Alshaya Hammad O, Al-Maghrabi Jaudah A, Kari Jameela A
Department of Pediatrics, King Abdul Aziz University Hospital, PO Box 80215, Jeddah 21589, Saudi Arabia.
Pediatr Nephrol. 2003 Nov;18(11):1143-6. doi: 10.1007/s00467-003-1279-x. Epub 2003 Sep 17.
Treatment of steroid-resistant nephrotic syndrome (SRNS) remains a challenge to pediatric nephrologists. Recently, intravenous cyclophosphamide (IV-CPM) infusion was shown to be effective, safe, and economical for the treatment of SRNS, particularly minimal change disease (MCD), as it results in more sustained remissions, longer periods without proteinuria, and fewer significant side effects at a lower cumulative dose. A prospective study was conducted to evaluate IV-CPM infusions in the management of children with SRNS secondary to MCD or IgM nephropathy. Five patients with SRNS (4 IgM nephropathy and 1 MCD) received six monthly IV-CPM infusions at a dose of 500 mg/m(2). No patient achieved complete or sustained remission. Three patients attained partial remission, which was not sustained for more than 1 month post therapy. One patient progressed rapidly to end-stage renal disease during treatment. Side effects included vomiting in four patients and alopecia in one patient.
IV-CPM pulse therapy at a dose of 500 mg/m(2) is unsuccessful in obtaining complete or sustained remission in children with SRNS secondary to IGM nephropathy or MCD. Further randomized controlled studies with higher doses are required.
对小儿肾脏病学家来说，类固醇抵抗性肾病综合征（SRNS）的治疗仍然是一项挑战。最近，静脉注射环磷酰胺（IV-CPM）被证明对SRNS的治疗有效、安全且经济，尤其是微小病变肾病（MCD），因为它能带来更持久的缓解、更长时间的无蛋白尿期，且在较低累积剂量下显著副作用更少。进行了一项前瞻性研究，以评估IV-CPM输注在治疗继发于MCD或IgM肾病的SRNS儿童中的作用。5例SRNS患者（4例IgM肾病和1例MCD）接受了6次每月一次的IV-CPM输注，剂量为500 mg/m²。没有患者实现完全或持续缓解。3例患者达到部分缓解，但缓解在治疗后持续不超过1个月。1例患者在治疗期间迅速进展为终末期肾病。副作用包括4例患者出现呕吐，1例患者出现脱发。
剂量为500 mg/m²的IV-CPM脉冲疗法在治疗继发于IGM肾病或MCD的SRNS儿童中未能实现完全或持续缓解。需要进一步开展更高剂量的随机对照研究。
