酶制剂Cerezyme治疗高雪氏病效果观察

目的　酶制剂cerezyme替代疗法治疗高雪氏病 4年小结。方法　 Cerezyme初剂量 6 0U/kg ,每 2周 1次静脉滴注 ,2年后疗效好转剂量改为 30U/kg。结果 　 1 5例用药 4年以上的患者 ,血红蛋白平均增加 2 1g/L ,1 4例血小板近 1 2月恢复正常 ;脾功能亢进得到改善 ,肝脾回缩 9.3%和 6 0 .6 % ,肝功能治疗 6个月后大多恢复正常 ;身高平均增长 2 5 6厘米 ,体重平均增加 1 4 5公斤 ,生活质量明显提高。结论　 在基因治疗尚未广泛应用之前 ,酶替代疗法仍是唯一改善症状的有效措施

Effectiveness of enzyme replacement therapy to patients with Gaucher Disease

Objective Summarize the effects of Cerezyme t re atment in Gaucher disease for 4 years. Methods Cerezyme was gi ven at an initial dose 60 U/kg by intravenous infusion every 2 weeks. The dose w as reduced to 30 U/kg when they had achieved the specified therapeutic goals aft er 2 years. Results After 15 patients have received enzyme rep lacement therapy for 4 years, all observed items have been more or less function assay after 6 months. The average height of the patients increases by 25.8 cm, average weigh increases by 14.5kg. All patients' life quality has been improved. Conclusion Given that gene cure has not been developed and widely used currently, enzyme replacement therapy is still the only effective managemen t.