BMJ Open Respiratory Research最新文献

Objective: Patient-reported experience measures (PREMs) are a key component of healthcare accountability frameworks, health policy, integrated care board commissioning and integrated care partnerships generating data which are crucial markers of patient care quality. The Rheumatoid Arthritis Patient-Reported Experience Measure (RA-PREM) incorporates the eight core elements of NHS Patient Experience Framework and is validated in a range of rheumatic conditions. Our objective is to determine the acceptability and feasibility of the RA-PREM for an interstitial lung disease (ILD) population.

Design: A mixed-methods patient-centred approach incorporating an interdisciplinary research steering group with patient partners. Patient surveys evaluated the language and meaning of the RA-PREM 8 domains, 24 statements and response categories. A patient focus group examined contentious statements. A consensus group of expert patient-partners agreed statements for the modified RA-PREM. Focus group participants reviewed the modified instrument (ILD-PREM) for acceptability and face/content validity.

Setting: A single NHSE-commissioned, regional ILD service/UK.

Results: Thirteen patients (10 male) diagnosed with ILD participated in focus group discussions. Critical discussion of the RA-PREM resulted in nuanced modifications of four statements of three domains. Five patients (three male) and three healthcare researchers attained consensus on the face/content validity of statements. Seventy-three patients completed the ILD-PREM following outpatient contact.

Conclusion: The ILD-PREM retains 24 statements representing the eight domains of the RA-PREM. It meets face/content validity criteria and is acceptable to an ILD population. Longitudinal validation of the ILD-PREM across ILD services including further testing in global minority groups will establish criterion and construct validity and objective measures of reliability.

Background: Interstitial lung disease (ILD) represents a group of complex parenchymal conditions characterised by varying clinical trajectories. The It's Not JUST Idiopathic Pulmonary Fibrosis Study seeks to identify genetic, proteomic and clinical biomarkers that distinguish rapidly progressive fibrotic phenotypes from stable phenotypes irrespective of aetiology. This manuscript presents baseline insights from the recruited cohort.

Methods: In this prospective, longitudinal study, participants with fibrotic ILDs, including idiopathic pulmonary fibrosis (IPF), fibrotic hypersensitivity pneumonitis, rheumatoid arthritis-associated ILD, asbestosis and unclassifiable ILD, were enrolled from 24 UK sites. Participants underwent comprehensive baseline evaluation including demographics, exposure history, lung function testing, 6-min walk tests, blood sampling and standardised questionnaires to assess symptoms and quality of life.

Results: A total of 272 participants were recruited, predominantly older white males with a smoking history. Baseline lung function showed comparable forced vital capacity (mean 89.0% predicted), diffusion of carbon monoxide (mean 57.9% predicted) and 6-min walk distance (mean 302 m) across ILD subtypes. Hypertension was the most prevalent comorbidity, affecting 40.8% of participants, with no significant differences across subtypes. Anxiety and depression were notably lower in IPF than non-IPF (4.5%; 21.0%). Previous occupational exposure was reported in 68.8% of participants, with asbestos exposure the most prevalent (36%). Bird exposure was reported by 40.4% of participants, with no significant differences across subtypes. No significant differences in health-related quality of life scores were observed across subtypes.

Conclusions: Despite varied aetiologies, fibrotic ILDs exhibit demographic and functional similarities, including lung function and health-related quality of life suggesting commonalities in disease mechanisms.

Trial registration number: NCT03670576.

Introduction: The EuroQol five dimensions questionnaire (EQ-5D) is widely used as a health-related quality of life (HRQoL) measure worldwide, but its sensitivity in detecting changes in health status among individuals with chronic obstructive pulmonary disease (COPD) remains suboptimal. Adding additional HRQoL dimensions (bolt-on items) to EQ-5D may enhance its sensitivity to specific diseases while preserving the original EQ-5D descriptive system. This study protocol documents the identification, development, selection and psychometric testing of respiratory-related bolt-on items for assessing COPD in China.

