Article
Endocrinology & Metabolism
Rebecca Hicks, Katie Larson Ode, Tim Vigers, Christine L. Chan
Summary: A study was conducted to examine the screening practices for cystic fibrosis related diabetes (CFRD) and identify barriers and strategies for improvement. Surveys were conducted among CF center directors and endocrinologists, and the results showed low screening rates and practice variations. Use of systematic reminders and further research are recommended to improve CFRD screening.
FRONTIERS IN ENDOCRINOLOGY
(2023)
Review
Medicine, General & Internal
Michal Gur, Mordechai Pollak, Ronen Bar-Yoseph, Lea Bentur
Summary: The introduction of mutation-specific therapy has revolutionized cystic fibrosis (CF) care, changing it from a severe and incurable disease to a treatable one with improved quality of life and survival. However, new concerns have arisen, such as fertility, pregnancy, and post-partum care. Although CFTR modulators show promising results in improving CF lung disease, their safety in pregnancy is still limited. This literature review explores past and ongoing studies to provide hope for improved outcomes in pregnancy for CF patients.
JOURNAL OF CLINICAL MEDICINE
(2023)
Article
Microbiology
Fabrice Jean-Pierre, Arsh Vyas, Thomas H. Hampton, Michael A. Henson, George A. O'Toole
Summary: Culture-independent studies have shown that chronic lung infections in persons with cystic fibrosis involve diverse microbial communities. Interactions among these communities influence clinical outcomes, and a single community cannot fully explain the diversity of outcomes. Combining computational and experimental approaches can help enhance our understanding of microbial community function and lead to the identification of new therapeutics for polymicrobial infections.
Article
Immunology
Gisli G. Einarsson, Bart M. Vanaudenaerde, Christopher D. Spence, Andrew J. Lee, Mieke Boon, Geert M. Verleden, J. Stuart Elborn, Lieven J. Dupont, Dirk Van Raemdonck, Deirdre F. Gilpin, Robin Vos, Stijn E. Verleden, Michael M. Tunney
Summary: This study analyzed the microbiota in the lung tissue and airway luminal mucus of PWCF patients and compared them to disease characteristics and lung damage. The results showed significant differences in microbial communities between different groups, but also overlap. The luminal mucus of PWCF had lower microbial diversity and higher dominance. Variability in ecological parameters was observed both between and within patients, but no clear relationship was found between ecological parameters and disease characteristics.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2022)
Review
Immunology
Veronica Roxana Flores-Vega, Silvia Yalid Vargas-Roldan, Jose Luis Lezana-Fernandez, Ricardo Lascurain, Jose Ignacio Santos-Preciado, Roberto Rosales-Reyes
Summary: Cystic fibrosis is a genetic disease caused by a mutation in the cftr gene, affecting chloride ion and water transport and leading to bacterial infections in the lungs. Autophagy plays a crucial role in pathogen clearance, but P. aeruginosa and B. cenocepacia have strategies to evade this pathway, resulting in chronic inflammatory immune responses.
FRONTIERS IN CELLULAR AND INFECTION MICROBIOLOGY
(2021)
Review
Infectious Diseases
Evanthia P. Perikleous, Despoina Gkentzi, Aris Bertzouanis, Emmanouil Paraskakis, Aleksandar Sovtic, Sotirios Fouzas
Summary: Patients with cystic fibrosis (CF) are frequently exposed to antibiotics, which can contribute to the development of antimicrobial resistance (AMR) and make treatment challenging. Conventional susceptibility-testing techniques may not accurately assess AMR in the lungs, and the dosing of antibiotics is complicated by factors such as biofilm formation and lung penetration. Novel diagnostic tools and effective treatment strategies are urgently needed to address the growing problem of AMR in CF.
Review
Critical Care Medicine
Christina M. Mingora, Patrick A. Flume
Summary: Cystic fibrosis is an autosomal recessive genetic condition with significant manifestations in the respiratory system. Despite advancements in treatment, pulmonary complications like pneumothorax and respiratory failure still occur. Palliative care and CF transmembrane conductance regulator therapies play evolving roles in managing the care of CF patients.
Article
Microbiology
Madeline Mei, Jacob Thomas, Stephen P. Diggle
Summary: The research revealed that heterogeneous populations of Pseudomonas aeruginosa exhibit varying susceptibility to R-pyocins, likely due to differences in the LPS core. Further studies suggest that there is heterogeneity in response to other types of LPS-binding antimicrobials as well.
