环磷酰胺与环孢霉素A治疗儿童激素耐药型肾病综合征临床疗效比较

目的:比较环磷酰胺(cyclophosphamide,CTX)与环孢霉素A(cyclosporine A,CsA)治疗儿童激素耐药型肾病综合征(steroid-resistant idiopathic nephrotic syndrome,SRNS)的临床疗效.方法:回顾性研究2001年1月～2006年12月我院收治并完整随访的SRNS37例,男女比例为5:1,平均年龄5.0岁(4个月～12岁).肾脏病理为微小病变(minimal change disease,MCD)28例,非微小病变9例,其中局灶节段性肾小球硬化(focal segmental glomerulosclerosis,FSGS)7例,系膜增生性肾小球肾炎(mesangial proliferative glomerulonephritis,MsPGN)2例.其中CTX治疗组30例(CTX冲击剂量500 mg/m2),CsA治疗组21例[CsA初始剂量3～5 mg/(kg·d)].期间联合泼尼松龙口服并逐渐减量.对两组患儿随访12个月内疗效及副作用进行比较.结果:(1)随访12个月CTX治疗组、CsA治疗组总有效率分别为40.0%(12/30)和85.7%(18/21).在MCD患儿中CsA治疗组有效率显著高于CTX治疗组(93.8% vs 36.3%,P＜0.05);而在FSGS患儿中CsA治疗组与CTX治疗组差异无统计学意义(75.0% vs 50.0%,P＞0.05).(2)无论在CTX治疗组或CsA治疗组,患儿的起病年龄、病理类型(MCD或FSGS)、临床分型(单纯型或肾炎型)均与疗效无显著相关(卡方检验,均P＞0.05).(3)CTX治疗组合并肝功能损害、外周血白细胞减低、消化道副反应比例分别为10%,16.7%及33.3%,无患儿出现出血性膀胱炎.CsA治疗组出现多毛、齿龈增生、高血压比例分别为71.4%,23.8%及9.5%.2例出现神经系统副作用.2例患儿分别在1年和5年时进入终末期肾衰.结论:儿童激素耐药型肾病综合征的治疗中,激素联合免疫抑制剂CTX或CsA取得了较好的疗效.特别是在MCD患儿中,CsA组在随访12个月内的总有效率优于CTX组.

Comparison of cyclophosphamide and cyclosporine in the treatmentof steroid-resistant idiopathic nephrotic syndrome in children

OBJECTIVE: To compare the therapeutic outcome of cyclophosphamide(CTX) and cyclosporine A (CsA) in the treatment of steroid-resistant idiopathic nephrotic syndrome (SRNS) in children. METHODS: Thirty-seven children with SRNS were analysed in a retrospective study from Jan 2001 to Dec. 2006. There was initial renal histology of minimal change (MCD) in 28 children, focal segmental glomerulosclerosis (FSGS) in 7 and mesangial proliferative glomerulonephritis (MsPGN) in the other 2. These patients were divided into 2 groups: one group received the induction therapy consisting of intravenous CTX and prednisolone, and the another group were treated with CsA [initial dose 3-5mg/(kg d)] and prednisolone.RESULTS: (1) Thirty children received CTX, while 21 received CsA, and the total efficacy was 40.0% and 85.7% respectively after the 12 month follow-up. Children with MCD receiving CsA had a better response than those treated with CTX (93.8% vs 36.3%, P<0.05). Children with FSGS receiving CsA did not show a significant difference compared with those treated with CTX (75.0% vs 50.0%, P>0.05). (2) There were no significant associations between age, pathology, clinic type and therapeutic outcome in the 2 groups(P>0.05). (3)The rate of liver functional impairment, leukocytopenia, vomiting and nausea was 10%, 16.7% and 33.3%, respectively in children receiving CTX. The rate of hypertrichosis, gingival hyperplasia and hypertension was 71.4%, 23.8% and 9.5% respectively in children receiving CsA. Two children had central adverse effect. Two patients with FSGS progressed into end-stage renal failure. CONCLUSION: For children with MCD, CsA combining prednisolone could result in a higher remission rate than intravenous CTX combining prednisolone.