Review
Pharmacology & Pharmacy
Alessandro Rimessi, Veronica A. M. Vitto, Simone Patergnani, Paolo Pinton
Summary: Cystic fibrosis is a genetic disorder characterized by airway issues and lung damage. Calcium signaling plays a crucial role in the pathophysiology of CF lung disease, with dysregulated calcium signals leading to impaired cell function and exacerbated inflammation.
FRONTIERS IN PHARMACOLOGY
(2021)
Review
Pediatrics
Argyri Petrocheilou, Aggeliki Moudaki, Athanasios G. Kaditis
Summary: Inflammation and infection are crucial factors in the pathophysiology of cystic fibrosis, with mucus accumulation and airway obstruction playing key roles. Recent research has focused on improving infection and inflammation, as well as correcting the genetic defect, to advance the treatment of the disease.
Review
Critical Care Medicine
Gemma E. Stanford, Kavita Dave, Nicholas J. Simmonds
Summary: Pulmonary exacerbations in cystic fibrosis patients are significant events associated with declining lung function, reduced quality of life, hospitalizations, and decreased survival. With the increasing adult CF population globally, the management of PExs needs to evolve to reflect changing demographics and improve outcomes. Studies are underway to address unmet needs and optimize treatment strategies for PExs in adults with CF.
Article
Critical Care Medicine
Heather D. Green, Andrew M. Jones
Summary: Cystic fibrosis is a disease characterized by chronic airway infection and progressive decline in respiratory function. Recent changes in the landscape of airway infection in CF have resulted in an increased prevalence of atypical gram-negative bacterial infections. The clinical relevance and treatment strategies for many of these lower prevalence organisms are still not well understood.
Review
Endocrinology & Metabolism
Sarah S. Malik, Diksha Padmanabhan, Rebecca L. Hull-Meichle
Summary: Cystic fibrosis related diabetes is a common complication in patients with cystic fibrosis, but the mechanisms behind its occurrence are not well understood. Cystic fibrosis affects the pancreatic/islet microenvironment, leading to the loss of beta cell function and pathological changes. Further research can help develop better treatment methods.
FRONTIERS IN ENDOCRINOLOGY
(2023)
Article
Pediatrics
Raphael Enaud, Eric Frison, Sophie Missonnier, Aude Fischer, Victor de Ledinghen, Paul Perez, Stephanie Bui, Michael Fayon, Jean-Francois Chateil, Thierry Lamireau
Summary: This study evaluated the repeatability and reproducibility of TE and pSWE VTQ in children with CF, finding both techniques to be reliable for assessing liver fibrosis. The high ICC values obtained suggest that TE and pSWE VTQ can be used effectively in the follow-up of CF patients, depending on their availability in CF centers.
PEDIATRIC RESEARCH
(2022)
Review
Biochemistry & Molecular Biology
Anca Daniela Pinzaru, Cristina Maria Mihai, Tatiana Chisnoiu, Alexandru Cosmin Pantazi, Vasile Valeriu Lupu, Mustafa Ali Kassim Kassim, Ancuta Lupu, Elena Grosan, Ahmed Zaki Naji Al Jumaili, Irina Ion, Gabriela Stoleriu, Ileana Ion
Summary: Cystic fibrosis (CF) is the most common inherited condition that leads to death, especially in those of Caucasian heritage. Around 20% of young adults diagnosed with CF will develop hyperglycemia as a complication, which is later classified as a disease associated with CF. Impaired insulin secretion and glucose intolerance are the primary mechanisms associated with diabetes (type 1 or type 2) and CF. Oxidative stress plays a crucial role in triggering other chronic diseases, including CF-related diabetes. Understanding the connection between CF-related diabetes and oxidative stress is essential for individualized therapies.
Review
Plant Sciences
Maria Angeles Castillejo, Jesus Pascual, Jesus V. V. Jorrin-Novo, Tiago Santana Balbuena
Summary: This review compiles proteomic studies on forest tree species in the past decade, with a focus on Eucalyptus, Pinus, and Quercus. Advances in equipment, platforms, and methods have improved the understanding of these species at molecular, organismal, and community levels. Integrating proteomics with other omics and systems biology approaches contributes to a comprehensive understanding of biological processes and can aid in climate mitigation strategies and molecular breeding programs.
FRONTIERS IN PLANT SCIENCE
(2023)
Review
Medicine, General & Internal
F. Frost, M. J. Walshaw, D. Nazareth
Summary: Cystic fibrosis (CF) is a common life-threatening inherited condition in the Caucasian population, with pulmonary disease being the main contributor to morbidity. While life expectancy has improved, new challenges have arisen, with an increasing prevalence of CF-related diabetes being a significant issue.
