共查询到20条相似文献，搜索用时 31 毫秒
1.
Janna W. Woestenenk Dorothea A. Schulkes Henk S. Schipper Cornelis K. van der Ent Roderick H.J. Houwen 《Journal of cystic fibrosis》2017,16(3):410-417
Background
Cystic fibrosis (CF) patients are advised to derive 35% of their daily energy intake from dietary fat. Whether this high fat intake is associated with dyslipidaemia is unknown. We described the lipid profile and dietary intake in paediatric patients with CF.Methods
110 fasting lipid concentrations of 110 Dutch patients with CF were studied, along with 86 measurements of dietary intake. For the total group and for boys and girls separately, the lipid profile and the dietary intake were investigated. The cross-sectional relationship between the lipid concentrations and dietary intake was determined.Results
The mean dietary fat intake was ≥ 35% of the total energy intake, along with a considerable consumption of saturated fat. We found lower concentrations of cholesterol, high-density lipoprotein cholesterol and low-density lipoprotein cholesterol, and increased concentrations of triglyceride and triglyceride to high-density lipoprotein cholesterol ratios. Lipid concentrations were not associated with dietary fat intake.Conclusion
This study lacks variation in dietary fat intake to exclude an effect on lipid concentrations as the distribution of dietary fat intake remained constant at a high level. Elevated triglyceride concentrations and triglyceride to high-density lipoprotein cholesterol ratios suggest an increased risk of cardiovascular disease. Any negative consequences of a high dietary fat intake on the overall lipid profile later in life cannot be excluded. 相似文献2.
Joaquim Calvo-Lerma Jessie M. Hulst Inês Asseiceira Ine Claes Maria Garriga Carla Colombo Victoria Fornés Sandra Woodcock Tiago Martins Mieke Boon Mar Ruperto Sylvia Walet Chiara Speziali Peter Witters Etna Masip Celeste Barreto Kris de Boeck Carmen Ribes-Koninckx 《Journal of cystic fibrosis》2017,16(4):510-518
Background
The New European guidelines have established the most updated recommendations on nutrition and pancreatic enzyme replacement therapy (PERT) in CF. In the context of MyCyFAPP project - a European study in children with CF aimed at developing specific tools for improvement of self-management - the objective of the current study was to assess nutritional status, daily energy and macronutrient intake, and PERT dosing with reference to these new guidelines.Methods
Cross sectional study in paediatric patients with CF from 6 European centres. SD-scores for weight-for-age (WFA), height-for-age (HFA) and body mass index-for-age (BMI) were obtained. Through a specific 4-day food and enzyme-dose record, energy and macronutrients intake and PERT-use (LU/g lipids) were automatically calculated by the MyCyFAPP system. Comparisons were made using linear regression models.Results
The lowest quartiles for BMI and HFA were between 0 and -1SD in all the centres with no significant differences, and 33.5% of the patients had a SD-score < 0 for all three parameters. The minimum energy intake recommendation was not reached by 40% of the children and mean nutrients intake values were 14%, 51% and 34% of the total energy for protein, carbohydrates and lipids respectively. When assessed per centre, reported PERT doses were in the recommended range in only 13.8% to 46.6% of the patients; from 5.6% up to 82.7% of children were above the recommended doses and 3.3% to 75% were below.Conclusion
Among the 6 centres, a large variability and inconsistency with new guidelines on nutrition and PERT-use was found. Our findings document the lack of a general criterion to adjust PERT and suggest the potential benefit of educational and self-managerial tools to ensure adherence to therapies, both for clinical staff and families. They will be taken into account when developing these new tools during the next stages of MyCyFAPP Project. 相似文献3.
