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Health-related quality of life (HRQL) in paediatric patients with X-linked hypophosphatemia (XLH) in France: analysis of observational real-world registry data

dc.rights.licenseopenen_US
dc.contributor.authorAMOUROUX, C.
dc.contributor.authorBACCHETTA, J.
hal.structure.identifierBordeaux population health [BPH]
dc.contributor.authorHARAMBAT, Jerome
dc.contributor.authorGUEORGUIEVA, I.
dc.contributor.authorSALLES, J. P.
dc.contributor.authorSANDILANDS, K.
dc.contributor.authorRYLANDS, A.
dc.contributor.authorWILLIAMS, A.
dc.contributor.authorISHII, H.
dc.contributor.authorDENT, J.
dc.contributor.authorNIXON, A.
dc.contributor.authorLINGLART, A.
dc.date.accessioned2025-03-03T13:42:41Z
dc.date.available2025-03-03T13:42:41Z
dc.date.issued2024-10-01
dc.date.conference2024-11-16
dc.identifier.issn1663-2818en_US
dc.identifier.urihttps://oskar-bordeaux.fr/handle/20.500.12278/205328
dc.description.abstractEnBackground: X-linked hypophosphatemia (XLH) is a rare, progressive, phosphate-wasting disorder that compromises skeletal development in childhood. The international XLH registry (NCT03193476) is a non-interventional, observational, real-world data collection programme established in August 2017 in Europe and Israel, with enrolment open to patients of any age with XLH. Objectives: To describe real-world registry data on HRQL in paediatric patients with XLH at centres in France. Methods: Data to 30 January 2024 were analysed for paediatric patients (<18 years at registry entry) who registered between March 2019 and January 2023. HRQL was measured using age-appropriate versions of the PedsQL, completed by parents for children aged <8 years and self-completed by older children. The PedsQL comprises four domains – Physical, Emotional, School and Social Functioning – which are combined into Physical health and Psychosocial health summary scores and a total score. Scores range from 0 to 100, with higher scores indicating better HRQL. The first PedsQL completed after registry entry was analysed. Results: Of 170 French paediatric patients registered at data cutoff (59% female; mean age 9.6 years [SD 4.96; range 0.2–17.9]), PedsQL data were available for 96 (59% female; mean age at completion 8.1 years [SD 4.43; range 0.3–16.0]). PedsQL scores are presented in Table 1. Psychosocial Health Summary scores worse than Physical Health Summary scores for all age bands, with the Emotional Functioning scores indicating greatest detriment. Total scores were worse for 5–7-year-olds. Conclusion: This real-world HRQL data from 96 paediatric patients with XLH in France show the greatest detriments in those aged 5–7 year. Psychosocial scores were lower than physical health scores in paediatric patients of all ages.
dc.language.isoENen_US
dc.title.enHealth-related quality of life (HRQL) in paediatric patients with X-linked hypophosphatemia (XLH) in France: analysis of observational real-world registry data
dc.typeCommunication dans un congrèsen_US
dc.subject.halSciences du Vivant [q-bio]/Santé publique et épidémiologieen_US
bordeaux.page216-217en_US
bordeaux.volume97en_US
bordeaux.hal.laboratoriesBordeaux Population Health Research Center (BPH) - UMR 1219en_US
bordeaux.institutionUniversité de Bordeauxen_US
bordeaux.institutionINSERMen_US
bordeaux.conference.title62nd Annual ESPE (ESPE 2024)en_US
bordeaux.countrygben_US
bordeaux.title.proceedingHormone research in pædiatricsen_US
bordeaux.teamLEHA_BPHen_US
bordeaux.conference.cityLiverpoolen_US
hal.identifierhal-04974348
hal.version1
hal.date.transferred2025-03-03T13:42:43Z
hal.proceedingsouien_US
hal.popularnonen_US
hal.audienceInternationaleen_US
hal.exporttrue
dc.rights.ccPas de Licence CCen_US
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