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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0.1mg/(kg·d),目标血药浓度5～8ng/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 observe the short-term curative effect of tacrolimus (FK506) on Alport syndrome (AS). Methods: We prospectively observed the short-term clinical efficacy and adverse reactions of 5 patients with AS treated with FK506. Five patients were diagnosed by renal biopsy, 4 males and 1 females, aged 8 to 42 years, of whom 4 cases of X-linked genetic and 1 case of autosomal recessive hereditary. The initial dose of FK506 was 0.15 mg / (kg · d), reduced to 0.1 mg / (kg · d) after 8 weeks, and the target plasma concentration was 5 ~ 8 ng / dl. Follow-up every 2 to 4 weeks to monitor the adverse reactions of FK506. Four patients were tested for CYP3A5 genotype. One patient whose genotype was CYP3A5- * 1 / * 1 did not reach the target range for the concentration of FK506. Results: The proteinuria of 5 patients decreased obviously and serum albumin increased in 2 to 4 weeks after treatment. However, with the prolongation of treatment time, proteinuria did not decrease or even increase again. 2 cases extended treatment time to 36 weeks and 40 weeks, serum albumin maintained at 33 ~ 35g / L, urine protein fluctuations in 0.5 ~ 2.5g / L. 2 cases of renal toxicity and (or) abnormal glucose metabolism, FK506 adverse reactions disappeared after reduction, 1 case of patients did not meet the target plasma concentration of treatment ineffective. Conclusion: Small clinical observation shows that short-term treatment with FK506 can improve proteinuria and hypoalbuminemia in patients with AS, and its long-term efficacy needs further observation.