News article
Persistence and evolution of Pseudomonas aeruginosa following initiation of highly effective modulator therapy in cystic fibrosis. With the widespread availability of elexacaftor/tezacaftor/ivacaftor (ETI) and dramatic improvement in respiratory symptoms, attention is turning to the role of therapies designed to maintain lung health. Some early studies have reported a reduced bacterial burden in the lungs after ETI, though results are easily confounded by the reduction in sputum samples and the impact on chronic P. aeruginosa infection remains unknown. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 16, 2024 Category: Respiratory Medicine Source Type: research
Safety and efficacy of elexacaftor/tezacaftor/ivacaftor in people with Cystic Fibrosis following liver transplantation: A systematic review
Cystic fibrosis (CF) is a multisystem disease caused by variants in the cystic fibrosis transmembrane conductance regulator (CFTR) gene and resulting in deficient or dysfunctional CFTR protein [1]. F508del is the most prevalent disease-causing variant in people with CF (pwCF), occurring in 80 –85 % of cases [2,3]. The prognosis has largely been determined by the pulmonary complications of the disease. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 13, 2024 Category: Respiratory Medicine Authors: Ilaria Testa, Giuseppe Indolfi, Rossa Brugha, Henkjan J Verkade, Vito Terlizzi Tags: Review Source Type: research
The evolving focus of cystic fibrosis microbiome research
For more than two decades, culture-independent microbiome analysis has been employed in efforts to improve clinical outcomes for those with CF and to gain a better fundamental understanding of polymicrobial infections. The use of these techniques began during the 1990s [1,2], in response, at least in part, to growing concerns over the emergence of new infectious syndromes. Pathogens such as Burkholderia cepacia [3] and rapidly growing nontuberculous mycobacteria [4] were increasingly associated with sudden and profound deterioration in lung function in persons with CF (PwCF) and the potential for other “novel” species ...
Source: Journal of Cystic Fibrosis - April 10, 2024 Category: Respiratory Medicine Authors: Steven L. Taylor, Geraint B. Rogers Source Type: research
Chronic rhinosinusitis in people with CF, a rapidly changing field
Chronic rhinosinusitis (CRS) has been for long addressed as a hallmark of cystic fibrosis (CF). It has been reported to affect the majority of people with CF (pwCF), with almost 100 % of patients revealing pathologic findings in CT scans, which are currently replaced by MRI scans to reduce exposure to ionising radiation and to enhance differentiation of soft tissue structures [1,2]. The burden of sinonasal symptoms markedly impairs a patient ´s well-being, and indeed, to a greater extent than “some rhinitis” is generally expected to bother general health. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 10, 2024 Category: Respiratory Medicine Authors: Jochen G. Mainz, Franziska Duckstein, Carlos Zagoya, Assen Koitschev Source Type: research
Azithromycin reduces bronchial wall thickening in infants with cystic fibrosis
The efficacy of azithromycin as an anti-inflammatory agent in infants with CF was investigated in the COMBAT-CF study [1]. Children with CF diagnosed by newborn screening were 1:1 randomly assigned to maintenance treatment with azithromycin or placebo from diagnosis until age of three, stratified by site. The primary outcome measure was structural airway disease as assessed on chest computed tomography scans (CTs) by the manual Perth-Rotterdam Annotated Grid Morphometric for CF (PRAGMA-CF) scoring method [2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 6, 2024 Category: Respiratory Medicine Authors: Yuxin Chen, Jean-Paul Charbonnier, Eleni-Rosalina Andrinopoulou, Peter D. Sly, Stephen M. Stick, Harm A.W.M. Tiddens, COMBAT-CF Study Group Source Type: research
Sexual & reproductive health in CF – A shared responsibility
Sexual and reproductive health (SRH) in cystic fibrosis (CF) is an expansive field that has recently gained more attention with the improved fertility and longevity of our CF population in the era of CFTR modulator therapy. People with cystic fibrosis (pwCF) – both men and women - have identified this field as a priority area of interest for clinicians to focus research and educational efforts [1]. SRH in CF is a broad topic encompassing not just fertility and sexuality, but also health outcomes associated with SRH events such as pregnancy/parenthood, and health services delivery of SRH needs in the unique context of spe...
