Prioritizing sexual and reproductive health research and care for people with cystic fibrosis: A 2023 workshop report from the Cystic Fibrosis Foundation Sexual Health, Reproduction, and Gender (SHARING) Research Working Group
Based on advances in therapy and care management, people with cystic fibrosis (CF) are living longer and healthier lives [1].. The median predicted survival in the United States (U.S.) is now in the mid-50 s, and most PwCF in the U.S. are adults. Thus, more PwCF have both general and CF-specific sexual and reproductive health (SRH) questions and are contemplating their reproductive futures [2,3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 13, 2024 Category: Respiratory Medicine Authors: Traci M Kazmerski, Christie Moy, Enid Aliaj, Jessica Hudson, Brandon Wright, Maddie Poranski, Jacqui Sjoberg, Jennifer L. Taylor-Cousar, Anna M. Georgiopoulos, Sigrid L. Ladores, Aaron Trimble, Vin Tangpricha, Farah Naz Khan, Ranjith Ramasamy, Danielle Ve Tags: Review Source Type: research
Health care resource utilization preceding death or lung transplantation in people with cystic fibrosis
Lung transplantation (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 12, 2024 Category: Respiratory Medicine Authors: Erika Guyot, Quitterie Reynaud, Manon Belhassen, Marjorie B érard, Clémence Dehillotte, Lydie Lemonnier, Marie Viprey, Eric Van Ganse, Pierre-Régis Burgel, Isabelle Durieu Tags: Original Article Source Type: research
Creation of a CF-specific antibiotic spectrum index (ASI) as an antimicrobial stewardship initiative
Antibiotics are routinely included in the treatment of cystic fibrosis (CF)-related pulmonary exacerbations (PEx) despite limited data to guide optimal selection. Unsurprisingly, this dearth of evidence has led to substantial variability in antibiotic choice[1,2]. Antibiotics are frequently selected based on prior successful PEx treatment regimens or on prior respiratory culture and susceptibility results. Although antibiotic use among people with CF (PwCF) has been associated with improved clinical outcomes[3,4], there are growing concerns related to antimicrobial resistance and other important antibiotic-related adverse ...
Source: Journal of Cystic Fibrosis - March 12, 2024 Category: Respiratory Medicine Authors: Jonathan D. Cogen, Sonya L. Heltshe, Adam W. Brothers, Donald R. VanDevanter, Jeffrey S. Gerber, Matthew P. Kronman, Ranjani Somayaji Tags: Short Communication Source Type: research
Symptoms and quality of life in adults with cystic fibrosis: A cross-sectional analysis of the InSPIRe:CF trial
People living with cystic fibrosis (CF) often report a high symptom burden comprising physical (e.g., cough, dyspnea), psychosocial (e.g., depression, anxiety) and existential (e.g., fear of dying) symptoms [1 –6]. Advances in CF care such as highly effective modulator therapy (HEMT), improved airway clearance therapies, nutritional support, and lung transplantation are projected to add decades to the lives of people with CF, with a median predicted survival of 56 years [7,8]. HEMT refers to a specific combination of drugs effective in correcting the malfunctioning protein made by the CFTR gene [9]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 9, 2024 Category: Respiratory Medicine Authors: Natalia Smirnova, Jane Lowers, Alexandre Cammarata-Mouchtouris, Elisabeth P Dellon, Anne Fitzpatrick, Dio Kavalieratos Tags: Original Article Source Type: research
Early onset of abnormal glucose tolerance in patients with cystic fibrosis: A systematic review and meta-analysis
Cystic fibrosis (CF, OMIM: 219,700) is the most prevalent autosomal recessive disease, affecting 1 in 3500 neonates [1]. Over 2000 CF-causing mutations have been discovered in the CF transmembrane conductance regulator (CFTR) gene, reducing chloride, sodium, and bicarbonate ion transport, which leads to excessive mucus production, disrupting the function of various organs (e.g. airways, gastrointestinal system), including the pancreas [2 –4]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 7, 2024 Category: Respiratory Medicine Authors: Adrienn F. K éri, Dorina Bajzát, Zita Andrásdi, Márk Félix Juhász, Rita Nagy, Tamás Kói, Gábor Kovács, Péter Hegyi, Andrea Párniczky Tags: Review Source Type: research
Alterations in the fecal microbiota in patients with advanced cystic fibrosis liver disease after 6 months of elexacaftor/tezacaftor/ivacaftor