Article
Pharmacology & Pharmacy
Carsten Schwarz, Claudio Procaccianti, Benjamin Mignot, Hosein Sadafi, Nicolas Schwenck, Xabier Murgia, Federico Bianco
Summary: This study used Functional Respiratory Imaging (FRI) to predict the lung deposition of Levofloxacin in CF patients, finding that shorter inspiratory times and higher inspiratory flow rates significantly reduced deposition in the lung periphery. A modest correlation was observed between the central-to-peripheral deposition (C/P) ratio of levofloxacin and FEV1, with FRI analysis revealing structural differences in airways of mild and moderate CF patients.
Review
Biochemistry & Molecular Biology
Carla M. P. Ribeiro, Matthew G. Higgs, Marianne S. Muhlebach, Matthew C. Wolfgang, Monica Borgatti, Ilaria Lampronti, Giulio Cabrini
Summary: Cystic fibrosis transmembrane conductance regulator (CFTR) modulators have provided a significant therapeutic advantage for people with cystic fibrosis (pwCF). However, these modulators face limitations in reducing chronic lung bacterial infection and inflammation, which are the main causes of respiratory damage and insufficiency in pwCF, especially in adults. This article revisits the debated issues of pulmonary bacterial infection and inflammatory processes in pwCF, highlighting the mechanisms favoring bacterial infection, the interplay between Pseudomonas aeruginosa and Staphylococcus aureus, and the cross-talk among bacteria, bronchial epithelial cells, and host immune defenses. The recent findings on the effect of CFTR modulators on bacterial infection and inflammation are also presented to identify potential therapeutic targets for respiratory pathology in pwCF.
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
(2023)
Article
Medicine, General & Internal
Anna Engell Holm, Hans Henrik Lawaetz Schultz, Helle Krogh Johansen, Tania Pressler, Thomas Kromann Lund, Martin Iversen, Michael Perch
Summary: This study monitored the time of bacterial re-colonization in lung transplant patients with cystic fibrosis and found that 70% of patients were re-colonized by bacteria after 2 years, with no observed impact on survival rates from re-colonization or different bacterial species.
JOURNAL OF CLINICAL MEDICINE
(2021)
Article
Pediatrics
Shivanthan Shanthikumar, Rasa Ruseckaite, Jen Corda, Siobhain Mulrennan, Sarath Ranganathan, Tonia Douglas
Summary: This study aimed to describe clinician experiences and attitudes towards telehealth in CF care among health professionals across Australia. The results showed that most clinicians had a positive attitude towards telehealth, but there were also concerns about its impact on health outcomes and the omission of core assessments. Guidelines are necessary to ensure the benefits of telehealth are realized without compromising the standard of care.
PEDIATRIC PULMONOLOGY
(2023)
Article
Microbiology
Thomas H. Hampton, Devin Thomas, Christopher van der Gast, George A. O'Toole, Bruce A. Stanton
Summary: This study identified five bacterial community types in CF patients, explaining 24% of lung function variability and suggesting that community stability is associated with a risk of lung function decline. Patients with severe disease experienced more shifts among community types, emphasizing the importance of understanding factors that drive shifts in community composition.
MICROBIOLOGY SPECTRUM
(2021)
Article
Critical Care Medicine
Shan Wang, Suchan Niroula, Ashley Hoffman, Melika Khorrami, Melina Khorrami, Feng Yuan, Grace N. Gasser, Soon Choi, Bovey Liu, Justin Li, Mark L. Metersky, Matthew Vincent, Christopher P. Crum, Richard C. Boucher, Harry Karmouty-Quintana, Howard J. Huang, Ajay Sheshadri, Burton F. Dickey, Kalpaj R. Parekh, John F. Engelhardt, Frank D. McKeon, Wa Xian
Summary: CF patients' lungs harbor proinflammatory stem cell variants that contribute to persistent inflammation and are resistant to CFTR modulator therapy.
AMERICAN JOURNAL OF RESPIRATORY AND CRITICAL CARE MEDICINE
(2023)
Article
Microbiology
Thomas Garrigos, Manon Dollat, Arnaud Magallon, Anais Folletet, Julien Bador, Maryam Abid, Marlene Amara, Clemence Beauruelle, Olivier Belmonte, Pierre Boyer, Emilie Cardot-Martin, Anne-Gaelle Cauchie, Sylvie Colin de Verdiere, Claire Daurel, Cecile Gaudru, Farida Hamdad, Genevieve Hery-Arnaud, Baptiste Hoellinger, Claudie Lamoureux, Marie-Frederique Lartigue, Damasie Malandain, Oceane Marchand, Caroline Piau, Sandrine Picot, Helene Revillet, Zeina Sabouni, Catherine Neuwirth, Lucie Amoureux
Summary: This study established a database for Achromobacter species using MALDI-TOF mass spectrometry, enabling rapid and accurate species identification and detection of multiresistant clones. Through the analysis of samples from French CF centers, the high prevalence of A. xylosoxidans in chronic colonization and the circulation of the clone A. xylosoxidans ST137 were confirmed. This approach holds potential for future large-scale epidemiological studies.
