Review
Nutrition & Dietetics
Susanna Esposito, Ilaria Testa, Elena Mariotti Zani, Daniela Cunico, Lisa Torelli, Roberto Grandinetti, Valentina Fainardi, Giovanna Pisi, Nicola Principi
Summary: Gut dysbiosis in patients with cystic fibrosis (CF) has been shown to have an impact on the disease, affecting both the intestinal and respiratory manifestations. Probiotics have the potential to improve gut dysbiosis and reduce negative effects, but further research is needed to determine their role in CF therapy.
Review
Pediatrics
Claire Edmondson, Christopher William Course, Iolo Doull
Summary: Cystic fibrosis, a genetic disorder, has been significantly improved by the new CFTR modulators, leading to increased life expectancy for patients. However, the high cost is the main barrier to their wider introduction.
ARCHIVES OF DISEASE IN CHILDHOOD
(2021)
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.
Review
Respiratory System
Nathan Ward, Scott Morrow, Kathy Stiller, Anne E. Holland
Summary: This study found that exercise can improve sputum clearance and ease of expectoration in patients with CF. In the short term, exercise may have similar effects to traditional ACTs, but data comparing them over the long term are lacking.
Review
Pediatrics
Marlou C. Bierlaagh, Danya Muilwijk, Jeffrey M. Beekman, Cornelis K. van der Ent
Summary: Cystic fibrosis is a genetic disease caused by a defect in the CFTR gene, with pulmonary manifestations being the most significant threat. However, the introduction of CFTR modulators since 2012 has significantly improved life expectancy and quality of life for cystic fibrosis patients.
EUROPEAN JOURNAL OF PEDIATRICS
(2021)
Review
Critical Care Medicine
Melanie Chin, Amanda L. Brennan, Scott C. Bell
Summary: Improved treatments for CF-related lung disease have led to increased longevity for patients, but also increased prevalence of extrapulmonary complications. Recognition of mild CF phenotypes has also changed the landscape of the disease.
Review
Cell Biology
Lucile Regard, Clemence Martin, Esperie Burnet, Jennifer Da Silva, Pierre-Regis Burgel
Summary: Cystic fibrosis is a rare genetic multisystemic disease caused by mutations in the CFTR gene, and CFTR modulators have shown clinical efficacy in restoring CFTR protein function. However, questions remain regarding their long-term safety and effectiveness, especially in patients with advanced lung disease, liver disease, renal insufficiency, or problematic bacterial colonization. Further research is needed to investigate the impact of CFTR modulators on important outcomes such as concurrent treatments, lung transplantation, chest imaging, and pregnancies.
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
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)
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.
Article
Critical Care Medicine
Pierre-Regis Burgel, Esperie Burnet, Lucile Regard, Clemence Martin
Summary: Cystic fibrosis (CF) is a genetic disease that affects the digestive and respiratory systems. Advances in disease management have significantly improved the prognosis, but there are still disparities in prognosis based on access to specialized care. The article describes the evolution of CF demographics, predicts future trends, and discusses the importance of specialized adult CF care.
Review
Cell Biology
Guoshun Wang
Summary: Cystic fibrosis (CF) is a genetic disorder caused by mutations in the CFTR gene. Recent advancements in CFTR modulators have improved treatment options for approximately 90% of CF patients. However, these treatments are not curative and cannot address all CFTR mutations. Therefore, there is a need to develop a comprehensive and long-lasting therapy, ideally involving in-situ repair of genetic lesions in the genome. Emerging technologies like CRISPR/Cas gene editing offer promising possibilities for genetic therapy in CF, and this review provides an update on the progress and application of this technology in CF treatment.
Review
Biochemistry & Molecular Biology
Vera Dora Izsak, Alexandra Soos, Zsolt Szakacs, Peter Hegyi, Mark Felix Juhasz, Orsolya Varannai, Agnes Rita Martonosi, Maria Foldi, Alexandra Kozma, Zsolt Vajda, James A. M. Shaw, Andrea Parniczky
Summary: Early recognition and prompt treatment of cystic fibrosis-related diabetes (CFRD) are crucial for improved outcomes. Two-day continuous glucose monitoring (CGM) appears to be a promising screening test for CFRD, showing the highest sensitivity and specificity among available screening tools.
Editorial Material
Medicine, General & Internal
James O'Connell, Joseph A. Murray, Katri Kaukinen
Summary: This article discusses a patient presenting with heartburn symptoms and a family history of celiac disease, offering different viewpoints on whether to recommend a gluten-free diet.
NEW ENGLAND JOURNAL OF MEDICINE
(2021)
Article
Medicine, General & Internal
Hannah Farley, Sarah Poole, Stephen Chapman, William Flight
Summary: A retrospective single-center cohort study identified 19 adult patients diagnosed with CF, all of whom had a history of chronic respiratory symptoms and the majority of whom had a CFTR genotype considered eligible for CFTR modulator therapy.
POSTGRADUATE MEDICAL JOURNAL
(2022)
