Evaluation of airway and circulating inflammatory biomarkers for cystic fibrosis drug development
Neutrophil-dominated airway inflammation is a hallmark feature of cystic fibrosis (CF) lung disease, playing a key role in its pathogenesis and progression [1]. Consequently, a major emphasis has been placed on evaluating drugs that target inflammation. Corticosteroids and high-dose ibuprofen, two of the first anti-inflammatory drugs studied in CF, both demonstrated clinical benefit [2-4], but side effects and other considerations have limited their use [5-7]. However, evidence that these anti-inflammatory agents can slow the progression of CF lung disease, suggests that strategies to modulate lung inflammation can be bene...
Source: Journal of Cystic Fibrosis - July 1, 2020 Category: Respiratory Medicine Authors: Raksha Jain, Arthur Baines, Umer Khan, Brandie D. Wagner, Scott D. Sagel Source Type: research

Disrupted local innervation results in less VIP expression in CF mice tissues
Vasoactive Intestinal Peptide (VIP), which was discovered in the 1970s by Said and Mutt, is a 28-amino-acid neuropeptide that belongs to a superfamily of brain-gut peptide hormones which are structurally related [2]. This family includes significant neuropeptides such as the secretin, glucagon and pituitary adenylyl cyclase-activating peptide (PACAP) [3,4]. VIP immunoreactive nerve fibers originate from the central, peripheral and intrinsic nervous systems innervating all exocrine glands and blood vessels throughout the body. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 27, 2020 Category: Respiratory Medicine Authors: Anna Semaniakou, Sarah Brothers, Grayson Gould, Mehrsa Zahiremani, Jamie Paton, Frederic Chappe, Audrey Li, Younes Anini, Roger P. Croll, Valerie Chappe Tags: Original Article Source Type: research

News Article
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 25, 2020 Category: Respiratory Medicine Source Type: research

JCF Year in Review
As our attention has been distracted to address issues related to the novel coronavirus (COVID-19) and we are hoping to be relieved of its disruption to our lives, we must remember that when it is all over, our patients and their families are still dealing with the consequences of cystic fibrosis (CF). All that we had been working on prior to the pandemic remains as important as before. Through all of this the Journal of Cystic Fibrosis (JCF) has continued to receive submitted manuscripts. We appreciate the dedication of our Associate Editors and the reviewers who have continued to respond to the work involved in the peer ...
Source: Journal of Cystic Fibrosis - June 25, 2020 Category: Respiratory Medicine Authors: Patrick Flume, Carlo Castellani Tags: Editorial Source Type: research

Have courage in using nasal potential difference for diagnostic decisions and clinical research
Measurement of the nasal potential difference (NPD) was introduced in 1995 by Mike Knowles and colleagues to quantify cystic fibrosis transmembrane conductance regulator (CFTR) function at the respiratory epithelia and assess CFTR modulation [1]. Five years later, Michael Wilschanski and colleagues described the effect of gentamicin on the NPD in cystic fibrosis (CF) patients with stop mutations [2] and the use of NPD as a diagnostic tool in atypical CF [3]. It took time and effort, from single center experiences to international harmonized protocols, to overcome differences in equipment, perfusion protocols, quality contr...
Source: Journal of Cystic Fibrosis - June 25, 2020 Category: Respiratory Medicine Authors: Lutz Naehrlich Source Type: research

Editorial Board
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 25, 2020 Category: Respiratory Medicine Source Type: research

From disease mechanisms to novel therapies and back
Never before has CF care and research been so filled with hope. Understanding of the pathogenesis cascade of this lethal disorder is still not complete, but since the early days it has become clear that an effective therapy needs to tackle the basic defect – either by correcting the gene or by repairing either CFTR mRNA or protein [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 25, 2020 Category: Respiratory Medicine Authors: Carlos M. Farinha Source Type: research

Fibrocyte accumulation in the lungs of cystic fibrosis patients
Cystic fibrosis (CF) is an inheritable multi-system disease due to deficient chloride ion transport at the cell membrane of epithelial cells, especially in the lower airways, that leads to significant lung disease. CF is caused by mutations in the gene coding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1,2]. CF-associated mutations result in defective CFTR protein synthesis, processing or function, and often lead to fibrosis in multiple organs including the liver, pancreas and lung [1,2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 24, 2020 Category: Respiratory Medicine Authors: Rajesh K. Kasam, Prathibha R. Gajjala, Anil G. Jegga, Jennifer A. Courtney, Scott H. Randell, Elizabeth L. Kramer, John P. Clancy, Satish K. Madala Source Type: research

