TY - JOUR
T1 - Health-related quality of life and medication use among individuals with Angelman syndrome
AU - On behalf of the STARS Investigators
AU - Khan, Nasreen
AU - Cabo, Raquel
AU - Burdine, Rebecca D.
AU - Tan, Wen Hann
AU - Keary, Christopher J.
AU - Ochoa-Lubinoff, Cesar
AU - Bird, Lynne M.
N1 - Funding Information:
The authors thank the patients in the STARS study, their families and the STARS investigators and participating study centres. Statistical assistance in the preparation of this manuscript was provided by Maria Reynolds and Beth Sherrill at RTI Health Solutions. Beth Sherrill also reviewed and provided comments on the first draft of the manuscript. Financial support was from Ovid. Editorial support was provided by Shalaka Samant, Ph.D. from RWEC, LLC.
Funding Information:
This study was funded by Ovid Therapeutics Inc, New York, NY, USA.
Funding Information:
Dr. Khan is a paid consultant for Ovid Therapeutics. Ms. Cabo is an employee of Ovid Therapeutics. Dr. Burdine consults on clinical trials supported by Ovid Therapeutics. Dr. Tan has participated in clinical trials supported by Ovid Therapeutics and Dimension Therapeutics and has also received research support from Ovid Therapeutics. Dr. Keary has consulted for Ovid Therapeutics and is participating in clinical trials supported by Ovid Therapeutics. Dr. Ochoa-Lubinoff has consulted for Ovid Therapeutics and participated in clinical trials supported by Ovid Therapeutics. Dr. Bird is participating in clinical trials supported by Ovid Therapeutics.
Publisher Copyright:
© 2023, The Author(s), under exclusive licence to Springer Nature Switzerland AG.
PY - 2023/7
Y1 - 2023/7
N2 - 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.
AB - 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.
KW - Angelman Syndrome
KW - EQ-5D
KW - HRQoL
KW - Healthcare burden
KW - Healthcare economics
KW - Medication utilization
KW - VAS
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U2 - 10.1007/s11136-023-03375-4
DO - 10.1007/s11136-023-03375-4
M3 - Article
C2 - 37039911
AN - SCOPUS:85152386519
SN - 0962-9343
VL - 32
SP - 2059
EP - 2067
JO - Quality of Life Research
JF - Quality of Life Research
IS - 7
ER -