The Prader-Willi Syndrome Anxiousness and Distress Behaviors Questionnaire: Development and Psychometric Validation |
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| Affiliation: | 1. Levo Therapeutics, Inc., Skokie, IL, USA;2. Foundation for Prader-Willi Research (FPWR), Walnut, CA, USA;3. Department of Rehabilitation Medicine, University of Washington School of Medicine, Seattle, WA, USA;4. Department of Genetics, University of Alabama at Birmingham, Birmingham, AL, USA;5. Department of Patient-Centered Outcomes Assessment, RTI Health Solutions, Research Triangle Park, NC, USA;1. The Comparative Health Outcomes, Policy, and Economics (CHOICE) Institute, School of Pharmacy, University of Washington, Seattle, WA, USA;2. Department of Health Services Research, Management & Policy, University of Florida, Gainesville, FL, USA;1. Decision Analysis and Support Unit, SGH Warsaw School of Economics, Warsaw, Poland;2. Erasmus School of Health Policy & Management, Erasmus University Rotterdam, Rotterdam, the Netherlands;3. EuroQol Research Foundation, Rotterdam, the Netherlands;4. School of Population Health, Curtin University, Perth, WA, Australia;5. Dalla Lana School of Public Health, University of Toronto, Toronto, ON, Canada;6. Child Health Evaluative Sciences, Hospital for Sick Children, Toronto, ON, Canada;7. Nuffield Department of Primary Care Health Sciences, University of Oxford, Oxford, England, UK;8. School of Nursing and Health Professions, University of San Francisco, San Francisco, CA, USA;2. Population Interventions, Centre for Epidemiology and Biostatistics, Melbourne School of Population and Global Health, University of Melbourne, Melbourne, Australia;1. The Ohio State University College of Medicine, Columbus, OH, USA;2. Erasmus University Rotterdam, Rotterdam, the Netherlands;3. Pfizer, Inc, New York, NY, USA;4. Evidera, London, England, UK;5. Janssen Research & Development, LLC, New Brunswick, NJ, USA;6. Beyond Celiac, Ambler, PA, USA;7. University of Pittsburgh, Pittsburgh, PA, USA;9. Novartis Pharma AG, Basel, Switzerland;10. Eric Low Consulting, Haddington, Scotland, UK;11. NICE, London, England, UK;12. University of Calgary, Calgary, AB, Canada;1. Department of Clinical and Experimental Sciences, University of Brescia, Brescia, Italy;2. Unit of Child Neurology and Psychiatry, ASST Spedali Civili of Brescia, Brescia, Italy;3. Department of Molecular and Translational Medicine, University of Brescia, Brescia, Italy;6. The Laboratory for Visual Neuroplasticity, Department of Ophthalmology, Massachusetts Eye and Ear, Harvard Medical School, Boston, MA, USA;4. Department of Neurological and Vision Sciences, ASST Spedali Civili of Brescia, Italy;5. University of Brescia, Eye Clinic, Brescia, Italy |
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| Abstract: | ObjectivesTo facilitate the development of new therapies for Prader-Willi syndrome (PWS), we sought to develop a reliable and valid assessment of anxiousness and distress, common characteristics that have a significant negative impact on individuals with PWS and their families.MethodsThe PWS Anxiousness and Distress Behaviors Questionnaire (PADQ) was developed with extensive input from clinical experts, as well as caregivers of individuals with PWS, who participated in iterative sets of qualitative interviews. The psychometric properties of the PADQ were subsequently demonstrated in a cross-sectional evaluation using data from the Global PWS Registry provided by > 400 caregivers and confirmed using data from a phase 3 clinical trial of an oxytocin analogue (intranasal carbetocin, LV-101).ResultsQualitative interview participants consistently endorsed the content of the PADQ and were confident they could accurately respond to each item based on their observations of their child’s behavior. Analysis of cross-sectional data supported the computation of a total PADQ score, as well as the reliability and validity of the measure. The results of analyses using longitudinal clinical trial data confirmed these properties and provided evidence for the responsiveness of the PADQ, further supporting its appropriateness for the evaluation of new treatments targeting anxiousness and distress in PWS.ConclusionsThe current body of evidence supports the conclusion that the PADQ measures observable behaviors that are meaningful to patients and their families and provides a valid and reliable method to assess beneficial treatment effects for some of the most challenging behaviors associated with PWS. |
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| Keywords: | anxiety anxiousness clinical trial Prader-Willi syndrome questionnaire validation |
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