BACKGROUND: Disease modifying therapies for progressive fibrotic interstitial lung diseases (F-ILDs) slow physiological decline but have not consistently shown to improve patient-reported symptoms or health-related quali...BACKGROUND: Disease modifying therapies for progressive fibrotic interstitial lung diseases (F-ILDs) slow physiological decline but have not consistently shown to improve patient-reported symptoms or health-related quality of life. Patients with F-ILD experience a substantial symptom burden that necessitates comprehensive supportive care alongside antifibrotic treatment. This review summarises the current evidence for management strategies for F-ILD to be considered in conjunction with disease-modifying therapies. SYMPTOM MANAGEMENT: Dyspnoea, chronic cough, fatigue, anxiety and depression are highly prevalent in F-ILD and significantly impair daily functioning. Dyspnoea management includes non-pharmacological interventions, personalised self-management strategies and selective use of low dose opioids in advanced disease though overall benefit may be modest. Chronic cough may be addressed through behavioural and speech therapy, treatment of contributing comorbidities such as gastro-oesophageal reflux disease and antitussive therapies, including neuromodulators and low dose opioids for refractory symptoms. Fatigue and psychological distress require routine screening, evaluation of modifiable factors and targeted interventions. OXYGEN THERAPY: Oxygen therapy remains a standard of care for patients with resting or exertional hypoxaemia. While it improves oxygenation, its effect on dyspnoea and symptoms is variable, underscoring the importance of individualised assessment and patient-centred decision making. PULMONARY REHABILITATION: Pulmonary rehabilitation provides evidence-based improvements in exercise capacity, symptom burden and health-related quality of life. It also offers important psychological benefits and should be integrated early and maintained as feasible throughout the disease course. PALLIATIVE CARE: Given the unpredictable trajectory of F-ILD, early integration of palliative care is essential. Discussions regarding prognosis, advance care planning and end of life preferences, supported by a multidisciplinary care model, enable holistic, goal concordant care across the progressive disease trajectory. A multidisciplinary care model is beneficial for providing individualised, holistic care throughout the patient's progressive disease trajectory.
BACKGROUND: In most chronic airway diseases, identifying phenotypes has advanced clinical practice and research. However, no such phenotypes exist for paediatric bronchiectasis. We therefore sought to develop robust paed...BACKGROUND: In most chronic airway diseases, identifying phenotypes has advanced clinical practice and research. However, no such phenotypes exist for paediatric bronchiectasis. We therefore sought to develop robust paediatric bronchiectasis phenotypes and to investigate whether they were associated with relevant clinical outcomes. METHODS: Latent class analysis was independently applied to two study cohorts of children with bronchiectasis. Data from 158 children from one study (BEST, development cohort) were used to identify phenotypes and their associations with relevant clinical outcomes. A cohort of 198 children from a second study (BAMP) was used to validate these phenotypes and compare findings for consistency. RESULTS: A three-class model was the best fit in the BEST cohort. Based on clinical characteristics, we labelled the phenotypes as 'Baseline-Well', 'Wheeze-Dyspnoea Predominant' and 'Daily Wet-Cough Predominant'. These phenotypes were validated in the BAMP cohort. In both development and validation cohorts, the 'Daily Wet-Cough Predominant' phenotype was associated with higher numbers of bronchiectatic lobes (OR 1.61, 95% CI 0.61 to 4.27; and 3.06, 95% CI 1.30 to 7.21 for BEST and BAMP cohorts, respectively) and more bronchiectasis-related hospitalisations ever at enrolment (incidence rate ratio (IRR) 4.33, 95% CI 1.94 to 9.66; and 2.96, 95% CI 1.07 to 8.20, respectively) and also within 2 years of enrolment (IRR 2.68, 95% CI 1.24 to 5.8; and 1.54, 95% CI 0.72 to 3.28, respectively) than the reference phenotype, although the strengths of evidential support differed. CONCLUSIONS: We identified and validated three novel paediatric bronchiectasis phenotypes, and linked them to distinct clinical outcomes. These phenotypes might enable targeted interventions and improve participant selection for clinical trials.
BACKGROUND: Asthma represents a major public health concern in childhood and adolescence. However, population-based estimates of trends in its epidemiology in Spain are limited. METHODS: We conducted a population-based c...BACKGROUND: Asthma represents a major public health concern in childhood and adolescence. However, population-based estimates of trends in its epidemiology in Spain are limited. METHODS: We conducted a population-based cohort study using primary care electronic health records in Catalonia, Spain, from 2010 to 2024. Asthma diagnoses, ascertained through Systematized Nomenclature of Medicine (SNOMED) codes, were leveraged to estimate annual incidence rates (IR) and prevalence among children aged 1-17 years. Results were stratified by sex, age and Socioeconomic Deprivation Index (SDI). Incidence rate ratios (IRR) were estimated to investigate differences in overall incidence of asthma in these sociodemographic groups. Joinpoint modelling was used to investigate time trends in incidence. RESULTS: We included 2 001 625 individuals (48.40% females, median age 7 years). Crude IR of asthma (2010-2024) was 687.46 (95% CI 683.09 to 691.85) per 100 000 person-years. Incidence was highest in males (IRR=1.35, 95% CI 1.33 to 1.37, vs females) until adolescence, after which the pattern reversed, youngest individuals (IRR=3.03 95% CI 2.96 to 3.11, for 1-4 versus 15-17) and in individuals living in the most deprived areas (IRR=1.14, 95% CI 1.11 to 1.16, vs least deprived SDI quintile). Incidence declined before the COVID-19 pandemic, dropped sharply during it, and then increased among children aged 5-14 years post-pandemic, while consistently decreased in children aged 1-4 years. Asthma prevalence showed an upward trend until 2019 followed by stabilisation until 2024, when its prevalence was 7.36% (7.3%-7.41%). CONCLUSION: Asthma remains a major public health burden in children and adolescents, with incidence and prevalence varying by age, sex and socioeconomic status, making continuous monitoring essential to guide prevention.
