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首页> 外文期刊>Orphanet journal of rare diseases >Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry
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Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry

机译：猎人结果调查（HOS）的十年：从全球患者登记处获得的见识，成就和经验教训

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Mucopolysaccharidosis type II (MPS II; Hunter syndrome; OMIM 309900) is a rare lysosomal storage disease with progressive multisystem manifestations caused by deficient activity of the enzyme iduronate-2-sulfatase. Disease-specific treatment is available in the form of enzyme replacement therapy with intravenous idursulfase (Elaprase?, Shire). Since 2005, the Hunter Outcome Survey (HOS) has collected real-world, long-term data on the safety and effectiveness of this therapy, as well as the natural history of MPS II. Individuals with a confirmed diagnosis of MPS II who are untreated or who are receiving/have received treatment with idursulfase or bone marrow transplant can be enrolled in HOS. A broad range of disease- and treatment-related information is captured in the registry and, over the past decade, data from more than 1000 patients from 124 clinics in 29 countries have been collected. Evidence generated from HOS has helped to improve our understanding of disease progression in both treated and untreated patients and has extended findings from the formal clinical trials of idursulfase. As a long-term, global, observational registry, various challenges relating to data collection, entry, and analysis have been encountered. These have resulted in changes to the HOS database platform, and novel approaches to maximize the value of the information collected will also be needed in the future. The continued evolution of the registry should help to ensure that HOS provides further insights into the burden of the disease and patient care and management in the coming years.
机译：II型粘多糖贮积病（MPS II; Hunter综合征; OMIM 309900）是一种稀有的溶酶体贮积病，其病因是异丁二酸-2-硫酸酯酶的活性不足，导致多系统进行性表现。特定疾病的治疗可以采用静脉注射艾杜硫酶（Elaprase？，Shire）进行酶替代治疗。自2005年以来，猎人结果调查（HOS）收集了有关该疗法安全性和有效性以及MPS II的自然史的真实，长期数据。确诊为MPS II的未经治疗或正在接受/已经接受艾杜硫酶或骨髓移植治疗的个体可以加入HOS。在注册表中捕获了与疾病和治疗有关的各种信息，并且在过去十年中，收集了来自29个国家/地区的124个诊所的1000多名患者的数据。 HOS产生的证据有助于增进我们对已治疗和未治疗患者疾病进展的了解，并扩展了艾杜硫酶正式临床试验的发现。作为长期的全球观察性注册表，已经遇到了与数据收集，输入和分析有关的各种挑战。这些都导致了居屋数据库平台的变化，将来也将需要新颖的方法来最大化所收集信息的价值。登记册的不断发展应有助于确保居屋在未来几年内对疾病的负担以及患者的护理和管理提供进一步的见解。

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