环磷酰胺静脉冲击联合泼尼松治疗儿童激素耐药性肾病综合征疗效分析

Effect and influencing factors of cyclophosphamide pulse therapy in children with steroid-resistant nephrotic syndrome

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 understand the short-term effect and relevant influencing factors of intravenous cyclophosphamide pulse therapy (CTX-PT) on children with steroid-resistant nephrotic syndrome (NS). Methods CTX-PT (500-750 mg/m 2, once every 4 weeks for 12 times) was given to 41 children with steroid-resistant NS (including 31 boys and 10 girls, aged from 31 months to 15-year, the average age was 7.66 years). The relevant data on clinical, pathological and laboratory tests were collected and analyzed. Results(1) Eleven cases (26%) got complete remission and 13 cases (31%) got partial remission, so the total effica of this therapy was 58%. In children with the complete remission, the numbers of cases obtained remission from the first to 5th course of CTX-PT were 2 (18%), 1 (9%), 4 (36%), 2 (18%) and 2 (18%), respectively, and the accumulated percentages of which were 18%, 27%, 63%, 82% and 100%, respectively. (2) The Scr level was the most important factor which influenced the effect of CTX-PT in this study. The Scr levels in the complete remission group, partial remission group and ineffective group were (43±11) μmol/L, (44±25) μmol/L and (71±23) μmol/L, respectively. There was no significant difference of Scr levels between the complete remission group and the partial remission group (q=0.327,P=0.576), but the Scr levels in both groups were significantly lower than that in the ineffiective group (q=8.949 and 5.923, P=0.008 and 0.023).The study showed that there was no relationship between the therapy and the clinical and pathological types of the disease, the age, the levels of Alb, Glb, Ch, IgG, IgA, IgM and excretions of urinary protein in the patients(P>0.05).(3)Twenty-eight cases (68%) were found to have side effects. Nausea and vomiting, losing hairs, the decrease of blood platelet counts and decrease of white blood cells, repeated infections and abnormal of liver functions occurred at 51%, 9%, 7% and 4%, 4% and 2%, respectively. These side effects were transient and could remit spontaneously or after symptomatic treatment. Conclusion The total efficacy of CTX-PT on children with steroid-resistant NS was 58%. The levels of Scr of patients was an important factor which influenced the efficacy of CTX-PT, the clinical and pathological types of this disease, the age, the levels of Alb, Glb, Ch, excretions of urinary protein of patients, etc, have all no relationship with the effect of this therapy.