Abdominal Symptoms Assessed With the CFAbd‐Score are Associated With Intestinal Inflammation in Patients With Cystic Fibrosis

Abstract

Objectives:This prospective study evaluated the relationship between fecal markers of intestinal inflammation and cystic fibrosis (CF)-associated abdominal symptoms. These were assessed using the CFAbd-Score, a CF-specific patient-related outcome measure developed and validated, following FDA guidelines.Methods:In feces from patients with CF (n = 41) and healthy volunteers (n = 27), concentrations of fecal calprotectin (FC), M2-pyruvate kinase (M2-PK), interleukins IL-1β, IL-6, IL-8, and neutrophilic elastase (NE) were measured. Abdominal symptoms during the 2 preceding weeks were recorded using the CFAbd-Score. This patient-reported outcome measure (PROM) for assessment of the multi-organic abdominal involvement in CF includes 28 items in five domains.Results:Inflammatory parameters FC, IL-1β, M2-PK, and NE in feces, as well as CFAbd-Scores resulted significantly higher in CF patients than in healthy controls (all P < 0.01). Furthermore, significant differences between both groups were found for pain-symptoms, disorders of bowel movement, impaired quality of life, as well as disorders of eating and appetite. With 83% sensitivity and 74% specificity, FC was the most reliable measure for CF-related intestinal inflammation, which, in the CFAbd-Score, was associated to significantly higher rates of abdominal pain, as well as to general quality of life items such as gastrointestinal-related impaired sleep and frustration.Conclusion:Using the CFAbd-Score as a CF-specific PROM for identification and quantification of abdominal symptoms revealed that abdominal pain and impaired quality of life are associated with intestinal inflammation in CF.

Elexacaftor-Tezacaftor-Ivacaftor Treatment Reduces Abdominal Symptoms in Cystic Fibrosis-Early results Obtained With the CF-Specific CFAbd-Score

“…Levels of single GI symptoms in pwCF, before ETI treatment initiation, were similar to those described in our previous studies, with predominant symptoms: pain, flatulence, smelly stools and frequency of bowel movements (Figure 2) (Tabori et al, 2017a;Jaudszus et al, 2019;Jaudszus et al, 2021). After ETI treatment, "Embarrassment due to GI symptoms" was increased but remained lower than those described by HCs.…”

“…More recently, we assessed intestinal inflammation in stool by faecal elastase, M2-pyruvate kinase (M2-PK), interleukins IL-1β, IL-6, IL-8, and neutrophil elastase (NE), together with the CFAbd-Score. Here we found that both, CFAbd-Scores and intestinal inflammation, most sensitively assessed by faecal elastase, are markedly higher in pwCF, compared to healthy controls (Jaudszus et al, 2021).…”

“…Given the higher levels of symptom burden observed in the United Kingdom cohort after a longer time of ETI administration, it is important to further investigate whether the significant reduction in GI symptoms seen after 24 weeks of ETI are sustained or attenuate with time and the impact of other intervening factors including COVID-19. (Tabori et al, 2017a;Tabori et al, 2017b;Jaudszus et al, 2019;Jaudszus et al, 2021). We repeatedly included focus groups with professionals from different fields of CF care, pwCF and their families/proxies, in order to build up a questionnaire that includes the most relevant fields and questions, weighting results optimally.…”

“…Elaborated in four published steps, the first version of this CF-specific questionnaire (initially named JenAbd-Score, and from then CFAbd-Score) aimed to assess and quantify abdominal involvement in pwCF, and its development included patients, proxies and health care specialists, as recommended by FDA guidelines (Tabori et al, 2017a;Tabori et al, 2017b;Jaudszus et al, 2019). At present, the CFAbd-Score is available in nine languages and implemented in many international studies (Boon et al, 2020;Jaudszus et al, 2021;Ng et al, 2021;Raun et al, 2022).…”

“…Frontiers in Pharmacology | www.frontiersin.org June 2022 | Volume 13 | Article 8771182019;Boon et al, 2020;Jaudszus et al, 2021;Ng et al, 2021;Raun et al, 2022).…”