Health-related quality of life and medication use among individuals with Angelman syndrome

PurposeThe primary goal of this analysis is to describe the health-related quality of life (HRQoL), medical history, and medication use among adolescents and adults individuals with Angelman syndrome (AS).MethodsThe analysis uses baseline data collected during the STARS study, a double-blind placebo controlled trial of gaboxadol (OV101) in adolescents and adults with AS. The HRQoL was estimated using EuroQoL 5-Dimension 5-Level (EQ-5D) health questionnaire proxy 1 version, which was completed by the caregivers. EQ-5D consists of two parts, a 5-dimension descriptive and a visual analogue scale (VAS) component. The utility score derived from EQ-5D ranges from 0 to 1 (perfect health) and VAS ranges from 0 to 100 (perfect health).Results87 individuals with AS were included in the present analysis. The mean utility score was 0.44 +/- 0.20 and VAS score was 84 +/- 1.5. The EQ-5D data indicated that the self-care, mobility and daily activities were most impacted. All adolescents (100%) and most adults (93%) had at least moderate problems with self-care activities, such as washing or dressing themselves. More than half (55%) of the adolescents and adults had at least moderate issues with mobility and usual activities. Approximately, 30% of adolescents and adults had moderate to extreme problems with anxiety/depression. High baseline concomitant use of medications was observed across both age groups with an average of 5 medications being used per person.ConclusionThis study highlights the impact of AS on HRQoL and medication utilization among adolescents and adults individuals with AS.

Automated summary

The purpose of this analysis is to study the quality of life, medical history, and medication use among individuals with Angelman syndrome (AS). The study used baseline data collected during a trial of gaboxadol (OV101) and found that AS has a significant impact on self-care, mobility, and daily activities. The study also showed that there is a high use of medications among AS patients.