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1.
Tools and Methods Used for the Assessment of Body Composition in Patients With Cystic Fibrosis: A Systematic Review.
Calella, P, Valerio, G, Brodlie, M, Taylor, J, Donini, LM, Siervo, M
Nutrition in clinical practice : official publication of the American Society for Parenteral and Enteral Nutrition. 2019;(5):701-714
Abstract
BACKGROUND Cystic fibrosis (CF) is characterized by changes in fat mass and lean body mass that may have important prognostic value. We aim to appraise the type and frequency of application of body composition (BC) methods in child and adult patients with CF. METHODS We used 4 databases (Embase, PubMed, Scopus, and Web of Science) to perform the literature search. The search was conducted from January 2017 to February 2017. Two independent reviewers selected articles based on titles and abstracts to check eligibility for inclusion. All study designs or types of articles (abstract, full text) were considered. RESULTS Eighty-four full-text articles and 40 studies presented only as abstracts were selected. Sixty-four studies included children and adolescents (age range of 0.1-18 years), and 41 studies recruited adults (range of 18-57 years); 13 studies included both age groups. Dual-energy X-ray absorptiometry (DXA) was used in 56 studies (33.9%), and bioelectric impedance analysis (BIA) was used in 12 studies (9.7%), whereas 38 studies (30.6%) combined different methods (up to 5 different methods) to assess BC. CONCLUSIONS The results show a large variability in the application of BC methods in patients with CF that makes the comparison between studies difficult. The only methods with a sufficient body of literature are DXA and BIA.
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2.
Gut Microbiota in Children With Cystic Fibrosis: A Taxonomic and Functional Dysbiosis.
Coffey, MJ, Nielsen, S, Wemheuer, B, Kaakoush, NO, Garg, M, Needham, B, Pickford, R, Jaffe, A, Thomas, T, Ooi, CY
Scientific reports. 2019;(1):18593
Abstract
Intestinal dysbiosis has been observed in children with cystic fibrosis (CF), yet the functional consequences are poorly understood. We investigated the functional capacity of intestinal microbiota and inflammation in children with CF. Stool samples were collected from 27 children with CF and 27 age and gender matched healthy controls (HC) (aged 0.8-18 years). Microbial communities were investigated by iTag sequencing of 16S rRNA genes and functional profiles predicted using Tax4Fun. Inflammation was measured by faecal calprotectin and M2-pyruvate kinase. Paediatric CF gastrointestinal microbiota demonstrated lower richness and diversity compared to HC. CF samples exhibited a marked taxonomic and inferred functional dysbiosis when compared to HC. In children with CF, we predicted an enrichment of genes involved in short-chain fatty acid (SCFA), antioxidant and nutrient metabolism (relevant for growth and nutrition) in CF. The notion of pro-inflammatory GI microbiota in children with CF is supported by positive correlations between intestinal inflammatory markers and both genera and functional pathways. We also observed an association between intestinal genera and both growth z-scores and FEV1%. These taxonomic and functional changes provide insights into gastrointestinal disease in children with CF and future gastrointestinal therapeutics for CF should explore the aforementioned pathways and microbial changes.
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3.
Cystic fibrosis bone disease treatment: Current knowledge and future directions.
Putman, MS, Anabtawi, A, Le, T, Tangpricha, V, Sermet-Gaudelus, I
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2019;:S56-S65
Abstract
Bone disease is a frequent complication in adolescents and adults with cystic fibrosis (CF). Early detection and monitoring of bone mineral density and multidisciplinary preventive care are necessary from childhood through adolescence to minimize CF-related bone disease (CFBD) in adult CF patients. Approaches to optimizing bone health include ensuring adequate nutrition, particularly intake of calcium and vitamins D and K, addressing other secondary causes of low bone density such as hypogonadism, encouraging weight bearing exercise, and avoiding bone toxic medications. Of the currently available anti-resorptive or anabolic osteoporosis medications, only bisphosphonates have been studied in individuals with CF. Future studies are needed to better understand the optimal approach for managing CFBD.
