Leclezio Loren, Jansen Anna, Whittemore Vicky H, de Vries Petrus J
Division of Child & Adolescent Psychiatry, Department of Psychiatry and Mental Health, University of Cape Town, Cape Town, South Africa.
Pediatric Neurology Unit, Department of Pediatrics, UZ Brussel, Vrije Universiteit Brussel, Brussels, Belgium.
Pediatr Neurol. 2015 Jan;52(1):16-24. doi: 10.1016/j.pediatrneurol.2014.10.006. Epub 2014 Oct 16.
Tuberous sclerosis complex is a multisystem disorder that includes a range of tuberous sclerosis-associated neuropsychiatric disorders (TAND). The lifetime prevalence rates of TAND are very high; yet surveys suggest that the majority of individuals with tuberous sclerosis never receive appropriate assessment or treatment for TAND. To aid systematic enquiry, a TAND Checklist was developed. Here, we performed pilot validation of the TAND Checklist.
Mixed methods were used across two stages. In stage 1, we gathered feedback on the Checklist from tuberous sclerosis "expert professionals" and "expert parents and caregivers." The aim was to examine face and content validity. Stage 2 involved the administration of the refined TAND Checklist to 20 parents of individuals with tuberous sclerosis concurrently with four widely used validated rating scales, to examine external validity and obtain qualitative feedback on face-to-face administration of the TAND Checklist.
Twenty professionals and 62 parents and caregivers from 28 countries participated in the pilot. The TAND Checklist demonstrated good face and content validity with high overall mean and median scores. Qualitative analysis highlighted concerns about the likely use of the TAND Checklist, suggesting that family members and individuals with tuberous sclerosis should drive usage. Stage 2 results showed moderate-to-very good external validity across TAND domain and key subdomains. Internal consistency of domains and subdomains was acceptable to very good. Ninety-three percent of all participants (93%) reported four or more lifetime TAND behavioral difficulties.
The pilot validation suggested that the TAND Checklist could provide a useful screening tool in clinical settings.
结节性硬化症是一种多系统疾病，包括一系列与结节性硬化症相关的神经精神障碍（TAND）。TAND的终生患病率非常高；然而，调查表明，大多数结节性硬化症患者从未接受过针对TAND的适当评估或治疗。为了有助于进行系统的调查，制定了一份TAND清单。在此，我们对TAND清单进行了初步验证。
在两个阶段使用了混合方法。在第1阶段，我们从结节性硬化症“专家专业人员”以及“专家家长和照顾者”那里收集了关于该清单的反馈。目的是检查表面效度和内容效度。第2阶段涉及向20位结节性硬化症患者的家长同时发放经过完善的TAND清单以及四种广泛使用的经过验证的评定量表，以检查外部效度，并获得关于TAND清单面对面施测的定性反馈。
来自28个国家的20名专业人员以及62名家长和照顾者参与了此次试点。TAND清单显示出良好的表面效度和内容效度，总体平均得分和中位数得分都很高。定性分析突出了对TAND清单可能用途的担忧，表明家庭成员和结节性硬化症患者应推动其使用。第2阶段的结果显示，在TAND领域和关键子领域，外部效度从中度到非常好。各领域和子领域的内部一致性从可接受到非常好。所有参与者中有93%报告在一生中出现过四种或更多TAND行为困难。
初步验证表明，TAND清单可在临床环境中提供一种有用的筛查工具。
