长疗程泼尼松治疗小儿重症肌无力的前瞻性研究

A prospective study of long-term prednisone therapy in juvenile myasthenia gravis

目的 探讨门诊长期使用醋酸泼尼松治疗小儿重症肌无力 (MG)的有效性、安全性和使用方法。方法 对 5 0 4例使用泼尼松“中剂量冲击、小剂量维持”口服疗法的MG患儿进行前瞻性研究 ,观察泼尼松使用剂量、撤药方式与疗效和副反应的关系 ,并分析MG类型、发病年龄、病程及伴发病对疗效的影响。结果 追踪 1～ 16年 (平均 5 5年 ) ,总有效率 95 0 %。终点的疗效与临床类型、发病年龄、病程及是否合并胸腺瘤或胸腺增生无明显关连。 195例患儿按计划撤药 ,其中 70例 (35 9% )症状复发 (药物依赖 )。主要副反应为Cushing反应和易患上呼吸道感染。结论 泼尼松可用于所有类型MG患儿的长期治疗。Cushing反应随药物减量逐渐消失 ,但易患上呼吸道感染和体格矮小不容忽视。药物依赖的发生率很高 。

Objective The study was designed to evaluate the efficacy,safety,and applying method of long term prednisone therapy in treating the outpatients with juvenile myasthenia gravis(MG).Methods From July 1983 to October 1999,504 cases juvenile MG patients treated with short courses of moderate doses of daily prednisone and followed by low dose daily maintainance therapy had been followed up to study the efficacy,side effects,and dependence.The influences of clinical types,age at onset,duration of the disorder,and the accompanying disorders on efficacy had been analysed.Results During a 1～16 year(mean 5 5 years) follow up,the total effectiveness rate was 95 0%,and the efficacy had no relations with the clinical types,age at onset,duration of the disorder,and the accompanying disorders.The therapy was scheduled to withdraw in 195 cases after a stable remission for at least a year,and 70 of them experienced recurrence of muscle weakness.The major side effects included Cushing's reaction and the liability to suffer from respiratory tract infection.The underdevelopment of stature was also observed.Conclusions (1)Prednisone can be applied to the treatment of all types of juvenile MG.(2)With the prednison taper,Cushing's reaction has become less apparent but the liability to suffer from the respiratory tract infection still remains a problem.(3)The concoction of Chinese herbs made by ourselves might be helpful in controlling recurrence.