ECEESPE2025 ePoster Presentations Growth Axis and Syndromes (132 abstracts)
Development of a minimum dataset (MDS) for the monitoring of growth hormone therapy in children with prader willi syndrome (PWS) - a globe-reg initiative
Ashley Leong 1 , Suet Ching Chen 2,3 , Malika Alimussina 2,3 , Jillian Bryce 3 , Minglu Chen 3 , Antony Fu 4 , Evelien Gevers 5 , Charlotte Höybye 6 , Marguerite Hughes 7 , Violeta Iotova 8 , Muhammad Yazid Jalaludin 9 , Gerthe Kerkhof 10 , Fei Hong Luo 11 , Jennifer Miller 12 , Ohn Nyunt 13 , Edna Roche 14 15 , M Guftar Shaikh 2 3 , Theresa Strong 16 , Maithé Tauber 17 , Latife Salomão Tyszler 18 & Faisal Ahmed 2,3
1University of Glasgow, School of Medicine, Dentistry and Nursing, Glasgow, United Kingdom; 2Royal Hospital for Children, Developmental Endocrinology Research Group, Glasgow, United Kingdom; 3University of Glasgow, Office for Rare Conditions, Glasgow, United Kingdom; 4Princess Margaret Hospital, Department of Paediatrics, Hong Kong, China; 5Barts Health and Queen Mary University of London, London, United Kingdom; 6Karolinska Institute, Department of Molecular Medicine and Surgery, Stockholm, Sweden; 7International Prader-Willi Syndrome Organisation (IPWSO), Ireland, Ireland; 8Medical University Varna, Department of Pediatrics, Varna, Bulgaria; 9Universiti Malaya, Department of Paediatrics, Faculty of Medicine, Kuala Lumpur, Malaysia; 10Erasmus University Medical Center-Sophia Children’s Hospital, Department of Pediatrics, Subdivision of Endocrinology, Rotterdam, Netherlands; 11Children’s Hospital of Fudan University, National Children’s Medical Center, Department of Pediatric Endocrinology and Inherited Metabolic Diseases, Shanghai, China; 12University of Florida, Department of Paediatrics, Division of Endocrinology, California, United States; 13Sydney Children’s Hospital, Department of Pediatric Endocrinology, Sydney, Australia; 14CHI @ Tallaght University Hospital, Department of Pediatric Endocrinology, Dublin, Ireland; 15University of Dublin, Trinity College Dublin, Discipline of Paediatrics, School of Medicine, Dublin, Ireland; 16Foundation for Prader-Willi Research, California, United States; 17Hôpital des Enfants, Toulouse, France; 18Instituto Estadual de Diabetes e Endocrinologia Luiz Capriglione, Rio de Janeiro, Brazil
JOINT682
Introduction: Recombinant Human Growth Hormone (GH) was approved in the US (2000) and Europe (2001) for children with Prader-Willi Syndrome (PWS) to improve growth and body composition, with reported benefits in cognition, motor skills and behaviour. However, longer term safety and efficacy data of GH in PWS are still lacking and may be difficult to interpret due to lack of consistency in data collection among studies. This study aimed to identify the minimum dataset (MDS) that could be measured in a routine clinical setting across the world, to minimise burden on clinician data entry and improve quality of data collection to facilitate future studies on long term outcomes.
Methods: The study was undertaken by the PWS Expert Working Group in GloBE-Reg, an international registry platform which supports studies on long-term safety and effectiveness of drugs. Twelve clinical experts on PWS from 10 countries and two patient representatives collaborated to develop this recommendation, based on previously published methodology (Chen et al. Horm Res Pediatr 2023). Data fields that achieved 70% consensus in terms of importance qualified for the MDS, provided <50% deemed the item difficult to collect. Several anomalies to the MDS rule were discussed to formulate the final MDS recommendation.
Results: In total, 294 items were compiled from routine clinical practice with 33 redundant items removed and 261 items subjected to the grading system. 151/261 items achieved consensus as important data to collect when monitoring children with PWS on GH treatment, while 218/261 items were deemed easy to collect. Combining both the criteria for importance and ease of collection, 126 items fulfilled the MDS requirement. Four items were designated as core data, two were computed fields, five reassigned as non-MDS, 13 removed as unrelated to safety and effectiveness and 65 were merged into 18 fields. Several anomalies which did not fulfill MDS criteria were also extensively discussed to determine its validity within the MDS, in particular family history of Type 2 diabetes, change of GH therapy (if applicable) and adherence, to produce the final MDS recommendations of 58 items; of which 24 are only to be completed once.
Conclusions: This exercise has identified by consensus the minimum dataset considered necessary, which can be collected through real-world data, to provide consistency and comparability in global studies for monitoring the safety and effectiveness of GH in children with PWS, applicable to the current daily preparations and potential newer long-acting GH.
Volume 110
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Endocrine Abstracts
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