Sci Rep. 2025 May 6;15(1):15789. doi: 10.1038/s41598-025-94171-2.

ABSTRACT

To translate, cross-culturally adapt, and evaluate the psychometric properties of the Cystic Fibrosis (CF) Stigma Scale. This exploratory methodological study involved the translation and cross-cultural adaptation using the translation, back-translation, review by experts committee, and pre-test steps. The psychometric properties were analyzed by applying the adapted instrument to a sample of 52 Brazilian individuals with CF over 18 years old. Moreover, the participants responded to the Short-Form 12-Item Survey - version 2 (SF-12v2), General Anxiety Disorder 7-item scale (GAD-7) and Cystic Fibrosis Quality of Life Questionnaire - Revised (CFQ-R). The content validity, test-retest reliability, and convergent validity were also assessed. The translation and cross-cultural adaptation obtained Cohen's kappa coefficients > 0.61 in the experts committee step and ranged between 0.48 and 0.72 in the pre-test. The Brazilian version of the CF Stigma Scale showed excellent psychometric properties, observed by the internal consistency (α = 0.836), mean correlation between items (0.3) and test-retest reliability (r = 0.886; p < 0.0001), and convergent validity (positive correlation with the anxiety scale and negative correlation with scores of overall and specific quality of life for CF). The Brazilian version of the CF Stigma Scale was accurately translated and cross-culturally adapted, with favorable psychometric properties for future studies involving the stigma experience in Brazilian individuals with CF over 18 years.

PMID:40328878 | DOI:10.1038/s41598-025-94171-2

J Cyst Fibros. 2025 May 5:S1569-1993(25)01463-8. doi: 10.1016/j.jcf.2025.04.008. Online ahead of print.

ABSTRACT

The introduction of cystic fibrosis transmembrane conductance regulator (CFTR) modulators has changed the landscape of therapy for persons with cystic fibrosis. However, the steep cost of targeted therapy poses significant financial burden for individuals and health systems. We aimed to determine the trends in Medicare and Medicaid spending on CFTR modulators between the years 2015 and 2022 through retrospective analysis of the Medicare and Medicaid claims data. The outcome measures included total dosage units prescribed, number of claims, spending per claim, and total spending on CFTR modulators for Medicare and Medicaid between 2015 to 2022. Average annual percentage changes (AAPC) were calculated for all outcome measures. Our results show that from 2015 to 2022, Medicaid consistently had higher total dosage units prescribed, number of claims, spending per claim, and overall spending on CFTR modulators compared to Medicare. Total spending for both Medicaid [AAPC 38.9, 95 % confidence interval [CI] 27.2-51.6, p < 0.01] and Medicare [AAPC 39.2, 95 % CI 30.2-55.1, p < 0.01] increased significantly during this period. Increases in CFTR spending was accelerated beginning in 2019, when the triple combination CFTR modulator therapy, elexacaftor/tezacaftor/ivacaftor, was introduced to the US market. This increase has been accompanied by a reduction in spending and use of other CFTR agents. This study evaluated the increases in spending for CFTR modulators over the years and the main drivers behind them, which may help inform future negotiations between healthcare systems and pharmaceutical companies, as well as policy makers and stakeholders.

PMID:40328584 | DOI:10.1016/j.jcf.2025.04.008

Klin Padiatr. 2025 May 6. doi: 10.1055/a-2551-2560. Online ahead of print.

NO ABSTRACT

PMID:40328282 | DOI:10.1055/a-2551-2560

J Patient Rep Outcomes. 2025 May 6;9(1):48. doi: 10.1186/s41687-025-00879-0.

ABSTRACT

BACKGROUND: Elexacaftor/Tezacaftor/Ivacaftor (ETI) is a therapy approved for cystic fibrosis (CF) that has given improved clinical outcomes in patients carrying the F508del mutation. There are few published data regarding ETI's effects on patients' quality of life (QoL). This study aims to (fill the data gap in current literature by assessing) evaluate the long-term effects of ETI on QoL.

