Diabetes is associated with increased burden of gastrointestinal symptoms in adults with cystic fibrosis

Caley, L.R.; Zagoya, Carlos; Duckstein, Franziska; White, Helen; Shimmin, D.; Jones, Andrew M.; Barrett, Joanne; Whitehouse, J.L.; Floto, R. Andres; Mainz, J.G.; Peckham, Daniel

doi:10.1016/j.jcf.2023.01.010

Cited by 5 publications

(5 citation statements)

References 29 publications

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“…Likewise, a similar improvement was observed in 108 pwCF from Ireland and the UK, assessed using the CFAbd-Score during a new therapy with ETI (Mainz et al, 2023). Therefore, the level of changes captured with the CF-specific CFAbd-Score can be considered clinically relevant (Caley et al, 2023b).…”

Section: Maximal Absolute Deviationsmentioning

confidence: 54%

“…These are complemented by factors like intestinal dysbiosis caused by frequent antibiotic treatments, cough-associated reflux, and maldigestion, resulting in malresorption of nutrients, which then allow bacterial fermentation, gases, and diarrhea as principal symptoms of untreated exocrine pancreatic insufficiency (Caley et al, 2023a). Finally, endocrine liberation of insulin is also hampered by the destruction of pancreatic beta cells, leading to CF-related diabetes in a substantial proportion of pwCF during adulthood (Caley et al, 2023b).…”

Section: Introductionmentioning

confidence: 99%

“…To fill this gap, we developed the CFAbd-Score in different steps following FDA guidelines for the development of a PROM (U.S. Department of Health and Human Services Food and Drug Adminitration, 2019), including CF patients and their families (community voice), as well as professional CF specialists at several time points. Initially, the PROM had been named JenAbd-Score (Tabori et al, 2017a), and after condensing it from a 5-sided questionnaire to a one-sided PROM, which comprises 28 symptoms grouped in five domains, it was renamed "CFAbd-Score" (Tabori et al, 2017b;Jaudszus et al, 2019;Mainz et al, 2023;Jaudszus et al, 2022;Mainz et al, 2022;Caley et al, 2023b). Presently, it is available in eleven languages and implemented in more than 25 studies around the world (Boon et al, 2020;Mainz et al, 2023;Ng et al, 2021;Mainz et al, 2022;Raun et al, 2022).…”

Section: Introductionmentioning

confidence: 99%

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Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire

Mainz,

Barucha,

Huang

et al. 2023

Front. Pharmacol.

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Background: Elexacaftor–tezacaftor–ivacaftor (ETI) is a novel, highly effective CFTR modulator combination proven to enhance lung function and body weight in people with cystic fibrosis (pwCF) carrying a F508del mutation. Recently, we revealed significant reductions in abdominal symptoms (AS) in German, British, and Irish pwCF after 24–26 weeks of ETI using the CFAbd-Score, the first patient-reported outcome measure (PROM) specifically developed and validated for pwCF following FDA guidelines. Notably, many pwCF reported marked changes in their AS during the first days of the new treatment. To capture these immediate effects, we developed the CFAbd-day2day, a CF-specific GI-diary, following FDA and COSMIN guidelines.Aim: To prospectively capture the immediate dynamics of AS using the CFAbd-day2day 14 days before and 14–28 days after ETI initiation. In addition, we aim to provide validation steps of the novel PROM concerning sensitivity to changes.Methods: To develop the CFAbd-day2day, focus groups (community voice = pwCF and their proxies and CF specialists from different fields) were repeatedly consulted. Before and during the new ETI therapy, pwCF prospectively scored AS on a daily basis with the CFAbd-day2day.Results: Altogether, 45 pwCF attended in five CF centers prospectively completed the CFAbd-day2day before (mean ± sd:14 ± 7 days) and after (mean ± sd: 28 ± 23 days) ETI initiation. On the one hand, cumulative scores significantly decreased during the 3–4-week time frame after ETI initiation, compared to 2 weeks prior to therapy. On the other hand, many patients who revealed a relatively stable level of AS before ETI reported changes during the first days of treatment with the highly effective CFTR modulators. Factors like pain and flatulence increased in up to 21% of patients during the first 14 days of therapy, but they improved during days 15–27.Conclusion: The CFAbd-day2day, specifically developed and in the process of validation to prospectively capture GI symptoms in pwCF, provides new substantial insights into the dynamics of AS in pwCF receiving a new treatment with ETI. This novel tool is also helpful in prospectively monitoring patients with specific GI problems. International implementation and further validation steps of the diary are ongoing.

