Health-related quality of life and medication use among individuals with Angelman syndrome. Author Nasreen Khan, Raquel Cabo, Rebecca Burdine, Wen-Hann Tan, Christopher Keary, Cesar Ochoa-Lubinoff, Lynne Bird, STARS Investigators Publication Year 2023 Type Journal Article Abstract PURPOSE: The 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).METHODS: The 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).RESULTS: 87 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.CONCLUSION: This study highlights the impact of AS on HRQoL and medication utilization among adolescents and adults individuals with AS. Keywords Angelman Syndrome, EQ-5D, HRQoL, Healthcare burden, Healthcare economics, Medication utilization, VAS Journal Quality of life research : an international journal of quality of life aspects of treatment, care and rehabilitation Volume 32 Issue 7 Pages 2059-2067 Date Published 07/2023 ISSN Number 1573-2649 DOI 10.1007/s11136-023-03375-4 Alternate Journal Qual Life Res PMID 37039911 PubMedGoogle ScholarBibTeXEndNote X3 XML