METHODOLOGY: A prospective observational study was conducted with thirty-seven severe patients that received ETI for compassionate use (group A), 184 received it for on-label use (group B). All carried one F508del mutation. Patients were assessed using the CFQ-R (Cystic Fibrosis Questionnaire-Revised). The evaluation time-points were pre-treatment (T0), and after 12 (T1) and 24 months (T2); group A was also assessed after 36 months (T3). Twenty-five patients completed 3 years of treatment and 65 patients completed 2 years of treatment, in groups A and B respectively.

RESULTS: At T1, median values for almost all areas of CFQ-R statistically significant increased in group A, particularly Physical Functioning (+ 25.0), Respiratory (+ 22.2) and Health Perception (+ 22.2).The Social Functioning area statistically significant increased at T2 (+ 5.6). At T3, these improvements remained stable. At T1, all areas of CFQ-R statistically significant increased in group B, particularly the Health Perception (+ 22,2) heading. At T2, these improvements remained stable. For both groups, the changes identified at the last follow-up showed no major differences by gender, age or genetic status.

CONCLUSIONS: Treatment with ETI significantly improved patients' QoL in both groups at 12-24 months, these improvements remaining stable in patients tested at 36 months.

PMID:40327240 | DOI:10.1186/s41687-025-00879-0

Allergy. 2025 May 6. doi: 10.1111/all.16581. Online ahead of print.

NO ABSTRACT

PMID:40326788 | DOI:10.1111/all.16581

Pediatr Pulmonol. 2025 May;60(5):e71110. doi: 10.1002/ppul.71110.

ABSTRACT

BACKGROUND: There have been significant improvements in the health of infants and children with cystic fibrosis (CF) since universal newborn screening (NBS) was implemented in the United States (US). However, a significant proportion of infants with CF are not evaluated in a timely manner, and delays disproportionately affect children from minoritized racial/ethnic groups. The aim of this study was to understand experiences of NBS in caregivers of young children with CF in the United States.

METHODS: We recruited caregivers of children (0-13 years) with CF through listservs and social media of CF organizations. The survey was administered online in 2023 and included questions about their recollections of their child's NBS and the process of getting a CF diagnosis.

RESULTS: Of 383 caregiver respondents, 43% reported being informed that their child's race or ethnicity was a predictor of the chances of their child having CF. Most reported that after they were notified of a positive NBS test, the initial evaluation for CF was scheduled ≥ 4 days later, 45% reported a delay of ≥ 8 days, and 5% reporting a delay of ≥ 15 days. Most (91%) felt the initial evaluation for CF was thorough, but 35% reported delays in getting information about their child's diagnosis.

CONCLUSIONS: Caregivers report delays in evaluation after a positive NBS. A significant proportion reported delays in receiving information about their child's diagnosis or being told that race or ethnicity were related to risk of CF. These findings show the need for education and practice changes in both primary care and CF center settings.

PMID:40326644 | DOI:10.1002/ppul.71110

Acta Physiol (Oxf). 2025 Jun;241(6):e70051. doi: 10.1111/apha.70051.

ABSTRACT

AIM: Amino acids, sugars, short-chain fatty acids (SCFA), vitamins, and other small molecules compose the extracellular metabolome on the airway lumen surface, but how the airway epithelium deals with these molecules has not been deeply studied. Due to the broad spectrum of metabolites transported by SLC5A8 and SLC5A12, we aim to determine if they are functionally expressed and participate in the absorption of Na+, short-chain fatty acids, and monocarboxylates in mouse and human airway epithelium.

METHODS: Tracheas isolated from male or female mice and human bronchial epithelial cells (HBECs) were used for electrophysiological studies in the Ussing chamber and to detect members of the SLC16 family by RT-PCR and bulk RNAseq. Additionally, cell lines expressing the human and murine SLC5A8 transporter were employed for uptake studies using a fluorescent lactate probe.

