长疗程环孢素A治疗儿童激素耐药型肾病综合征的疗效和预后

目的 观察激素耐药的儿童肾病综合征(SRNS)应用环孢素A(CsA)长疗程治疗的疗效并分析影响疗效的相关因素.方法 回顾性分析疗程2年的CsA治疗SRNS病例20例,男:女为3:1;平均年龄5.5岁,肾病病理类型微小病变(MCNS)15例,局灶节段肾小球硬化(FSGS)4例,系膜增生性肾炎(MsPGN)1例.结果 (1)完全缓解13例(65%),部分缓解4例(20%),CsA无效3例(15%),总有效率85%.平均随访时间40.5个月,复发率45%.(2)CsA治疗微小病变肾病的有效率为93%,非微小病变为60%,但两者差异无统计学意义.(3)毒副作用:多毛、齿龈增生和高血压的发生率分别为75%、25%和10%,2例出现可逆性的肾损伤,4例出现尿NAG/Cr增高,神经系统症状2例.3例重复肾活检未发现CsA相关的肾小管间质纤维化病变.结论 对儿童激素耐药型肾病综合征CsA长疗程治疗有良好的疗效.小剂量CsA长期治疗未发现药物相关的肾小管间质纤维化和肾功能损伤.CsA长期治疗的安全性和预后有待进一步研究.

Long-term effects of cyclosporine A in children with steroid-resistant idiopathic nephrotic syndrome and outcomes of the patients

Objective To observe the effects of long-term cyclosparine A(CsA)treatment in 20 children with steroid-resistant idiopathic nephrotic syndrome(SRNS)and analyse the relevant influencing factom of CsA therapy.Methods Twenty children with SRNS received CsA therapy for 2 years between February 2001 and October 2006 in the Department of Nephmlogy.The mean age of children at initiation of CsA therapy was 5.5 years(30 months to 12 years).The initial renal histology showed minimal change (MCNS)in 15 patients,focal segmental glomerulosclerosis(FSGS)in 4 patients and mesangial proliferative with a trough level of 40-70 μg/L wfs applied to mainrain remission for 1 year.Liver function,serum albumin,serum cholesterol,serum creatinine,urinary NAG/Cr,24 h urinary protein excretion and CsA whole blood trough level of the patients were monitoted every one or three months.Results (1)Complete remission(proteinuria≤0.1 g/d or negative by dipstick for 3 consecutive days),partial remission of 100μg/L)were observed in 65%,20% and 15%,respectively.Eleven patients who had complete remission discontinued CsA,in 5(45%)patients the disease relapsed,and resumption of CsA thempy was foilowed by remission in th.ree of them.(2)MCNS showed a 93%response to CsA therapy while non-MCNS showed a 60% response,but the difference was not significant(P＞0.05).(3)Hypertrichosis,gingival hyperplasia and hypertension occurred in 75%,25%and 10%of the patients,respectively.Two patients were found to have renal impairment(＞30% rise of serum creatinine)and recovered in 2 weeks. Four patients showed a rise of urinary NAG/Cr.The central nervous system adverse event occurred in 2 cages.Post-thempy biopsies performed in 3 patients(2 with FSGS and one with MCNS)did not show any relevant tubulointerstitial fibrosis.Two patients with FSGs ofthe twenty cases progressed into end-stage renal failure.Conclusions CsA treatment was confirmed to be effective in children with SRNS.Rehal fibrosis was rare in patients treated with a low dose of CsA for 2 years.