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The impact of liver transplantation on health-related quality of life in (acute) intoxication-type inborn errors of metabolism

  • Organic acidurias (OAs), urea-cycle disorders (UCDs), and maple syrup urine disease (MSUD) belong to the category of intoxication-type inborn errors of metabolism (IT-IEM). Liver transplantation (LTx) is increasingly utilized in IT-IEM. However, its impact has been mainly focused on clinical outcome measures and rarely on health-related quality of life (HRQoL). Aim of the study was to investigate the impact of LTx on HrQoL in IT-IEMs. This single center prospective study involved 32 patients (15 OA, 11 UCD, 6 MSUD; median age at LTx 3.0 years, range 0.8–26.0). HRQoL was assessed pre/post transplantation by PedsQL-General Module 4.0 and by MetabQoL 1.0, a specifically designed tool for IT-IEM. PedsQL highlighted significant post-LTx improvements in total and physical functioning in both patients' and parents' scores. According to age at transplantation (≤3 vs. >3 years), younger patients showed higher post-LTx scores on Physical (p = 0.03), Social (p < 0.001), and Total (p =0.007) functioning. MetabQoL confirmed significant post-LTx changes in Total and Physical functioning in both patients and parents scores (p ≤ 0.009). Differently from PedsQL, MetabQoL Mental (patients p = 0.013, parents p = 0.03) and Social scores (patients p = 0.02, parents p = 0.012) were significantly higher post-LTx. Significant improvements (p = 0.001–0.04) were also detected both in self- and proxy-reports for almost all MetabQoL subscales. This study shows the importance of assessing the impact of transplantation on HrQoL, a meaningful outcome reflecting patients' wellbeing. LTx is associated with significant improvements of HrQol in both self- and parentreports. The comparison between PedsQL-GM and MetabQoL highlighted that MetabQoL demonstrated higher sensitivity in the assessment of diseasespecific domains than the generic PedsQL tool.

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Metadaten
Author:Benedetta Greco, Stefania Caviglia, Diego Martinelli, Teresa Grimaldi Capitello, Daniela Liccardo, Francesca De Nictolis, Andrea Pietrobattista, Martina Huemer, Simone Piga, Giorgia Olivieri, Gionata Spagnoletti, Marco Spada, Carlo Dionisi-Vici
DOI:https://doi.org/10.1002/jimd.12648
ISSN:0141-8955
ISSN:1573-2665
Parent Title (English):Journal of Inherited Metabolic Disease
Document Type:Article
Language:English
Year of publication:2023
Release Date:2024/01/31
Volume:46
Issue:5
First Page:906
Last Page:915
Organisationseinheit:Soziales & Gesundheit
DDC classes:600 Technik, Medizin, angewandte Wissenschaften / 610 Medizin, Gesundheit
Open Access?:nein
Peer review:wiss. Beitrag, peer-reviewed
Huemer, Martina
Licence (German):License LogoCreative Commons - CC BY-NC - International - Attribution - NonCommercial - Namensnennung - Nicht kommerziell 4.0