Cystic fibrosis (CF) associated liver disease (CFLD) is a significant cause of morbidity and mortality for patients for cystic fibrosis (PwCF), accounting for 2.6 % of overall mortality and the fourth leading cause of death for PwCF in the United States [1]. CFLD describes a wide spectrum of disease, ranging from elevated serum transaminases to hepatic steatosis, focal biliary cirrhosis, and advanced CF liver disease. Advanced CFLD (aCFLD) can be further categorized as multilobular cirrhosis with or without portal hypertension or noncirrhotic portal hypertension [2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 6, 2024 Category: Respiratory Medicine Authors: Jennifer T Duong, Christopher E Pope, Hillary S Hayden, Carson Miller, Stephen J Salipante, Steven M Rowe, George M Solomon, David Nichols, Lucas R Hoffman, Michael R Narkewicz, Nicole Green Tags: Original Article Source Type: research
Clinical efficacy of CFTR modulator therapy in people with cystic fibrosis carrying the I1234V mutation
CFTR modulators (CFTRm) have revolutionized the world of many people with cystic fibrosis (pwCF), but not for all. The most common mutation, the F508del mutation, leads to a processing and trafficking defect resulting in reduced functioning and quantity of the CFTR on the cell membrane. Subjects homozygous for the F508del mutation are defined as having minimal function of the CFTR protein, and tend to present with more severe phenotype. In the heterozygous states with a residual function mutation, subjects tend to have milder phenotype [1]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 4, 2024 Category: Respiratory Medicine Authors: Bat El Bar Aluma, Joel Reiter, Ori Efrati, Yael Bezalel, Shlomit Keler, Moshe Ashkenazi, Adi Dagan, Yael Buchnik, Ido Sadras, Malena Cohen-Cymberknoh Tags: Original Article Source Type: research
Letter to the editor: A contemporary assessment of CFTR modulator use and eligibility
The treatment of cystic fibrosis (CF) changed dramatically with the advent of cystic fibrosis transmembrane conductance regulator (CFTR) modulator therapies that address the underlying cause of the disease. With the approval of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA), CFTR modulators can now treat most people with CF (pwCF). Clinical benefits observed in trials and in real-world data demonstrate substantial improvements in lung function, pulmonary exacerbations, nutritional measures, and transplant and mortality outcomes in patients treated with ELX/TEZ/IVA [1,2]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 4, 2024 Category: Respiratory Medicine Authors: Jaime L. Rubin, James L. Kreindler Tags: Letter to the Editor Source Type: research
Year in review 2023 – Back to the future
This review synthesizes articles published in 2023, focusing on the impact of elexacaftor-tezacaftor-ivacaftor (ETI) in cystic fibrosis (CF) care. Real-world data highlights sustained benefits of ETI across age groups, while challenges like neuropsychological side effects persist. Beyond CFTR modulators, research explores telemedicine and novel therapies. Prioritizing equitable access and addressing unmet needs remain crucial for comprehensive CF management. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - March 1, 2024 Category: Respiratory Medicine Authors: Luca Cristiani, Fl ávia Fonseca Fernandes Source Type: research
Remote endpoints for clinical trials in cystic fibrosis: Report from the U.S. CF foundation remote endpoints task force
The COVID-19 pandemic forced a rapid pivot to virtual visits and remote endpoint assessment for clinical research. This pivot provides a key opportunity to optimize the use of remote endpoints in cystic fibrosis (CF) clinical research in order to minimize participant burden, engage more diverse participants and obtain more granular data in a “real world” setting. To better understand remote endpoint measurements for CF clinical research, the Cystic Fibrosis Foundation convened the Remote Endpoint Task Force (Supplement 1), a multidisciplinary group of CF researchers with remote endpoint expertise and community members ...
Source: Journal of Cystic Fibrosis - February 29, 2024 Category: Respiratory Medicine Authors: Jordana E. Hoppe, Jacquelyn Sjoberg, Gina Hong, Katie Poch, Edith T. Zemanick, Stephanie Thee, Claire Edmondson, Dhiren Patel, Meghana Sathe, Drucy Borowitz, Melissa S. Putman, Noah Lechtzin, Kristin A. Riekert, Melissa Basile, Christopher H. Goss, Mary E Tags: Review Source Type: research
Role of viable but non culturable cells in patients with cystic fibrosis in the era of highly effective modulator therapy
Cystic fibrosis (CF) is a genetic disease that results in the accumulation and the lack of clearance of dehydrated mucus, mostly in the respiratory system, and hence in bronchiectasis [1]. This thick mucus traps most inhaled bacteria and viruses in the airways, making patients more susceptible to infection. During the first years of life CF sputum cultures show the presence of Haemophilus influenzae and Staphylococcus aureus, presumed to be the source of the lung morbidity in children [2]. Meticulous daily management of lung infections combining airway clearance techniques together with prompt, aggressive antibiotic treatm...