QJM-AN INTERNATIONAL JOURNAL OF MEDICINE
(2022)
Article
Obstetrics & Gynecology
Rita F. Padoan, Serena Quattrucci, Annalisa Amato, Marco Salvatore, Donatello Salvatore, Giuseppe Campagna
Summary: The study found that preconceptional respiratory function of women with cystic fibrosis (CF) is associated with the duration of pregnancy and birthweight of newborns, and cesarean deliveries are common among young CF women with normal respiratory function.
ACTA OBSTETRICIA ET GYNECOLOGICA SCANDINAVICA
(2021)
Review
Pharmacology & Pharmacy
Yizi Wang, Bin Ma, Wenya Li, Peiwen Li
Summary: Triple combination therapy for cystic fibrosis patients achieves better clinical results and comparable adverse events compared to the control group.
FRONTIERS IN PHARMACOLOGY
(2022)
Article
Microbiology
Jacob Vieira, Sirus Jesudasen, Lindsay Bringhurst, Hui-Yu Sui, Lauren McIver, Katrine Whiteson, Kurt Hanselmann, George A. O'Toole, Christopher J. Richards, Leonard Sicilian, Isabel Neuringer, Peggy S. Lai
Summary: This study found that increased oxygen in persons with cystic fibrosis led to reduced total numbers and diversity of microbes, while common CF pathogens such as Pseudomonas aeruginosa and Staphylococcus aureus were relatively spared. Supplemental oxygen use may promote the growth of lung pathogens and should be further studied in the clinical setting.
Review
Endocrinology & Metabolism
Amir Moheet, Antoinette Moran
Summary: Cystic fibrosis-related diabetes (CFRD) is a common complication of cystic fibrosis (CF) and is associated with poor health outcomes. The pathophysiology of CFRD is complex and not fully understood, but factors such as inflammation, alterations in the hormone axis, insulin resistance, and genetic factors may contribute to its development. Further research is needed to better understand the mechanisms underlying CFRD and the impact of diabetes on clinical outcomes in CF.
JOURNAL OF CLINICAL ENDOCRINOLOGY & METABOLISM
(2022)
Article
Respiratory System
Nicola Jane Rowbotham, Sherie Smith, Zoe C. Elliott, Belinda Cupid, Lorna J. Allen, Katherine Cowan, Lucy Allen, Alan Robert Smyth
Summary: Through online surveys and workshops, patients and clinicians selected the revised top 10 priorities for cystic fibrosis clinical research from 971 new research questions and 15 questions from 2018, in order to determine the new focus of research in cystic fibrosis.
Review
Biology
Filippo Scialo, Felice Amato, Gustavo Cernera, Monica Gelzo, Federica Zarrilli, Marika Comegna, Lucio Pastore, Andrea Bianco, Giuseppe Castaldo
Summary: The defective mucociliary clearance in CF patients due to CFTR malfunctioning predisposes them to colonization by pathogens, leading to recurrent inflammation and rapid deterioration of lung function, impacting the lung microbiome composition. Understanding of microbial interactions in the lung and how they change during disease progression is still in its early stages, but manipulating the lung microbiome could offer promising prospects for improving therapies for CF patients.
Article
Health Care Sciences & Services
Laura Liliana Obregon, Kwonho Jeong, Zachariah P. Hoydich, Jonathan Yabes, Joseph Pilewski, Connie Richless, Laura T. Moreines, Elisabeth P. Dellon, Christopher H. Goss, Robert M. Arnold, Dio Kavalieratos
Summary: Adults living with cystic fibrosis (CF) have significant unmet supportive care needs, particularly related to anxiety, sadness, pain, and uncertainty about the future. These needs are associated with demographic characteristics, with insufficient income being a significant factor. This highlights the importance of developing treatment approaches that consider patient demographics when addressing unmet supportive care needs in adults with CF.
BMJ SUPPORTIVE & PALLIATIVE CARE
(2022)
Article
Respiratory System
Paul D. Robinson, Renee Jensen, Ryan A. Seeto, Sanja Stanojevic, Clare Saunders, Christopher Short, Jane C. Davies, Felix Ratjen
JOURNAL OF CYSTIC FIBROSIS
(2022)
Editorial Material
Respiratory System
Lucy Mary Perrem, Paul McNally, Felix Ratjen
Editorial Material
Pediatrics
Hartmut Grasemann, Nancy McDonald, Xi Zhou Yuan, Sharon Dell, Valerie Waters, Felix Ratjen
Summary: Treating exacerbations of primary ciliary dyskinesia resulted in increased sputum NO metabolites and decreased neutrophilic inflammation. The association between the two suggests that neutrophilic inflammation contributes to airway NO deficiency in primary ciliary dyskinesia, and reducing inflammation may improve airway NO homeostasis.
