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首页> 中文期刊> 《肾脏病与透析肾移植杂志》 >他克莫司治疗Alport综合征的近期疗效

他克莫司治疗Alport综合征的近期疗效

         
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摘要

目的:观察他克莫司(tacrolimus,FK506)治疗Alport综合征(Alport syndrome,AS)的近期疗效.方法:前瞻性观察5例接受FK506治疗的AS患者的近期临床疗效与不良反应.5例患者均经肾活检确诊,男4例,女1例,年龄8～42岁,其中X连锁遗传患者4例,常染色体隐性遗传患者1例.起始FK506治疗剂量为0.15mg/(kg·d),8周后减至0.1 mg/(kg·d),目标血药浓度5～8 ng/dl.随访中每2～4周监测一次FK506的不良反应.4例患者行CYP3A5基因型检测,一例基因型为CYP3A5-* 1/* 1的患者FK506谷浓度始终未达到目标范围.结果:治疗2～4周5例患者蛋白尿明显减少,血清白蛋白升高,但随治疗时间延长,蛋白尿未进一步减少,甚至再次增多.2例延长治疗时间至36周和40周,血清白蛋白维持在33～35g/L,尿蛋白波动于0.5～2.5g/L.2例出现肾毒性和(或)糖代谢异常,FK506减量后不良反应消失,1例未达到目标血药浓度者治疗无效.结论:小样本临床观察显示短期FK506治疗能改善AS患者的蛋白尿及低白蛋白血症,其长期疗效有待进一步观察.%Objective:To evaluate the short term effects of tscrolimus (FK506), a kind of calcineurin inhibitors,in patients with Alport syndrome. Methodology:Five 5 cases with Alport syndrome who proved non-responsive to ACEI and traditional Chinese herbal medicine were enrolled this study. All of them were performed percutanious biopsy and satisfied the Flinter's diagnostic criteria for Alport syndrome, and had pathologic evidence of collagen Ⅳ chains abnormalities both in kidney and/or skin biopsy specimen. They were 4 male and one female with an average age of 18.5 years old. Their inheritance modes were 4 X-linkage and one autosomal recessive AS(ARAS). FK506 was administered at a dosage of 0.15 mg/(kg·d) for the first 8 weeks and then tapered to 0.10 mg/(kg·d) for the maintenance therapy. The target trough serum concentration of FK506 was 5 ～ 8 ng/dl. The efficacy and side-effects of FK506 were observed at an interval of 2 ～4 weeks during the follow-up. Results:In 4 of the five patients whose CYP3A5 polymorphic genotypes were 2 of CYP3A5-*1/*3, one of CYP3A5-* 3/* 3 and one of CYP3A5- * 1/* 1, the target trough level of FK506 was achieved in the proposed therapeutic window range, except one patient of CYP3A5- * 1/* 1 genotype. In the end of the second and the fourth week of follow-up, a marked reduction of 24-hour urinary protein excretion, and an elevation of serum albumin level were observed in all of patients. Two cases withdrew FK506 because of side-effects as renal toxicity and/or abnormal glucose metabolism. One patient dropped out of the study because of inefficacy associated with failure to achieve targeted trough serum level of FK506. Another 2 patients prolonged the therapy of FK506 for 36 weeks and 40 weeks showed that the level of serum albumin maintained at 33 ～ 35 g/L and proteinuria fluctuating between 0. 5 ～ 2. 5 g/L. The side - effects of renal toxicity, gastro - intestinal disorders, and abnormality of glucose metabolism were observed during the first 8 weeks. Conclusion:The proteinuria and hypoalbuminemia was improved by the short term of FK506 therapy in the patients with Alport syndrome. The long-term efficacy of FK506 therapy for patients with Alport syndrome remained further investigation.

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  • 来源
    《肾脏病与透析肾移植杂志》 |2011年第4期|338-341|共4页
  • 作者

    姚小丹; 陈欣; 黄高渊; 许书添; 黄倩; 曾彩虹; 陈惠萍; 胡伟新; 刘志红;

  • 作者单位

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

    南京军区南京总医院全军肾脏病研究所,南京,210002;

  • 原文格式 PDF
  • 正文语种 chi
  • 中图分类
  • 关键词

    Alport综合征; 他克莫司; 临床疗效;

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