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Abstract：
OBJECTIVE: Enzyme replacement therapy with idursulfase has been shown to improve somatic signs and symptoms of mucopolysaccharidosis type II (MPS II). Idursulfase is available in Japan (since 2007), based on the outcome of clinical trials conducted in the United States, but data from Japanese patients are limited.
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.
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.
摘要：
目的：艾杜硫酸酯酶的酶替代疗法已被证明可以改善II型粘多糖贮积症（MPSII）的躯体体征和症状。Idursulfase在日本有售（自2007年起），根据在美国进行的临床试验的结果，但日本患者的数据有限。
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。
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。
