重新审视威尔逊和荣格纳:筛选标准适合 21 世纪吗?

IF 4 Q1 GENETICS & HEREDITY International Journal of Neonatal Screening Pub Date : 2024-09-13 DOI:10.3390/ijns10030062
Elena Schnabel-Besson, Ulrike Mütze, Nicola Dikow, Friederike Hörster, Marina A Morath, Karla Alex, Heiko Brennenstuhl, Sascha Settegast, Jürgen G Okun, Christian P Schaaf, Eva C Winkler, Stefan Kölker
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引用次数: 0

摘要

在技术革新的推动下,新生儿筛查(NBS)面板不断扩大,基因组 NBS 试点项目的发展也日新月异。有关 NBS 疾病选择的决策仍以 1968 年发布的 Wilson 和 Jungner(WJ)标准为基础。尽管有这一统一的参考标准,但对 WJ 标准的解释和 NBS 项目的实际疾病选择却千差万别。我们进行了系统的文献检索[PubMED 搜索 "Wilson "和 "Jungner";最后一次搜索 16.07.22],以评估 WJ 标准对当前和未来 NBS 计划的适用性以及调整的必要性。至少有两名审稿人对 105 篇出版物(系统文献检索,77 篇;人工检索,28 篇)的相关内容进行了筛选,最后对 38 篇出版物进行了评估。受定性文本分析研究设计的限制,没有进行统计评估,而是有条理地收集整理了所报告的批评意见和改进建议。这揭示了 WJ 标准的一系列普遍局限性,如术语不准确、缺乏可衡量性和客观性、缺乏对儿科的关注以及缺乏对计划管理的指导。此外,它还揭示了在临床、诊断、治疗和经济方面受到批评的具体方面。研究发现,一个主要障碍是在实施 NBS 之前,人们对罕见病的自然史和表型多样性了解不全面,导致病例定义、风险分层和治疗适应症方面的不确定性。通过系统地收集和评估有关 NBS 的质量、安全性和(成本)有效性以及受筛查者长期获益的实际证据,可以弥补这一不足。一个旨在不断学习的综合性 NBS 公共卫生项目将满足这些要求,而一个整合了医学、伦理、法律和社会视角的多维度未来 NBS 项目框架也已迫在眉睫。
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Wilson and Jungner Revisited: Are Screening Criteria Fit for the 21st Century?

Driven by technological innovations, newborn screening (NBS) panels have been expanded and the development of genomic NBS pilot programs is rapidly progressing. Decisions on disease selection for NBS are still based on the Wilson and Jungner (WJ) criteria published in 1968. Despite this uniform reference, interpretation of the WJ criteria and actual disease selection for NBS programs are highly variable. A systematic literature search [PubMED search "Wilson" AND "Jungner"; last search 16.07.22] was performed to evaluate the applicability of the WJ criteria for current and future NBS programs and the need for adaptation. By at least two reviewers, 105 publications (systematic literature search, N = 77; manual search, N = 28) were screened for relevant content and, finally, 38 publications were evaluated. Limited by the study design of qualitative text analysis, no statistical evaluation was performed, but a structured collection of reported aspects of criticism and proposed improvements was instead collated. This revealed a set of general limitations of the WJ criteria, such as imprecise terminology, lack of measurability and objectivity, missing pediatric focus, and absent guidance on program management. Furthermore, it unraveled specific aspects of criticism on clinical, diagnostic, therapeutic, and economical aspects. A major obstacle was found to be the incompletely understood natural history and phenotypic diversity of rare diseases prior to NBS implementation, resulting in uncertainty about case definition, risk stratification, and indications for treatment. This gap could be closed through the systematic collection and evaluation of real-world evidence on the quality, safety, and (cost-)effectiveness of NBS, as well as the long-term benefits experienced by screened individuals. An integrated NBS public health program that is designed to continuously learn would fulfil these requirements, and a multi-dimensional framework for future NBS programs integrating medical, ethical, legal, and societal perspectives is overdue.

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来源期刊
International Journal of Neonatal Screening
International Journal of Neonatal Screening Medicine-Pediatrics, Perinatology and Child Health
CiteScore
6.70
自引率
20.00%
发文量
56
审稿时长
11 weeks
期刊最新文献
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