Specific Antibodies to Recombinant Allergens of \u3cem\u3eAspergillus fumigatus\u3c/em\u3e in Cystic Fibrosis Patients with ABPA

Background Aspergillus fumigatus, a widely distributed fungus, has been implicated in causing life threatening infections as well as severe asthma and allergic diseases in man. Allergic affliction like allergic bronchopulmonary aspergillosis (ABPA) is a disabling lung disease frequently seen in patients with asthma and cystic fibrosis. Immunodiagnosis of the former is comparatively easier due to the availability of purified antigens and sensitive methods. However, this is not true with cystic fibrosis patients where the prevalence of ABPA is fairly high and the morbidity and mortality are significant. Methods In the present study, we have evaluated purified recombinant allergens from A. fumigatus, namely Asp f 1, f 2, f 3, f 4, and f 6 using ELISA and a semi-automated method (ImmunoCAP). We studied 17 patients each from cystic fibrosis with ABPA, and cystic fibrosis with asthma, 22 cystic fibrosis with no ABPA or asthma, and 11 age matched controls. Results The results indicate that no antigen, antibody or method is capable of differentiating cystic fibrosis (CF) with ABPA from other CF patients, although some allergens showed strong reaction or showed more prevalence among the patients studied. Conclusion When results of several allergens such as Asp f 1, f 2, f 3, f 4, and f 6 in their binding to IgA, IgG, and IgE antibodies were analyzed, a more strong discrimination of CF patients with ABPA was possible from the other groups studied

Cystic Fibrosis Foundation and European Cystic Fibrosis Society Survey of cystic fibrosis mental health care delivery

Background: Psychological morbidity in individuals with cystic fibrosis (CF) and their caregivers is common. The Cystic Fibrosis Foundation (CFF) and European Cystic Fibrosis Society (ECFS) Guidelines Committee on Mental Health sought the views of CF health care professionals concerning mental health care delivery. Methods: An online survey which focused on the current provision and barriers to mental health care was distributed to CF health care professionals. Results: Of the 1454 respondents, many did not have a colleague trained in mental health issues and 20% had no one on their team whose primary role was focused on assessing or treating these issues. Insufficient resources and a lack of competency were reported in relation to mental health referrals. Seventy-three percent of respondents had no experience with mental health screening. Of those who did, they utilized 48 different, validated scales. Conclusions: These data have informed the decision-making, dissemination and implementation strategies of the Mental Health Guidelines Committee sponsored by the CFF and ECFS

Cystic Fibrosis

Cystic Fibrosis - Heterogeneity and Personalized Treatment provides the latest research and clinical evidence for clinicians, scientists and researchers involved in the care of patients with cystic fibrosis (CF). This book outlines the burden of the CF microbiome, utilisation of CF registries to impact future care, the sequelae of hepatobiliary complication, the use of upcoming technologies to provide patient-centred care, and provides an overview of cystic fibrosis transmembrane regulator (CFTR) modulators. Looking after patients with CF is highly rewarding, allowing those of us to combine our dedication and problem-solving skills to create a personalized approach. This book is invaluable for those involved in the care of CF patients

Relationship between pulmonary exacerbations and daily physical activity in adults with cystic fibrosis

The aim of this study was to examine the relationship between pulmonary exacerbations and physical activity (PA) in adults with cystic fibrosis (CF)

Mechanisms of endothelial cell dysfunction in cystic fibrosis

Although cystic fibrosis (CF) patients exhibit signs of endothelial perturbation, the functions of the cystic fibrosis conductance regulator (CFTR) in vascular endothelial cells (EC) are poorly defined. We sought to uncover biological activities of endothelial CFTR, relevant for vascular homeostasis and inflammation. We examined cells from human umbilical cords (HUVEC) and pulmonary artery isolated from non-cystic fibrosis (PAEC) and CF human lungs (CF-PAEC), under static conditions or physiological shear. CFTR activity, clearly detected in HUVEC and PAEC, was markedly reduced in CF-PAEC. CFTR blockade increased endothelial permeability to macromolecules and reduced trans‑endothelial electrical resistance (TEER). Consistent with this, CF-PAEC displayed lower TEER compared to PAEC. Under shear, CFTR blockade reduced VE-cadherin and p120 catenin membrane expression and triggered the formation of paxillin- and vinculin-enriched membrane blebs that evolved in shrinking of the cell body and disruption of cell-cell contacts. These changes were accompanied by enhanced release of microvesicles, which displayed reduced capability to stimulate proliferation in recipient EC. CFTR blockade also suppressed insulin-induced NO generation by EC, likely by inhibiting eNOS and AKT phosphorylation, whereas it enhanced IL-8 release. Remarkably, phosphodiesterase inhibitors in combination with a β2 adrenergic receptor agonist corrected functional and morphological changes triggered by CFTR dysfunction in EC. Our results uncover regulatory functions of CFTR in EC, suggesting a physiological role of CFTR in the maintenance EC homeostasis and its involvement in pathogenetic aspects of CF. Moreover, our findings open avenues for novel pharmacology to control endothelial dysfunction and its consequences in CF

