他克莫司治疗儿童重症肌无力临床观察

摘要

目的 观察应用他克莫司(FK506)治疗儿童重症肌无力(MG)患者的临床疗效及安全性.方法 对2009年7月至2016年12月于火箭军总医院神经内科就诊的年龄≤14岁的MG患儿进行筛选,选择其中应用FK506治疗患儿,对临床资料进行分析与随访,监测其实验室指标,临床疗效,不良反应.根据临床好转程度,分为痊愈组及未痊愈组,对两组临床资料进行分析.结果 符合入组条件MG患儿31例,男性15例,女性16例,平均年龄(9.59±3.89)岁.服药时间4~71个月,平均用药时间(26.65±19.85)个月.FK506用药量0.02mg·kg-1·d-1到0.12mg·kg-1·d-1,平均0.05±0.02mg·kg-1·d-1.血药浓度0.46~8.5ng·mL-1,平均血药浓度3.91±1.99ng·mL-1.MG相对评分好转≥95%患儿12例,80%~94%为7例,50%~79%为5例, 25%~49%为2例,< 5%为5例.监测肝肾功能、血糖、血尿常规化验指标无恶化.所有患儿均成功停用激素.治愈组19例,未治愈组12例,两组患儿在性别比例、临床分型、病程、用药时MG绝对评分,FK506用药量、FK506血药浓度差异无统计学意义,在发病年龄、服用FK506疗程及服药前复发次数方面差异有统计学意义(P<0.05).结论 FK506用于治疗儿童MG患者疗效确切,具有好的安全性,不良反应少.发病年龄越早,复发次数越多,FK506治疗效果差,FK506服药疗程影响临床疗效.%Objective? To evaluate the effectiveness and safety of tacrolimus(FK506)for myasthenia gravis(MG)in children.Method Clinical information of children with MG,who were diagnosed between July 2009 to December 2016 and received oral tacrolimus treatment,were collected and retrospectively analyzed. During the treatment , the plasma concentration of FK506, urine, serum creatinine and liver function were regularly investigated. Relative scores of the MG patients were recorded to evaluate the effectiveness. The cases were divided into two groups,according to relative scores of the MG patients. Clinical indexes were compared between two groups. Results? Among the 31 cases,15 were males and 16 females.The duration of treatment was 4-71 months, and the average duration was 26.65±19.85months. The dosage of FK506 fluctuated from 0.02mg·kg-1·d-1 to 0.12mg·kg-1·d-1, with an average dosage of 0.05±0.02mg·kg-1·d-1. The plasma concentration of FK506 fluctuated at 0.46-8.5ng·mL-1, with an average blood concentration of 3.91±1.99ng·mL-1. The relative score of MG improved in 12 children with≥ 95%, 7 cases with 80%-94%, 5 cases with 50%-79%, 2 cases with 25%-49%, and 5 cases with <25% cases. We monitored liver and kidney function, blood glucose, blood routine, urine routine, and there was no deterioration in these parameters. All of the patients successfully stopped using glucocorticoids. 19 cases were cured, 12 cases were not completely cured. There was no significant difference between the two groups in terms of sex ratio, clinical type, course of disease, absolute score of MG, dosage of FK506 and blood concentration of FK506. There were significant differences in the age of onset, the course of taking tacrolimus and the number of relapses before treatment with FK506 (P<0.05). Conclusion? FK506 is effective in the treatment of MG in children, with good safety and less adverse reactions. The FK506 treatment is relatively poor with earlier onset of the disease,andmore times of relapses. FK506 treatment course affects the clinical efficacy.