No detection of SARS-CoV-2 in cystic fibrosis patients at the Regional (Lazio) Reference Center for CF in Italy
The newly emergent human coronavirus (HCoV), known as severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has become a worldwide threat and the most severe public healthcare concern. The impact of SARS-CoV-2 on cystic fibrosis (CF) remains poorly understood, still there are serious concerns over its potential effect due to deficiencies in respiratory tract physiology of CF patients that prevent efficient clearance of pathogens from the airways. SARS-CoV-2 infections have been observed in few CF patients and were associated to a not severe infection [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 23, 2020 Category: Respiratory Medicine Authors: Carolina Scagnolari, Camilla Bitossi, Federica Frasca, Agnese Viscido, Giuseppe Oliveto, Mirko Scordio, Corrado De Vito, Maria Trancassini, Fabio Midulla, Giuseppe Cimino, Alessandra Pierangeli, Guido Antonelli Source Type: research

Tezacaftor/ivacaftor in people with cystic fibrosis who stopped lumacaftor/ivacaftor due to respiratory adverse events
Cystic fibrosis (CF) —a rare, autosomal recessive, life-shortening disease—affects more than 90,000 people worldwide [1]. CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene that lead to decreased quantity and/or defective function of epithelial cell–surface CFTR proteins, resulting in reduced ion transport and dysfunction in numerous organ systems [2,3]. Phe508del is the most prevalent CFTR mutation worldwide; approximately 45% of people with CF (pwCF) in the United States [4] and 38% with CF worldwide are homozygous for the Phe508del-CFTR mutation [5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 23, 2020 Category: Respiratory Medicine Authors: Carsten Schwarz, Sivagurunathan Sutharsan, Ralph Epaud, Ross C. Klingsberg, Rainald Fischer, Steven M. Rowe, Paul K. Audhya, Neil Ahluwalia, Xiaojun You, Thomas J. Ferro, Margaret E. Duncan, Bote G. Bruinsma Tags: Original Article Source Type: research

Clinical response to lumacaftor-ivacaftor in patients with cystic fibrosis according to baseline lung function
Cystic fibrosis (CF) is a genetic autosomal recessive disease involving mutations in the gene encoding for the cystic fibrosis transmembrane conductance regulator (CFTR) protein [1-3]. CFTR dysfunction is responsible for a multisystem disease dominated by respiratory manifestations with chronic airway infection, accelerated decline in lung function and frequent respiratory exacerbations, and by impaired nutritional status [1, 2]. Over the past decades, CF management has consisted in symptomatic treatment, which includes airway clearance techniques, systemic and inhaled antibiotics, pancreatic enzyme replacement and high fa...
Source: Journal of Cystic Fibrosis - June 23, 2020 Category: Respiratory Medicine Authors: Pierre-R égis Burgel, Isabelle Durieu, Raphaël Chiron, Laurent Mely, Anne Prevotat, Marlene Murris-Espin, Michele Porzio, Michel Abely, Philippe Reix, Christophe Marguet, Julie Macey, Isabelle Sermet-Gaudelus, Harriet Corvol, Stéphanie Bui, Tiphaine Bi Tags: Original Article Source Type: research

No detection of  SARS-CoV-2 in cystic fibrosis patients at the Regional (Lazio) Reference Center for CF in Italy
The newly emergent human coronavirus (HCoV), known as severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2), has become a worldwide threat and the most severe public healthcare concern. The impact of SARS-CoV-2 on cystic fibrosis (CF) remains poorly understood, still there are serious concerns over its potential effect due to deficiencies in respiratory tract physiology of CF patients that prevent efficient clearance of pathogens from the airways. SARS-CoV-2 infections have been observed in few CF patients and were associated to a not severe infection [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 23, 2020 Category: Respiratory Medicine Authors: Carolina Scagnolari, Camilla Bitossi, Federica Frasca, Agnese Viscido, Giuseppe Oliveto, Mirko Scordio, Corrado De Vito, Maria Trancassini, Fabio Midulla, Giuseppe Cimino, Alessandra Pierangeli, Guido Antonelli Source Type: research

