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目的 了解大剂量环磷酰胺静脉冲击疗法 (CTX PT)治疗儿童激素耐药性肾病的近期疗效以及影响疗效的相关因素。方法 总结 1991年 9月～ 2 0 0 0年 3月接受CTX PT治疗的激素耐药性肾病 41例 ,并就其疾病类型、临床观察指标等与疗效的关系进行分析。结果 (1)本组CTX PT完全缓解 11例 (2 6 %) ,部分缓解 13例 (31%) ,总有效率 5 8%。完全缓解的患儿尿蛋白转阴发生在第 3、4、5个疗程的累计百分率分别为 6 3%、82 %和 10 0 %。 (2 )治疗前Scr水平较低者效果较好。CTX PT疗效与疾病的临床、病理类型以及性别、发病年龄、病程、治疗前血清白蛋白、球蛋白、胆固醇、免疫球蛋白、2 4h尿蛋白排泄量等无关 (P >0 0 5 )。 (3)毒副作用 :本组 13例 (31%)未见明显CTX毒副作用 ,2 8例 (6 8%)出现不同程度的副作用。最常见的副作用是胃肠道反应 ,占 5 1%,其次为轻度脱发 (9%) ,7%的患儿表现为血小板下降 ,血白细胞减少和反复感染均各为 4%,肝功能异常及出血性膀胱炎各 1例 (2 %)。这些毒副作用均为一过性 ,能自行缓解或通过对症处理后很快缓解。结论 CTX PT治疗儿童激素耐药性肾病总有效率为 5 8%;影响疗效的因素主要为Scr水平 ,疾病的临床、病理类型、性别、发病年龄、2 4h尿蛋白排泄量等与疗效无关。
Objective To investigate the short-term effect of high-dose cyclophosphamide (CTX PT) in children with steroid-resistant nephropathy and the related factors that influence the curative effect. Methods From September 1991 to March 2000, 41 cases of steroid-resistant nephropathy treated with CTX PT were analyzed. The relationship between the type of disease, the clinical observation indexes and the curative effect was analyzed. Results (1) CTX PT completely relieved in 11 cases (26%), partial remission in 13 cases (31%), the total effective rate was 58%. Urine protein in children with complete remission occurred in the third, fourth and fifth courses cumulative percentage were 63%, 82% and 100%. (2) Scr lower level before treatment is better. The efficacy of CTX PT was not related to the clinical and pathological type of the disease, sex, age of onset, duration of disease, serum albumin, globulin, cholesterol, immunoglobulin, 24 h urinary protein excretion, etc. before treatment (P> 0.05). (3) Toxicity and side effects: No obvious side effects of CTX were observed in 13 cases (31%) in this group and 28% (68%) had different degrees of side effects. The most common side effects were gastrointestinal reactions, accounting for 51%, followed by mild alopecia (9%), 7% of children with thrombocytopenia, 4% of leukopenia and recurrent infections, respectively, and liver dysfunction And hemorrhagic cystitis in 1 case (2%). These toxic and side effects are transient, can relieve itself or through symptomatic treatment soon relief. Conclusions The total effective rate of CTX PT in children with steroid-resistant nephropathy is 58%. The main factors influencing the curative effect are Scr level. The clinical, pathological type, sex, age of onset, urinary protein excretion at 24 hours and so on are irrelevant to the curative effect.