Adhikari M, Bhimma R, Coovadia H M
Department of Paediatrics and Child Health, University of Natal, Faculty of Medicine, Congella, South Africa.
Pediatr Nephrol. 1997 Aug;11(4):423-8. doi: 10.1007/s004670050309.
Seven children with steroid-resistant focal segmental glomerulosclerosis (SR-FGS) were placed on a therapeutic protocol of methylprednisolone (MP), oral prednisone (pred) and oral cyclophosphamide (CYC) given over 16 months (regimen A). Another 5 children with SR-FGS were treated with a shorter course of intravenous CYC (monthly doses over 6 months), intravenous MP (3 consecutive daily doses) and oral pred 2 mg/kg (alternate days) (regimen B). With regimen A, 1 child had a short remission, and in the others, oedema subsided, the urine protein/ creatinine ratio decreased, haematuria disappeared and the estimated glomerular filtration rate (GFR) increased. The observation period was 21-42 months and the drugs were well tolerated. With regimen B, 2 patients went into complete remission, 1 had partial remission, 1 failed to respond and another died because of severe concurrent infections. In the responding children, oedema cleared, the urine protein/ creatinine ratio decreased, haematuria disappeared and the GFR rose. The follow-up was between 3 and 34 months. Minor side effects were alopecia and transient hypertension. Both regimens improved the quality of life of most children. Compared with regimen A, regimen B is six times less costly with a quarter of the number of hospital visits. These observations may be of value in designing appropriate multicentre controlled trials, which have been advocated recently, for the rational and optimum management of SR-FGS.
7名患有激素抵抗性局灶节段性肾小球硬化症(SR-FGS)的儿童接受了为期16个月的甲基强的松龙(MP)、口服泼尼松(pred)和口服环磷酰胺(CYC)治疗方案(方案A)。另外5名SR-FGS儿童接受了较短疗程的静脉注射CYC(6个月内每月给药)、静脉注射MP(连续3天每日给药)和口服pred 2mg/kg(隔日给药)(方案B)。采用方案A时,1名儿童有短期缓解,其他儿童水肿消退、尿蛋白/肌酐比值降低、血尿消失且估计肾小球滤过率(GFR)升高。观察期为21至42个月,药物耐受性良好。采用方案B时,2例患者完全缓解,1例部分缓解,1例无反应,另1例因严重并发感染死亡。在有反应的儿童中,水肿消退、尿蛋白/肌酐比值降低、血尿消失且GFR升高。随访时间为3至34个月。轻微副作用为脱发和短暂性高血压。两种方案均改善了大多数儿童的生活质量。与方案A相比,方案B成本低六倍,就诊次数减少四分之一。这些观察结果可能对设计合适的多中心对照试验有价值,最近有人提倡进行此类试验,以合理、优化地管理SR-FGS。