Assessing gastro-intestinal related quality of life in cystic fibrosis: Validation of PedsQL GI in children and their parents

English version

Autoři: Mieke Boon ^aff001; Ine Claes ^aff001; Trudy Havermans ^aff001; Victoria Fornés-Ferrer ^aff002; Joaquim Calvo-Lerma ^aff002; Inês Asseiceira ^aff003; Anna Bulfamante ^aff004; María Garriga ^aff005; Etna Masip ^aff002; Sandra Woodcock ^aff006; Sylvia Walet ^aff006; Celeste Barreto ^aff003; Carla Colombo ^aff004; Paula Crespo ^aff002; Els Van der Wiel ^aff006; Jessie Hulst ^aff006; Sandra Martinez-Barona ^aff002; Rita Nobili ^aff004; Luisa Pereira ^aff003; Mar Ruperto ^aff005; Saioa Vicente ^aff005; Kris De Boeck ^aff001; Carmen Ribes-Koninckx ^aff002;
Působiště autorů: Cystic Fibrosis Center, Department of Pediatrics, University Hospital Gasthuisberg, Leuven, Belgium ^aff001; Instituto de Investigación Sanitaria La Fe de Valencia, Spain ^aff002; Associação para a Investigação e Desenvolvimento da Faculdade de Medicina, Lisbon, Portugal ^aff003; Università degli Studi di Milano, Fondazione IRCCS Ca’ Granda, Ospedale Maggiore Policlinico, Milan, Italy ^aff004; Hospital Universitario Ramón y Cajal, Madrid, Spain ^aff005; Erasmus Medical Center, Sophia Children’s Hospital, Rotterdam, Netherlands ^aff006
Vyšlo v časopise: PLoS ONE 14(12)
Kategorie: Research Article
prolekare.web.journal.doi_sk: https://doi.org/10.1371/journal.pone.0225004

Souhrn

Background

Most patients with cystic fibrosis (CF) suffer from pancreatic insufficiency, leading to fat malabsorption, malnutrition and abdominal discomfort. Until recently, no specific tool was available for assessing gastro-intestinal related quality of life (GI QOL) in patients with CF. As the Horizon2020 project MyCyFAPP aims to improve GI QOL by using a newly designed mobile application, a sensitive and reliable outcome measure was needed. We aimed to study the applicability of the existing child-specific Pediatric Quality of Life Inventory, Gastrointestinal Symptoms Scales and Module (PedsQL GI) in children with CF.

Methods

A multicenter, prospective observational study was performed in 6 European centers to validate the PedsQL GI in children with CF during 3 months.

Results

In total, 248 children and their parents were included. Within-patient variability of PedsQL GI was low (24.11), and there was reasonable agreement between children and parents (ICC 0.681). Nine of 14 subscales were informative (no ceiling effect). The PedsQL GI and the median scores for 4 subscales were significantly lower in patients compared to healthy controls. Positive associations were found between PedsQL GI and age (OR = 1.044, p = 0.004) and between PedsQL GI and BMI z-score (OR = 1.127, p = 0.036). PedsQL GI correlated with most CFQ-R subscales (r 0.268 to 0.623) and with a Visual Analogue Scale (r = 0.20).

Conclusions

PedsQL GI is a valid and applicable instrument to assess GI QOL in children with CF. Future research efforts should examine the responsiveness of the CF PedsQL GI to change in the context of clinical interventions and trials.

Klíčová slova:

Pediatrics – Quality of life – Age groups – Cystic fibrosis – Diarrhea – Stomach – Constipation – PERT

Zdroje

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