Lumacaftor/ivacaftor improves liver cholesterol metabolism but does not influence hypocholesterolemia in patients with cystic fibrosis
Among a myriad of clinical alterations, most patients with cystic fibrosis (CF) have low plasma cholesterol due to intestinal malabsorption, despite the pancreatic enzyme supplementation [1,2]. In particular, we previously found that CF patients with pancreatic insufficiency (PI) have reduced plasma phytosterols levels, markers of intestinal absorption of cholesterol, that were positively correlated with vitamin E [2]. The serum level of vitamin E represents an indirect pancreatic function test that allows to evaluate the intestinal digestion/absorption of lipids [3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 22, 2020 Category: Respiratory Medicine Authors: Monica Gelzo, Paola Iacotucci, Mafalda Caputo, Gustavo Cernera, Marika Comegna, Vincenzo Carnovale, Gaetano Corso, Giuseppe Castaldo Source Type: research

Lung clearance index-triggered intervention in children with cystic fibrosis – A randomised pilot study
Cystic fibrosis (CF) lung disease affects the small airways even early in life and is characterized by progressive, irreversible changes due to recurrent, multi-pathogenic infections and inflammation that may take place in absence of symptoms [1 –3]. In school-age children, CF lung disease is conventionally monitored by the forced expired volume in 1 s (FEV1) assessed by spirometry [4]. However, many children with CF and an abnormal lung clearance index (LCI) assessed by multiple breath inert gas washout (MBW) show a normal FEV1 [5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 20, 2020 Category: Respiratory Medicine Authors: Christian Voldby, Kent Green, Thomas Kongstad, Astrid Madsen Ring, Rikke Mulvad Sandvik, Marianne Skov, Frederik Buchvald, Tacjana Pressler, Kim Gjerum Nielsen Source Type: research

Influenza B outbreak at an adult cystic fibrosis centre - Clinical impact and factors influencing spread
Cystic fibrosis (CF) is the commonest life-threatening genetic disease in the developed world [1]. Over 30,000 people have CF in Europe, with the majority of these being adults [2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 19, 2020 Category: Respiratory Medicine Authors: Jordan B Dennis, Andrew M Jones, Emma A Davies, William Welfare, Peter J Barry, Lisa Collier, Andrew Turner, Rowland J Bright-Thomas Source Type: research

In vitro 3D culture lung model from expanded primary cystic fibrosis human airway cells
Cystic fibrosis (CF) is a monogenic disease affecting the human respiratory, digestive and reproductive systems. Caused by over 2,000 genetic mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, airway symptoms include excessive production of thick mucus in the lungs, which can lead to pathogenic infections and ultimately lung damage and respiratory failure. The most common mutation is deletion of phenylalanine at position 508 (F508del; present in ~85% of individuals with CF) [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 18, 2020 Category: Respiratory Medicine Authors: Rachael E. Rayner, Jack Wellmerling, Wissam Osman, Sean Honesty, Maria Alfaro, Mark E. Peeples, Estelle Cormet-Boyaka Source Type: research

Antimicrobial resistance: Concerns of healthcare providers and people with CF
Antimicrobial resistance (AMR) broadly defined, refers to the inability of antimicrobials to effectively inhibit the growth of or kill a given microorganism [1]. AMR is increasing among microbial opportunists, which is a major concern for healthcare providers (HCP), patients, and the general public. In CF, the emergence of AMR is multi-factorial due to chronic lung infections with bacterial adaptation and to the use of chronic suppressive antibiotic therapy with additional antibiotics for exacerbations [2 –5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 17, 2020 Category: Respiratory Medicine Authors: Wendy Bullington, Sarah Hempstead, Alan R. Smyth, Pavel Drevinek, Lisa Saiman, Valerie J. Waters, Scott C. Bell, Donald R. VanDevanter, Patrick A. Flume, Stuart Elborn, Marianne S Muhlebach, Antimicrobial Resistance International Working Group in Cystic F Tags: Original Article Source Type: research

