Institute of Child Health, 9 Greek Church Row Extension, Kolkata, India.
Pediatr Nephrol. 2013 Jan;28(1):93-7. doi: 10.1007/s00467-012-2278-6. Epub 2012 Aug 14.
Mycophenolate mofetil (MMF) is used as a steroid-sparing agent in pediatric nephrotic syndrome (NS). However, data about its long-term efficacy and safety is limited.
We report the long-term outcome of MMF therapy in 46 NS patients who remained steroid dependent (SD) despite previous treatment with levamisole and cyclophosphamide.
After 1 year of MMF initiation, 32 (70 %) patients had reduced steroid requirement: 12 with decreased threshold dose and 20 were able to stop steroids. At follow-up of mean 3.56 (standard deviation + 1.76) years, 25 (54 %) children required no further alternative immunosuppression (IS), having infrequent or no relapses, of which 14 stopped MMF after a mean 2.4 (standard deviation + 0.9) years; 11 are continuing on MMF for a median of 2.25 years (range 1.33-7.75 years). One patient had a psoriasis flare, and MMF was stopped. No other patient required permanent drug withdrawal due to side effects. The outcome of patients who did not require further alternate IS was significantly better than those who did, with 56 % vs. 10.5 %, respectively, being off regular medications at last follow-up.
We conclude that MMF therapy is safe in the long term and allows >50 % of severe SDNS patients to avoid further toxic IS.
霉酚酸酯（MMF）被用作小儿肾病综合征（NS）的类固醇保肾药物。然而，关于其长期疗效和安全性的数据有限。
我们报告了 46 例肾病综合征患者的长期 MMF 治疗结果，这些患者在使用左旋咪唑和环磷酰胺治疗后仍然对类固醇依赖（SD）。
在 MMF 治疗开始后的 1 年内，32 名（70%）患者减少了类固醇的需求：12 名患者降低了阈值剂量，20 名患者能够停用类固醇。在平均 3.56 年（标准差+1.76 年）的随访中，25 名（54%）儿童不需要进一步的替代免疫抑制（IS），复发频率较低或无复发，其中 14 名在平均 2.4 年（标准差+0.9 年）后停用 MMF；11 名患者继续服用 MMF 中位数为 2.25 年（范围 1.33-7.75 年）。1 名患者出现银屑病发作，停用了 MMF。没有其他患者因副作用而需要永久性停药。不需要进一步替代 IS 的患者的结局明显好于需要替代 IS 的患者，分别为 56%和 10.5%的患者在最后一次随访时停用常规药物。
我们的结论是，MMF 治疗在长期内是安全的，可以使超过 50%的严重 SDNS 患者避免进一步的毒性 IS。
