Nat Rev Drug Discov. 2025 Jan 13. doi: 10.1038/d41573-025-00008-y. Online ahead of print.

NO ABSTRACT

PMID:39806010 | DOI:10.1038/d41573-025-00008-y

Sci Rep. 2025 Jan 13;15(1):1849. doi: 10.1038/s41598-025-85948-6.

ABSTRACT

Approaches to mitigate the severity of infections and of immune responses are still needed for the treatment of cystic fibrosis (CF) even with the success of highly effective modulator therapies. Previous studies identified reduced levels of melatonin in a CF mouse model related to circadian rhythm dysregulation. Melatonin is known to have immunomodulatory properties and it was hypothesized that treatment with melatonin would improve responses to bacterial infection in CF mice. Data demonstrate that CF mice (G542X/G542X) treated with melatonin (10 µg/mL) in drinking water for 10 weeks had improved responses to airway infection with a clinical isolate of Pseudomonas aeruginosa. Melatonin-treated mice exhibited improved bacterial clearance, reduced inflammatory markers. Mice treated in drinking water for 1 week had improved bacterial clearance but no improvement in inflammation. Wild type (WT) control mice showed no response to melatonin treatment suggesting melatonin is eliciting a CF-specific response in this model. The efficacy of direct melatonin (1 µM) treatment to the airways was also tested and found to be ineffective. In conclusion, long-term systemic treatment with melatonin is an effective therapy in a CF mouse model that normalizes the response to airway infection to a WT pattern.

PMID:39805903 | DOI:10.1038/s41598-025-85948-6

NPJ Biofilms Microbiomes. 2025 Jan 13;11(1):14. doi: 10.1038/s41522-024-00644-z.

ABSTRACT

Biofilms are critical in the persistence of Pseudomonas aeruginosa infections, particularly in cystic fibrosis patients. This study explores the adaptive mechanisms behind the phenotypic switching between Small Colony Variants (SCVs) and revertant states in P. aeruginosa biofilms, emphasizing hypermutability due to Mismatch Repair System (MRS) deficiencies. Through experimental evolution and whole-genome sequencing, we show that both wild-type and mutator strains undergo parallel evolution by accumulating compensatory mutations in factors regulating intracellular c-di-GMP levels, particularly in the Wsp and Yfi systems. While wild-type strains face genetic constraints, mutator strains bypass these by accessing alternative genetic pathways regulating c-di-GMP and biofilm formation. This increased genetic accessibility, driven by higher mutation rates and specific mutational biases, supports sustained cycles of SCV conversion and reversion. Our findings underscore the crucial role of hypermutability in P. aeruginosa adaptation, with significant implications for managing persistent infections in clinical settings.

PMID:39805827 | DOI:10.1038/s41522-024-00644-z

Arch Dis Child Educ Pract Ed. 2025 Jan 13:edpract-2024-328203. doi: 10.1136/archdischild-2024-328203. Online ahead of print.

ABSTRACT

Transformative changes in the health of children with cystic fibrosis (CF) mean that more sensitive outcome measures are needed to monitor paediatric CF lung disease. Multiple breath washout (MBW) and its primary readout lung clearance index are gaining increasing traction as an endpoint for clinical trials in the CF space and show promise as a clinical investigation. In this article, we use four clinically based questions to explore what MBW can and cannot (yet) do and highlight some of its strengths and weaknesses as an investigation. We end by discussing how we can increase the utility of MBW as an investigation in children with CF.

PMID:39805677 | DOI:10.1136/archdischild-2024-328203

Lancet Respir Med. 2025 Jan 10:S2213-2600(24)00364-3. doi: 10.1016/S2213-2600(24)00364-3. Online ahead of print.

ABSTRACT

BACKGROUND: Bronchiectasis is a disease with a global impact, but most published data come from high-income countries. We aimed to describe the clinical characteristics of patients with bronchiectasis in China.

