使用智能手机应用程序评估威廉姆斯-贝伦综合征焦虑症治疗效果：单病例实验方案

Evaluation of the Effectiveness of Therapy for Anxiety in Williams Beuren Syndrome Using a Smartphone App: Protocol for a Single-Case Experiment.

作者信息

Lehman Natacha, Trouillet Raphaël, Genevieve David

机构信息

EA4556 Laboratoire Epsylon, Université Paul Valery Montpellier 3, Montpellier, France.

Genetic Department, Montpellier Hospital, Montpellier, France.

出版信息

JMIR Res Protoc. 2023 Apr 3;12:e44393. doi: 10.2196/44393.

DOI:10.2196/44393

PMID:37010888

原文链接:https://pmc.ncbi.nlm.nih.gov/articles/PMC10131632/

Abstract

BACKGROUND

Williams syndrome (WS-OMIM 194050, orphaned number: Orpha 904) is a rare condition mostly associated with intellectual disability. People with Williams syndrome are 8 times more likely to have anxiety disorders than the general population. Therapeutic solutions to treat the anxiety remain limited, particularly nonpharmacological therapy. However, cognitive behavioral therapy (CBT) has been found efficacious in managing anxiety disorders and can be used for people with intellectual disability.

OBJECTIVE

This paper describes a protocol to assess the efficiency of a CBT program based on digital support for people with Williams syndrome and anxiety based on a research methodology designed for rare diseases.

METHODS

We will recruit 5 individuals with Williams syndrome and anxiety. They will participate in 9 CBT sessions. Participants will perform daily self-assessments of anxiety using a digital app, which will allow for ecological and repeated evaluation of their anxiety. This digital app will provide support for each therapy session. Anxiety and quality of life will be externally assessed before and after the program and at a 3-month follow-up. This is a single-case intervention research design with multiple baselines implying repeated measures of judgment criteria. The present protocol ensures high internal validity and will help identify encouraging contributions for later clinical trials.

RESULTS

Participant recruitment and data collection began in September 2019, and we project that the study findings will be available for dissemination by spring 2023.

CONCLUSIONS

This study will allow the assessment of the efficiency of a CBT program based on digital support to treat anxiety in people with Williams syndrome. Finally, the program could be used as an example of nonpharmacological therapy for rare diseases.

TRIAL REGISTRATION

ClinicalTrials.gov ID: NCT03827525; https://clinicaltrials.gov/ct2/show/NCT03827525.

INTERNATIONAL REGISTERED REPORT IDENTIFIER (IRRID): DERR1-10.2196/44393.

摘要

背景

威廉姆斯综合征（WS - OMIM 194050，孤儿编号：Orpha 904）是一种罕见病症，大多与智力残疾相关。患有威廉姆斯综合征的人患焦虑症的可能性比普通人群高8倍。治疗焦虑症的解决方案仍然有限，尤其是非药物治疗。然而，认知行为疗法（CBT）已被证明在管理焦虑症方面有效，并且可用于智力残疾患者。

目的

本文描述了一种方案，该方案基于针对罕见病设计的研究方法，评估基于数字支持的CBT项目对患有威廉姆斯综合征并伴有焦虑症的患者的疗效。

方法

我们将招募5名患有威廉姆斯综合征并伴有焦虑症的患者。他们将参加9次CBT治疗课程。参与者将使用一款数字应用程序进行每日焦虑自我评估，这将允许对他们的焦虑进行生态学和重复性评估。该数字应用程序将为每次治疗课程提供支持。在项目前后以及3个月随访时，将由外部评估焦虑症和生活质量。这是一项单病例干预研究设计，具有多个基线，意味着对判断标准进行重复测量。本方案确保了较高的内部效度，并将有助于为后续临床试验确定有启发性的贡献。