Source: Journal of Cystic Fibrosis - April 5, 2024 Category: Respiratory Medicine Authors: Bethany Collins, Aaron Trimble Source Type: research
Elexacaftor/Tezacaftor/Ivacaftor use in Pediatric Cystic Fibrosis Patients with Advanced Liver Disease
Cystic fibrosis (CF) is an autosomal recessive genetic disease that affects about 30,000 individuals in the United States [1,2]. Advances in the treatment of CF, including cystic fibrosis transmembrane conductance regulator (CFTR) modulators, have increased the life expectancy, lung function, and nutritional status in people with CF (pwCF). With increased longevity, extrapulmonary manifestations, such as CF-related liver disease (CFLD), have become increasingly relevant in the management and care of pwCF [3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 4, 2024 Category: Respiratory Medicine Authors: Hannah E Protich, Jean P Molleston, Molly Bozic, Rebecca S Pettit Tags: Original Article Source Type: research
Safety and efficacy of ivacaftor in infants aged 1 to less than 4 months with cystic fibrosis
Cystic fibrosis (CF) transmembrane conductance regulator (CFTR) modulators are small-molecule therapeutics that target the underlying cause of CF. Ivacaftor, a CFTR potentiator, improves CFTR function by increasing channel gating activity at the cell surface [1,2]. In clinical trials and real-world studies, ivacaftor has been shown to be safe and efficacious in children as young as 4 months of age with CFTR gating pathogenic variants, with early and sustained improvements in lung function, CFTR function, and respiratory symptoms [3 –6]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - April 4, 2024 Category: Respiratory Medicine Authors: Paul McNally, Alvin Singh, Susanna A. McColley, Jane C. Davies, Mark Higgins, Meng Liu, Jennifer Lu, Violeta Rodriguez-Romero, Judy L. Shih, Margaret Rosenfeld, VX15-770-124 Study Group Tags: Original Article Source Type: research
The impact of switching to race-neutral reference equations on FEV1 percent predicted among people with cystic fibrosis,
Racial and ethnic disparities in health, shaped by implicit and overt racism, are pervasive and substantial. Societal structures such as health care, economic and legal systems, education and housing profoundly impact health [1 –3]. Experiences of racism, both direct and vicarious, also contribute to poorer health outcomes among minoritized individuals [4]. With the growing emphasis on improving health equity over the past two decades has come increased attention to the racist origins and racial biases in a range of clin ical tests [5], including spirometry [6], glomerular filtration rate [7] and pulse oximetry [8]. (Sou...
Source: Journal of Cystic Fibrosis - March 30, 2024 Category: Respiratory Medicine Authors: Margaret Rosenfeld, Elizabeth A. Cromwell, Michael S. Schechter, Clement Ren, Patrick A. Flume, Rhonda D. Szczesniak, Wayne J. Morgan, Raksha Jain Tags: Original Article Source Type: research
Elexacaftor/tezacaftor/ivacaftor improves nasal nitric oxide in patients with cystic fibrosis
Nasal nitric oxide (nNO) is known to be reduced in people with primary ciliary dyskinesia (PwPCD) and the measurement is used in PCD's diagnostic workup [1]. It was found, that nNO is not only reduced in PCD, but also in people with cystic fibrosis (PwCF), and in chronic rhinosinusitis (CRS) even without underlying CF and nasal polyps [2,3]. It is increased in people with allergic rhinitis and asthma compared to healthy controls (HC) [2]. People with reduced nNO levels and a history of lung disease should therefore not only be evaluated for PCD, but also for CF and other diseases associated with chronic inflammation of the...
Source: Journal of Cystic Fibrosis - March 21, 2024 Category: Respiratory Medicine Authors: Charlotte O. Pioch, Niklas Ziegahn, Christine Allomba, Leonie M. Busack, Alexandra N. Schnorr, Apolline Tosolini, Bent R. Fuhlrott, Styliani Zagkla, Till Othmer, Zulfiya Syunyaeva, Simon Y. Graeber, Mehrak Yoosefi, Stephanie Thee, Eva Steinke, Jobst R öh Tags: Original Article Source Type: research
Covid-19 in cystic fibrosis patients compared to the general population: Severity and virus-host cell interactions
Cystic fibrosis (CF), the most frequent autosomal recessive disease in the Caucasian population, is caused by mutations that disrupt chloride (Cl −) and bicarbonate transport by CFTR protein, a plasma membrane channel mainly expressed in epithelial cells of respiratory and gastrointestinal systems [1,2]. Most people with CF (pwCF) are affected by chronic obstructive pulmonary disease that is the result of the vicious cycle of respiratory ba cterial infection, mucus hypersecretion, and inflammation. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 21, 2024 Category: Respiratory Medicine Authors: Fabiana Ciciriello, Francesco Panariello, Paola Medino, Arianna Biffi, Federico Alghisi, Chiara Rosazza, Patrizia Annunziata, Valentina Bouch è, Antonio Grimaldi, Daniela Guidone, Arianna Venturini, Gianfranco Alicandro, Massimo Oggioni, Pellegrino Cerin Tags: Original Article Source Type: research
ECFS standards of care on CFTR-related disorders: Identification and care of the disorders
The European Cystic Fibrosis Society (ECFS) and its Standards of Care Committee and Diagnostic Network Working Group have launched a project to update recommendations on the diagnosis and management of cystic fibrosis transmembrance conductance regulator (CFTR)-related disorders (CFTR-RD). Two of the four planned documents addressing the general considerations of the definition and management of CFTR-RD have been published in the Journal of Cystic Fibrosis [1,2]. This third paper of the series examines in more depth the characteristics of the well established disorders that are either occasionally or commonly related to CF...