METHODS: The Chinese Bronchiectasis Registry (BE-China) is a prospective, observational cohort enrolling patients from 111 hospitals in China. Data on demographics, comorbidities, and aetiological testing results were collected from adult patients with bronchiectasis at baseline and annual follow-up. Patients who met the inclusion criteria (age ≥18 years; received chest high-resolution CT in the past year showing bronchiectasis affecting one or more lung lobes; and clinical history consistent with bronchiectasis, including chronic cough, daily sputum production, and history of exacerbations) were included. Patients with known cystic fibrosis were excluded. To investigate variations according to different economic regions, two groups were compared based on whether per capita disposable income of residents was greater than US$5553. Clinical characteristics were compared with the European (EMBARC) registry and other national registries.

FINDINGS: Between Jan 10, 2020, and March 31, 2024, 10 324 patients from 97 centres were included in the study. Among 9501 participants with available data, the most common cause of bronchiectasis was post-infective disease (4101 [43·2%] patients), followed by idiopathic (2809 [29·6%] patients). 6676 (70·0%) of 9541 patients with available data had at least one exacerbation in the year before enrolment and 5427 (57·2%) of 9489 patients with available data were hospitalised at least once due to exacerbations. Treatments commonly used in high-income countries, such as inhaled antibiotics and macrolides, were infrequently used in China. Implementation of airway clearance in China was scarce, with only 1177 (12·2%) of 9647 patients having used at least one method of airway clearance. Compared with upper-middle-income regions, patients from lower-middle-income regions were younger (61·0 years [SD 14·0] vs 63·9 years [14·2]) with a higher proportion of pulmonary comorbidities (521 [17·8%] of 2922 patients vs 639 [8·6%] of 7402 with chronic obstructive pulmonary disease and 194 [6·6%] of 2922 patients vs 364 [4·9%] of 7402 patients with asthma), a higher tuberculosis burden (442 [16·0%] of 2768 patients vs 715 [10·6%] of 6733 patients), more severe radiological involvement (1160 [42·4%] of 2736 patients vs 2415 [35·4%] of 6816 patients with cystic bronchiectasis), more exacerbations (median 1·4 [IQR 0-2] in both groups; mean 1·4 [SD 1·6] vs 1·2 [1·4] in the previous year) and hospitalisations (1662 [60·6%] of 2743 patients vs 3765 [55·8%] of 6746 patients hospitalised at least once in the previous year), and poorer quality of life (median 57·4 [IQR 53·5-63·1] vs 58·7 [54·8-64·8] assessed by the Bronchiectasis Health Questionnaire).

INTERPRETATION: The clinical characteristics of patients with bronchiectasis in China show differences compared with cohorts in Europe and India. Bronchiectasis is more severe with a higher burden of exacerbations in lower-income regions. The management of patients with bronchiectasis in China urgently needs standardisation and improvement.

FUNDING: National Natural Science Foundation of China, Innovation Program of the Shanghai Municipal Education Commission, Program of the Shanghai Municipal Science and Technology Commission, and Program of the Shanghai Shenkang Development Center.

TRANSLATION: For the Chinese translation of the abstract see Supplementary Materials section.

PMID:39805296 | DOI:10.1016/S2213-2600(24)00364-3

Lancet Respir Med. 2025 Jan 10:S2213-2600(24)00427-2. doi: 10.1016/S2213-2600(24)00427-2. Online ahead of print.

NO ABSTRACT

PMID:39805295 | DOI:10.1016/S2213-2600(24)00427-2

Semin Thromb Hemost. 2025 Jan 13. doi: 10.1055/s-0044-1801825. Online ahead of print.