Postprandial changes in gastrointestinal function and transit in cystic fibrosis assessed by Magnetic Resonance Imaging
Cystic fibrosis (CF) is an autosomal recessive disorder, affecting over 72,000 people worldwide [1 –3]. The mutation with the highest prevalence is p.Phe508del and between 85 and 90% of CF patients have at least one copy of this gene mutation [2,3]. The mutation leads to a dysfunctional CF transmembrane conductance regulator (CFTR) protein which disrupts the passage of chloride ions and bicarbo nate, causing increased viscosity of epithelial mucus. CFTR is expressed on many epithelia; including the gastrointestinal mucosa [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 16, 2020 Category: Respiratory Medicine Authors: Christabella Ng, Neele S. Dellschaft, Caroline L. Hoad, Luca Marciani, Lu Ban, Andrew P. Prayle, Helen L. Barr, Anke Jaudszus, Jochen G. Mainz, Robin C. Spiller, Penny Gowland, Giles Major, Alan R. Smyth Tags: Original Article Source Type: research

Tezacaftor/ivacaftor in people with cystic fibrosis heterozygous for minimal function CFTR mutations
Cystic fibrosis (CF) is a life-shortening, multisystem disease that affects ≈85,000 people worldwide [1]. CF is caused by mutations in the CF transmembrane conductance regulator (CFTR) gene that reduce the quantity and/or the function of the CFTR protein (a chloride channel) on epithelial cell surfaces. The F508del-CFTR mutation is the most common CFTR mutation [2]. This mutation impairs the processing of the CFTR protein and its trafficking to the epithelial cell surface and also reduces the function of the mutated CFTR protein that reaches the cell surface [3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 13, 2020 Category: Respiratory Medicine Authors: Anne Munck, Eitan Kerem, Helmut Ellemunter, Daniel Campbell, Linda T. Wang, Neil Ahluwalia, Caroline A. Owen, Claire Wainwright Source Type: research

The fungal airway microbiome in cystic fibrosis and non-cystic fibrosis bronchiectasis
Cystic fibrosis (CF) and non-CF bronchiectasis are chronic suppurative lung diseases characterised by permanent bronchial dilatation, variable mucociliary clearance and recurrent infections [1,2]. Recent culture-independent studies have illustrated a far more complex bacterial airway biodiversity than revealed by conventional microbiology [3]. By contrast the extent of fungal infection of the airways is much less clear, and the prevalence and clinical significance of fungal communities in sputum is comparatively poorly understood [4,5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 13, 2020 Category: Respiratory Medicine Authors: Leah Cuthbertson, Imogen Felton, Phillip James, Michael J. Cox, Diana Bilton, Silke Schelenz, Michael R. Loebinger, William O.C. Cookson, Nicholas J. Simmonds, Miriam F. Moffatt Source Type: research

Newborn screening alone insufficient to improve pulmonary outcomes for cystic fibrosis
Although convincing evidence exists that newborn screening (NBS) for cystic fibrosis (CF) leads to better nutritional outcomes [1 –4], there has been a paucity of studies evaluating long-term pulmonary and mortality outcomes. To our knowledge, only one previously published study compared cohort mortality with or without screening, and this evaluation suggested that better survival occurred after early diagnosis in New South Wales, Australia [5]. Our previous comparative assessment of lung function parameters in children up to the age of 16 years old enrolled and followed in the Wisconsin CF Neonatal Screening Project...
Source: Journal of Cystic Fibrosis - June 13, 2020 Category: Respiratory Medicine Authors: Christina B. Barreda, Philip M. Farrell, Anita Laxova, Jens C. Eickhoff, Andrew T. Braun, Ryan J. Coller, Michael J. Rock Source Type: research

Immunotyping of clinically divergent p.Phe508del homozygous monozygous cystic fibrosis twins
Cystic fibrosis (CF) is the only common monogenic disease of autosomal recessive inheritance in Caucasian populations in whom a large proportion of affected individuals carries the same disease-causing lesion on both alleles, i.e. the mutation p.Phe508del in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene [1]. The clinical course of p.Phe508del homozygous patients is variable which has been attributed to genetic factors other than CFTR and to environmental factors such as climate, living conditions, socioeconomic status, therapeutic measures and adherence to treatment [2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 13, 2020 Category: Respiratory Medicine Authors: Esther Schamschula, Wolfgang Hagmann, Yassen Assenov, Silke Hedtfeld, Ahmed K. Farag, Lennart M. R ösner, Lutz Wiehlmann, Frauke Stanke, Sebastian Fischer, Angela Risch, Burkhard Tümmler Source Type: research

