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Quality of Life Research
Gepubliceerd in: Quality of Life Research 7/2023

11-04-2023

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

Auteurs: Nasreen Khan, Raquel Cabo, Rebecca D. Burdine, Wen-Hann Tan, Christopher J. Keary, Cesar Ochoa-Lubinoff, Lynne M. Bird, On behalf of the STARS Investigators

Gepubliceerd in: Quality of Life Research | Uitgave 7/2023

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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.
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Metagegevens
Titel
Health-related quality of life and medication use among individuals with Angelman syndrome
Auteurs
Nasreen Khan
Raquel Cabo
Rebecca D. Burdine
Wen-Hann Tan
Christopher J. Keary
Cesar Ochoa-Lubinoff
Lynne M. Bird
On behalf of the STARS Investigators
Publicatiedatum
11-04-2023
Uitgeverij
Springer International Publishing
Gepubliceerd in
Quality of Life Research / Uitgave 7/2023
Print ISSN: 0962-9343
Elektronisch ISSN: 1573-2649
DOI
https://doi.org/10.1007/s11136-023-03375-4

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