Methods and analysis: We will conduct a content review of the COPD disease-specific instruments to identify potential deficiencies in dimensions covered by the EQ-5D. Drawing from the dimensions identified in the content review, we will consult with clinical experts and conduct qualitative interviews with patients to identify and develop candidate bolt-on items that are relevant to COPD. Then, in the quantitative phase, we will assess the psychometric properties of the candidate bolt-on items among patients with COPD in China. Test-retest reliability will be examined using intraclass correlation coefficients for total scores and weighted kappa statistics for item-level agreement. Construct validity will be evaluated through convergent and divergent validity analyses using Spearman's rank correlations. Known-groups validity will be tested by comparing five-level EQ-5D version (EQ-5D-5L) and EQ-5D-5L plus bolt-on scores across clinically defined subgroups using analysis of variance. The dimensional structure will be explored using exploratory factor analysis with maximum likelihood extraction and Promax rotation. Explanatory power will be assessed through ordinary least-squares regression with EQ visual analogue scale as the dependent variable, supported by Shorrocks-Shapley decomposition to quantify the contribution of each item to the explained variance.

Discussion: Based on the EQ-5D, Our study offers a new approach to measuring HRQoL in patients with COPD. Furthermore, it may offer valuable insights and serve as a reference for the development of bolt-on items for other diseases.

Ethics and dissemination: This study has received ethical approval from the Ethics Committee of the Centre for Health Management and Policy Research at Shandong University (no. ECSHCMSDU20240301).

Background: To facilitate shared decision-making (SDM), it is important that doctors clearly present the decision to be made and the available treatment options to the patient. This study explored how treatment options after first-line therapy were presented in advanced lung cancer consultations.

Methods: Audio recordings of 12 advanced lung cancer consultations between patients, their companions and doctors in three Norwegian hospitals were transcribed and analysed using Conversation Analysis. Data were collected between November 2019 and March 2022.

Results: Doctors employed three strategies when presenting treatment options to patients. These were (1) open option presentation, (2) selective option presentation and (3) recommended option presentation. Strategy (1) involved the doctor presenting all the possible treatment options in a balanced manner to the patient. With strategy (2), the doctor presented selected treatment options to the patient, and, for example, did not articulate the possibility of refraining from further therapy. Strategy (3) included the doctor's explicit preference for certain treatment options.

Conclusions: Strategies that doctors employ to present treatment options to patients facilitated SDM to different degrees, where some can challenge core principles of SDM, such as creating choice awareness. Doctors must be aware of their strategies to ensure that treatment options are presented to patients in a way that supports SDM.

Background: Maternal asthma has been associated with a higher risk of asthma in early childhood compared with paternal asthma, but it is unclear whether this difference persists into adolescence.

Methods: We analysed 55 643 children from the Norwegian Mother, Father and Child Cohort Study. Parental asthma was self-reported during pregnancy; offspring asthma was reported by mothers at ages 3, 7 and 14 years. Logistic regression models estimated associations between parental asthma and offspring asthma at each age, adjusting for maternal age, parental prepregnancy body mass index, parental education, parental smoking and parental atopic conditions, including atopic eczema and pollen/hay fever.

Results: Asthma prevalence among offspring was 6.5% at age 3, 5.2% at age 7 and 5.4% at age 14. Compared with children of non-asthmatic parents, adjusted ORs for asthma at age 3 were 3.11 (95% CI 2.73 to 3.54) for maternal asthma only and 2.25 (95% CI 1.97 to 2.56) for paternal asthma only. Similar patterns were observed at ages 7 (maternal OR 2.96 (95% CI 2.54 to 3.45); paternal OR 2.36 (95% CI 2.03 to 2.75)) and 14 (maternal OR 3.03 (95% CI 2.46 to 3.73); paternal OR 1.95 (95% CI 1.57 to 2.43)). At age 3, maternal asthma was associated with higher odds in boys (OR 3.30) than girls (OR 2.82), and higher absolute risk (19.2% vs 12.2%). However, interaction tests by offspring sex were not statistically significant.

Conclusions: Maternal asthma conferred a consistently stronger risk of offspring asthma than paternal asthma, from early childhood into adolescence. This effect appeared slightly stronger in boys in early childhood, though sex differences were not statistically significant.