JOURNAL OF CLINICAL MICROBIOLOGY
(2022)
Article
Radiology, Nuclear Medicine & Medical Imaging
Andrew McCallister, Sang Hun Chung, Michael Antonacci, Margret Z. Powell, Agathe S. Ceppe, Scott H. Donaldson, Yueh Z. Lee, Rosa Tamara Branca, Jennifer L. Goralski
Summary: In CF subjects, ventilation abnormalities can be identified by both (19)F and (HPXe)-Xe-129 imaging, but these abnormalities are not entirely congruent. (19)F and (HPXe)-Xe-129 imaging provide complementary information that allows for differentiation of normally ventilated, slowly ventilated, and non-ventilated regions in the lungs.
MAGNETIC RESONANCE IN MEDICINE
(2021)
Review
Respiratory System
Dave P. Nichols, Scott H. Donaldson, Carla A. Frederick, Steven D. Freedman, Daniel Gelfond, Lucas R. Hoffman, Andrea Kelly, Michael R. Narkewicz, Jessica E. Pittman, Felix Ratjen, Scott D. Sagel, Margaret Rosenfeld, Sarah Jane Schwarzenberg, Pradeep K. Singh, George M. Solomon, Michael S. Stalvey, Shannon Kirby, Jill M. VanDalfsen, John P. Clancy, Steven M. Rowe
Summary: Highly effective CFTR modulator drug therapy is increasingly available for cystic fibrosis patients, with multiple observational research studies underway to better understand its impacts. The PROMISE study focuses on the broad impacts of starting elexacaftor/tezacaftor/ivacaftor in the US population, aiming to address important health outcomes, clinical care priorities, and research needs.
JOURNAL OF CYSTIC FIBROSIS
(2021)
Article
Respiratory System
Charlotte Rigauts, Juliana Aizawa, Steven L. Taylor, Geraint B. Rogers, Matthias Govaerts, Paul Cos, Lisa Ostyn, Sarah Sims, Eva Vandeplassche, Mozes Sze, Yves Dondelinger, Lars Vereecke, Heleen Van Acker, Jodie L. Simpson, Lucy Burr, Anne Willems, Michael M. Tunney, Cristina Cigana, Alessandra Bragonzi, Tom Coenye, Aurelie Crabbe
Summary: The presence of Rothia mucilaginosa in the lower airways has an inhibitory effect on pathogen-induced inflammation and is negatively correlated with pro-inflammatory markers in bronchiectasis patients. Mechanistically, R. mucilaginosa inhibits inflammation by reducing NF-kappa B pathway activation. These findings suggest that R. mucilaginosa in the lower airways could potentially mitigate inflammation and impact the severity and progression of chronic respiratory disorders.
EUROPEAN RESPIRATORY JOURNAL
(2022)
Article
Respiratory System
Scott H. Donaldson, Beth L. Laube, Peter Mogayzel, Timothy E. Corcoran, Joseph M. Pilewski, Agathe Ceppe, Jihong Wu, Pradeep G. Bhambhvani, Felix Ratjen, Scott D. Sagel, J. P. Clancy, Steven M. Rowe, William D. Bennett
Summary: This observational study investigated the effect of lumacaftor-ivacaftor on mucociliary and cough clearance in CF patients. The results showed no effect on whole lung mucociliary clearance, but cough-assisted clearance was significantly increased. Sweat chloride improved, indicating a modest restoration of CFTR activity, but there was no demonstrable change in lung function indices. The authors speculate that the modest effect of lumacaftor-ivacaftor on CFTR function was insufficient to improve mucociliary clearance.
JOURNAL OF CYSTIC FIBROSIS
(2022)
Article
Respiratory System
Cecilia Kristensson, Annika Astrand, Scott Donaldson, Ron Goldwater, Raolat Abdulai, Naimish Patel, Philip Gardiner, Ulrika Tehler, Anne-Kristina Mercier, Marita Olsson, Eva Ersdal, Jukka Maenpaa, Tobias Bramer, Anna Malmgren, William Bennett, Christina Keen
Summary: AZD5634 demonstrated favorable pharmacokinetics and safety in both healthy subjects and patients with CF, although proof of mechanism was not achieved in patients with CF.
JOURNAL OF CYSTIC FIBROSIS
(2022)
Review
Biochemistry & Molecular Biology
Andrew P. Shoubridge, Jocelyn M. Choo, Alyce M. Martin, Damien J. Keating, Ma-Li Wong, Julio Licinio, Geraint B. Rogers
Summary: The gut microbiome has a significant influence on human neurophysiology and mental health, playing a role in the development of psychiatric conditions and the effectiveness of common therapies. With an increasing acceptance of its role, research is now shifting towards using this knowledge to improve clinical outcomes and understanding the mechanisms of gut microbiome-brain interactions.