ABSTRACT

The purpose of this study is to (1) estimate and compare the prevalence of venous thromboembolism (VTE) in children (age 0 to ≤21) with versus without cystic fibrosis (CF); (2) investigate putative associations between specific gastrointestinal (GI) manifestations and the development of VTE among children with CF. This was a multicenter case-control analysis among patients aged 0 to ≤ 21 years between 2010 and 2020, using the TriNetX Research Network. Data queries included ICD-9/10 (International Classification of Diseases-9th/10th Revision) diagnosis codes. Bivariate associations with VTE among CF patients were compared using Chi-square testing for categorical variables and Student's t-test for continuous variables. We used multivariable logistic regression to test for independent associations of GI manifestations with VTE among children with CF, with adjustment for other salient covariates. There was a total of 7,689 children with and 22,327,660 without CF. The frequency of occurrence of VTE was increased nearly 20-fold among those with, as compared with without CF (130 vs. 7 per 10,000 patients). Acute pancreatitis (adjusted odd ratio [aOR] = 3.80, [95% confidence interval, CI: 2.00-7.22]), biliary disease (aOR = 2.17 [95% CI: 1.17-4.03]), gastrostomy status (aOR = 2.01 [95% CI: 1.27-3.18]), and malabsorption/malnutrition (aOR = 2.41 [95% CI: 1.52-3.82]) were each associated with a higher likelihood of VTE among children with CF. In conclusion, we found a significantly increased frequency of VTE occurrence and association of specific GI diseases as independent risk factors for VTE among children with CF compared with those without.

PMID:39805291 | DOI:10.1055/s-0044-1801825

Turk Arch Pediatr. 2025 Jan 2;60(1):22-28. doi: 10.5152/TurkArchPediatr.2025.24003.

ABSTRACT

Objective: Nebulizer contamination has potential harmful effects on the respiratory system. The aim was to investigate the contamination profile of the nebulizers in cystic fibrosis patients and evaluate the relationship between hygiene practices and microbial contamination. Materials and Methods: Microbiological swab samples were taken from 3 different locations of the nebulizers of 102 patients. A questionnaire regarding nebulizer hygiene practices was applied to participants. Results: Contamination rate was 40.2%, while chambers were the most contaminated area. The bacterial contamination rate was 37.3%, with gram-negative bacterial growth being predominant. The organisms identified were mostly environmental or floral. Only 3 of the patients were performing the whole steps correctly. This number was not sufficient to assess the relationship between nebulizer cleaning and disinfection practices and microbial growth from nebulizers. When the relationship between nebulizer cleaning/disinfection frequencies, methods, and storage locations was evaluated separately with microbial growth from nebulizers, no statistically significant relationship was found for all (P > .05 for all). Conclusion: The nebulizer contamination rate with pathogenic microorganisms is low in the present study. Regular educational interventions regarding nebulizer hygiene practices should be implemented in all Cystic Fibrosis Centers.

PMID:39803923 | DOI:10.5152/TurkArchPediatr.2025.24003

J Cyst Fibros. 2025 Jan 12:S1569-1993(24)01861-7. doi: 10.1016/j.jcf.2024.12.010. Online ahead of print.

ABSTRACT

BACKGROUND: Elexacaftor-tezacaftor-ivacaftor (ETI) has significantly improved the clinical course of people with cystic fibrosis (pwCF) and eligible CFTR variants. In this study, we prospectively evaluated liver elastography, liver fibrosis indices and liver tests in children with CF aged 6-12 years started on ETI therapy.

METHODS: Body mass index, sweat test, percent predicted forced expiratory volume in one second, serum markers of liver injury or portal hypertension, liver fibrosis indices, controlled attenuation parameter and liver stiffness were assessed before starting ETI and three and twelve months post-ETI, according to new international guidelines.

RESULTS: 27 children with CF were enrolled, 14 with liver involvement and 13 without liver involvement at baseline. A significant improvement in sweat chloride after ETI was observed in all subjects. In those with liver involvement, liver stiffness significantly decreased at 12 months of ETI, with all individuals achieving normalization or near-normalization of liver stiffness. The majority of individuals with abnormal AST, ALT, GGT, or liver fibrosis indices at baseline experienced normalization by 12 months of ETI (AST: 67%, ALT: 100%, GGT: 50%, APRI: 100%, GPR: 100%). In the no liver involvement group, the only significant change in liver health metrics at 12 months was a significant reduction in platelets (P<0.05) that remained within the normal range.

