Mekahli Djalila, Liutkus Aurelia, Ranchin Bruno, Yu Anchalee, Bessenay Lucie, Girardin Eric, Van Damme-Lombaerts Rita, Palcoux Jean-Bernard, Cachat François, Lavocat Marie-Pierre, Bourdat-Michel Guylhène, Nobili François, Cochat Pierre
Service de Pédiatrie, Centre de référence des maladies rénales rares, Hôpital Femme Mère Enfant and Université de Lyon, Lyon, France.
Pediatr Nephrol. 2009 Aug;24(8):1525-32. doi: 10.1007/s00467-009-1138-5. Epub 2009 Mar 12.
Long-term outcome of idiopathic steroid-resistant nephrotic syndrome was retrospectively studied in 78 children in eight centers for the past 20 years. Median age at onset was 4.4 years (1.1-15.0 years) and the gender ratio was 1.4. Median follow-up period was 7.7 years (1.0-19.7 years). The disease in 45 patients (58%) was initially not steroid-responsive and in 33 (42%) it was later non-responsive. The main therapeutic strategies included administration of ciclosporine (CsA) alone (n = 29; 37%) and CsA + mycophenolate mofetil (n = 18; 23%). Actuarial patient survival rate after 15 years was 97%. Renal survival rate after 5 years, 10 years and 15 years was 75%, 58% and 53%, respectively. An age at onset of nephrotic syndrome (NS) > 10 years was the only independent predictor of end-stage renal disease (ESRD) in a multivariate analysis using a Cox regression model (P < 0.001). Twenty patients (26%) received transplants; ten showed recurrence of the NS: seven within 2 days, one within 2 weeks, and two within 3-5 months. Seven patients lost their grafts, four from recurrence. Owing to better management, kidney survival in idiopathic steroid-resistant nephrotic syndrome (SRNS) has improved during the past 20 years. Further prospective controlled trials will delineate the potential benefit of new immunosuppressive treatment.
在过去20年里，八个中心对78例儿童特发性类固醇抵抗性肾病综合征的长期预后进行了回顾性研究。发病时的中位年龄为4.4岁（1.1 - 15.0岁），性别比为1.4。中位随访期为7.7年（1.0 - 19.7年）。45例患者（58%）疾病最初对类固醇无反应，33例（42%）后来无反应。主要治疗策略包括单独使用环孢素（CsA）（n = 29；37%）和CsA + 霉酚酸酯（n = 18；23%）。15年后的精算患者生存率为97%。5年、10年和15年后的肾脏生存率分别为75%、58%和53%。在使用Cox回归模型的多变量分析中，肾病综合征（NS）发病年龄>10岁是终末期肾病（ESRD）的唯一独立预测因素（P < 0.001）。20例患者（26%）接受了移植；10例出现NS复发：7例在2天内，1例在2周内，2例在3 - 5个月内。7例患者移植肾失功，4例因复发。由于管理改善，特发性类固醇抵抗性肾病综合征（SRNS）的肾脏生存率在过去20年中有所提高。进一步的前瞻性对照试验将阐明新免疫抑制治疗的潜在益处。
