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目的观察Myozyme对依赖呼吸机辅助通气的晚期晚发型糖原贮积病Ⅱ型患者的治疗效果。方法参照药品说明书要求,对1例晚发型糖原贮积病Ⅱ型患者进行Myozyme治疗,剂量20 mg/kg(1次/2～4周),连续治疗6次,末次给药距首次给药间隔4个月;首次给药前须经静脉注射地塞米松5 mg预处理。随访16个月,记录每日运动功能、脱机时间和呼吸机参数变化。结果患者对Myozyme耐受良好,治疗期间未发生药物不良反应。首次给药第2天肩部压痛感即减轻,随着治疗时间的延长和给药次数的增加,脱机时间延长,原地踏步速度增快和时间延长、双上肢上举力量增加、自主呼吸下行走距离增加和时间延长。疗效评价显示,以治疗7个月时(停药后3个月)疗效最佳,至8个月时(停药后4个月)降至治疗前水平;肩部压痛感最先改善,也最快回复至治疗前水平。结论 Myozyme对依赖呼吸机辅助通气的晚发型糖原贮积病Ⅱ型患者有一定疗效。
Objective To investigate the therapeutic effect of Myozyme on type Ⅱ patients with late-stage late-onset glycogen storage disease that is dependent on ventilator-assisted ventilation. Methods One patient with type Ⅱ of late-onset glycogen storage disease was treated with Myozyme at a dose of 20 mg / kg (once / 2 to 4 weeks) with continuous treatment for 6 times according to the requirements of the drug specification. The last administration was from the first administration Intervals of 4 months; before the first dose of intravenous dexamethasone 5 mg pretreatment. After 16 months of follow-up, daily motor function, off-line time and ventilator parameters were recorded. Results Myozyme was well tolerated and no adverse drug reactions occurred during the treatment. Shoulder tenderness was relieved on the second day after the first administration. With the prolongation of treatment time and number of administration, the time of off-line was extended, the speed of standing-in and the extension of time were prolonged. Under walking distance and time extension. Efficacy evaluation showed that the treatment effect was best at 7 months (3 months after drug withdrawal), and dropped to pre-treatment level at 8 months (4 months after drug withdrawal); the shoulder tenderness first improved and The fastest return to pre-treatment levels. Conclusion Myozyme has a certain effect on type Ⅱ patients with late-onset glycogen storage disease that is dependent on ventilator-assisted ventilation.