Accumulation and persistence of ivacaftor in airway epithelia with prolonged treatment
The basic defect in cystic fibrosis (CF) reflects loss of function of the cystic fibrosis transmembrane conductance regulator (CFTR), a plasma membrane protein involved in chloride and bicarbonate transport across cellular membranes. A key therapeutic strategy for CF focuses on CFTR modulators, drugs designed to improve the function of abnormal CFTR. All currently approved CFTR modulators include the potentiator compound ivacaftor (discovered as VX-770, [1]), which improves the gating efficiency of CFTR. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 11, 2020 Category: Respiratory Medicine Authors: Tara N. Guhr Lee, Deborah M. Cholon, Nancy L. Quinney, Martina Gentzsch, Charles R. Esther Source Type: research

Defective BACH1/HO-1 regulatory circuits in cystic fibrosis bronchial epithelial cells
Cystic fibrosis (CF) is a genetic disorder resulting in severe pulmonary impairment. It is caused by mutations in the gene encoding the cystic fibrosis transmembrane conductance regulator (CFTR) [1,2]. Defective CFTR results in impaired chloride ion export that eventually alters several metabolic and homeostatic processes [3]. One central consequence of impaired ion homeostasis in CF in vivo is the accumulation of dehydrated thick sticky mucus on the airway epithelium [4]. Due to high viscosity of the mucus there is a gradual decrease in mucus clearance resulting in recurrent infections [5] and increased inflammation and o...
Source: Journal of Cystic Fibrosis - June 11, 2020 Category: Respiratory Medicine Authors: Shashipavan Chillappagari, Virajith Garapati, Poornima Mahavadi, Lutz Naehrlich, Bernd T. Schmeck, M. Lienhard Schmitz, Andreas Guenther Source Type: research

Clinimetric evaluation of muscle function tests for individuals with cystic fibrosis: A systematic review
Cystic fibrosis (CF) is a genetic multisystemic disease caused by a mutation of the CFTR (Cystic Fibrosis Transmembrane Regulator) gene [1]. CF is associated with a progressive decline in lung function, but it also impacts on systemic functions, including muscle function [2]. Quadriceps strength (along with fat-free mass) is strongly related to reduced maximal aerobic capacity (measured by cardio-pulmonary exercise testing), which leads to a progressive reduction of quality of life and an increased rate of mortality [3 –5]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 10, 2020 Category: Respiratory Medicine Authors: Yann Combret, Clement Medrinal, Tristan Bonnevie, Francis-Edouard Gravier, Pascal Le Roux, Bouchra Lamia, Guillaume Prieur, Gregory Reychler Source Type: research

Cystic fibrosis drug trial design in the era of CFTR modulators associated with substantial clinical benefit: stakeholders ’ consensus view
CFTR modulators, which treat the basic CF defect improve key clinical outcomes in PWCF, including quality of life (QoL). These drugs demonstrate disease modulation by decreasing pulmonary exacerbations as well as lung function decline, a correlate of survival [1 –5]. Registry data already indicate survival benefit for the first of these [6]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 8, 2020 Category: Respiratory Medicine Authors: K. De Boeck, T. Lee, M. Amaral, P. Drevinek, J.S. Elborn, I. Fajac, E. Kerem, J.C. Davies Source Type: research

Building global development strategies for cf therapeutics during a transitional cftr modulator era
The cystic fibrosis (CF) community is experiencing transformative changes that will forever alter the landscape of care with the discovery and confirmation of clinically effective CF transmembrane conductance regulator (CFTR) modulator therapies potentially effective for approximately 90% of the CF population [1]. Although tremendously promising, critical goals remain to identify disease modifying therapies for all, develop new therapeutics to address the remaining complications of a multifaceted disease and, equally as important, ensure the equitable access of these therapies to all individuals with CF worldwide. (Source:...
Source: Journal of Cystic Fibrosis - June 7, 2020 Category: Respiratory Medicine Authors: N. Mayer-Hamblett, S. van Koningsbruggen-Rietschel, D.P. Nichols, D.R. VanDevanter, J.C. Davies, T. Lee, A.G. Durmowicz, F. Ratjen, M.W. Konstan, K. Pearson, S.C. Bell, J.P. Clancy, J.L. Taylor-Cousar, K. De Boeck, S.H. Donaldson, D.G. Downey, P.A. Flume, Source Type: research