Lasantha Gunasekara Mustafa Al-Saiedy Francis Green Ryan Pratt Candice Bjornson Ailian Yang W. Michael Schoel Ian Mitchell Mary Brindle Mark Montgomery Elizabeth Keys John Dennis Grishma Shrestha Matthias Amrein 《Journal of cystic fibrosis》2017,16(5):565-572
Background
Airway surfactant is impaired in cystic fibrosis (CF) and associated with declines in pulmonary function. We hypothesized that surfactant dysfunction in CF is due to an excess of cholesterol with an interaction with oxidation.Methods
Surfactant was extracted from bronchial lavage fluid from children with CF and surface tension, and lipid content, inflammatory cells and microbial flora were determined. Dysfunctional surfactant samples were re-tested with a lipid-sequestering agent, methyl-β-cyclodextrin (MβCD).Results
CF surfactant samples were unable to sustain a normal low surface tension. MβCD restored surfactant function in a majority of samples.Mechanistic studies showed that the dysfunction was due to a combination of elevated cholesterol and an interaction with oxidized phospholipids and their pro-inflammatory hydrolysis products.Conclusion
We confirm that CF patients have impaired airway surfactant function which could be restored with MβCD. These findings have implications for improving lung function and mitigating inflammation in patients with CF. 相似文献4.
5.
Michelle N. Eakin Shang-En Chung Jessica Hoehn Belinda Borrelli Devin Rand-Giovannetti Kristin A. Riekert 《Journal of cystic fibrosis》2017,16(5):637-644
Background
Beliefs about medication have been associated with adherence in other diseases but there are no existing disease-specific medication beliefs questionnaires for CF. This mixed-methods validated the Cystic Fibrosis Medication Belief Questionnaire (CF-MBQ), based on social cognitive theory.Methods
Based on previous research, items were developed for five domains: motivation, self-efficacy, perceived importance, and decisional balance to take or miss medications. Cognitive interviews were conducted with 15 adult patients with CF to refine item development. 128 patients with CF completed an online survey and objective medication adherence was measured using pharmacy refill data.Results
The five subscales demonstrated strong psychometric properties, with adequate-to-good internal consistency scores. More importantly, each domain demonstrated construct validity with adherence.Conclusions
These theoretically-derived measures may be important for clinical purposes to provide guidance on appropriate interventions to improve adherence and for research to provide enhanced understanding on patient determinants of medication adherence. 相似文献6.
Lily Paemka Brian N. McCullagh Mahmoud H. Abou Alaiwa David A. Stoltz Qian Dong Christoph O. Randak Robert D. Gray Paul B. McCray 《Journal of cystic fibrosis》2017,16(4):471-474
Background
We sought to address whether CF macrophages have a primary functional defect as a consequence of CFTR loss and thus contribute to the onset of infection and inflammation observed in CF lung disease.Methods
Monocyte derived macrophages (MDMs) were prepared from newborn CF and non-CF pigs. CFTR mRNA expression was quantified by rtPCR and anion channel function was determined using whole cell patch clamp analysis. IL8 and TNFα release from MDMs in response to lipopolysaccharide stimulation was measured by ELISA.Results
CFTR was expressed in MDMs by Q-rtPCR at a lower level than in epithelial cells. MDMs exhibited functional CFTR current at the cell membrane and this current was absent in CF MDMs. CF MDMs demonstrated an exaggerated response to lipopolysaccharide stimulation.Conclusions
In the absence of CFTR function, macrophages from newborn CF pigs exhibit an increased inflammatory response to a lipopolysaccharide challenge. This may contribute to the onset and progression of CF lung disease. 相似文献7.
Kathryn A. Ramsey Caroline McGirr Stephen M. Stick Graham L. Hall Shannon J. Simpson 《Journal of cystic fibrosis》2017,16(6):713-718
Background
We assessed the effect of posture on ventilation distribution and the impact on associations with structural lung disease.Methods
Multiple breath washout (MBW) was performed in seated and supine postures in 25 healthy children and 21 children with CF. Children with CF also underwent a chest CT scan. Functional residual capacity (FRC), lung clearance index (LCI) and moment ratios were calculated from the MBW test. CT scans were evaluated for CF-related structural lung disease.Results
FRC was lower in the supine than in the seated posture, whereas LCI was higher in the supine than in the seated posture. In children with CF, associations between LCI and the extent of structural lung disease were stronger when performed in the supine posture.Conclusions
Body posture influences lung volumes and ventilation distribution in both healthy children and children with CF. MBW testing in the supine posture strengthened associations with structural lung damage. 相似文献8.
