Luminal Gastrointestinal Manifestations of Cystic Fibrosis

Burton, Samuel; Hachem, Christine; Abraham, James

doi:10.1007/s11894-021-00806-5

Cited by 14 publications

(9 citation statements)

References 93 publications

Supporting

Mentioning

Contrasting

Order By: Relevance

“…The improvement in respiratory symptoms and life expectancy in pwCF has shifted focus towards better understanding of the non-pulmonary CF-related manifestations, especially GI involvement (Bell et al, 2020;Balfour-Lynn and King, 2020;J Burton et al, 2021). Consequently, abdominal symptoms resulting from loss or altered function of CFTR in the pancreas, gut and hepatobiliary system, impairing transport of chloride and sodium, bicarbonate and water, have recently come into scientific and clinical focus (Bodewes et al, 2015;Ooi and Durie, 2016;Freeman and Ooi, 2017;Bolia et al, 2018;Rowbotham et al, 2018).…”

Section: Introductionmentioning

confidence: 99%

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

et al. 2022

View full text Add to dashboard Cite

Background: The novel and highly effective CFTR modulator combination of elexacaftor-tezacaftor-ivacaftor (ETI) has been shown to improve lung function and body weight in people with Cystic Fibrosis (pwCF) carrying a F508del mutation. However, the impact of these modulators on gastrointestinal (GI) symptoms is relatively unknown. Therefore, the CFAbd-Score was developed and validated following FDA recommendations for development of a PROM including focus groups, multidisciplinary CF specialists, people with CF and their families. The aim of this study was to assess effects of ETI on GI symptoms using the CFAbd-Score.Methods: Gastrointestinal symptoms were prospectively assessed in pwCF using the CFAbd-Score before and up to 26 weeks during therapy. The CFAbd-Score was also administered to a healthy control (HC) group. The one-sided questionnaire includes 28 items grouped in five domains. Data analysis included calculation of scores with a weighting tool, developed according to FDA recommendations.Results: A total of 107 pwCF attended in four CF centres in Germany and four centres in the UK completed the CFAbd-Score on at least two occasions. Results were compared to those obtained from the questionnaire of 45 HCs. Despite differences in demographics, age and proportion of pancreatic insufficiency between German and UK patients, analyses based on linear mixed-effects models at week 24 of ETI therapy revealed that estimated marginal means (EMMs) of total CFAbd-Scores significantly reduced (mean ± SE: 14.9 ± 1.2→10.6 ± 1.4; p < 0.01). Also EMMs of all five domains significantly declined (“pain” 16.3 ± 1.6→10.2 ± 2.3, “GERD” 15.8 ± 1.8→8.2 ± 1.9, “disorders of bowel movement” 20.9 ± 1.5→16.0 ± 1.7, “disorders of appetite” 7.9 ± 1.1→2.6 ± 1.1 and “quality of life impairment” 10.1 ± 1.92→3.9 ± 1.9). However, during 24 weeks, CF participants’ symptoms mostly still did not reach the reference levels of HCs.Discussion: Using the CFAbd-Score, the first PROM specifically developed for assessment of CF-related abdominal symptoms, we demonstrate comprehensive improvements in GI symptoms after initiation of the highly effective modulator therapy ETI.

show abstract

Section: Introductionmentioning

confidence: 99%

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

et al. 2022

View full text Add to dashboard Cite

show abstract

“…Significant weight gain is seen in pwCF on modulating drug which should be aware of the problem of obesity. Increased risk for intestinal cancer and cardiovascular complications will ask to develop preventive screenings programs and early interventions when pwCF grows older in the near future [61,62,63].…”

Section: Prognosismentioning

confidence: 99%

A new era for people with cystic fibrosis

et al. 2021

View full text Add to dashboard Cite

Cystic fibrosis is the most prevalent inherited disease caused by a defect in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The impaired electrolyte homeostasis caused by the mutated or absent protein leads to symptoms in multiple organ systems. However, the pulmonary manifestation with chronic infections and eventually respiratory failure remains the most important threat. Until one decade ago, only symptomatic treatment was available. However, since 2012, different combinations of CFTR modulators are available for people with cystic fibrosis (pwCF) that carry different mutations. The advent of these drugs has impressively changed life expectancy and quality of life in people with cystic fibrosis and raised new challenges regarding long-term complications and tapering of conventional therapies.Conclusion: In this review, we provide an update on the latest developments around diagnostics, treatment, and prognosis of pwCF. What is Known:• Cystic fibrosis is an incurable and life-shortening disease asking for life-long symptomatic treatment.• Three combination CFTR modulating drugs has gained marked approval over the last 10 years. What is New:• The emerge of new (modulating) therapies contribute to the increasing life expectancy.• A high unmet need to develop new therapies for people with CF who cannot access or benefit from these drugs remains. This review gives an update on the current status.

show abstract

“…Treatment goals include reduction in bacteria and amelioration of nutritional deficiencies. Antimicrobials are the first-line treatment [ 1 ] and can include metronidazole and rifaximin [ 61 , 66 , 67 ]. Amoxicillin/clavulanate, which can play a role in treating CF pulmonary exacerbations, also treats SIBO by stimulating contraction of the duodenum and increasing gut motility.…”

Section: Small Bowel Bacterial Overgrowthmentioning

confidence: 99%

Understanding Cystic Fibrosis Comorbidities and Their Impact on Nutritional Management

et al. 2022

View full text Add to dashboard Cite

Cystic fibrosis (CF) is a chronic, multisystem disease with multiple comorbidities that can significantly affect nutrition and quality of life. Maintaining nutritional adequacy can be challenging in people with cystic fibrosis and has been directly associated with suboptimal clinical outcomes. Comorbidities of CF can result in significantly decreased nutritional intake and intestinal absorption, as well as increased metabolic demands. It is crucial to utilize a multidisciplinary team with expertise in CF to optimize growth and nutrition, where patients with CF and their loved ones are placed in the center of the care model. Additionally, with the advent of highly effective modulators (HEMs), CF providers have begun to identify previously unrecognized nutritional issues, such as obesity. Here, we will review and summarize commonly encountered comorbidities and their nutritional impact on this unique population.

show abstract

Luminal Gastrointestinal Manifestations of Cystic Fibrosis

Cited by 14 publications

References 93 publications

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

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

A new era for people with cystic fibrosis

Understanding Cystic Fibrosis Comorbidities and Their Impact on Nutritional Management

Contact Info

Product

Resources

About