Page 92 - Demo

Chapter 490best practice and recommended to measure for refractory or difficult to treat behavioral manifestations in this population. The shortened version Vineland Adaptive Behavior Scales-III (VABS-III)58 will be filled out by the clinicians to determine whether the SRS-2 could be filled out during the trial.59 Optionally, electroencephalogram (EEG) recordings will be performed at baseline to detect changes in brain resting-state EEG for stratification of responsiveness post hoc.Rationale for outcome measures As a primary outcome measure, the ABC-I was selected. The ABC is a caregiver-completed rating scale for assessing problem behaviors in children and adults, which has robust psychometric properties in intellectually impaired and developmentally delayed populations.47,60,61The empirically derived and widely used irritability subscale consists of 15 items and comprises items reflecting temper outbursts, aggression, negative affect, and self-harm behavior. Irritability has been identified as a prominent behavioral correlate of ASD. The ABC-I has often been used as an outcome measure in treatment studies of behavioral problems in individuals with ASD and ID.62–64 The ABC, including the irritably subscale, has been shown to be sensitive to treatment change in previous clinical trials of FXS.65,66 The other domains of the ABC, including lethargy/social withdrawal, stereotypic behavior, hyperactivity/noncompliance, and inappropriate speech, will serve as generalization measures to evaluate transfer effects of the intervention to a broader domain of functioning.67 A generalization measure is an outcome measure that is related to the target behavior (irritability), for example irritability at school and at home (another setting), or interventional effects on a completely different behavior, such as less hyperactive when the target behavior is irritability.By including an extensive set of secondary outcome measures in this study, we aim to explore the effectiveness of CBD on several behavioral and psychiatric domains as it is yet unclear which manifestations specifically respond to treatment. Moreover, we aim to explore if these outcome measures are appropriate and useful in these patient groups and in RGNDs in general. We chose outcome measures at different levels, such as disorder-specific, personalized and generalizable measures. As most Annelieke Muller sHL.indd 90 14-11-2023 09:07