SAT-206 Development of a Minimum Dataset (MDS) for the Monitoring of Growth Hormone Therapy in Children with Prader Willi Syndrome (PWS) - A GloBE-Reg Initiative
Notice bibliographique
Résumé
Abstract Disclosure: A. Leong: None. S. Chen: None. M. Alimussina: None. J. Bryce: None. M. Chen: None. A. Fu: None. E.F. Gevers: None. C. Hoybye: None. M. Hughes: None. V. Iotova: None. M.Y. Jalaludin: None. G.F. Kerkhof: None. F. Luo: None. J. Miller: None. O. Nyunt: None. E.F. Roche: None. M.G. Shaikh: None. T. Strong: None. M. Tauber: None. L.S. Tyszler: None. S.F. Ahmed: None. 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. Conclusion: 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. Presentation: Saturday, July 12, 2025
Récupéré en direct depuis OpenAlex et désinversé. Les résumés ne sont pas conservés dans cette base de données : les index inversés représentent 8,6 Go des 9,3 Go de texte de la base, et le serveur dispose de 13 Go libres.
Comment cette classification a été obtenuedéplier
Prédiction distillée sur la base complète
Imitation des enseignantsNi prévalence calibrée, ni vérité terrain. Validation humaine à venir. Apprise à partir de 10 348 étiquettes directes de Codex et de 10 348 étiquettes directes de Gemma. Le mode candidate est l'union des têtes enseignantes seuillées; le consensus est leur intersection. Ces sorties portent le statut machine_predicted_unvalidated et ne sont ni des étiquettes humaines ni des étiquettes directes de modèles de pointe.
Scores Codex et Gemma par catégorie
| Catégorie | Codex | Gemma |
|---|---|---|
| Métarecherche | 0,000 | 0,000 |
| Méta-épidémiologie (sens strict) | 0,000 | 0,000 |
| Méta-épidémiologie (sens large) | 0,000 | 0,000 |
| Bibliométrie | 0,000 | 0,000 |
| Études des sciences et des technologies | 0,000 | 0,000 |
| Communication savante | 0,000 | 0,000 |
| Science ouverte | 0,000 | 0,000 |
| Intégrité de la recherche | 0,000 | 0,000 |
| Charge utile insuffisante (le modèle a refusé de juger) | 0,000 | 0,000 |
Scores machine (provisoires)
Les deux têtes enseignantes du modèle étudiant, lues sur ce travail. Un score ordonne la base pour la relecture; il n'affirme jamais une catégorie, et le statut de validation accompagne chaque rangée tel quel.
Scores de référence d'un modèle non mature (critères de maturité non atteints, 7 itérations). Un score ordonne; il n'affirme jamais une catégorie.
score_only:v0-immature-baseline · tel quel depuis la passe de notation : score_only signifie que le nombre peut ordonner les travaux, et qu'aucune étiquette de catégorie n'en découleClassification
machine, non validéePrédiction automatique; un appel candidat d’une seule tête enseignante, pas un consensus.
Le détail, modèle par modèle et score par score, se trouve en fin de page sous « Comment cette classification a été obtenue ».