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本文引用的文献

1
Adaptive Skills of Individuals with Angelman Syndrome Assessed Using the Vineland Adaptive Behavior Scales, 2nd Edition.使用《韦氏适应行为量表》第二版评估安格曼综合征个体的适应技能。
J Autism Dev Disord. 2024 Oct;54(10):3863-3887. doi: 10.1007/s10803-023-06090-8. Epub 2023 Aug 15.
2
Electrophysiological Abnormalities in Angelman Syndrome Correlate With Symptom Severity.天使综合征的电生理异常与症状严重程度相关。
Biol Psychiatry Glob Open Sci. 2021 Sep;1(3):201-209. doi: 10.1016/j.bpsgos.2021.05.003. Epub 2021 May 25.
3
Nutritional Formulation for Patients with Angelman Syndrome: A Randomized, Double-Blind, Placebo-Controlled Study of Exogenous Ketones.《用于 Angelman 综合征患者的营养配方:外源性酮体的随机、双盲、安慰剂对照研究》
J Nutr. 2021 Dec 3;151(12):3628-3636. doi: 10.1093/jn/nxab284.
4
Therapies in preclinical and clinical development for Angelman syndrome.用于治疗 Angelman 综合征的临床前和临床开发疗法。
Expert Opin Investig Drugs. 2021 Jul;30(7):709-720. doi: 10.1080/13543784.2021.1939674. Epub 2021 Jun 28.
5
Using Applied Conversation Analysis in Patient Education.在患者教育中运用应用会话分析
Glob Qual Nurs Res. 2021 May 28;8:23333936211012990. doi: 10.1177/23333936211012990. eCollection 2021 Jan-Dec.
6
Developmental Skills of Individuals with Angelman Syndrome Assessed Using the Bayley-III.使用贝利婴幼儿发展量表第三版评估天使综合征个体的发育技能。
J Autism Dev Disord. 2023 Feb;53(2):720-737. doi: 10.1007/s10803-020-04861-1. Epub 2021 Jan 30.
7
Comprehensive Assessment of Individuals With Significant Levels of Intellectual Disability: Challenges, Strategies, and Future Directions.全面评估具有显著智力障碍水平的个体:挑战、策略和未来方向。
Am J Intellect Dev Disabil. 2020 Nov 1;125(6):434-448. doi: 10.1352/1944-7558-125.6.434.
8
Angelman syndrome genotypes manifest varying degrees of clinical severity and developmental impairment.天使综合征的基因型表现出不同程度的临床严重程度和发育障碍。
Mol Psychiatry. 2021 Jul;26(7):3625-3633. doi: 10.1038/s41380-020-0858-6. Epub 2020 Aug 13.
9
The REDCap consortium: Building an international community of software platform partners.REDCap 联盟:构建软件平台合作伙伴的国际社区。
J Biomed Inform. 2019 Jul;95:103208. doi: 10.1016/j.jbi.2019.103208. Epub 2019 May 9.
10
Maladaptive behaviors in individuals with Angelman syndrome.患有安格曼综合征个体的适应不良行为。
Am J Med Genet A. 2019 Jun;179(6):983-992. doi: 10.1002/ajmg.a.61140. Epub 2019 Apr 3.

在天使综合征中使用贝利婴幼儿发展量表第4版和文兰适应行为量表第3版:障碍、解决方案及具有挑战性的项目

Using the Bayley-4 and Vineland-3 in Angelman syndrome: barriers, solutions, and challenging items.

作者信息

Halpin Sean N, Potter Sarah Nelson, Gwaltney Angela, Sadhwani Anjali, Okoniewski Katherine C, Wheeler Anne C

机构信息

GenOmics and Translational Research Center, RTI International, 3040 East Cornwallis Road, Research Triangle Park, Durham, NC, 27709-2194, USA.

Department of Psychiatry and Behavioral Sciences, Boston Children's Hospital, Boston, MA, USA.

出版信息

Orphanet J Rare Dis. 2025 Jun 3;20(1):273. doi: 10.1186/s13023-025-03817-x.

DOI:10.1186/s13023-025-03817-x
PMID:40462183
原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC12135291/
Abstract

OBJECTIVE

The Bayley Scales of Infant and Toddler Development- 4th Edition (Bayley-4) and Vineland Adaptive Behavior Scales - 3rd Edition (Vineland-3) are outcome measures often considered as primary endpoints in clinical trials for Angelman syndrome (AS). We explored barriers encountered when administering these instruments to individuals with AS and associated guidance for their use in trials and research studies.

METHODS

We interviewed nine clinicians who have administered the Bayley-4 and/or the Vineland-3 to individuals with AS and analyzed their transcripts using a quasi-deductive analysis approach.

RESULTS

Barriers to administering the Bayley-4 included participant's lack of interest, overexcitement, emotional impact on caregiver, the mental workload of administering the Bayley-4, and environmental factors (e.g., administration setting). Barriers to administering the Vineland-3 included determining the most appropriate start point, emotional impact on caregiver, distractions, conflicting answers from two caregivers, and the mental workload of administering the Vineland-3. Participants provided potential solutions to each barrier. Lastly, we identified overarching item-level concerns for both the Bayley-4 (i.e., administration challenges, items not aligned with abilities) and the Vineland-3 (i.e., misalignment of assessment criteria and condition characteristics, limitations in observation and contextual understanding, requires specialized training).

CONCLUSION

Clinical trials often rely on the Bayley-4 and Vineland-3 assessments as outcome measures, yet our identified barriers threaten their validity. The associated solutions provide a path forward for improving administration of the Bayley-4 and Vineland-3 in clinical practice, research, and future trials focused on individuals with AS and other intellectual and developmental disabilities.

摘要

目的

贝利婴幼儿发展量表第4版(Bayley-4)和文兰适应行为量表第3版(Vineland-3)是通常在天使综合征(AS)临床试验中被视为主要终点的结果测量工具。我们探讨了在对AS患者使用这些工具时遇到的障碍以及在试验和研究中使用它们的相关指导。

方法

我们采访了9位曾对AS患者使用过Bayley-4和/或Vineland-3的临床医生,并使用准演绎分析方法分析了他们的访谈记录。

结果

使用Bayley-4的障碍包括参与者缺乏兴趣、过度兴奋、对照顾者的情绪影响、使用Bayley-4的心理工作量以及环境因素(如施测环境)。使用Vineland-3的障碍包括确定最合适的起始点、对照顾者的情绪影响、干扰因素、两位照顾者给出的相互矛盾的答案以及使用Vineland-3的心理工作量。参与者针对每个障碍提供了潜在的解决方案。最后,我们确定了Bayley-4(即施测挑战、项目与能力不匹配)和Vineland-3(即评估标准与病情特征不匹配、观察和情境理解方面的局限性、需要专业培训)在总体项目层面的问题。

结论

临床试验通常依赖Bayley-4和Vineland-3评估作为结果测量工具,但我们发现的障碍威胁到了它们的有效性。相关解决方案为在临床实践、研究以及未来针对AS及其他智力和发育障碍患者的试验中改进Bayley-4和Vineland-3的使用提供了一条前进的道路。