Moncrieff M W, White R H, Oggs C S, Cameron J S
Br Med J. 1969 Mar 15;1(5645):666-71. doi: 10.1136/bmj.1.5645.666.
Forty-six children with the nephrotic syndrome whose renal biopsy specimens showed minimal changes and whose response to corticosteroid therapy was unsatisfactory were treated with cyclophosphamide. Three patients were completely steroid-resistant from the outset and the remainder were steroid-dependent. In several patients steroids controlled the condition less effectively with time. Most patients showed signs of steroid toxicity, and growth retardation was striking.A moderate leucopenia was induced with cyclophosphamide, and treatment was maintained for three to four months in the majority of cases. Thirty-eight children (83%) have remained in complete remission off all treatment for periods of 3 to 23 months, 33 after one course of cyclophosphamide and five after a second course. Two other patients who remitted but relapsed later are still on treatment. In only six patients was full remission not obtained, and three of these were steroid-resistant from the start. Two died from pneumonia and adrenal failure and four continued to have proteinuria, though in one an impressive reduction occurred.The results indicate that cyclophosphamide therapy is an effective alternative for nephrotic children with normal glomeruli on light microscopy who develop steroid dependence or resistance, and who exhibit toxic effects of steroid therapy.
46例肾活检标本显示微小病变且对皮质类固醇治疗反应不佳的肾病综合征患儿接受了环磷酰胺治疗。3例患者从一开始就完全对类固醇耐药，其余患者为类固醇依赖型。随着时间的推移，在一些患者中类固醇对病情的控制效果变差。大多数患者出现了类固醇毒性的迹象，生长发育迟缓明显。环磷酰胺诱导了中度白细胞减少，大多数病例的治疗持续了三到四个月。38名儿童（83%）在停止所有治疗后完全缓解了3至23个月，33名在接受一个疗程的环磷酰胺治疗后缓解，5名在接受第二个疗程后缓解。另外两名缓解但后来复发的患者仍在接受治疗。只有6例患者未获得完全缓解，其中3例从一开始就对类固醇耐药。2例死于肺炎和肾上腺衰竭，4例继续有蛋白尿，尽管其中1例蛋白尿有明显减少。结果表明，对于光镜下肾小球正常、出现类固醇依赖或耐药且表现出类固醇治疗毒性作用的肾病患儿，环磷酰胺治疗是一种有效的替代方法。