JOURNAL OF PEDIATRICS
(2022)
Article
Respiratory System
Gyde Nissen, Elad Ben-Meir, Matthias Kopp, Michelle Shaw, Felix Ratjen, Hartmut Grasemann
Summary: Airway nitric oxide (NO) deficiency is a characteristic of cystic fibrosis (CF), and this study found that ivacaftor therapy significantly reduces IL-1β concentration in sputum, which is closely related to the improvement in NO metabolism, suggesting a potential interaction between transepithelial chloride conductance, IL-1β, and airway NO production.
JOURNAL OF CYSTIC FIBROSIS
(2022)
Letter
Respiratory System
Renee Jensen, Michelle Shaw, Angel Phanthanourak, Felix Ratjen
JOURNAL OF CYSTIC FIBROSIS
(2022)
Article
Respiratory System
Andrew Zikic, Felix Ratjen, Michelle Shaw, Elizabeth Tullis, Valerie Waters
Summary: Changing antibiotics during treatment for pulmonary exacerbations in cystic fibrosis patients with poor clinical response did not lead to improved lung function recovery or return to baseline.
JOURNAL OF CYSTIC FIBROSIS
(2022)
Article
Respiratory System
Lucy Perrem, Sanja Stanojevic, Melinda Solomon, Hartmut Grasemann, Neil Sweezey, Valerie Waters, Don B. Sanders, Stephanie D. Davis, Felix Ratjen
Summary: The changes in lung clearance index (LCI) have different thresholds for clinical relevance in children with cystic fibrosis (CF), and these thresholds can also be applied to healthy children.
Article
Respiratory System
Isabel Gascon Casaredi, Michelle Shaw, Valerie Waters, Ryan Seeto, Ana C. Blanchard, Felix Ratjen
Summary: This study conducted long-term follow-up observation on cystic fibrosis patients and found that antibiotic eradication therapy (AET) can improve lung function in patients with early Pseudomonas aeruginosa infection.
JOURNAL OF CYSTIC FIBROSIS
(2023)
Editorial Material
Pediatrics
Ediane De Queiroz Andrade, Brooke Bailey, Jane C. C. Davies, Renee Jensen, Felix Ratjen, Clare J. J. Saunders, Christopher Short, Paul D. D. Robinson
PEDIATRIC PULMONOLOGY
(2023)
Review
Respiratory System
Lucy Perrem, Isaac Martin, Felix Ratjen
Summary: Pulmonary exacerbations in children with cystic fibrosis are common and even mild clinical events contribute to the progression of lung disease. Prompt diagnosis and management are necessary to preserve lung function.
EXPERT REVIEW OF RESPIRATORY MEDICINE
(2023)
Article
Cell Biology
Tarini N. A. Gunawardena, Zoltan Bozoky, Claire Bartlett, Hong Ouyang, Paul D. W. Eckford, Theo J. Moraes, Felix Ratjen, Tanja Gonska, Christine E. Bear
Summary: In vitro studies on nasal epithelial cultures derived from cystic fibrosis patients suggest that the rescue effect of CFTR modulator drugs can be predicted through these studies. There is interest in evaluating different measuring methods for in vitro modulator responses in patient-derived nasal cultures. The fluorescence-based Fl-ACC method shows effectiveness in detecting positive responses to interventions for all genotypes and has the potential for greater sensitivity in detecting responses to pharmacological rescue strategies targeting W1282X.
Editorial Material
Respiratory System
Christine Bear, Felix Ratjen
EUROPEAN RESPIRATORY JOURNAL
(2023)
Article
Pediatrics
Dvir Gatt, Michelle Shaw, Valerie Waters, Fiona Kritzinger, Melinda Solomon, Sharon Dell, Felix Ratjen
Summary: This retrospective study analyzed 52 pulmonary exacerbation events in 28 children with PCD between 2010 and 2022. The majority of lung function recovery occurred within the first week of IV therapy. Lower BMI was associated with nonresponse to treatment.
PEDIATRIC PULMONOLOGY
(2023)
Letter
Respiratory System
Elias Seidl, Johann-Christoph Licht, Wallace B. Wee, Martin Post, Felix Ratjen, Hartmut Grasemann
ANNALS OF THE AMERICAN THORACIC SOCIETY
(2023)
