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


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
doi: 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:

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


Zdroje

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