Early childhood lung function is a stronger predictor of adolescent lung function in cystic fibrosis than early Pseudomonas aeruginosa infection

Pseudomonas aeruginosa has been suggested as a major determinant of poor pulmonary outcomes in cystic fibrosis (CF), although other factors play a role. Our objective was to investigate the association of early childhood Pseudomonas infection on differences in lung function in adolescence with CF

Cystic fibrosis: What’s new in South Africa in 2019

Cystic fibrosis (CF) is one of the most common autosomal recessive  disorders worldwide. The incidence of CF depends on the prevalence of CFTR (cystic fibrosis transmembrane conductance regulator) gene  mutations in the population, which is determined by genetic diversity and ethnicity. Over 2 000 CFTR mutation variants, divided into six distinct functional classes, are now identified, but not all cause CF disease.  Advancements in the molecular diagnosis of CF and recognition of a wider spectrum of CF severity have led to recent revision of CF diagnostic nomenclature and criteria. Identifying which CFTR mutations people with CF carry is important, as novel treatments that target the specific CFTR  dysfunction at a molecular level are now available, and many new drugs are in the pipeline. These and other advancements in CF are comprehensively covered in the revised 5th edition of the South African Cystic Fibrosis Consensus Guidelines, published in 2017. In addition, the South African Cystic Fibrosis  Registry Initiative (SACFRI) was launched in April 2018. SACFRI is a multicentre public-private collaboration of CF healthcare  providers across South Africa (SA), which will prospectively collect data relating to CF diagnosis and  outcomes in SA. Local SA registry data are critical to understanding the epidemiology of CF in SA, and SACFRI willbe an important tool to identify and prioritise areas of CF care that require intervention

Cystic Fibrosis - Related Diabetes

Cystic fibrosis (CF) is life-shortening autosomal recessive disease, caused by mutations in the cystic fibrosis transmembrane conductance regulator protein. The most common of CF complications is cystic fibrosis-related diabetes (CFRD). The pathophysiology of CFRD is complex. The best test for screening and diagnosis of CFRD is the oral glucose tolerance test (OGTT). Insulin therapy is a treatment of choice in CFDR pharmacotherapy. An inseparable element of CFRD therapy is also physical activity and diet

Measuring and reporting quality of life outcomes in clinical trials in cystic fibrosis: a critical review

Good quality clinical trials are essential to inform the best cystic fibrosis (CF) management and care, by determining and comparing the effectiveness of new and existing therapies and drug delivery systems. The formal inclusion of quality of life (QoL) as an outcome measure in CF clinical trials is becoming more common. Both an appropriate QoL measure and sound methodology are required in order to draw valid inferences about treatments and QoL. A review was undertaken of randomised controlled trials in cystic fibrosis where QoL was measured. EMBASE, MEDLINE and ISI Web of Science were searched to locate all full papers in the English language reporting randomised controlled trials in cystic fibrosis, published between January 1991 and December 2004. All Cochrane reviews published before December 2004 were hand searched. Papers were included if the authors had reported that they had measured QoL or well being in the trial. 16 trials were identified. The interventions investigated were: antibiotics (4); home versus hospital administration of antibiotics (1); steroids (1); mucolytic therapies (6); exercise (3) and pancreatic enzymes (1). Not one trial evaluated in this review provided conclusive results concerning QoL. This review highlights many of the pitfalls of QoL measurement in CF clinical trials and provides constructive information concerning the design and reporting of trials measuring QoL

Low-grade Appendiceal Mucinous Neoplasm (Appendiceal Villous Adenoma) with Cystic Fibrosis: A Case Report

Cystic fibrosis (CF) is an autosomal dominant disease characterized by the dysfunction of exocrine secretory glands resulting from a mutation in the transmembrane regulator protein (CFTR) gene. As life expectancy increases in patients with cystic fibrosis secondary to advances in treatment, advanced age malignancies secondary to cystic fibrosis emerge. Especially, the frequency of gastrointestinal system malignancies and colon cancers increases with aging. Appendiceal tumors are a rare entity and constitute less than 1% of gastrointestinal tumors. We presented a villous adenoma encountered in an 18-year-old male patient with CF accompanied by clinical and radiological findings. Our case is the first reported appendiceal tumor that emerged in patients with cystic fibrosis