
Continuous MMF therapy achieved remission in 83% of patients. MMF was well tolerated in the study population and discontinuation of MMF resulted in 100% relapse.
激素依赖型肾病综合征（SDNS）患者在长期接受激素或环磷酰胺治疗时经常复发或出现不良反应。本研究评估了霉酚酸酯（MMF）治疗我国儿童肾病综合征的疗效和副作用情况。
对接受MMF治疗至少1年且在马德拉斯医学院附属儿童健康研究所和儿童医院肾病科定期随访的SDNS患儿进行回顾性研究。
该研究纳入87例患者，男女比例为2:1。肾病综合征诊断时的中位年龄为3岁[95%置信区间（CI）：1 - 8岁]，这被发现是MMF治疗失败的一个具有统计学意义的危险因素。开始MMF治疗后的中位随访时间为3年3个月（95%CI：1年3个月至6年6个月）。在初始评估时，31例（36%）患者表现为SDNS，其余患者为频繁复发的肾病综合征并进展为SDNS。82例患者将静脉注射环磷酰胺用作一线治疗，其中24例患者持续存在蛋白尿，其余58例达到缓解，中位缓解期为6个月。MMF的中位治疗时间为2年6个月（95%CI：1年3个月至4年6个月）。MMF的平均使用剂量为28.5mg/kg。72例（83%）患者对MMF敏感，这些患者的泼尼松龙平均剂量从1.28mg/kg降至0.35mg/kg（P < 0.05）。在对MMF敏感的患者中，31例在至少2年后停用MMF，此后他们的中位缓解期为5个月（95%CI：1 - 8个月）。15例（17%）患者出现MMF治疗失败。19例（22%）患者记录有不良事件。
持续MMF治疗使83%的患者达到缓解。在研究人群中MMF耐受性良好，停用MMF导致100%复发。
