The impact of liver transplantation on health-related quality of life in (acute) intoxication-type inborn errors of metabolism.
Benedetta GrecoStefania CavigliaDiego MartinelliTeresa Grimaldi CapitelloDaniela LiccardoFrancesca De NictolisAndrea PietrobattistaMartina HuemerSimone Piga StatGiorgia OlivieriGionata SpagnolettiMarco SpadaCarlo Dionisi-ViciPublished in: Journal of inherited metabolic disease (2023)
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 parent-reports. The comparison between PedsQL-GM and MetabQoL highlighted that MetabQoL demonstrated higher sensitivity in the assessment of disease-specific domains than the generic PedsQL tool. This article is protected by copyright. All rights reserved.