CONCLUSIONS: ETI is associated with improvement in liver stiffness, liver function tests and fibrosis indices in pwCF and liver involvement. ETI may reduce the development of advanced CF liver disease, but longer observations with larger cohorts are needed.

PMID:39800644 | DOI:10.1016/j.jcf.2024.12.010

J Cyst Fibros. 2025 Jan 11:S1569-1993(25)00005-0. doi: 10.1016/j.jcf.2025.01.005. Online ahead of print.

ABSTRACT

BACKGROUND: Cystic Fibrosis-related Bone Disease is an emerging challenge faced by 50 % of adult people with cystic fibrosis (CF). The multifactorial causes of this comorbidity remain elusive. However, congenital bone defects have been observed in animal models with CFTR mutations, suggesting its importance. The role of CFTR in bone cells development is unknown. Studies from human cells remain somewhat controversial depending on the cells used and the disease state of the patients from which the cells derived.

METHODS: Therefore, we investigated the role of CFTR in osteoblast development using induced pluripotent stem cells generated from homozygous CF donors for F508del and non-CF controls. This approach allows for a clear understanding towards how the CFTR mutation may influence osteoblast differentiation independently from other confounding factors.

RESULTS: We observed a lower capacity of differentiation in CF cells as compared to control, already from mesenchymal stem cells (MSC) stage, whereby they retained expression of the pluripotency marker OCT4. Furthermore, our results demonstrated a delayed osteoblast commitment and altered expression of specific markers, such as an increased RANKL/OPG ratio and decreased BMP2, suggesting a potentially perturbed bone homeostasis associated with CFTR mutation.

CONCLUSIONS: This is the first study of its kind, clearly demonstrating a role for CFTR mutation in delaying osteoblast differentiation and/or regeneration.

PMID:39800643 | DOI:10.1016/j.jcf.2025.01.005

J Cyst Fibros. 2025 Jan 11:S1569-1993(24)01852-6. doi: 10.1016/j.jcf.2024.12.001. Online ahead of print.

ABSTRACT

INTRODUCTION: The Lung Clearance Index (LCI) is an established research test, but its role in clinical decision-making is not well defined. This study estimated the proportion of treatment decisions that are changed or supported by the added information provided by LCI.

METHODS: A mixed methods prospective observational study was conducted in North America. Providers were invited to participate in a clinical vignette survey consisting of 10 hypothetical scenarios involving pediatric cystic fibrosis (CF) management. First, they made a clinical decision based on information captured in routine clinical visits. Then, the LCI value was made available, and providers were asked whether the LCI changed or supported their decision. A prospective study was also conducted at three CF centres to determine how often physicians make pulmonary treatment decisions at CF clinic visits and how often they perceive additional lung function data would be helpful for these decisions.

RESULTS: We received 522 vignette responses from 62 participants. LCI changed the decision in 18.4 % of cases, supported the decision in 57.1 % and did not impact the decision in 24.5 %. Data from patient encounters in the prospective study demonstrated that changes to pulmonary treatments were considered in 98/322 (30.4 %) visits; additional lung function information could potentially have helped in 64.3 % of the treatment decisions.

CONCLUSION: LCI changes or supports a significant proportion of treatment decisions. Providers perceive that additional information about lung function could be helpful at the majority of encounters where changes in treatment are considered.

PMID:39800642 | DOI:10.1016/j.jcf.2024.12.001

Dig Liver Dis. 2025 Jan 11:S1590-8658(24)01145-9. doi: 10.1016/j.dld.2024.12.020. Online ahead of print.

ABSTRACT

BACKGROUND: In pediatric patients, celiac disease (CD) may influence the health-related quality of life (HRQoL).

AIMS: The study aimed to assess HRQoL and further characterise the clinical factors associated with reduced HRQoL, in a large multicenter pediatric cohort with CD.

METHODS: The disease-specific questionnaire CD Dutch Questionnaire (CDDUX) and the generic questionnaire Paediatric Quality of Life Inventory (PedsQL) were used to assess the HRQoL. Clinical and sociodemographic characteristics were analyzed, univariate and multivariate analysis were conducted.