Introduction: Targeted maximum likelihood estimation (TMLE) is a semiparametric doubly-robust estimator that integrates the SuperLearner in the estimation process, an ensemble method that allows us to model the exposure-outcome relationship combining multiple parametric and non-parametric methods.

Aim: We applied TMLE to assess the effect of maternal paracetamol use during the first trimester of pregnancy on child wheezing during the first 18 months of life, using data of the Italian NINFEA birth cohort.

Methods: We included three progressively larger sets of covariates for confounding adjustment. Set 1 included baseline socioeconomic and maternal characteristics, conditions and disorders. Set 2 additionally included maternal respiratory infections in the first pregnancy trimester. Set 3 added prepregnancy maternal mental health disorders.The effect was estimated with three TMLE implementations, differing in the methods used to model the exposure-outcome relationship: (1) parametric; (2) SuperLearner with parametric and semiparametric approaches and (3) SuperLearner with parametric, semiparametric and non-parametric approaches, and with hyperparameters tuning. We compared TMLE with multivariable regression, propensity score regression adjustment and inverse probability weighting.

Results: All methods provided similar results, suggesting a weak positive association that attenuated toward the null as progressively more covariates were adjusted for, from set 1 (TMLE 3: risk ratio, RR 1.15 (95% CI 1.03 to 1.29)) to set 3 (TMLE 3: RR 1.10 (95% CI 0.97 to 1.26), N=4099).

Conclusions: Such an association could be interpreted as a small positive effect or incomplete control for residual or unmeasured confounding, and its consistency across methods suggests it is unlikely to be driven by model misspecification.

Objective: Polypeptide N-acetylgalactosaminyltransferase 7 (GALNT7) is a glycopeptide transferase which is closely involved in the development and progression of cancer. This study aimed to investigate the effects of GALNT7 inhibition on non-small cell lung cancer (NSCLC) cell proliferation, apoptosis and invasiveness and to determine whether these effects are mediated by the protein kinase B (AKT) pathway.

Methods: Negative control small interfering RNA (siRNA) (si-NC) and siRNA targeting GALNT7 (si-GALNT7) were transfected into A549 and NCI-H1650 cells. Afterwards, the AKT activator '740 Y-P' was added to treat the cells with or without siRNA transfection.

Results: Cell proliferation was reduced after si-GALNT7 transfection compared with si-NC transfection at 24 hours, 48 hours and 72 hours in A549 cells, and at 48 hours and 72 hours in NCI-H1650 cells. Furthermore, the cell apoptosis rate was increased, but the cell invasive number was decreased after si-GALNT7 transfection compared with si-NC transfection in A549 cells and NCI-H1650 cells. Phosphorylated (p)-AKT/AKT expression was lower after si-GALNT7 transfection compared with si-NC transfection in A549 cells and NCI-H1650 cells. Worth noting, the effects of si-GALNT7 transfection on the above-mentioned cell proliferation, apoptosis and invasiveness were repressed by the addition of 740 Y-P in A549 cells and NCI-H1650 cells.

Conclusion: GALNT7 inhibition suppresses NSCLC cell proliferation and invasiveness while increasing apoptosis through inactivation of the AKT pathway.

Background: The burden of severe asthma due to asthma exacerbation is increasing. However, recent studies identifying exacerbation-related disease burden among patients with severe asthma in South Korea are lacking.

Objective: To evaluate the burden of asthma exacerbations in patients with severe asthma in South Korea.

Methods: A retrospective cohort study using National Health Insurance data from 2016 to 2019 was conducted. Patients with severe asthma were defined as satisfying the following criteria: (1) having been prescribed ≥1 inhaled corticosteroid-long-acting β-2 agonist and long-acting muscarinic antagonist with a diagnosis of asthma; (2) having experienced ≥4 asthma exacerbation events within 1 year, after meeting the first criterion. Exacerbation was defined as a corticosteroid burst. Healthcare utilisation, direct medical costs and case fatality rates related to asthma exacerbations were identified during the follow-up period.

