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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Data availability
Ovid Therapeutics Inc (Ovid) is committed to providing qualified scientific researchers appropriate access to anonymized data and clinical study information from the company’s clinical trials for the purpose of conducting legitimate scientific research. Additionally, Ovid shall post all company-sponsored safety and efficacy studies of clinical trial participants on the clinical trial registries of the U.S. National Institutes of Health (NIH) and European Medicines Agency (EMA), as mandated by applicable laws and regulations. Ovid supports an approach to sharing data that responsibly reflects the interests of all parties involved in clinical trials, including protecting (i) the rights and privacy of trial participants, (ii) the innovator’s intellectual property rights, and (iii) other incentives for innovation, and as such, will evaluate requests for sharing company clinical trial data with qualified external scientific researchers. Data will be made available for request either (i) after product approval in the United States and European Union, (ii) after product development is discontinued or (iii) as otherwise required by law or regulation. There are circumstances that may prevent Ovid from sharing the requested data as the product is still investigational at this time.
Code availability
Not applicable.
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Acknowledgements
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
This study was funded by Ovid Therapeutics Inc, New York, NY, USA.
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All authors read and approved the final manuscript. All authors helped interpret the data and contributed to the manuscript. NK designed the analyses, interpreted the data and wrote the manuscript.
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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.
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Khan, N., Cabo, R., Burdine, R.D. et al. Health-related quality of life and medication use among individuals with Angelman syndrome. Qual Life Res 32, 2059–2067 (2023). https://doi.org/10.1007/s11136-023-03375-4
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DOI: https://doi.org/10.1007/s11136-023-03375-4