RESULTS: Eleven different Italian pediatric centers and 871 families were involved. Mean age at interview was 12.9 ± 2.9 years. The mean total CDDUX score of CD patients was 47.1 ± 18.8, revealing a neutral HRQoL (47.1 ± 18.8), and a good to very good HRQoL according to the PedsQL (81.4 ± 12.6), parents indicated lower scores (p = 0.03) with both questionnaires (CDDUX 45.1 ± 18.6 and PedsQL 79.9 ± 14.5). Patients with lower HRQoL were mainly female, living in Northern Italy, with lower parent's education level and non-biopsy diagnosis of CD. In multivariate analysis, the main predictor of lower CDDUX score was non-biopsy diagnosis.

CONCLUSIONS: The HRQoL in a large cohort of Italian children is reported as neutral-good. This indicates a high level of adaptive behaviors in response to the daily challenges of CD. Parents tend to underestimate their children's HRQoL. Specific clinical factors, including non-biopsy diagnosis, may be associated to lower HRQoL.

PMID:39800588 | DOI:10.1016/j.dld.2024.12.020

J Control Release. 2025 Jan 10:S0168-3659(25)00019-7. doi: 10.1016/j.jconrel.2025.01.017. Online ahead of print.

ABSTRACT

Aerosol delivery represents a rapid and non-invasive way to directly reach the lungs while escaping the hepatic first-pass effect. The development of pulmonary drugs for respiratory diseases such as cystic fibrosis, lung infections, pulmonary fibrosis or lung cancer requires an enhanced understanding of the relationships between the natural physiology of the respiratory system and the pathophysiology of these conditions. This knowledge is crucial to better predict and thereby control drug deposition. Moreover, aerosol administration faces several challenges, including the pulmonary tract, immune system, mucociliary clearance, the presence of fluid on the airway surfaces, and, in some cases, bacterial colonisation. Each of them directly influences on the bioavailability of the active molecule. In addition to these challenges, particle size and the device used to administer the treatment are critical factors that can significantly impact the biodistribution of the drugs. Nanoparticles are very promising in the development of new formulations for aerosol drug delivery, as they can be fine-tuned to reach the entire pulmonary tract and overcome the difficulties encountered along the way. However, to properly assess drug delivery, preclinical studies need to be more thorough to efficiently enhance drug delivery.

PMID:39800241 | DOI:10.1016/j.jconrel.2025.01.017

Glycoconj J. 2025 Jan 11. doi: 10.1007/s10719-024-10174-7. Online ahead of print.

ABSTRACT

Cystic Fibrosis (CF) is a life-threatening hereditary disease resulting from mutations in the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) gene that encodes a chloride channel essential for ion transport in epithelial cells. Mutations in CFTR, notably the prevalent F508del mutation, impair chloride transport, severely affecting the respiratory system and leading to recurrent infections. Recent therapeutic advancements include CFTR modulators such as ETI, a combination of two correctors (Elexacaftor and Tezacaftor) and a potentiator (Ivacaftor), that can improve CFTR function in patients with the F508del mutation. This study investigated ETI's impact on the maturation of the mutated CFTR, the expression levels of its scaffolding proteins, and lipid composition of cells using bronchial epithelial cell lines expressing both wild-type and F508del CFTR. Our findings revealed that ETI treatment enhances CFTR and its scaffolding proteins expression and aids in rescuing mature F508del CFTR, causing also significant alterations in the lipid profile including reduced levels of lactosylceramide and increased content of gangliosides GM1 and GD1a. These changes were linked to ETI's influence on enzymes involved in the sphingolipid metabolism, in particular GM3 synthase and sialidase. Through this work, we aim to deepen understanding CFTR interactions with lipids, and to elucidate the mechanisms of action of CFTR modulators. Our findings may support the development of potential therapeutic strategies contributing to the ongoing efforts to design effective correctors and potentiators for CF treatment.