Zoé Cavalli Quitterie Reynaud Romain Bricca Raphaële Nove-Josserand Stéphane Durupt Philippe Reix Marie Perceval Michèle Pérouse de Montclos Gérard Lina Isabelle Durieu 《Journal of cystic fibrosis》2017,16(5):579-584
Background
We evaluated the prevalence of non-tuberculous mycobacteria (NTM)-positive cultures among our cystic fibrosis (CF) center patients, reviewed risk factors for NTM positivity, and determined its impact on lung function evolution.Methods
From 2009 to 2014, CF adults and children attending the CF center of Lyon (France) and having at least one positive NTM isolate were included. Each case was matched by age and gender with two CF patients with no NTM isolate (controls).Results
48 CF patients with NTM-positive isolates were matched to 96 controls. The age group for whom incident NTM was higher was young adolescents aged 13 to 17. A significant association for NTM positivity was found with Staphylococcusaureus in multivariate analysis and with allergic bronchopulmonary aspergillosis, corticosteroid and itraconazole in univariate analysis. Mean annual FEV1 decline was faster for NTM-positive patients compared to controls.Conclusion
These data highlight the high incidence of NTM-positive cultures among young adolescents with CF. 相似文献9.
Sebastian Fischer Leonie Greipel Jens Klockgether Marie Dorda Lutz Wiehlmann Nina Cramer Burkhard Tümmler 《Journal of cystic fibrosis》2017,16(3):346-352
Background
Early antimicrobial chemotherapy can prevent or at least delay chronic cystic fibrosis (CF) airways infections with Pseudomonas aeruginosa.Methods
During a 10-year study period P. aeruginosa was detected for the first time in 54 CF patients regularly seen at the CF centre Hannover. Amplicon sequencing of 34 loci of the P. aeruginosa core genome was performed in baseline and post-treatment isolates of the 15 CF patients who had remained P. aeruginosa – positive after the first round of antipseudomonal chemotherapy.Results
Deep sequencing uncovered coexisting alternative nucleotides at in total 33 of 55,284 examined genome positions including six non-synonymous polymorphisms in the lasR gene, a key regulator of quorum sensing. After early treatment 42 of 50 novel nucleotide substitutions had emerged in exopolysaccharide biosynthesis, efflux pump and porin genes.Conclusions
Early treatment selects pathoadaptive mutations in P. aeruginosa that are typical for chronic infections of CF lungs. 相似文献10.
Background
Cystic fibrosis (CF, mucoviscidosis) is caused by mutations in the gene encoding CF transmembrane conductance regulator (CFTR), which is a chloride and bicarbonate channel necessary for fluid secretion and extracellular alkalization. For a long time, research concentrated on abnormal Cl- and Na+ transport, but neglected bicarbonate as a crucial factor in CF.Methods
The present short review reports early findings as well as recent insights into the role of CFTR for bicarbonate transport and its defects in CF.Results
The available data indicate impaired bicarbonate transport not only in pancreas, intestine, airways, and reproductive organs, but also in salivary glands, sweat duct and renal tubular epithelial cells. Defective bicarbonate transport is closely related to the impaired mucus properties and mucus blocking in secretory organs of CF patients, causing the life threatening lung disease.Conclusions
Apart from the devastating lung disease, abrogated bicarbonate transport also leads to many other organ dysfunctions, which are outlined in the present review. 相似文献11.
Jacqueline L. Anderson Caitlin Miles Audrey C. Tierney 《Journal of cystic fibrosis》2017,16(2):186-197
Background
An increasing body of research investigating the use of probiotics to improve health outcomes in patients with cystic fibrosis (CF) prompted the need to systematically assess and summarise the relevant literature.Methods
An electronic search of five databases and three trial databases was conducted. Studies describing the administration of probiotics to patients with CF older than 2 years, with a comparator group on respiratory, gastrointestinal and nutritional outcomes were included.Results
Three pre–post studies and six randomised controlled trials met the inclusion criteria. Overall studies showed a positive effect of probiotics on reducing the number of pulmonary exacerbations and decreasing gastrointestinal inflammation. There was limited effect of probiotics on other outcomes and inadequate evidence for the effects of specific probiotic species and strains.Conclusion
The findings suggest that probiotics may improve respiratory and gastrointestinal outcomes in a stable CF clinic population with no reported evidence of harm. There is inadequate evidence at this time to recommend a specific species, strain or dose of probiotic as likely to be of significant benefit. 相似文献12.