MOLECULAR PSYCHIATRY
(2022)
Article
Pharmacology & Pharmacy
Katherine A. Despotes, Scott H. Donaldson
Summary: Small molecular modulators have transformed the care of cystic fibrosis patients, improving lung function, respiratory symptoms, and nutrition. Efforts are being made to expand these therapies to more pediatric patients. Alternative approaches are needed for those with ineligible mutations.
CURRENT OPINION IN PHARMACOLOGY
(2022)
Article
Respiratory System
Sarah Jane Schwarzenberg, Phuong T. Vu, Michelle Skalland, Lucas R. Hoffman, Christopher Pope, Daniel Gelfond, Michael R. Narkewicz, David P. Nichols, Sonya L. Heltshe, Scott H. Donaldson, Carla A. Frederick, Andrea Kelly, Jessica E. Pittman, Felix Ratjen, Margaret Rosenfeld, Scott D. Sagel, George M. Solomon, Michael S. Stalvey, John P. Clancy, Steven M. Rowe, Steven D. Freedman
Summary: The study aims to investigate the effect of ETI on gastrointestinal symptoms in patients with cystic fibrosis. The results show that there was an overall improvement in gastrointestinal symptoms after 6 months of ETI treatment.
JOURNAL OF CYSTIC FIBROSIS
(2023)
Article
Medicine, General & Internal
Othman AlOmeir, Nilesh Patel, Parastou Donyai
Summary: The objective of this study was to visually represent women's experiences with oral hormone therapy for breast cancer using data synthesized in the literature. Three schematics were drawn and validated through interviews with healthcare professionals and breast cancer survivors. The final schematics could aid patient-professional communication and support decisions regarding hormone medication in breast cancer.
PATIENT PREFERENCE AND ADHERENCE
(2022)
Article
Oncology
Othman AlOmeir, Nilesh Patel, Parastou Donyai
Summary: The study developed a grounded theory based on a literature review and meta-synthesis of qualitative research to explain the difficulties faced by breast cancer survivors in making the initial decision to accept long-term endocrine therapy and the challenges of continuing or stopping treatment. The findings indicated that some women changed their minds about treatment due to medication side effects over time.
SUPPORTIVE CARE IN CANCER
(2022)
Article
Health Care Sciences & Services
Hamzeh Almomani, Nilesh Patel, Parastou Donyai
Summary: More people are purchasing prescription medicines from internet pharmacies, which carries the risk of buying fake medicines. This study explores how newspapers cover this issue and highlights the complexity of personal beliefs and external circumstances that influence these purchases.
JMIR FORMATIVE RESEARCH
(2023)
Article
Health Care Sciences & Services
Hamzeh Almomani, Nilesh Patel, Parastou Donyai
Summary: This study aimed to identify why people in the United Kingdom purchase prescription-only medicines (POMs) from the internet and their perceptions of risks associated with fake medicines. The factors influencing the decision to buy POMs online were grouped into themes, including advantages, disadvantages, social influencing factors, barriers, and facilitators. The findings could inform the development of public awareness campaigns and interventions to minimize the purchase of POMs online.
JMIR FORMATIVE RESEARCH
(2023)
Editorial Material
Respiratory System
Scott H. Donaldson
Summary: Higher concentrations of hypertonic saline provide better protection against pulmonary exacerbation and respiratory symptom improvement in cystic fibrosis patients, and are generally well tolerated when delivered with rapid nebulizer systems.
EUROPEAN RESPIRATORY JOURNAL
(2023)
Article
Pharmacology & Pharmacy
Mohamed Obiedalla, Nilesh Patel, Parastou Donyai
Summary: Drug shortages have a negative impact on patients and pharmacists, and need to be addressed. The survey revealed that the majority of pharmacists experience drug shortages on a weekly basis, and these shortages increase their workload and potentially affect the quality of patient care.
Article
Respiratory System
Nicole Mayer-Hamblett, David P. Nichols, Katherine Odem-Davis, Kristin A. Riekert, Greg S. Sawicki, Scott H. Donaldson, Felix Ratjen, Michael W. Konstan, Noah Simon, Daniel B. Rosenbluth, George Retsch-Bogart, John P. Clancy, Jill M. VanDalfsen, Rachael Buckingham, Alex H. Gifford
Summary: The study aims to evaluate whether the daily treatment burden can be reduced for individuals with cystic fibrosis receiving new triple-combination therapy, by comparing the effects of discontinuing versus continuing two commonly used chronic therapies.
ANNALS OF THE AMERICAN THORACIC SOCIETY
(2021)