Farmer Cristan, Lewis Monica, Farhat Nicole, Robbins Kendall P, Joseph Lisa, Albert Orsolya K, Bianconi Simona, Hoffmann Anne, Giserman-Kiss Ivy, Alexander Derek M, Thurm Audrey, Porter Forbes D, Kravis Elizabeth Berry
National Institute of Mental Health, Bethesda, MD, USA.
Rush University Medical Center, Chicago, IL, USA.
J Child Neurol. 2025 Jun 17:8830738251346348. doi: 10.1177/08830738251346348.
The 5-Domain Niemann Pick Type C Clinical Severity Scale (5DNPCCSS) is used in clinical practice and trials. Although psychometric data support the clinical meaningfulness of the concepts and the scale's interrater reliability, more information is needed to support its construct validity. Here, we evaluated the convergent validity of the Cognition, Speech, and Fine Motor domains. Data from 121 individuals with Niemann-Pick disease type C were drawn from several studies conducted at 2 US sites. Direct standardized assessments included the Nine-Hole pegboard or Purdue pegboard, a portion of the Clinical Evaluation of Language Fundamentals, and the age-appropriate Wechsler IQ test or the Mullen Scales of Early Learning. The 5DNPCCSS domains were significantly related in the expected directions to their respective direct assessments, supporting their construct validity. In combination with previous evidence presented for the Ambulation and Swallow domains, these results support the fitness of purpose of the (5DNPCCSS for clinical studies in Niemann-Pick disease type C. ClinicalTrials.gov: NCT00344331, NCT01747135, NCT02534844.
5 领域尼曼匹克 C 型临床严重程度量表（5DNPCCSS）用于临床实践和试验。尽管心理测量数据支持这些概念的临床意义以及该量表的评分者间信度，但仍需要更多信息来支持其结构效度。在此，我们评估了认知、言语和精细运动领域的收敛效度。来自美国 2 个地点进行的多项研究的 121 名尼曼匹克 C 型病患者的数据。直接标准化评估包括九孔插板或普渡插板、语言基本能力临床评估的一部分，以及适合年龄的韦氏智商测试或莫伦早期学习量表。5DNPCCSS 领域在预期方向上与其各自的直接评估显著相关，支持其结构效度。结合先前为行走和吞咽领域提供的证据，这些结果支持 5DNPCCSS 适用于尼曼匹克 C 型病临床研究的目的。ClinicalTrials.gov：NCT00344331、NCT01747135、NCT02534844。