William Poncin Florian Singer Anne-Sophie Aubriot Patrick Lebecque 《Journal of cystic fibrosis》2017,16(2):258-266
Background
Comparability of multiple breath washout (MBW) systems has been little explored. We assessed agreement in lung clearance index (LCI) from two similar, commercial nitrogen MBW setups in patients with Cystic Fibrosis (CF) and controls.Methods
The EasyOne Pro (NDD) and Exhalyzer D (EM) were randomly applied in 85 adults (34 with CF) and 97 children (47 with CF and normal forced expiratory volume in one second). We assessed differences between setups in LCI, lung volumes and breathing pattern and diagnostic performance for detecting abnormal lung function.Results
Compared to NDD, EM measured higher LCI, functional residual capacity and cumulative expired volume while respiratory rate was lower. Mean difference (limits of agreement) in LCI was 1.30 (? 2.34 to 4.94). In CF, prevalence of abnormal LCI was greater in children and similar in adults using EM compared to NDD.Conclusions
Agreement of MBW outcomes between setups is poor and explained by nitrogen measurement techniques and breathing pattern. 相似文献13.
L. Nkam J. Lambert A. Latouche G. Bellis PR. Burgel M.N. Hocine 《Journal of cystic fibrosis》2017,16(6):702-708
Background
Therapeutic progress in patients with cystic fibrosis (CF) has resulted in improved prognosis over the past decades. We aim to reevaluate prognostic factors of CF and provide a prognostic score to predict the risk of death or lung transplantation (LT) within a 3-year period in adult patients.Methods
We developed a logistic model using data from the French CF Registry and combined the coefficients into a prognostic score. The discriminative abilities of the model and the prognostic score were assessed by c-statistic. The prognostic score was validated using a 10-fold cross-validation.Results
The risk of death or LT within 3 years was related to eight characteristics. The development and the validation provided excellent results for the prognostic score; the c-statistic was 0.91 and 0.90 respectively.Conclusion
The score developed to predict 3-year death or LT in adults with CF might be useful for clinicians to identify patients requiring specialized evaluation for LT. 相似文献14.
Jeff R. Crosby Chenguang Zhao Chong Jiang Dong Bai Melanie Katz Sarah Greenlee Hiroshi Kawabe Michael McCaleb Daniela Rotin Shuling Guo Brett P. Monia 《Journal of cystic fibrosis》2017,16(6):671-680
Background
Epithelial sodium channel (ENaC, Scnn1) hyperactivity in the lung leads to airway surface dehydration and mucus accumulation in cystic fibrosis (CF) patients and in mice with CF-like lung disease.Methods
We identified several potent ENaC specific antisense oligonucleotides (ASOs) and tested them by inhalation in mouse models of CF-like lung disease.Results
The inhaled ASOs distributed into lung airway epithelial cells and decreased ENaC expression by inducing RNase H1-dependent degradation of the targeted Scnn1a mRNA. Aerosol delivered ENaC ASO down-regulated mucus marker expression and ameliorated goblet cell metaplasia, inflammation, and airway hyper-responsiveness. Lack of systemic activity of ASOs delivered via the aerosol route ensures the safety of this approach.Conclusions
Our results demonstrate that antisense inhibition of ENaC in airway epithelial cells could be an effective and safe approach for the prevention and reversal of lung symptoms in CF and potentially other inflammatory diseases of the lung. 相似文献15.
16.
Isobel E.R. MacKenzie Valerie Paquette Frances Gosse Sheenagh George Frederic Chappe Valerie Chappe 《Journal of cystic fibrosis》2017,16(3):335-341
Background
The progression of cystic fibrosis (CF) in patients with the rare mutation P67L was examined to determine if it induced a milder form of CF compared to the common severe ΔF508 mutation.Methods
Parameters of lung function, level of bacterial infection, nutritional status and hospitalization were used to represent CF progression. Age at diagnosis and pancreatic status were used to assess CF presentation. Analysis of data from the CF Canada Registry collected over a 15-year period included 266 ΔF508/ΔF508 homozygote patients from CF clinics in Atlantic Canada and 26 compound heterozygote patients with the rare P67L mutation from clinics across Canada.Results
Late age at diagnosis, high incidence of pancreatic sufficiency, maintained Body Mass Index (BMI) with age, delayed life-threatening bacterial infection, and fewer days in hospital were observed for P67L heterozygote patients included in this study. Although the decline of lung function did not differ from ΔF508 homozygotes, the fact that a greater proportion of P67L heterozygotes live to an older age suggests that lung function is not the primary factor determining CF progression for P67L heterozygote patients.Conclusion
The P67L mutation is associated with a mild disease, even when combined with the severe ΔF508 mutation. 相似文献17.
