University of Colorado School of Medicine Department of Pediatrics, Section of Pediatric Hospital Medicine, 13123 East 16th Avenue, Box 302, Aurora, CO, United States; Children's Hospital Colorado Precision Medicine Institute, 13123 East 16th Avenue, Box 155, Aurora, CO 80045 United States.
University of Colorado School of Medicine Department of Pediatrics, Section of Neurology, 13123 East 16th Avenue, Box 155, Aurora, CO 80045, United States.
Epilepsy Res. 2023 Nov;197:107231. doi: 10.1016/j.eplepsyres.2023.107231. Epub 2023 Sep 20.
CDKL5 Deficiency Disorder (CDD) is a severe X-linked developmental and epileptic encephalopathy. Existing developmental outcome measures have floor effects and cannot capture incremental changes in symptoms. We modified the caregiver portion of a CDD clinical severity assessment (CCSA) and assessed content and response-process validity.
We conducted cognitive interviews with 15 parent caregivers of 1-39-year-old children with CDD. Caregivers discussed their understanding and concerns regarding appropriateness of both questions and answer options. Item wording and questionnaire structure were adjusted iteratively to ensure questions were understood as intended.
The CCSA was refined during three rounds of cognitive interviews into two measures: (1) the CDD Developmental Questionnaire - Caregiver (CDQ-Caregiver) focused on developmental skills, and (2) the CDD Clinical Severity Assessment - Caregiver (CCSA-Caregiver) focused on symptom severity. Branching logic was used to ensure questions were age and skill appropriate. Initial pilot data (n = 11) suggested no floor effects.
This study modified the caregiver portion of the initial CCSA and provided evidence for its content and response process validity.
CDKL5 缺乏症（CDD）是一种严重的 X 连锁发育性和癫痫性脑病。现有的发育结果测量方法存在下限效应，无法捕捉症状的增量变化。我们修改了 CDD 临床严重程度评估（CCSA）的照顾者部分，并评估了其内容和反应过程的有效性。
我们对 15 名 1-39 岁患有 CDD 的儿童的照顾者进行了认知访谈。照顾者讨论了他们对问题和答案选项的适当性的理解和关注。通过迭代调整问题措辞和问卷结构，以确保问题得到了预期的理解。
在三轮认知访谈中，CCSA 被细化为两个衡量标准：（1）关注发育技能的 CDD 发育问卷-照顾者（CDQ-Caregiver），（2）关注症状严重程度的 CDD 临床严重程度评估-照顾者（CCSA-Caregiver）。采用分支逻辑来确保问题适合年龄和技能。初步试点数据（n=11）表明不存在下限效应。
本研究对初始 CCSA 的照顾者部分进行了修改，并提供了其内容和反应过程有效性的证据。