Source: Journal of Cystic Fibrosis - March 21, 2024 Category: Respiratory Medicine Authors: N.J. Simmonds, K.W. Southern, E. De Wachter, K. De Boeck, F. Bodewes, J.G. Mainz, P.G. Middleton, C. Schwarz, V. Vloeberghs, M. Wilschanski, E. Bourrat, J.D. Chalmers, C.Y. Ooi, D. Debray, D.G. Downey, P. Eschenhagen, E. Girodon, G. Hickman, A. Koitschev, Tags: Original Article Source Type: research
Correlation between trough concentration and AUC for elexacaftor, tezacaftor and ivacaftor
Cystic Fibrosis Transmembrane conductance Regulator (CFTR) modulators are the first drugs to directly target the underlying defect of the disease, and have been transforming treatment of people with Cystic Fibrosis (pwCF). The combination of elexacaftor-tezacaftor-ivacaftor (ETI) is the newest and most effective CFTR modulating medicine [1]. ETI is administered at a fixed dose in adolescents and adults (aged 12 years and older) and adjusted to age and/or weight in the pediatric population (under 12 years) [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 16, 2024 Category: Respiratory Medicine Authors: Steffie E.M. Vonk, Josje Altenburg, Ron A.A. Math ôt, E. Marleen Kemper, Amsterdam Mucociliary Clearance Disease (AMCD) Research Group Source Type: research
Association between CFTR modulators and changes in iron deficiency markers in cystic fibrosis
Extrapulmonary manifestations are a major source of morbidity and poor quality of life for people with cystic fibrosis (pwCF), and include pancreatic exocrine and endocrine dysfunction, intestinal obstruction, chronic liver disease, reproductive dysfunction, and significant nutritional deficiencies [1,2]. Small, single center studies have reported that iron deficiency (ID) is a common extrapulmonary comorbidity in pwCF, with a reported prevalence of 40 –80 % [3–5]. The presence of ID increases with advancing age and has been associated with markers for poor outcomes, including shorter time to pulmonary exacerbations, l...
Source: Journal of Cystic Fibrosis - March 14, 2024 Category: Respiratory Medicine Authors: Shijing Jia, Yizhuo Wang, Melissa H Ross, Jonathan B Zuckerman, Susan Murray, MeiLan K Han, Shannon E Cahalan, Blair E Lenhan, Ryan N Best, Jennifer L Taylor-Cousar, Richard H Simon, Linda J Fitzgerald, Jonathan P Troost, Suman L Sood, Alex H Gifford Tags: Original Article Source Type: research
Education, employment, and income among people living with cystic fibrosis across three decades – A matched cohort study using Danish health registries
Over the past thirty years, there have been remarkable incremental improvements in cystic fibrosis (CF) care, resulting in improved patient outcomes and a notable increase in median life expectancy from 28 years in 1990 to 45 years in 2018 [1,2]. However, the management of CF poses a significant burden, with adults typically dedicating almost 2 h daily to complete their CF treatments [3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 14, 2024 Category: Respiratory Medicine Authors: Camilla Bj ørn Jensen, Kristoffer Jarlov Jensen, Tacjana Pressler, Terese L. Katzenstein, Marianne Skov, Tavs Qvist, Mette Frahm Olsen, Majbritt Jeppesen, Søren Jensen-Fangel, Hanne Vebert Olesen, Simon Bertram Reuter, Hans Kristian Råket Pedersen, Joa Tags: Original Article Source Type: research
