METHODS: This was a postmarketing study of Japanese MPS II patients treated with 0.5 mg/kg intravenous idursulfase weekly, conducted over a period of 8 years after initial administration. Assessments included the safety profile, survival rate, degree of clinical improvement, change in urinary uronic acid (UA) concentration, and 6-minute walk test (6MWT).
RESULTS: The safety and efficacy analysis populations included 145 and 143 patients, respectively. The incidence of serious adverse events was 42.8% and the incidence of adverse drug reactions was 48.3%. The 7-year survival rate was 82.7%. Improvements in the clinical features of hepatosplenomegaly, skin, joint, and respiratory disorders were reported (per investigator\'s assessment). The mean change in urinary UA concentration was -128.39 mg/g creatinine, and that of 6MWT walking distance was +31.8 m.
CONCLUSIONS: Long-term idursulfase treatment was well tolerated, and effective in improving clinical features, reducing urinary UA, and slowing disease progression in Japanese MPS II patients.
方法:这是一项针对日本MPSII患者的上市后研究,该患者每周接受0.5mg/kg静脉注射艾杜硫酸酯酶,在初次给药后8年内进行。评估包括安全概况,存活率,临床改善程度,尿糖醛酸(UA)浓度的变化,和6分钟步行测试(6MWT)。
结果:安全性和有效性分析人群包括145和143名患者,分别。严重不良事件发生率为42.8%,药物不良反应发生率为48.3%。7年生存率为82.7%。改善肝脾肿大的临床特征,皮肤,接头,并报告了呼吸系统疾病(根据研究者的评估).尿UA浓度的平均变化为-128.39mg/g肌酐,6MWT的步行距离为+31.8m。
结论:长期艾杜硫酸酯酶治疗耐受性良好,并有效改善临床特征,降低尿UA,和减缓日本MPSII患者的疾病进展。