Search Results (3)

Background/Objectives: Health-related quality of life has come to the forefront of the process of treatment. As a non-quantitative value, it requires the use of specialized tools to be measured. Despite the availability of general HRQoL assessment tools in Polish, a specific instrument for children with gastrointestinal (GI) disorders has not been developed. This study aims to describe the linguistic validity and reliability of the Polish version of the PedsQL™ Gastrointestinal Symptoms Module, which measured health-related quality of life (HRQoL) in children with gastrointestinal (GI) disorders. Methods: The PedsQL™ module, originally in English, was translated according to a structured process. Professional medical translators conducted the translations, and cognitive debriefing was performed with 35 respondents. Field testing involved 371 completed questionnaires (203 adults and 168 children). Results: Internal consistency was assessed using Cronbach’s alpha, with values generally exceeding 0.8 indicating high reliability. Conclusions: The final Polish version of the PedsQL™ GI Symptoms Module exhibits strong linguistic and content validity, making it suitable for both clinical trials and routine practice. Its use enables a comprehensive, culturally sensitive assessment of HRQoL in pediatric patients with GI disorders, thereby supporting individualized patient care and enhancing the physician–patient communication that is essential for effective treatment. Full article

(1) Background: The introduction of highly effective CFTR-modulating therapies (HEMT) has changed the course of the disease for many people with Cystic Fibrosis (pwCF). Attention previously focused on life-threatening conditions of the respiratory system has broadened, bringing the involvement of the digestive system into the clinical and scientific focus. This emphasized the need for sensitive tools to capture and quantify changes in abdominal symptoms (AS), ideally applying patient-reported outcome measures (PROMs). (2) Methods: The present review focuses on studies addressing AS assessment deriving from the multi-organic abdominal involvement in pwCF. Among 5224 publications retrieved until Nov. 2022, 88 were eligible, and 39 were finally included. (3) Results: The review reveals that for a long time, especially before HEMT availability, AS in pwCF were assessed by single questions on abdominal complaints or non-validated questionnaires. PROMs focusing on quality of life (QOL) including a few GI-related questions were applied. Likewise, PROMs developed and partially validated for other non-CF GI pathologies, such as chronic inflammatory bowel diseases, irritable bowel syndrome, gastroesophageal reflux, constipation, or pancreatitis, were implemented. (4) Conclusions: Only lately, CF-specific GI-PROMs have been developed and validated following FDA guidelines, showing high sensitivity to changes and capturing marked and statistically significant reductions in the burden of AS achieved with HEMT implementation. Full article

Blenderised tube feeds (BTF) have become a popular alternative to commercial formula (CF) for enterally fed children. This study sought to compare gastrointestinal (GI) symptoms, GI inflammation, and stool microbiome composition between children receiving BTF or CF. This prospective cohort study involved 41 gastrostomy-fed children, aged 2–18 years, receiving either BTF (n = 21) or CF (n = 20). The Paediatric Quality of Life Inventory Gastrointestinal Symptoms Scale (GI-PedsQL) was used to compare GI symptoms between the groups. Anthropometric data, nutritional intake, nutritional blood markers, faecal calprotectin levels, stool microbiota, and parental satisfaction with feeding regimen were also assessed. Caregivers of children on BTF reported greater GI-PedsQL scores indicating significantly fewer GI symptoms (74.7 vs. 50.125, p = 0.004). Faecal calprotectin levels were significantly lower for children receiving BTF compared to children on CF (33.3 mg/kg vs. 72.3 mg/kg, p = 0.043) and the BTF group had healthier, more diverse gut microbiota. Subgroup analysis found that 25% of caloric intake from BTF was sufficient to improve GI symptoms. The CF group had better body mass index (BMI) z-scores (−0.7 vs. 0.5, p = 0.040). Although growth was poorer in children receiving only BTF in comparison to the CF group, this was not seen in children receiving partial BTF. A combination of BTF and CF use may minimise symptoms of tube feeding whilst supporting growth. Full article