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4.
Enteral tube feeding for cystic fibrosis.
Shimmin, D, Lowdon, J, Remmington, T
The Cochrane database of systematic reviews. 2019;(7):CD001198
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Abstract
BACKGROUND Enteral tube feeding is routinely used in many cystic fibrosis centres when oral dietary and supplement intake has failed to achieve an adequate nutritional status. The use of this method of feeding is assessed on an individual basis taking into consideration the patients age and clinical status. This is a final update of a previously published review. OBJECTIVES To examine the evidence that in people with cystic fibrosis, supplemental enteral tube feeding improves nutritional status, respiratory function, and quality of life without significant adverse effects. SEARCH METHODS We searched the Cochrane Cystic Fibrosis and Genetic Disorders Group Trials Register which comprises references identified from comprehensive electronic database searches and handsearches of relevant journals and abstract books of conference proceedings. We also contacted the companies that market enteral feeds and reviewed their databases.Date of the most recent search of the Group's Cystic Fibrosis Trials Register: 10 July 2019.Date of the most recent hand search of PubMed: 26 October 2018. SELECTION CRITERIA All randomised controlled trials comparing supplemental enteral tube feeding for one month or longer with no specific intervention in people with cystic fibrosis. DATA COLLECTION AND ANALYSIS The searches identified 44 trials; however, none were eligible for inclusion in this review. MAIN RESULTS There are no trials included in this review. AUTHORS' CONCLUSIONS Supplemental enteral tube feeding is widely used throughout the world to improve nutritional status in people with cystic fibrosis. The methods mostly used, nasogastric or gastrostomy feeding, are expensive and may have a negative effect on self-esteem and body image. Reported use of enteral tube feeding suggests that it results in nutritional and respiratory improvement; but, efficacy has not been fully assessed by randomised controlled trials. It is acknowledged, however, that performing a randomised controlled trial would be difficult due to the ethics of withholding an intervention in a group of people whose nutritional status necessitates it.
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Course of lung function in children with cystic fibrosis in their first 3 years of life.
Balinotti, JE, Chang, DV, Lubovich, S, Rodríguez, V, Zaragoza, S, Escobar, N, Kofman, C, Pérez, GL, Ardiles, V, Teper, A
Archivos argentinos de pediatria. 2019;(5):323-329
Abstract
INTRODUCTION The early prevention of respiratory complications in children with cystic fibrosis is determining for a longer survival. The implementation of lung function tests in the first months of life allows to detect respiratory involvement, even in asymptomatic children. OBJECTIVE To assess the course of lung function in children with cystic fibrosis in their first 3 years of life and identify the factors affecting it. POPULATION AND METHODS Observational, retrospective, analytical study. Children younger than 36 months with at least 2 lung function tests were included. RESULTS Between 2008 and 2016, 48 patients were included; 85 % of them had been diagnosed by newborn screening. The first lung function test was done at 5 months old. The median Z-score of maximal flow at functional residual capacity was -0.05 (interquartile range: -1.09 to 1.08). The median change in the maximal flow Z-score between tests was -0.32 (interquartile range: -1.11 to 0.25), p = 0.045. Patients with Staphylococcus aureus respiratory infections, especially methicillin-resistant SA, evidenced a greater deterioration of lung function compared to those without infection. Neither sex nor the type of genetic mutation were associated with the course of lung function. Nutritional recovery throughout the study was really good. CONCLUSION Lung function in children with cystic fibrosis worsens progressively during their first 3 years of life. These findings are associated with Staphylococcus aureus respiratory infections.
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The role of small intestinal bacterial overgrowth in cystic fibrosis: a randomized case-controlled clinical trial with rifaximin.