Changes in LCI in F508del/F508del patients treated with lumacaftor/ivacaftor: Results from the prospect study
The PROSPECT study, a post-approval observational study in the U.S., showed no significant changes in lung function as measured by spirometry with clinical initiation of lumacaftor/ivacaftor. A sub-study within the PROSPECT study assessed the lung clearance index (LCI), as measured by multiple breath washout (MBW), a measure of lung function demonstrated to be sensitive among people with normal spirometry. Participants performed MBW prior to clinically initiating lumacaftor/ivacaftor therapy and for one year of follow-up. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 6, 2020 Category: Respiratory Medicine Authors: Michelle Shaw, Umer Khan, John P. Clancy, Scott H. Donaldson, Scott D. Sagel, Steven M. Rowe, Felix Ratjen, PROSPECT Investigators of the Cystic Fibrosis Foundation Therapeutics Development Network Tags: Short Communication Source Type: research

Pseudomonas aeruginosa antimicrobial susceptibility test (AST) results and pulmonary exacerbation treatment responses in cystic fibrosis
People with CF are prone to airway bacterial infection and experience pulmonary exacerbations (PEx), periods of acute increases in respiratory signs and symptoms often coupled with an acute lung function drop, throughout their lives. [1,2] PEx are considered important clinical events; [1-3] guidelines recommend treatment with increased airway clearance, antimicrobial therapy, and nutritional and psychosocial support, [4-5] with antimicrobials chosen by in vitro antimicrobial susceptibility testing (AST) of patient bacterial isolates. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 4, 2020 Category: Respiratory Medicine Authors: Donald R. VanDevanter, Sonya L. Heltshe, Jay B. Hilliard, Michael W. Konstan Source Type: research

In the South, if you give us lemons, we will make you lemonade
In February 2020 the MUSC Adult Cystic Fibrosis Center saw an average of 14 patients per clinic, many of whom traveled to our clinic in coastal Charleston, South Carolina (SC), United States of America (USA) from interior rural regions of our state including the towns of Hemmingway, Rockland, and Aiken among others. The majority of SC, including these towns, is designated as Medically Underserved and are designated as having Health Professional Shortages by the US Health Resources and Services Administration (HRSA). (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 3, 2020 Category: Respiratory Medicine Authors: Bryan Garcia, Lillian Christon, Sue Gray, MUSC Adult CF Program Source Type: research

Eligibility of CFTR modulators for the adult-diagnosed cystic fibrosis population
To the Editor: (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 3, 2020 Category: Respiratory Medicine Authors: Sameer Desai, Grace  Y. Lam, Jenna Sykes MMath, Anne L. Stephenson, Bradley S. Quon Tags: Letter to the Editor Source Type: research

Laboratory reporting on the clinical spectrum of CFTR p.Arg117His: Still room for improvement
Cystic fibrosis (CF) is a common autosomal recessive disorder in Caucasian populations, caused by pathogenic variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Currently, more than 2000 CFTR variants have already been identified [1]. CFTR variant LRG_663t1: c.350G>A (p.Arg117His, traditional name: R117H) is a missense variant in exon 4, leading to conductance (class IV) as well as CFTR gating (class III) defects [2,3]. It has an estimated allele frequency of 0.3% in Caucasian populations [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 3, 2020 Category: Respiratory Medicine Authors: Nele Laudus, Marie-Pierre Audr ézet, Emmanuelle Girodon, Michael A Morris, Dragica Radojkovic, Caroline Raynal, Manuela Seia, Alexandra Štambergová, Heike Torkler, Raina Yamamoto, Elisabeth M C Dequeker Source Type: research

Comment on "Effect of one-year lumacaftor-ivacaftor treatment on glucose tolerance abnormalities in cystic fibrosis patients"
With great interest we read the article by Misgault et  al [1]. In a multicentre observational study, they described favourable changes in weight and pulmonary function as known effects of Lumacaftor-Ivacaftor treatment. The new aspect of their study was the impact on oral glucose tolerance (OGT) as measured by OGTT before and one year after initiation of Lumacaftor-Ivacaftor treatment. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 2, 2020 Category: Respiratory Medicine Authors: Manfred Ballmann, Nicole Prinz, Aenne Glass, Reinhard Holl Source Type: research

WS01.1 Low frequency of confirmed hypersensitivity to antibiotics in cystic fibrosis patients
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: C. Braun, P. Reix, I. Durieu, R. Nove-Josserand, S. Durupt, C. Ohlmann, C. Mainguy, J.-F. Nicolas, A. Nosbaum, V. Jubin Tags: Workshops Source Type: research

