环磷酰胺冲击治疗儿童激素耐药型肾病综合征的疗效

目的 探讨激素耐药型肾病综合征(SRNS)患儿大剂量环磷酰胺(CTX)冲击治疗的疗效及影响疗效的相关因素.方法 总结本院2004年12月～2009年12月收治的资料完整并进行CTX静脉冲击治疗的38例SRNS患儿的病例资料及随访情况,并就其缓解情况及与临床分型、病理类型、临床指标之间的关系及药物不良反应进行回顾性分析.结果 1.本组完全缓解18例(47%),部分缓解11例(29%),总有效率76%;部分缓解患儿均发生于疗程6个月内,延长疗程无累积缓解率增加;未缓解患儿1例在起病5 a内进展至终末期肾病.2.SRNS患儿单纯型肾病组缓解率高于肾炎型肾病组.3.SRNS病理改变以非微小病变肾病为主,即以系膜增生性肾小球肾炎(MsPGN)、局灶节段性肾小球硬化(FSGS)为主;其中MsPGN缓解率较高,而FSGS缓解率相对较低.4.不良反应发生率为53%,最常见不良反应为一过性胃肠道反应,占36%,其次为轻度脱发(10%)、白细胞降低(7%),无严重感染、出血性膀胱炎发生.结论 CTX冲击联合激素治疗SRNS疗效肯定;临床类型、病理类型等因素可能与疗效及预后有关,单纯型肾病、MsPGN有效率较高,FSGS患儿CTX冲击疗效较差,发展为终未期肾病的风险较高.

Effect of Cyclophosphamide Pulse Therapy on Steroid-Resistant Nephrotic Syndrome in Children

Objective To investigate the effect and relevant influencing factors of intravenous cyclophosphamide(CTX) pulse therapy on steroid-resistant nephrotic syndrome(SRNS) in children.Methods The retrospective study was conducted in the Children′s Hospital Affiliated to Chongqing Medical University from Dec.2004 to Dec.2009.Thirty-eight well-documented clinical cases of SRNS treated by CTX pulse therapy were consecutively enrolled.The clinical data of the cases and follow-up recording were collected.The relationship among clinical remission and clinical staging,pathologic type,clinical index and adverse drug reactions were analyzed.Results 1.Eighteen patients(47%) had achieved complete remission,11 patients(29%) had partial remission,and the response rate was 76%.All the cases that obtained partial remission occurred within 6 months of therapy and there was no effect of increasing remission to extend the course.2.Of the patients with SRNS,the remission rates were significantly higher in patients with simple nephrotic syndrome than those in patients with nephritis nephrotic syndrome.3.The predominantly pathological changes of SRNS were non-minimal change nephropathy,and the latter showed mesangial proliferative glomerulonephritis(MsPGN) and focal segmental glomerulosclerosis(FSGS).The patients with MsPGN demonstrated higher remission rates,while those of FSGS had relatively lower remission rates.4.The incidence of adverse drug reactions was 53%,and the main clinical manifestations included transient gastrointestinal disorders(36%),light loss of hair(10%) and leukocytopenia(7%).There were no severe sepsis and hemorrhagic cystitis.Conclusions CTX pulse therapy has positive effects on patients with SRNS.The factors,including clinical staging and pathologic type,may be correlated with the therapy and prognosis of SRNS.The patients with simple nephrotic syndrome and MsPGN had better curative effects,while the patients with FSGS who were not effectively controlled by CTX pulse therapy,had a higher risk of end stage renal disease.