RESULTS: We showed for the first time that human and murine airway epithelium express a functional SLC5A8 transporter, facilitating the absorption of glucose metabolites and SCFAs. The Na+-coupled monocarboxylate transport was not additive with ENaC-mediated Na+ absorption in mouse trachea. We observed that valproate acts as an inhibitor of the murine but not of the human SLC5A8 transporter.

CONCLUSIONS: Our results demonstrate that several metabolites derived from bacterial and cellular metabolism can be transported from the airway lumen into the epithelial cells, participating in a homeostatic relation of the tissue with its environment.

PMID:40326639 | DOI:10.1111/apha.70051

Pediatr Pulmonol. 2025 May;60(5):e71117. doi: 10.1002/ppul.71117.

ABSTRACT

BACKGROUND: Previous research showed that lung function abnormalities are common in infants with cystic fibrosis (IwCF) but real-world data are missing.

METHODS: This single-center retrospective study analyzed infant lung function results from IwCF born in 2012-2018. The tests were conducted at Great Ormond Street Hospital, London, as part of routine care at 3 months, 1 year, and 2 years of age. Z-scores for SF6 Lung Clearance Index (zLCI), plethysmographic FRC (zFRCpleth) and FEV0.5 were derived. Microbiology and antibiotics prescription from 3 months before lung function assessments, up to the closest medical review following the lung function encounter, were analyzed, along with changes in management advised by the physician.

RESULTS: A total of 126 lung function encounters (n = 43 at 3 months, 46 at 1 year, 37 at 2 years) from 60 IwCF were included. LCI was abnormal (zLCI > 1.96) in 31% (12/39) of 3-month-olds (mean± zLCI 1.21 ± 1.08), 28% (12/43) of 1-year-olds and 19% (7/36) of 2-year-olds (mean± zLCI 1.13 ± 1.10). Among 74 cases with recent positive microbiology or abnormal chest findings at medical review, 100% (31/31) of those with abnormal lung function and 86% (37/43) of those with normal lung function (p = 0.04) had a recent antibiotic prescription or a change in clinical management. Conversely, in encounters with abnormal lung function but normal clinical findings, management changes occurred in only 12% (2/16) of cases.

CONCLUSION: In this real-word cohort of IwCF, clinical management was mainly influenced by clinical findings and only marginally by abnormal lung function (elevated FRC or LCI).

PMID:40326637 | DOI:10.1002/ppul.71117

Pediatr Pulmonol. 2025 May;60(5):e71102. doi: 10.1002/ppul.71102.

ABSTRACT

BACKGROUND: Cystic fibrosis (CF) is a chronic genetic disorder requiring regimented visits for maintenance of care. The COVID-19 pandemic accelerated the accessibility of telehealth (TH) and forced a trial of incorporating remote care into routine CF care. The CF Learning Network (CFLN) organized for data sharing into a telehealth innovation lab (TH-iLab) to improve access to the interdisciplinary care team and co-produced shared agenda-setting.

METHODS: All persons with CF (PwCF) with a CF diagnosis in the CF Foundation Registry (CFFPR) from 1/2020-12/2021 were included and categorized into CFLN TH-iLab, CFLN TH-iLab non-participants, and non-CFLN programs.

HYPOTHESIS: standardized TH implementation in the CFLN TH-iLab is associated with increased access to the CF care model and results in similar lung function and nutrition health outcomes.

RESULTS: In 2020 and 2021, the average number of TH visits per person per year and the percentage of PwCF with one or more TH visits per year were higher in the CFLN TH-iLab than in the other groups. Lung function was highest in PwCF, followed by a program that was part of the CFLN TH-iLab in 2020 and 2021. Anthropometric measurements, spirometry, and attainment of microbiology cultures were similar among all three groups. Access to interdisciplinary care was highest in the CFLN non-TH-iLab group.