Furnari, M, De Alessandri, A, Cresta, F, Haupt, M, Bassi, M, Calvi, A, Haupt, R, Bodini, G, Ahmed, I, Bagnasco, F, et al
Journal of gastroenterology. 2019;(3):261-270
Abstract
BACKGROUND Scientific literature shows a high prevalence of Small Intestinal Bacterial Overgrowth (SIBO) in patients with Cystic Fibrosis (CF). The role of SIBO in nutritional status and gastrointestinal symptoms in CF is not known. Our aim was to study epidemiology and clinical impact of SIBO while assessing the efficacy of rifaximin in eradicating SIBO in CF patients. METHODS Symptoms questionnaire and Glucose Breath Test (GBT) were given to 79 CF patients (median age 19.6 years; 9.2-36.9). Subjects with a positive GBT were enrolled in a randomized controlled trial and received rifaximin 1200 mg for 14 days or no treatment. Questionnaire and GBT were repeated 1 month after the end of treatment or 45 days after the first negative GBT. RESULTS Out of 79 patients, 25 were affected by SIBO (31.6%) with a significant correlation with lower BMI, SDS-BMI (p < 0.05) and serum albumin levels (p < 0.05), independently from pancreas insufficiency. Twenty-three patients took part in the randomized trial, 13 patients (56.5%) in rifaximin group and 10 patients (43.5%) in control group. Eradication rate of SIBO was 9/10 (90%) in rifaximin group and 2/6 (33.3%) in control group (p < 0.05). In the rifaximin group, gastrointestinal symptom improvement was observed in 4/5 patients aged ≤ 14 years and in 0/5 patients aged > 14 years (p < 0.05); in 2/6 patients in the control group. CONCLUSIONS CF patients show a high prevalence of SIBO, related to a poorer nutritional status. Rifaximin therapy is well tolerated and the results are promising in terms of efficacy in eradicating small intestinal bacterial overgrowth in CF.
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Effects of Exercise Intervention Program on Bone Mineral Accretion in Children and Adolescents with Cystic Fibrosis: A Randomized Controlled Trial.
Gupta, S, Mukherjee, A, Lodha, R, Kabra, M, Deepak, KK, Khadgawat, R, Talwar, A, Kabra, SK
Indian journal of pediatrics. 2019;(11):987-994
Abstract
OBJECTIVE To evaluate effect of one year exercise intervention program on bone mineral accrual in children and adolescent with cystic fibrosis (CF). METHODS Fifty-two CF children (mean age 149.79 mo) were randomized into experimental (15 boys and 10 girls) and control groups (15 boys and 12 girls). Experimental group performed prescribed exercises three times/week, while control group continued with routine physical activities for one year. Following were assessed at baseline and at one year: Bone mineral density (BMD) of whole body and lumbar spine, pulmonary function, exercise capacity, quality of life and habitual activity. RESULTS Change in whole body and lumbar spine BMD over 12 mo in experimental group was lower by 0.006 g/cm2 (95% CI -0.02 to 0.02) and higher by 0.001 g/cm2 (95% CI -0.04 to 0.03) than controls, respectively. However, difference between groups was non-significant for both parameters. Experimental group had a significant improvement in their exercise capacity (p = 0.006), quality of life, and serum vitamin D (p = 0.007) levels. Differences between groups for changes in pulmonary function and habitual activity were non-significant. CONCLUSIONS Exercise regime was not associated with significant improvement in BMD of CF patients, but it had a positive impact on both physical and psychological health of these patients.
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First clinical trials of novel ENaC targeting therapy, SPX-101, in healthy volunteers and adults with cystic fibrosis.