WS01.2 Non-invasive ventilation in adult cystic fibrosis patients with severe lung disease
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: C. Biglia, S. Demichelis, R. Di Tria, C. Bena, M. Sciolla, B. Messore Tags: Workshops Source Type: research

WS01.3 Chronic rhinosinusitis with nasal polyps (CRSwNP) in adult cystic fibrosis patients: optimal medical and surgical strategies
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: G. Shumkova, E. Amelina, V. Svistushkin, S. Krasovskiy, E. Sinkov Tags: Workshops Source Type: research

WS01.4 Survival after pulmonary re-transplantation in cystic fibrosis patients: 10-year single centre experience
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: F.F. R önsholt, K. Jensen, T.K. Lund, H.H.L. Schultz, T. Pressler, M. Perch Tags: Workshops Source Type: research

WS01.5 Assessment of inhaler technique in inpatients on an adult cystic fibrosis ward
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: E.J. Williams Tags: Workshops Source Type: research

WS01.6 Antifibrinolytics therapy for treatment of hemoptysis in adults with cystic fibrosis; does it affect lung function?
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: L. Cardoni, R. Perkins, K. Shin, H. Al-Samkari, E. Pighetti, S. Rits, L. McMahon, J. Connors, A. Uluer Tags: Workshops Source Type: research

WS02.1 CF BOOST - engaging the disengaged
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: H. Green, M. Clegg, J. Hildage, V. Kendall, L. Kinsey, H. Oxley, J. Pickles, A. Tansinda, A. Jones Tags: Workshops Source Type: research

WS02.2 Specific psychological symptoms of cystic fibrosis patients following lung transplantation
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: O. Poletaeva Tags: Workshops Source Type: research

WS02.3 Cystic fibrosis in adolescence: the experience in first person
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: M.C. Reisinho Tags: Workshops Source Type: research

WS02.4 Developing an enhanced transition pathway for young people with additional needs at the Manchester Adult Cystic Fibrosis Centre (MACFC)
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: E. Shaw N úñez, A. Myrtle, D. McKenna, L. Brown, J. Fauset-Jones, M. McKenna Tags: Workshops Source Type: research

WS02.5 Setting up a therapeutic education course for male patients presenting cystic fibrosis infertility
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: S. Ramel, D. Beauvillard, J. Le Bihan, A. Valeri, M.-T. Le Martelot, A. Caroff, F. Touilin, M. Kerbrat Tags: Workshops Source Type: research

WS02.6 Living with cystic fibrosis: patients ’ experiences of diagnosis in adulthood
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: N. Sharma, D. Harcourt, E. Jenkinson, A. Pearce Tags: Workshops Source Type: research

WS03.1 Do blood biomarkers perform better than traditional methods for identifying adults with cystic fibrosis and liver fibrosis?
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: D. Amaxilati, M. Allenby, T. Daniels, M. Carroll, J. Patel Tags: Workshops Source Type: research

WS03.2 A multicentre cohort study on ursodeoxycholic acid and liver disease associated with cystic fibrosis
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: C. Colombo, G. Alicandro, H. Evans, M. Oliver, C.Y. Ooi, F. Alghisi, N. Kashirskaya, E. Kondratyeva, G. Ramm, I. de Monestrol, R. Padoan, I. Asherova, A. Lindblad, CF UDCA Study Group Tags: Workshops Source Type: research

WS03.3 A review of ursodeoxycholic acid prescribing in an adult cystic fibrosis population
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: C. Bradley, C. Rutherford, C. McParland, C. Addy, D.G. Downey, C. McKeown, S. Caskey Tags: Workshops Source Type: research

WS03.4 Lumacaftor/ivacaftor improves the intestinal inflammation in children with cystic fibrosis
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: C. Tetard, M. Mittaine, F. Beaufils, S. Bui, H. Clouzeau, F. Galod é, C. Collet, M. Fayon, T. Lamireau, P.-R. Burgel, L. Delhaes, E. Mas, R. Enaud Tags: Workshops Source Type: research

WS03.5 Gut microbiome and intestinal inflammation in infants in the FIRST cohort through 6 years of age
(Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - June 1, 2020 Category: Respiratory Medicine Authors: H. Lai, S. Murali, S. Sudakaran, FIRST Study Group Tags: Workshops Source Type: research