Adaptive behavior assessed by Vineland-3 as comprehensive outcome measure in vanishing white matter

Abstract

Objectives: Investigate the results and usability of the Vineland-3 as an outcome measure in vanishing white matter patients.

Methods: A cross-sectional investigation of the Vineland-3 based on interviews with caregivers, the Health Utilities Index, and the modified Rankin Scale in 64 vanishing white matter patients.

Results: Adaptive behavior measured with the Vineland-3 is impaired in the vast majority of vanishing white matter patients and significantly impacts daily life. Typically, the daily living skills domain is most severely affected and the socialization domain is the least affected. Based on the metric properties and the clinical relevance, the standard scores for the daily living skills domain and Adaptive Behavior Composite have the best properties to be used as an outcome measure.

Interpretation: The Vineland-3 appears to be a useful outcome measure to explore and quantify complex cognitive, behavioral, and psychiatric impairments affecting daily functioning in vanishing white matter. Further research should address the longitudinal evaluation of this tool and its additional value to standard neuropsychological and clinical examination.

Figure 1

Scatter plot of current age by age at onset. Legend: Current age groups: Yellow dot, <6 years. Blue dot, 6–<18 years. Red dot, ≥18 years.

Figure 2

V‐scale scores of subdomains by disease duration. The R² of the exponential regression lines are the following: receptive: 0.336, expressive: 0.220, written: 0.340, personal: 0.269, domestic: 0.530, community: 0.474, interpersonal relationships: 0.192, play and leisure: 0.168, and coping: 0.251. Legend: Current age groups: Yellow dot, <6 years. Blue dot, 6–<18 years. Red dot, ≥18 years.

Figure 3

Standard scores of ABC and domains by disease duration. Exponential regression lines are shown with an R² of ABC: 0.487, communication: 0.436, daily living skills: 0.578, and socialization: 0.372. Legend: Current age groups: Yellow dot, <6 years. Blue dot, 6–<18 years. Red dot, ≥18 years.

Figure 4

The ABC is plotted versus the HUI generic score (A) and modified Rankin Scale (B). The quadratic regression line for the correlation with the HUI generic score is shown and has an R² of 0.581. The levels of the modified Rankin Scale correspond to the following clinical status: 0, no symptoms at all; 1, no significant disability despite symptoms, able to carry out all usual duties and activities; 2, slight disability, unable to carry out all previous activities, but able to look after own affairs without assistance; 3, moderate disability, requiring some help, but able to walk without assistance; 4, moderately severe disability, unable to walk and attend to bodily needs without assistance; 5, severe disability, bedridden, incontinent, and requiring constant nursing care and attention; and 6, dead. Legend: Current age groups: Yellow dot, <6 years. Blue dot, 6–<18 years. Red dot, ≥18 years.