Couroux, P, Farias, P, Rizvi, L, Griffin, K, Hudson, C, Crowder, T, Tarran, R, Tullis, E
Pulmonary pharmacology & therapeutics. 2019;:101819
Abstract
BACKGROUND ENaC inhibition has been investigated as a CF treatment; however, small molecule inhibitors of ENaC lack efficacy and/or have shown dose-limiting hyperkalemia. SPX-101 is a novel, investigational small peptide (SPLUNC1 mimetic) that regulates ENaC density with the potential for efficacy without systemic effects. METHODS Two trials are presented: The first was a Phase 1, 2-part, randomized, double-blind, placebo-controlled, ascending-dose study of nebulized SPX-101 in healthy adults. Part 1 evaluated 4 single doses of SPX-101 ranging from 20 to 240 mg. Part 2 evaluated a 14-day regimen of SPX-101 at 4 doses of SPX-101 ranging from 10 to 120 mg BID. Pharmacokinetics, adverse events, spirometry, vital signs, electrocardiograms, pulse oximetry, and clinical laboratory values were assessed. The second trial was a tolerability-confirming, Phase 1b, open-label study conducted in 5 adult subjects with CF. Ascending doses of SPX-101 inhalation solution (10 mg-120 mg BID) were administered for 7 days. Safety was assessed as described above. RESULTS All 64 healthy volunteers (32 in each Part) completed the single and multiple dose study. SPX-101 was well tolerated with little/no systemic exposure and with no hyperkalemia. Adverse events were generally mild with reported respiratory events associated with the purported pharmacological activity of SPX-101. Tolerability of SPX-101 was similarly observed in adults with CF; all 5 subjects treated with SPX-101 completed the study. CONCLUSIONS SPX-101 was well-tolerated across a range of doses and had little/no systemic exposure in healthy adults and adults with CF, thus supporting further study in patients with CF. CLINICALTRIAL. GOV REGISTRATION NCT03056989.
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[Diagnostic possibilities of modern biochemical study of sputum from patients with cystic fibrosis (literature review).].
Kozlov, AV, Gusyakova, OA, Ereshchenko, AA, Khaliulin, AV
Klinicheskaia laboratornaia diagnostika. 2019;(1):24-28
Abstract
The review presents the pathobiochemical and molecular mechanisms of sputum formation in patients with cystic fibrosis associated with the pathophysiological features of the disease. Statistical data on the prevalence of this pathology in the world and in the Russian Federation are presented. The mechanisms of sputum formation and disorders of the mucociliary apparatus, leading to the accumulation of viscous bronchopulmonary secret in cystic fibrosis, are considered. The principles of the relationship between the rheological properties of sputum and the formation of inflammation in the lungs with the addition of a concomitant specific microflora in the bronchopulmonary system in patients with cystic fibrosis are presented. Describes the opportunities for biochemical studies of sputum of patients with this pathology: determining the activity of enzymes (myeloperoxidase), the content of proteinase inhibitors (α2-macroglobulin and α1-antitrypsin) and proinflammatory cytokines (IL-8 and TNFa), concentrations of iron and ferriferous proteins (lactoferrin and ferritin), which makes biochemical studies of sputum available, non-invasive, quick and cost-effective method of diagnosis, which can be widely used as an auxiliary laboratory method and makes it possible to use these metabolites as diagnostic markers to assess the severity of inflammation and infection of the lower respiratory tract and predict the development of respiratory complications in patients with cystic fibrosis.
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10.
Hypoglycemia in cystic fibrosis: Prevalence, impact and treatment.
Moheet, A, Chan, CL, Granados, A, Ode, KL, Moran, A, Battezzati, A
Journal of cystic fibrosis : official journal of the European Cystic Fibrosis Society. 2019;:S19-S24
Abstract
Hypoglycemia is a common and feared complication of insulin therapy. As in type 1 and type 2 diabetes, people with cystic fibrosis related diabetes are also at risk for hypoglycemia related to insulin therapy. Spontaneous hypoglycemia is also common in patients with CF without diabetes, who are not on glucose lowering medications. Spontaneous hypoglycemia in CF may also occur during or after an oral glucose tolerance test. In this review, we will discuss the definition, epidemiology, pathophysiology and impact of hypoglycemia, with a focus on people with cystic fibrosis. We will also review strategies to manage and prevent hypoglycemia.