PMID:39797966 | DOI:10.1007/s10719-024-10174-7

J Paediatr Child Health. 2025 Jan 11. doi: 10.1111/jpc.16773. Online ahead of print.

ABSTRACT

BACKGROUND: Children with cystic fibrosis are more likely to become severely unwell with influenza-associated illness compared to children without chronic lung disease. The provision of accessible influenza vaccinations is essential in the prevention of infection.

OBJECTIVES: To describe the prevalence of the influenza vaccine uptake in children with cystic fibrosis from 2016 to 2020 at a single tertiary paediatric hospital site and determine if the COVID pandemic of 2020 and the introduction of telehealth encounters affected the vaccine uptake.

METHODS: A retrospective study of children with cystic fibrosis aged 6 months to 18 years who reside in South Australia was performed using the Women's and Children's Hospital (WCH) Respiratory Department cystic fibrosis database from 2016 to 2020. The Australian Immunisation Register (AIR) was used to determine vaccination status during this period.

RESULTS: One hundred and eighty two children with cystic fibrosis were identified, one hundred and seventy two of whom vaccination records were available on the Australian Immunisation Register. Proportion of eligible patients who were vaccinated ranged from 66% to 88% in the years 2016-2019. There was no significant decrease in uptake during the COVID-19 pandemic in 2020 (75%). Despite the introduction of Telehealth reviews in 2020 majority (66%) of patients continued to have exclusive face-to-face appointments, of which 73% received the vaccination. Vaccination coverage of those who received combination of Telehealth and face-to-face was 81%.

CONCLUSION: The high influenza vaccination rate of South Australian children with cystic fibrosis is consistent with rates seen in other tertiary centres globally. This study demonstrated that the pandemic and introduction of Telehealth appointments did not have any impact in the uptake of the influenza vaccination in our South Australian population.

PMID:39797528 | DOI:10.1111/jpc.16773

Mol Ther. 2025 Jan 9:S1525-0016(25)00015-2. doi: 10.1016/j.ymthe.2025.01.011. Online ahead of print.

ABSTRACT

Cystic Fibrosis (CF) is a life-shortening autosomal recessive disease caused by mutations in the CFTR gene, resulting in functional impairment of the encoded ion channel. F508del mutation, a trinucleotide deletion, is the most frequent cause of CF affecting approximately 80% of persons with cystic fibrosis (pwCFs). Even though current pharmacological treatments alleviate the F508del-CF disease symptoms there is no definitive cure. Here we leveraged revertant mutations (RMs) in cis with F508del to rescue CFTR protein folding and restore its function. We developed CRISPR base editing strategies to efficiently and precisely introduce the desired mutations in the F508del locus. Both editing and CFTR function recovery were verified in CF cellular models including primary epithelial cells derived from pwCFs. The efficacy of the CFTR recovery strategy was validated in cultures of pseudostratified epithelia from pwCF cells showing full recovery of ion transport. Additionally, we observed an additive effect by combining our strategy with small molecules that enhance F508del activity, thus paving the way to combinatorial therapies.

PMID:39797401 | DOI:10.1016/j.ymthe.2025.01.011

Int J Mol Sci. 2025 Jan 6;26(1):425. doi: 10.3390/ijms26010425.

ABSTRACT

Granulomatous mastitis (GM) is a rare, benign, but chronic and recurrent inflammatory breast disease that significantly impacts physical and psychological well-being. It often presents symptoms such as pain, swelling, and discharge, leading to diagnostic confusion with malignancy. The etiology of GM remains unclear, though autoimmune and multifactorial components are suspected. This study aimed to explore the genetic underpinnings of GM using whole-exome sequencing (WES) on 22 GM patients and 52 healthy controls to identify single nucleotide variants (SNVs) and copy number variations (CNVs) potentially linked to the disease. WES analysis revealed novel SNVs in six genes: BRCA2 (rs169547), CFTR (rs4727853), NCF1 (rs10614), PTPN22 (rs2476601), HLA-DRB1 (seven variants), and C3 (rs406514). Notably, most of these variants are associated with immune regulation and inflammatory pathways, supporting the hypothesis that GM is an autoimmune disease. However, all identified variants were classified as benign according to the American College of Medical Genetics and Genomics (ACMG) guidelines, necessitating further investigation into their potential functional effects. Despite conducting CNV analysis, no significant variations were identified. This study represents a foundational step in linking genetic predisposition to GM and highlights the need for integrating genetic, clinical, and functional data to better understand GM's pathophysiology. Future research should focus on larger cohorts, functional studies, and exploring multifactorial contributors to GM, including hormonal and environmental factors.