Source: Journal of Cystic Fibrosis - February 28, 2024 Category: Respiratory Medicine Authors: Natalia Cirilli, Valentina Schiavoni, Valentina Tagliabracci, Rosaria Gesuita, Luca Tiano, Benedetta Fabrizzi, Anastasia D'Antuono, Arianna Peruzzi, Nicholas Cedraro, Flavia Carle, Marco Moretti, Luigi Ferrante, Carla Vignaroli, Francesca Biavasco, Gianma Tags: Original Article Source Type: research
SPLUNC1 as a biomarker of pulmonary exacerbations in children with cystic fibrosis
Cystic fibrosis (CF) is an autosomal recessive multiorgan disease that in the lung leads to impaired muco-ciliary clearance, chronic inflammation as well as acute and chronic bacterial infections, resulting in progressive loss of lung function [1,2]. Acute CF pulmonary exacerbations (PEx) usually are treated with anti-infectives; oral antibiotics for mild events and intravenous (IV) antibiotics for severe PEx [3,4]. However, while there is currently no universally accepted definition of severe PEx a [5,6], PEx episodes in children with CF treated with oral antibiotics are often not associated with significant spirometry ch...
Source: Journal of Cystic Fibrosis - February 26, 2024 Category: Respiratory Medicine Authors: E. Ben-Meir, L. Perrem, M. Shaw, F. Ratjen, H. Grasemann Tags: Original Article Source Type: research
Effectiveness of lumacaftor/ivacaftor initiation in children with cystic fibrosis aged 2 through 5 years on disease progression: Interim results from an ongoing registry-based study
Cystic fibrosis (CF), an autosomal recessive disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affects an estimated 105,000 people worldwide, nearly half being children (under the age of 18 years) [1,2]. Clinical manifestations of CF begin early in life and include progressive structural lung damage and pancreatic insufficiency [3 –5]. In the lungs, mucus hyperconcentration and decreased mucociliary clearance can lead to air flow obstruction, infection, and inflammation and eventually the development of severe bronchiectasis and respiratory failure [6], while pancreatic d...
Source: Journal of Cystic Fibrosis - February 23, 2024 Category: Respiratory Medicine Authors: Claire Kim, Mark Higgins, Lingyun Liu, Nataliya Volkova, Anna Zolin, Lutz Naehrlich, ECFSPR Study Group Tags: Original Article Source Type: research
Repurposing DNase I and alginate lyase to degrade the biofilm matrix of dual-species biofilms of Staphylococcus aureus and Pseudomonas aeruginosa grown in artificial sputum medium: In-vitro assessment of their activity in combination with broad-spectrum antibiotics
Lung infections are a leading cause of morbidity and mortality in patients with cystic fibrosis (CF). The sticky mucus that accumulates in their airways provides a favorable environment for microorganisms to form biofilms that lead to chronic infections[1]. Biofilms are bacterial aggregates encased in self-produced matrix mainly composed of extracellular DNA (eDNA), polysaccharides and proteins[2,3]. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - February 23, 2024 Category: Respiratory Medicine Authors: Zhifen Wang, Rita Vanbever, Joseph H. Lorent, Jessica Solis, Christiane Knoop, Fran çoise Van Bambeke Tags: Original Article Source Type: research
ECFS standards of care on CFTR-related disorders: Towards a comprehensive program for affected individuals
In 2020, the ECFS Standards of Care Committee appointed a team from the Diagnostic Network Working Group to produce a model for the diagnosis and care of people with a CFTR-related disorder (pwCFTR-RD). Since the beginning of this undertaking, it was clear that such a large topic could not be encompassed within a single document. Four papers were planned; the first two on diagnostic criteria and CFTR dysfunction assessment have been published in the Journal of Cystic Fibrosis [1,2]. The third paper covers the individual disorders. (Source: Journal of Cystic Fibrosis)
Source: Journal of Cystic Fibrosis - February 21, 2024 Category: Respiratory Medicine Authors: E De Wachter, K De Boeck, I Sermet-Gaudelus, NJ Simmonds, A Munck, L Naehrlich, J Barben, C Boyd, SJ Veen, SB Carr, I Fajac, PM Farrell, E Girodon, T Gonska, WW Grody, M Jain, A Jung, E Kerem, KS Raraigh, S van Koningsbruggen-Rietschel, MD Waller, KW Sout Tags: Original Article Source Type: research