Lung volume reduction procedures are an established evidence-based aspect of chronic obstructive pulmonary disease (COPD) care. We conducted a research prioritisation exercise involving people with COPD and healthcare pr...Lung volume reduction procedures are an established evidence-based aspect of chronic obstructive pulmonary disease (COPD) care. We conducted a research prioritisation exercise involving people with COPD and healthcare professionals across a range of disciplines, to identify a clear set of 10 questions to guide the development of research proposals in this area. Priorities were identified using an iterative approach based on the James Lind Alliance methodology. The final set of 10 priorities address the identification, assessment and optimisation of patients with COPD prior to any procedure, how lung volume reduction procedures are conducted and how care after the procedure has taken place should be organised.
INTRODUCTION: Congenital central hypoventilation syndrome (CCHS) is a rare disorder characterised by autonomic dysfunction and impaired respiratory control. While advances in ventilatory support have improved survival, r...INTRODUCTION: Congenital central hypoventilation syndrome (CCHS) is a rare disorder characterised by autonomic dysfunction and impaired respiratory control. While advances in ventilatory support have improved survival, risk factors for mortality remain incompletely understood. Our objective was to investigate the impact of genetic and clinical parameters on mortality in children and young adults with genetically confirmed CCHS. METHODS: Data were collected from the European CCHS Consortium registry, encompassing patient information collected from 2012 to 2021. This dataset included information for 240 patients with genetically confirmed CCHS. Complete outcome data were available for 211 patients. Survival outcomes were analysed using Kaplan-Meier curves and Cox proportional hazards regression models. RESULTS: The overall mortality rate was 14% (95% CI 9% to 19%), with most deaths occurring in early childhood. The 25-year survival probability was 89% (95% CI 79% to 100%) for children and young adults with isolated CCHS, compared with 26% (95% CI 6% to 100%) for those with CCHS and Hirschsprung disease (HSCR), corresponding to a 6.8-fold increased risk of mortality (95% CI 2.2 to 21.1) associated with HSCR. In multivariable Cox models including genotype and HSCR as covariates, mortality risk was consistently associated with HSCR. While respiratory signs at initial presentation were comparable between groups, children with both CCHS and HSCR exhibited signs of more severe autonomic dysfunction, requiring more frequent resuscitation and intubation at birth. CONCLUSIONS: HSCR is a key determinant of increased mortality in children with CCHS, likely because of its association with more extensive autonomic dysfunction.
INTRODUCTION: The current cystic fibrosis (CF) care era, while hugely welcome, raises new challenges, particularly the need for more sensitive pulmonary outcome measures. Seeking further optimisation, we previously devel...INTRODUCTION: The current cystic fibrosis (CF) care era, while hugely welcome, raises new challenges, particularly the need for more sensitive pulmonary outcome measures. Seeking further optimisation, we previously developed a Short extension to multiple breath washout measure (MBW) which captures previously overlooked, under-ventilated lung units but lacks regional information. Functional lung MRI addresses this limitation. We hypothesised these measures would be more sensitive to change in tracking CF lung disease than usual clinical respiratory function tests. METHODS: Forty-six people with (pw)CF, median age 15 (range 6-55) years were recruited to a single-centre study. While clinically stable, pwCF performed OE-MRI, MBW+/- and spirometry at baseline and at 6 monthly intervals over 18 months of follow-up. A subgroup of pwCF (n=20) and age-matched healthy controls (HC, n=20) performed two repeatability visits within 6 weeks. RESULTS: OE-MRI/MBW were well tolerated, differentiated HC and CF groups, and were repeatable with negligible differences between two visits <6 weeks apart. OE-MRI/MBW parameters worsened at 12 months (p<0.05) and 18 months (p<0.01). In contrast, conventional measures of pulmonary function (FEV+ LCI) did not change significantly. CONCLUSIONS: OE-MRI/MBW are novel, sensitive tools to track progression of abnormalities in lung structure/function. Such progression may not be detected by conventional outcome measures. CF transmembrane conductance regulator (CFTR) modulators have been transformative for many pwCF and generally lead to substantial improvements in lung health. Stable FEV over longer time periods and, during mucoactive treatment withdrawal, may give false reassurance. OE-MRI/MBW reveal the likely less welcome reality that lung-disease progresses despite CFTR modulators. These measures could be considered in future studies when enhanced sensitivity is required.
BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal interstitial lung disease characterised by excessive extracellular matrix deposition, leading to respiratory failure. While the exact aetiology of I...BACKGROUND: Idiopathic pulmonary fibrosis (IPF) is a progressive, fatal interstitial lung disease characterised by excessive extracellular matrix deposition, leading to respiratory failure. While the exact aetiology of IPF remains unclear, growing evidence suggests that immune dysregulation plays a pivotal role in its pathogenesis. Recent research highlights the involvement of conventional (CD4, CD8 T cells as well as B cells), and unconventional T cell subsets, including natural killer T cells and mucosal-associated invariant T cells in fibrotic lung disease. Unconventional T cell subsets are known for their rapid response to stress signals, antigen-independent activation and involvement in tissue homeostasis and repair. Their potential to modulate fibrosis through interactions with fibroblasts, macrophages and epithelial cells positions them as key players in lung injury and repair mechanisms. METHODS: This review explores the current knowledge of both conventional and unconventional lymphocytes in IPF, examining their contributions to fibrosis development and their potential as therapeutic targets. CONCLUSIONS: By elucidating their functions and interactions within the lung microenvironment, we provide insights into novel immunomodulatory strategies for IPF treatment.