Results: Outpatient visits accounted for 81.26% of all exacerbation events, followed by general ward hospitalisation (14.55%). The exacerbation-related costs were as follows: outpatient visit, $29.89; emergency room visit, $95.02; general ward hospitalisation, $1929.32; and intensive care unit admission, $7918.86. The case fatality rates were 2.65% for patients with asthma, 3.16% for patients who had exacerbations and 6.96% for patients who had exacerbations with hospitalisation.

Conclusion: This study is the first population-based cohort study observing exacerbation-related burdens in patients with severe asthma, using recent data and generating real-world evidence. The results of this study can be used as evidence for further research on the burden of asthma and to inform decision-making in healthcare policy.

Background: This study aims to evaluate the association of Life's Essential 8 (LE8) with chronic lower respiratory disease (CLRD)-specific mortality and impaired lung health outcomes.

Methods: This population-based cohort study used data from the National Health and Nutrition Examination Survey (NHANES, 2007-2018), including adults aged 20-79 years (n=10 135), with lung function measurements available for a subset (n=3188). Multivariable Cox proportional hazards and restricted cubic spline models were employed to assess the associations between LE8 scores and CLRD-specific mortality. Logistic and linear regression models evaluated the associations between LE8 scores and lung health. All models were adjusted for sociodemographic variables (age, sex, race/ethnicity, education, income-to-poverty ratio), cardiovascular disease, respiratory disease and smoking history. Sensitivity analyses were conducted to assess the stability of the results. The primary outcome was CLRD-specific mortality, and the secondary outcome was lung health.

Results: Over a median follow-up of 7.83 years, 50 CLRD-specific deaths were recorded. Higher LE8 scores were associated with reduced risks of CLRD-specific mortality (adjusted HR (aHR), 0.56 (0.40-0.79)), with a linear dose-response relationship observed (P for non-linear=0.574). Furthermore, each 10-point increase in total LE8 score was associated with impaired lung health, including lower odds of asthma (adjusted OR (aOR), 0.88 (0.83-0.93)), chronic bronchitis (aOR, 0.81 (0.74-0.88)), emphysema (aOR, 0.59 (0.52-0.65)), chronic obstructive pulmonary disease (aOR, 0.63 (0.45-0.89)) and lower relative risk of a restrictive spirometry pattern (adjusted relative risk ratio (aRRR), 0.66 (0.56-0.79)). Positive correlations were observed between total LE8 scores and lung function (p<0.001). The findings were robust in sensitivity analyses and consistent across key subgroups.

Conclusions: Higher LE8 scores were associated with reduced CLRD-specific mortality and improved lung health. Promoting LE8 adherence could significantly alleviate respiratory disease burdens and mortality.

Background: Birth weight (BW) Z-score is associated with outcomes in very preterm infants (VPIs). This study aimed to investigate the association between BW Z-score and the adverse outcomes in VPIs.

Methods: This retrospective cohort study included VPIs admitted to a tertiary neonatal intensive care unit between 1 January 2014 and 31 December 2023. Restricted cubic splines and multivariable logistic regression models were employed to assess associations between BW Z-score and primary outcomes. Infants were categorised based on the identified turning point of Z=-0.35 in the Z-score distribution, where the risk gradient changed most sharply. The primary outcome was bronchopulmonary dysplasia (BPD) or mortality at 36 weeks postmenstrual age or discharge.

Results: Among 4632 included VPIs, a turning point at Z=-0.35 was identified. Compared with those with Z≥-0.35, VPIs with Z<-1 exhibited higher risks of primary outcomes (OR 3.10, 95% CI 2.53 to 3.79), while those with BW Z-score between -1 and -0.35 also showed increased risks (OR 1.81, 95% CI 1.52 to 2.15). Subgroup and sensitivity analyses further supported the robustness of these findings.

Conclusion: Compared with BW Z-score above -0.35, both substantially negative BW Z-score<-1 and moderate lower BW Z-score between -1 and -0.35 are associated with increased risk of BPD and mortality in VPIs. The findings underscore the importance of considering BW Z-score as a continuous variable in risk stratification and management of VPIs.