CONCLUSION: Integrating TH into CF care in the CFLN TH-iLab provided access to care during the COVID-19 pandemic without compromising clinical outcomes. Further research on optimizing the telehealth experience for PwCF can help better understand TH's long-term impact on CF care.

PMID:40325945 | DOI:10.1002/ppul.71102

Pediatr Pulmonol. 2025 May;60(5):e71101. doi: 10.1002/ppul.71101.

ABSTRACT

BACKGROUND: Cystic fibrosis (CF) is a genetic disorder that necessitates high-calorie, protein-rich diets, leading to nutritional deficiencies. Food insecurity (FI) poses a significant challenge for people with CF (pwCF), impacting their ability to maintain the necessary dietary intake. This study aims to explore FI and dietary patterns among pwCF in Turkey.

METHODS: A cross-sectional study involving 290 pwCF from the Marmara University Selim Çöremen Cystic Fibrosis Center was conducted between April 2023 and February 2024. The "US Household Food Security Survey Module" and the "Your Current Life Situation" survey were used to assess FI and socioeconomic status among the participants. Nutritional data, including BMI, FEV1 values, and dietary intake, were recorded.

RESULTS: Among the participants, 52.7% were female, with a mean age of 13.3 ± 8.1 years. FI was detected in 46.8% of pwCF, with 18% facing very low food security. Higher income levels were associated with better food security (p = 0.008). Nutritional inadequacies were observed even among food-secure individuals, particularly in the consumption of legumes, nuts, and fish. BMI and BMI percentile values were significantly lower in the very low FS group compared to the high FS group (p = 0.03 and p = 0.02, respectively).

CONCLUSION: Ensuring adequate nutrition and calorie intake is crucial for pwCF. Our study highlights significant FI among pwCF in Turkey, with income levels influencing food security status. Nutritional inadequacies persist even among those classified as food secure. Based on these findings, targeted nutritional support will be provided to those in need to improve overall health and well-being.

PMID:40325925 | DOI:10.1002/ppul.71101

PLoS One. 2025 May 5;20(5):e0321996. doi: 10.1371/journal.pone.0321996. eCollection 2025.

ABSTRACT

Cystic fibrosis (CF) is the most common genetic diseases in the Caucasian population. CFTR defects, the most common being F508del, lead to abnormal mucus accumulation. Respiratory failure caused by the resulting chronic infections is the leading cause of death in people with cystic fibrosis (pwCF). Pseudomonas aeruginosa is a major pathogen in CF and is responsible for a deterioration of respiratory function in pwCF. The increase of antibiotic-resistant P. aeruginosa strains encourages the search for alternative therapeutics for treating P. aeruginosa infection. In vitro studies have shown an interest in (R)-roscovitine (roscovitine) in the fight against bacterial infection in pwCF. Here we show a nuanced effect of roscovitine on ASL bactericidal activity and CF bronchial epithelium protection against P. aeruginosa. Using a 3D model of fully differentiated and functional F508del-CFTR human bronchial epithelium, we evidenced (i) an enhancement of the bactericidal activity of the airway surface liquid for 25 μM roscovitine but (ii) no limitation of the dynamic of the epithelium destruction upon roscovitine treatment whatever the concentrations. Our findings shed light on reasons for the lack of beneficial effects to prevent P. aeruginosa infection in pwCF treated with roscovitine in the ROSCO-CF clinical trial. We anticipate that our findings would have significant therapeutic implications in seeking to optimize roscovitine analogs.

PMID:40323902 | DOI:10.1371/journal.pone.0321996

J Vis Exp. 2025 Apr 18;(218). doi: 10.3791/67477.

