部份中文艾杜硫酸酯处方资料（仅供参考）
商品名：Elaprase Concentrado solucion
英文名：Idursulfase
中文名：艾杜硫酸酯冻干粉注射液
生产商：希雷制药
药品介绍
Elaprase是首个用于治疗享特综合征(Hunter syndrome，又名Mucopolysaccharidosis II［Ⅱ型粘多糖贮积症］，或MPS Ⅱ)的产品，享特综合征是一种罕见遗传疾病，它可能导致过早死亡。
作用机制
亨特综合征是一种X连锁疾病，由溶酶体酶iduronate-2-硫酸酯酶水平不足引起。Iduronate-2-硫酸酯酶通过切割寡糖连接的硫酸盐部分来分解代谢糖胺聚糖（GAG）硫酸皮肤素和硫酸乙酰肝素。由于Hunter综合征患者的iduronate-2-硫酸酯酶缺失或缺陷，糖胺聚糖在细胞中逐渐积累，导致细胞充血、器官肥大、组织破坏和器官系统功能障碍。
Idursulfate是溶酶体酶iduronate-2-硫酸酯酶的一种纯化形式，在人类细胞系中产生，提供人类糖基化特征，类似于天然存在的酶。Idursulfate是一种525个氨基酸的糖蛋白，含有8个N-连接的糖基化位点，这些位点被复杂、杂交和高甘露糖型寡糖链占据。Idursulfate的分子量约为76kD。
静脉注射伊杜硫酶治疗亨特综合征患者可提供外源酶，使其进入细胞溶酶体。寡糖链上的甘露糖-6-磷酸（M6P）残基允许酶与细胞表面的M6P受体特异性结合，导致酶的细胞内化，靶向细胞内溶酶体，随后分解代谢积累的GAG。
适应症
用于亨特综合征（粘多糖病II，MPS II）患者的长期治疗。
在临床试验中未对杂合雌性进行研究。
用法与用量
该治疗应由医生或其他在MPS II疾病或其他遗传性代谢紊乱患者管理方面有经验的医疗保健专业人员监督。
剂量
每周以0.5mg/kg体重的剂量通过静脉输注给予依拉普酶，持续3小时，如果未观察到输注相关反应，可逐渐减少至1小时。
有关使用说明。
对于在诊所接受了几个月治疗且对输液耐受良好的患者，可以考虑在家输液。家庭输液应在医生或其他医疗专业人员的监督下进行。
特殊人群
老年患者
65岁以上的患者没有临床经验。
肾或肝损害患者
目前尚无肾或肝功能不全患者的临床经验。
儿科人群
儿童和青少年的剂量与成人相同，每周0.5mg/kg体重。
给药方法
有关给药前药物稀释的说明。
禁忌症
如果超敏反应不可控，则对活性物质或列出的任何赋形剂产生严重或危及生命的超敏反应。
保质期
3年。
在25°C条件下，化学和物理使用稳定性已证明8小时。
稀释后
从微生物安全的角度来看，稀释后的产品应立即使用。如果未立即使用，则使用前的使用储存时间和条件由用户负责，在2至8°C下不得超过24小时。
储存的特殊预防措施
储存在冰箱中（2°C–8°C）。
不要冻结。
有关药物稀释后的储存条件。
容器的性质和内容
5ml小瓶（I型玻璃），带有塞子（氟树脂涂层丁基橡胶）、一体式密封件和蓝色翻盖。每个小瓶含有3ml用于输液的浓缩液。
包装为1、4和10瓶。并非所有包装尺寸均可上市。
请参阅随附的ELAPRASE完整处方信息：
https://www.medicines.org.uk/emc/medicine/19764/SPC/Elaprase+2+mg+ml+concentrate+for+
solution+for+infusion/
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ELAPRASE(idursulfase) is indicated for patients with Hunter syndrome (Mucopolysaccharidosis II, MPS II).
ELAPRASE has been shown to improve walking capacity in these patients.
Important Safety Information
Some patients in the clinical trials experienced life-threatening immediate allergic reactions to ELAPRASE (idursulfase) infusion.
Patients who have experienced severe allergic reactions may experience another allergic reaction approximately 24 hours after the initial reaction and may require prolonged observation.
Patients with compromised respiratory function or acute respiratory disease may have a higher risk of life-threatening reactions to ELAPRASE infusion and require additional monitoring.
Life-threatening allergic reactions have been seen in some patients during ELAPRASE infusions.
Reactions included difficulty breathing, lack of oxygen, seizure or loss of consciousness, hives, and/or swelling of the throat or tongue. Patients received an antihistamine to reverse the allergic reaction or corticosteroid to decrease inflammation prior to or during later infusions.
ELAPRASE was given more slowly or was stopped early if patients had serious reactions.
With these measures, no patients stopped treatment permanently due to an infusion reaction.
Because of the potential for life-threatening complications, the ELAPRASE infusion may have to be delayed for patients who have respiratory illness and/or fever.
The most common side effects that required intervention had to do with infusions. These included muscle and joint aches, headache, fever, rash, hives, itching and increased blood pressure.
These side effects decreased over time with continued ELAPRASE treatment.
In clinical trials, patients given ELAPRASE once a week for 52 weeks had the following side effects at a rate greater than placebo and at least 10%.
Approximately:
2 out of 3 patients had a fever or headache.
1 out of 3 patients had joint pain.
1 out of 4 patients had itching or high blood pressure.
1 out of 5 patients had general discomfort or disturbed vision or wheezing.
1 out of 6 patients had abscess or muscle/bone pain or chest wall pain or hives.
1 out of 7 patients had anxiety/irritability or irregular heart rhythm or stomach upset or swelling at the infusion site or a skin disorder or itchy rash or slight injury or a side effect from injury.
About half of the patients in clinical studies produced antibodies to treatment with ELAPRASE and these patients had an increase in infusion reactions. The presence of antibodies on the effectiveness of ELAPRASE is unknown.
Please see full Prescribing Information including Boxed Warning