laronidase治疗的长期疗效和安全性黏多糖病。
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克拉克拉,幽灵我,贝克M, Kolodny呃,牧师通用,Muenzer J,拉波波特DM,伯杰KI, Sidman M, Kakkis ED,考克斯的女朋友
laronidase治疗的长期疗效和安全性黏多糖病。
儿科。2009年1月,123 (1):229 - 40。doi: 10.1542 / peds.2007 - 3847。
- PubMed ID
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19117887 (在PubMed]
- 文摘
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目的:我们的目标是评估重组人类的长期安全性和有效性alpha-l-iduronidase (laronidase)患者的黏多糖病即病人和方法:所有45完成了26周的患者,双盲,安慰剂对照试验laronidase被注册在一个3.5年里开放的扩展研究。意味着患者基线年龄16岁(范围:6-43)年。所有患者减毒疾病(Scheie表型Hurler-Scheie 84%, 16%)。临床、生化和健康结果的措施进行评估通过扩展阶段。变化了的意思是+ / - SEM。结果:40例(89%)完成了试验获得至少80%的计划注入。正如前面所示的试验、尿粘多糖水平下降在头12周内和肝体积在第一年内减少。预测百分比用力肺活量保持稳定,每年-0.78的线性斜率。6分钟步行距离的增加31.7 + / - 10.2米在第一个两年,最终获得17.1 + / - 16.8米。改善呼吸暂停/呼吸不足指数(减少每小时7.6 + / - 4.5事件的重要基线睡眠呼吸暂停患者)和肩膀弯曲(增加17.4度+ / - 3.6度)是最快速的在第一个两年。 Improvements in the Child Health Assessment Questionnaire/Health Assessment Questionnaire disability index (decrease of 0.31 +/- 0.11, signifying a clinically meaningful improvement in activities of daily living) were gradual and sustained over the treatment period. Laronidase infusions were generally well tolerated except in 1 patient who experienced an anaphylactic reaction. Infusion-associated reactions, which occurred in 53% of the patients, were mostly mild, easily managed, and decreased markedly after 6 months. One patient died as a result of an upper respiratory infection unrelated to treatment. Antibodies to laronidase developed in 93% of the patients; 29% of the patients were seronegative at their last assessment. CONCLUSIONS: This trial demonstrates the long-term clinical benefit and safety of laronidase in attenuated patients with mucopolysaccharidosis I and highlights the magnitude and chronology of treatment effects. Prompt diagnosis and early treatment will maximize treatment outcomes.
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