ABSTRACT

Standard pre-clinical testing methods for novel antimicrobial therapeutics used to treat chronic lung infections in people with cystic fibrosis do not reflect the environmental conditions of the hostile lung niche. Current reductionist testing conditions can lead to the progression of compounds along a preclinical pipeline without evidence of their activity under cystic fibrosis lung niche-appropriate conditions. Several approaches used to study traditional antimicrobials may not be suitable for antibiotic alternatives, including anti-virulence therapeutics like anti-quorum sensing agents and siderophore inhibitors. This protocol documents an aggregate biofilm model of Pseudomonas aeruginosa to compare resistance and infection-relevant gene expression in single-species and multi-species cultures (Staphylococcus aureus and Candida albicans), examining colony-forming unit (CFU) reductions and changes in gene expression, using algD as an exemplar. The model was optimized for small, static volumes of bacterial cultures to allow the study of novel compounds in the discovery phase of the drug development pipeline, where compound quantities may be limited. Single-species P. aeruginosa biofilms were formed in Synthetic Cystic Fibrosis Medium 2 (SCFM2) for 24 h before treatment with meropenem at different concentrations (1, 16, and 256 µg/mL) for a further 24 h. Polymicrobial biofilms were established by growing Staphylococcus aureus and Candida albicans together in SCFM2, then inoculating with P. aeruginosa for an additional 24 h and treating with meropenem. The lack of a direct connection between compound efficacy measures in pre-clinical testing and clinical trial results has cast doubt on the applicability of current laboratory screening tools. This model allows us to understand the impact of relevant factors on P. aeruginosa gene expression, including genes contributing to resistance and virulence, thereby bridging this gap.

PMID:40323888 | DOI:10.3791/67477

Clin Pharmacol Ther. 2025 May 5. doi: 10.1002/cpt.3705. Online ahead of print.

ABSTRACT

The use of elexacaftor/tezacaftor/ivacaftor (ETI) has been associated with increased fertility in women with cystic fibrosis (CF) and is increasingly used during pregnancy to support both maternal and fetal health. However, little is known about the pharmacokinetics (PK) of ETI during pregnancy, which is crucial for optimizing its efficacy and safety. This study aimed to predict the PK of ETI during pregnancy and to determine the maternal dose required to achieve therapeutic concentrations in both the maternal and fetus. The pregnancy physiological-based pharmacokinetic (PBPK) model within the Simcyp Simulator was used to predict the maternal and feto-placental exposure of ETI. Placental kinetics were parameterized using permeability parameters determined from the physicochemical properties of these compounds. The model closely predicted the observed data, with the observed ETI maternal plasma concentrations, cord concentrations, and infant plasma concentrations mostly falling within the range of predicted 5th to 95th percentiles. Steady-state simulations up to gestational week 40 predicted a continuous decline in ETI concentrations, with the AUC declining to 32.4-37.5% of baseline levels by week 40. However, the 5th percentile of trough concentrations for ETI consistently remained above the efficacy thresholds, both in mother and fetus. Therefore, it appears reasonable to maintain standard dosing regimen during pregnancy, complemented by careful monitoring. A clinical trial, such as the ongoing Maternal and Fetal Outcomes in the Era of Modulators (MAYFLOWERS) study, is required to further confirm the efficacy and safety of ETI in this population.

PMID:40323155 | DOI:10.1002/cpt.3705

Biomicrofluidics. 2025 May 1;19(3):034101. doi: 10.1063/5.0255847. eCollection 2025 May.