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Section: Maximal Absolute Deviationsmentioning

confidence: 54%

Section: Introductionmentioning

confidence: 99%

Section: Introductionmentioning

confidence: 99%

See 1 more Smart Citation

Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire

Mainz,

Barucha,

Huang

et al. 2023

Front. Pharmacol.

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“…Our prior study focused on detecting CFRD using changes in voice characteristics and showed significantly higher VTI in males with CFRD compared to male CF patients without CFRD 10 whereas this study demonstrated that there is no significant difference of VTI in individuals with CFRD according to glycemic control status or POCT glucose level. This can be explained by gastrointestinal acid reflux in individuals with CFRD leading to laryngopharyngeal reflux 24 . However, there is no specific sex differences in prevalence of acid reflex endoscopy-based study in CFRD.…”

Section: Discussionmentioning

confidence: 99%

Predicting glycemic control status and high blood glucose levels through voice characteristic analysis in patients with cystic fibrosis-related diabetes (CFRD)

Suppakitjanusant,

Kasemkosin,

Sivapiromrat

et al. 2023

Sci Rep

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Cystic fibrosis-related diabetes (CFRD) is associated with reduced life expectancy in adults with cystic fibrosis (CF). Voice analysis may be a convenient method for diagnosing and monitoring CFRD. This study aims to determine the relationship between voice characteristics and markers of glucose and glycemic control and to identify if voice analysis can predict high blood glucose levels and glycemic control in adults with CFRD. We conducted a prospective cross-sectional study in adults with CF from March to December 2021. We recorded 3-second voice samples of a sustained /a/ vowel and analyzed voice characteristic using the Computerized Speech Lab with the Multi-Dimensional Voice Program. In female participants with CFRD, the noise-to-harmonic ratio was significantly lower in those with HbA1c ≥ 7. Furthermore, fundamental frequency variation was significantly lower in both male and female participants with CFRD who had a glucose level of 200 mg/dL or higher at the time of collection. This finding was also associated with a high level of point-of-care glucose. The human voice has potential as a non-invasive tool for measuring glucose levels and glycemic control status in CFRD patients in the future.

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“…These marked differences also resulted in significantly higher scores for domains “GERD” +46% (19.2 versus 35.3 points), “Disorders of appetite” +53% (7.4 versus 15.9 points), and “GI-related QOL” (+42% with diabetes). Differences between the two groups were also marked, albeit not reaching significance, for the “Pain” (+ 21%) and “Bowel movement” domains, which, however, only revealed a +4% difference between both groups [ 84 ].…”

Section: Resultsmentioning

confidence: 99%

Scoring Abdominal Symptoms in People with Cystic Fibrosis

Tabori,

Barucha,

Zagoya

et al. 2024

JCM

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(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.

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Diabetes is associated with increased burden of gastrointestinal symptoms in adults with cystic fibrosis

Cited by 5 publications

References 29 publications

Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire

Dynamics of abdominal symptoms during the start of a new therapy with elexacaftor/tezacaftor/ivacaftor using the novel CFAbd-day2day questionnaire

Predicting glycemic control status and high blood glucose levels through voice characteristic analysis in patients with cystic fibrosis-related diabetes (CFRD)

Scoring Abdominal Symptoms in People with Cystic Fibrosis

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