他克莫司联合小剂量激素治疗儿童激素抵抗型肾病综合征21例临床分析

目的 分析评估他克莫司对治疗儿童激素抵抗型肾病综合征的疗效及其安全性.方法 采用回顾性纵向研究分析21例激素抵抗型肾病综合征患儿,他克莫司初始剂量0.10 ～0.15 mg/(kg·d),每12小时1次,定期监测血药浓度、尿常规、血常规及肝肾功能等指标.同时口服小剂量泼尼松0.20 ～0.75 rmg/( kg·d).结果 1～3个月后观察近期疗效,完全缓解者14例,部分缓解者7例,完全缓解率66.7％.16例患儿接受了肾活检,其中6例微小病变型肾病患儿中3例完全缓解,3例部分缓解;4例局灶节段性肾小球硬化患儿中2例完全缓解,2例部分缓解;5例lgM肾病及1例系膜增生性肾小球肾炎患儿均完全缓解.服药期间6例患儿出现一过性不良反应,经对症处理后均缓解.20例患儿获随访,1年内共4例复发,第2年共4例6次出现复发.结论 他克莫司对儿童激素抵抗型肾病综合征有较好的疗效,不良反应较少,大多可耐受,但服药1～2年内复发率较高,因此其长期疗效仍有待于进一步随访观察.

Clinical application of tacrolimus with low-dose prednisone in 21 children with steroid-resistant nephrotic syndrome

Objective To analyze and evaluate the efficacy and safety of tacrolimus and low-dose steroids in the treatment of steroid-resistant nephrotic syndrome in children.Method Twenty-one children with steroid-resistant nephrotic syndrome enrolled from October 2008 to July 2010 into this retrospective longitudinal study received oral tacrolimus treatment,0.1 to 0.15 mg/kg per day and once every 12 hours,and prednisone 0.2 to 0.75 mg/kg per day simultaneously.During the treatment,the plasma concentration of tacrolimus,urine volume,urine,serum creatinine and liver function were regularly monitored.Result After 1 to 3 months treatment,14 cases showed complete remission and 7 cases had partial remission.Sixteen patients received renal biopsy,of whom 6 revealed minimal change nephropathy with complete remission in 3 cases,3 cases had partial remission ;4 cases revealed focal segmental glomerulosclerosis with 2 complete remission and 2 partial remission; other 5 children with IgM nephropathy and 1 mesangial proliferative glomerulonephritis achieved complete remission.Within treatment period,6 patients presented transient adverse reactions,without altering the principle treatment strategy,but only taking the symptomatic treatment.During follow-up,1 case was lost to follow-up and the remaining 20 cases were followed up from 2 months to 21 months.In 4 patients the disease relapsed within 1st-year follow-up,while at 2nd-year follow-up,4 cases had (6 times) recurrence.Conclusion Tacrolimus showed a reliable effect in children with steroid-resistant nephrotic syndrome.Less adverse reactions were seen,and most of them could be tolerated.Nevertheless,the patients had a higher relapse rate after 1 to 2 years treatment.Therefore,the long-term effects of tacrolimus for steroid-resistant nephrotic syndrome remains to be further evaluated.