ABSTRACT

Remote health monitoring has the potential to enable individuals to take control of their own health and well-being and to facilitate a transition toward preventative and personalized healthcare. Sweat can be sampled non-invasively and contains a wealth of information about the metabolic state of an individual, making it an excellent candidate for remote health monitoring. An accurate, rapid, and low-cost biofluid characterization technique is required to enable the widespread use of remote health monitoring. We previously introduced microfluidic impedance spectroscopy for the detection of electrolyte concentration in fluids, whereby a novel device architecture, measurement method, and analysis technique were presented for the characterization of cationic species. The purely electrical nature of this measurement technique removes the intermediate steps inherent in common rival technologies such as optical and electrochemical sensing, offering a range of advantages. In this work, we investigate the effect of temperature on microfluidic impedance spectroscopy of ionic species commonly present in biofluids. We find that the impedance spectra and concentration determination are temperature-dependent; remote health monitoring devices must be calibrated appropriately as they are likely to experience temperature fluctuations. Importantly, we demonstrate the ability of the method to measure the concentration of anionic species alongside that of cationic species, enabling the detection of chloride and lactate, which are useful biomarkers for hydration, cystic fibrosis, fatigue, sepsis, and hypoperfusion. We show that the presence of neutral species does not impair accurate determination of ionic concentration, thus, demonstrating the suitability of microfluidic impedance spectroscopy for non-invasive biofluid characterization.

PMID:40322639 | PMC:PMC12048175 | DOI:10.1063/5.0255847

Open Respir Med J. 2025 Feb 12;19:e18743064341009. doi: 10.2174/0118743064341009241210045737. eCollection 2025.

ABSTRACT

Despite medical science advancements in recent years, pulmonary diseases are still hard to control and can be potentially life-threatening. These include asthma, COPD, lung cancer, cystic fibrosis, pneumonia, pleurisy, and sarcoidosis. These illnesses often cause severe breathing problems, which can be fatal if not treated properly. While some chemical drugs are used to treat these conditions, they can cause side effects and are not always effective. Herbal medicine offers an alternative treatment option with fewer side effects and has shown promise in treating respiratory issues. Certain medicinal plants, such as garlic (Allium sativum), hawthorn (Crataegus rhipidophylla), moringa (Moringa oleifera), and ashwagandha (Withania somnifera), may help manage lung diseases. Natural compounds found in plants, like apple polyphenol, ligustrazine, salidroside, resveratrol, and quercetin, can also help reduce symptoms. These plants and compounds work by reducing cell overgrowth, fighting oxidative stress, lowering inflammation, stopping tumor growth, improving blood flow, and relaxing the airways. This review outlines the types of plants and compounds that can be utilized in treating pulmonary conditions, along with their respective mechanisms of action.

PMID:40322495 | PMC:PMC12046236 | DOI:10.2174/0118743064341009241210045737

Mol Cell Pediatr. 2025 May 5;12(1):6. doi: 10.1186/s40348-025-00194-0.

ABSTRACT

BACKGROUND: Cystic fibrosis (CF) is a systemic disorder of exocrine glands that is caused by mutations in the CFTR gene.

MAIN BODY: The basic defect in people with CF (pwCF) leads to impaired epithelial transport of chloride and bicarbonate that can be assessed by CFTR biomarkers, i.e. the β-adrenergic sweat rate and sweat chloride concentration (SCC), chloride conductance of the nasal respiratory epithelium (NPD), urine secretion of bicarbonate, intestinal current measurements (ICM) of chloride secretory responses in rectal biopsies and in bioassays of chloride transport in organoids or cell cultures. CFTR modulators are a novel class of drugs that improve defective posttranslational processing, trafficking and function of mutant CFTR. By April 2025, triple combination therapy with the CFTR potentiator ivacaftor (IVA) and the CFTR correctors elexacaftor (ELX) and tezacaftor (TEZ) has been approved in Europe for the treatment of all pwCF who do not carry two minimal function CFTR mutations. Previous phase 3 and post-approval phase 4 studies in pwCF who harbour one or two alleles of the major mutation F508del consistently reported significant improvements of lung function and anthropometry upon initiation of ELX/TEZ/IVA compared to baseline. Normalization of SCC, NPD and ICM correlated with clinical outcomes on the population level, but the restoration of CFTR function was diverse and not predictive for clinical outcome in the individual patient. Theratyping of non-F508del CF genotypes in patient-derived organoids and cell cultures revealed for most cases clinically meaningful increases of CFTR activity upon exposure to ELX/TEZ/IVA. Likewise, every second CF patient with non-F508del genotypes improved in SCC and clinical outcome upon exposure to ELX/TEZ/IVA indicating that triple CFTR modulator therapy is potentially beneficial for all pwCF who do not carry two minimal function CFTR mutations. This group who is not eligible for CFTR modulators may opt for gene addition therapy in the future, as the first-in-human trial with a recombinant lentiviral vector is underway.

