ECEESPE2025 Poster Presentations MTEabolism, Nutrition and Obesity (125 abstracts)
The impact of prader-willi syndrome (PWS) on caregivers and the healthcare system: a burden of illness study design
Evelien Gevers 1 , Kathryn Obrynba 2 , Dairine Dempsey 3 , Kristen Yen 3 , Maria Hall 4 , Jackie Lodge 5 , Catherine Shaw 6 , Helen Ramage 6 , Theresa Strong 7 , Stacy Ward 7 , Lorraine Munetsi 8 & Tom Blenkiron 8
1Barts and the London Medical School, London, United Kingdom; 2The Research Institute at Nationwide Children’s Hospital, Columbus, United States; 3Soleno Therapeutics Inc., Redwood City, United States; 4Maria Hall Consulting, London, United Kingdom; 5PWSA UK, Derby, United Kingdom; 6FPWR UK, Bradford, United Kingdom; 7FPWR USA, Covina, United States; 8PRIME HCD, Daresbury, United Kingdom
JOINT1567
Background: Prader-Willi syndrome (PWS) is a rare, genetic, neurobehavioural-metabolic disorder characterised by hyperphagia and behavioural/psychological complications. Managing PWS symptoms through strict supervision, behavioral management and specialised supports imposes significant demands on caregivers and healthcare systems. However, data are lacking on the real-world burden of PWS.
Objectives: The objective was to design a study that characterises the clinical, humanistic, and economic burden of PWS on caregivers, families, and the healthcare system.
Study design: This study is a descriptive, retrospective, cross-sectional, multi-site, micro-costing burden of illness study. The study will be conducted in USA, UK, France, Germany, and Italy. To assess the burden of PWS, a retrospective review will be conducted by consenting approximately 330 healthcare professionals (HCPs) who meet the following inclusion criteria: 1) primary specialty in paediatrics, endocrinology, or psychiatry; 2) qualified in their medical specialty for at least three years and 3) personally responsible for the management of patients with PWS. HCPs who qualify and consent to participate will complete electronic case record forms (eCRFs) for eligible patients under their care. Patients are eligible for inclusion if they are aged 4 years and over, with a diagnosis of PWS >12 months, and their caregiver is aged 18 years or over and capable of providing informed consent. The eCRF will collect data on PWS disease history, management, and burden during the past 12 months from the date of consultation with the HCP. Aspects captured by the eCRF will include socio-demographics, symptoms, disease history, comorbidities, management, interventions, and care requirements. HCPs will invite caregivers of these patients to complete corresponding Patient Public Involvement & Engagement caregiver forms (PPIE-c). The PPIE-c will capture health-related quality of life (HRQoL) through EuroQoL 5-Dimensions 5-levels (EQ-5D-5L); the Work Productivity and Activity Impairment (WPAI) Questionnaire; the Hyperphagia Questionnaire for Clinical Trials (HQ-CT), the Zarit Burden Interview (ZBI) and the Food Safe Zone (FSZ) Questionnaire. The PPIE-c will help provide insights into the wider impact of PWS on caregivers and families. All eCRF and PPIE-c data will be collected anonymously. The mean per-patient cost of PWS will be calculated based on a comprehensive resource utilisation analysis incorporating unit cost estimates for direct medical and non-medical, and indirect resource requirements. Key factors to be evaluated include hospitalisations, HCP visits, treatments, PWS-related transportation, specialised equipment, education status, and dietary modifications. This study is sponsored by Soleno Therapeutics, Inc.
Volume 110
Article tools
My recent searches
My recently viewed abstracts
Authors
Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
© Bioscientifica 2025 |
Privacy policy |
Cookie settings
BiosciAbstracts
Bioscientifica Abstracts is the gateway to a series of products that provide a permanent, citable record of abstracts for biomedical and life science conferences.
Find out more