Dataset Information

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

ABSTRACT:

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.

SUBMITTER: Boon M

PROVIDER: S-EPMC6924691 | biostudies-literature | 2019

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

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

Boon Mieke M Claes Ine I Havermans Trudy T Fornés-Ferrer Victoria V Calvo-Lerma Joaquim J Asseiceira Inês I Bulfamante Anna A Garriga María M Masip Etna E Woodcock Sandra S Walet Sylvia S Barreto Celeste C Colombo Carla C Crespo Paula P Van der Wiel Els E Hulst Jessie J Martinez-Barona Sandra S Nobili Rita R Pereira Luisa L Ruperto Mar M Vicente Saioa S De Boeck Kris K Ribes-Koninckx Carmen C

PloS one 20191220 12

<h4>Background</h4>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 ...[more]

PMID: 31860639

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Project description:Cystic fibrosis (CF) is a heritable disease that causes altered physiology at mucosal sites; these changes result in chronic infections in the lung, significant gastrointestinal complications as well as dysbiosis of the gut microbiome, although the latter has been less well explored. Here, we describe the longitudinal development of the gut microbiome in a cohort of children with CF (cwCF) from birth through early childhood (0-4 years of age) using 16S rRNA gene amplicon sequencing of stool samples as a surrogate for the gut microbiota. Similar to healthy populations, alpha diversity of the gut microbiome increases significantly with age, but diversity plateaus at ~2 years of age for this CF cohort. Several taxa that have been associated with dysbiosis in CF change with age toward a more healthy-like composition; notable exceptions include Akkermansia, which decreases with age, and Blautia, which increases with age. We also examined the relative abundance and prevalence of nine taxa associated with CF lung disease, several of which persist across early life, highlighting the possibility of the lung being seeded directly from the gut early in life. Finally, we applied the Crohn's Dysbiosis Index to each sample, and found that high Crohn's-associated dysbiosis early in life (<2 years) was associated with significantly lower Bacteroides in samples collected from 2 to 4 years of age. Together, these data comprise an observational study that describes the longitudinal development of the CF-associated gut microbiota and suggest that early markers associated with inflammatory bowel disease may shape the later gut microbiota of cwCF. IMPORTANCE Cystic fibrosis is a heritable disease that disrupts ion transport at mucosal surfaces, causing a buildup of mucus and dysregulation of microbial communities in both the lungs and the intestines. Persons with CF are known to have dysbiotic gut microbial communities, but the development of these communities over time beginning at birth has not been thoroughly studied. Here, we describe an observation study following the development of the gut microbiome of cwCF throughout the first 4 years of life, during the critical window of both gut microbiome and immune development. Our findings indicate the possibility of the gut microbiota as a reservoir of airway pathogens and a surprisingly early indication of a microbiota associated with inflammatory bowel disease.

Project description:BackgroundCystic fibrosis (CF) is caused by mutations in the CFTR gene that impair the function of CFTR, a cAMP-regulated anion channel. In the small intestine loss of CFTR function creates a dehydrated, acidic luminal environment which is believed to cause an accumulation of mucus, a phenotype characteristic of CF. CF mice have small intestinal bacterial overgrowth, an altered innate immune response, and impaired intestinal transit. We investigated whether lubiprostone, which can activate the CLC2 Cl- channel, would improve the intestinal phenotype in CF mice.MethodsCftr(tm1UNC) (CF) and wildtype (WT) littermate mice on the C57BL/6J background were used. Lubiprostone (10 μg/kg-day) was administered by gavage for two weeks. Mucus accumulation was estimated from crypt lumen widths in periodic acid-Schiff base, Alcian blue stained sections. Luminal bacterial load was measured by qPCR for the bacterial 16S gene. Gastric emptying and small intestinal transit in fasted mice were assessed using gavaged rhodamine dextran. Gene expression was evaluated by Affymetrix Mouse430 2.0 microarray and qRT-PCR.ResultsCrypt width in control CF mice was 700% that of WT mice (P < 0.001). Lubiprostone did not affect WT crypt width but, unexpectedly, increased CF crypt width 22% (P = 0.001). Lubiprostone increased bacterial load in WT mice to 490% of WT control levels (P = 0.008). Conversely, lubiprostone decreased bacterial overgrowth in CF mice by 60% (P = 0.005). Lubiprostone increased gastric emptying at 20 min postgavage in both WT (P < 0.001) and CF mice (P < 0.001). Lubiprostone enhanced small intestinal transit in WT mice (P = 0.024) but not in CF mice (P = 0.377). Among other innate immune markers, expression of mast cell genes was elevated 4-to 40-fold in the CF intestine as compared to WT, and lubiprostone treatment of CF mice decreased expression to WT control levels.ConclusionsThese results indicate that lubiprostone has some benefits for the CF intestinal phenotype, especially on bacterial overgrowth and the innate immune response. The unexpected observation of increased mucus accumulation in the crypts of lubiprostone-treated CF mice suggests the possibility that lubiprostone increases mucus secretion.

Project description:BackgroundThe Pediatric Quality of Life™ 3.0 Neuromuscular Module is an instrument to assess health-related quality of life (HRQoL) among children with neuromuscular diseases (NMDs) aged 2-18 years. This study aimed to determine whether the PedsQL™ 3.0 Neuromuscular Module Indonesian Version is valid and reliable.MethodsThis study used the Indonesian translation of the PedsQL™ 3.0 Neuromuscular Module after getting formal permission from the inventor, and the translation process followed the Mapi linguistic translation guidelines. This study administered the PedsQL™ 3.0 Neuromuscular Module Indonesian Version to 84 parents and 71 children. In addition, we used the Rasch model to analyze the psychometric properties.ResultsThe reliability of the total scale of the PedsQL™ Neuromuscular Module Indonesian Version shows good to very good criteria. On the parent proxy-report, Cronbach alpha was 0.95, person reliability was 0.84, item reliability was 0.93, person separation was 2.32, item separation was 3.61, person strata separation was 4 levels, and item strata separation was 5 levels. On the child self-report, Cronbach alpha was 0.93, person reliability was 0.81, item reliability was 0.81, person separation was 2.08, item separation was 2.06, person strata separation was 3 levels, and item strata separation was 3 levels. The total scale of the PedsQL Neuromuscular Module Indonesian Version shows fair to good construct validity in parent proxy-report (explained variance 51.9%; unexplained variance 8.4%) and child self-report (explained variance 40.9%; unexplained variance 12.6%). There were no misfit items in the parent proxy-report (infit 0.66-1.49; outfit 0.51-1.81; point measure correlation 0.36-0.93) and child self-report (infit 0.53-1.65; outfit 0.50-1.73; point measure correlation 0.31-0.90) identified by the Rasch models.ConclusionsThe PedsQL™ 3.0 Neuromuscular Modul Indonesian Version is a valid and reliable instrument in measuring HRQoL in Indonesian children with neuromuscular diseases.

Dataset Information

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

Background

Methods

Results

Conclusions

Publications

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

Similar Datasets

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