Janice Kelly 《Journal of cystic fibrosis》2017,16(3):367-370
Background
Cystic fibrosis (CF) is a rare fatal genetic disease, affecting 70,000 to 100,000 people worldwide [1]. Numerous countries have specific charitable organizations dedicated to CF, with many funding research to find a cure or effective control for the disease. Cystic Fibrosis Canada, the largest charity in Canada dedicated to funding research and care in CF, conducted an environmental scan of these organizations to learn and better understand their research goals and funding process.Methods
A set of questions was sent to 25 CF charitable organizations around the world by email. Responses were consolidated to identify common practices and innovative approaches.Results
Among respondents, there were variations in the amount of funds invested in research annually and the number of studies supported. Common themes identified included practicing an open call for research applications, evaluating applications using a peer review process, and placing an increased emphasis on patient engagement. Innovative approaches included funding one larger project; funding a series of sub-projects on a common theme; partially funding a research project; and, indefinitely funding part of a researcher's salary.Conclusions
Among CF charitable organizations, there are numerous approaches to research funding. Both similarities and differences were noted between these organizations, all of which share the common goal of working towards improving quality of life and survival for people with CF. 相似文献18.
George M. Solomon Bo Liu Isabelle Sermet-Gaudelus Isabelle Fajac Michael Wilschanski Francois Vermeulen Steven M. Rowe 《Journal of cystic fibrosis》2017,16(5):573-578
Background
Nasal Potential Difference (NPD) is a biomarker of CFTR activity used to diagnose CF and monitor experimental therapies. Limited studies have been performed to assess agreement between expert readers of NPD interpretation using a scoring algorithm.Methods
We developed a standardized scoring algorithm for “interpretability” and “confidence” for PD (potential difference) measures, and sought to determine the degree of agreement on NPD parameters between trained readers.Results
There was excellent agreement for interpretability between NPD readers for CF and fair agreement for normal tracings but slight agreement of interpretability in indeterminate tracings. Amongst interpretable tracings, excellent correlation of mean scores for Ringer's Baseline PD, Δamiloride, and ΔCl-free + Isoproterenol was observed. There was slight agreement regarding confidence of the interpretable PD tracings, resulting in divergence of the Ringers and Δamiloride, and ΔCl-free + Isoproterenol PDs between “high” and “low” confidence CF tracings.Conclusion
A multi-reader process with adjudication is important for scoring NPDs for diagnosis and in monitoring of CF clinical trials. 相似文献19.
Rhonda Szczesniak Lidija Turkovic Eleni-Rosalina Andrinopoulou Harm A.W.M. Tiddens 《Journal of cystic fibrosis》2017,16(2):175-185
Background
The dawn of precision medicine and CFTR modulators require more detailed assessment of lung structure in cystic fibrosis (CF) clinical studies. Various imaging markers have emerged and are measurable, but clarity is needed to identify what markers should count for clinical studies. High-resolution chest computed tomography (CT) scoring has yielded sensitive markers for the study of CF disease progression. Once completed, CT scores from ongoing randomized controlled trials can be used to examine relationships between imaging endpoints and therapeutic effectiveness. Similarly, Magnetic Resonance Imaging (MRI) is in development to generate structural as well as functional markers.Results
The aim of this review is to characterize the role of currently available CT and MRI markers in clinical studies, and to discuss study design, data processing and statistical challenges unique to these endpoints in CF studies. Suggestions to overcome these challenges in CF studies are included.Conclusions
To maximize the potential of CT and MRI markers in clinical studies and advance treatment of CF disease progression, efforts should be made to conduct longitudinal randomized controlled trials including these modalities, develop data repositories, promote standardization and conduct reproducible research. 相似文献20.
Danielle Usatin Elizabeth H. Yen Catherine McDonald Fadi Asfour John Pohl Jacob Robson 《Journal of cystic fibrosis》2017,16(4):503-509