FUTURE DIRECTIONS: The upcoming generation of pwCF will probably experience a rather normal life in childhood and adolescence. To classify the upcoming personal signatures of CF disease in the times of efficient modulators, we need more sensitive CFTR biomarkers that address the long-term course of airway and gut microbiome, host defense, epithelial homeostasis and multiorgan metabolism.

PMID:40320452 | DOI:10.1186/s40348-025-00194-0

J Cyst Fibros. 2025 May 3:S1569-1993(25)01465-1. doi: 10.1016/j.jcf.2025.04.010. Online ahead of print.

ABSTRACT

BACKGROUND: Despite widespread availability of modulator therapies and improved lung function in many people with cystic fibrosis (CF), physical symptoms may remain burdensome for some people with CF (PwCF). This study identifies the impact of ivacaftor (IVA) and elexacaftor/tezacaftor/ivacaftor (ETI) on self-reported physical well-being and burden of care among adults with CF.

METHODS: We conducted a secondary analysis of data from the Well-ME Survey. Participants included adults with CF (age≥18) who reported taking IVA or ETI. We used a mixed methods approach to identify self-reported health status, physical well-being, and experience of CF care while taking IVA or ETI.

RESULTS: Among 414 eligible respondents, overall health status was reported very good/excellent by 59 % (n = 243), good by 26 % (n = 114), and poor/fair by 14 % (n = 57). While the majority of respondents experienced improvements in respiratory symptoms, PwCF reporting poor/fair health were less likely to report improvement in overall physical health, fatigue, and ability to exercise compared to those with good or very good/excellent health and less likely to report improvement in pain, sinus issues, and cough than those with very good/excellent health. PwCF reporting poor/fair health or good health were less likely to report improvements in gastrointestinal issues or experience reductions in CF medications or treatments, compared to those reporting very good/excellent health.

CONCLUSIONS: Despite improvements in respiratory symptoms, some adults with CF taking IVA or ETI report their health is poor/fair. A better understanding of physical well-being and burden of care may help identify underrecognized comorbidities to improve care.

PMID:40320360 | DOI:10.1016/j.jcf.2025.04.010

Clin Ther. 2025 May 2:S0149-2918(25)00121-3. doi: 10.1016/j.clinthera.2025.04.002. Online ahead of print.

NO ABSTRACT

PMID:40318987 | DOI:10.1016/j.clinthera.2025.04.002

Pathology. 2025 Mar 26:S0031-3025(25)00123-0. doi: 10.1016/j.pathol.2025.02.004. Online ahead of print.

NO ABSTRACT

PMID:40318963 | DOI:10.1016/j.pathol.2025.02.004

BMC Pulm Med. 2025 May 2;25(1):212. doi: 10.1186/s12890-025-03621-0.

ABSTRACT

Cystic fibrosis (CF) is a genetic disorder characterized by progressive lung disease and extra-pulmonary manifestations with notable sex disparities in disease outcomes. In this review we summarize the underlying mechanisms driving this sex disparity, with a particular focus on the role of sex hormones on CF lung disease pathophysiology. We explore how the introduction of highly effective modulator therapies (HEMT) may impact sex differences in outcomes and assess whether they have the potential to close the sex gap. While treatment with HEMT has led to better outcomes in the CF population as a whole, females with CF continue to experience worse pulmonary morbidity than males. There is a need for continued research in this area, particularly into the influence and therapeutic potential of sex hormones.

PMID:40316939 | DOI:10.1186/s12890-025-03621-0