PMID:39796280 | DOI:10.3390/ijms26010425

Diagnostics (Basel). 2025 Jan 4;15(1):107. doi: 10.3390/diagnostics15010107.

ABSTRACT

Objectives: To determine the association between airflow limitation and the quantification of lung attenuation in computed tomography (CT) in adult patients with cystic fibrosis (CF). Methods: A cross-sectional study in a single center between January 2013 and December 2018 in adult patients with stable CF. We collected clinical data and the results of spirometry and plethysmography. A chest CT at inspiration and expiration, using a specific software that automatically measured the lung attenuation, was performed. Results: In total, 73 patients (63% males) were included. The mean age was 31.6 ± 12.3 years and the FEV1 was 67.8 ± 25.9% pred. An airflow limitation was found in 63%, the mean residual volume was 159.9% pred, and air trapping was observed in 50 (87.7%) of the patients. The patients with airflow limitations showed a higher bulla index and a percentage of lung voxels in the range of emphysema. The FEV1 and the FEV1/FVC correlated with the percentage of the lungs at a high attenuation value (HAV), the range of emphysema, and the bulla index at inspiration, as well as the mean lung density at expiration and the inspiratory-expiratory variation of the mean lung density (MLDi-e). Finally, in the multivariate model, the MLDi-e and the HAV at inspiration were associated with airflow limitations. Conclusions: The measurements obtained from the automated quantification of lung parenchymal attenuation predicts airflow limitation in CF.

PMID:39795635 | DOI:10.3390/diagnostics15010107

J Cyst Fibros. 2025 Jan 9:S1569-1993(24)01857-5. doi: 10.1016/j.jcf.2024.12.003. Online ahead of print.

NO ABSTRACT

PMID:39794183 | DOI:10.1016/j.jcf.2024.12.003

J Cyst Fibros. 2025 Jan 9:S1569-1993(24)01855-1. doi: 10.1016/j.jcf.2024.12.005. Online ahead of print.

ABSTRACT

BACKGROUND: Food insecurity (FI) is more prevalent in people with cystic fibrosis (PwCF) than the reported national prevalence, but there are limited data on the relationship between FI and health outcomes in PwCF. The objective of this study was to analyze the relationship between FI in PwCF and pulmonary and nutritional status.

METHODS: We leveraged an electronic cross-sectional survey that ascertained FI status and gave participants the option to link their survey data to their records in the Cystic Fibrosis Foundation Patient Registry (CFFPR). Linear regression and negative binomial models were used to estimate the associations in mean differences between FI and percent predicted FEV1 (ppFEV1), nutritional indices, and hospitalizations.

RESULTS: There were 1,856 respondents, 1,234 (66.5 %) of whom granted permission to link to the CFFPR. FI was present in 352 (28 %) of the respondents. FI was associated with lower ppFEV1 (-6.5; 95 % CI -9.9, -3.1); however, this was no longer statistically significant after adjusting for confounders. FI was independently associated with increased hospitalizations. Higher weight for age was significantly associated with FI in the adjusted model, but there were no significant associations between height for age or absolute weight and body mass index (BMI) in adults.

CONCLUSIONS: FI in PwCF is associated with adverse health outcomes. These results support screening for FI during routine visits. Further studies are needed to investigate causal relationships between FI and adverse clinical outcomes.

PMID:39794182 | DOI:10.1016/j.jcf.2024.12.005