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Distinct trajectories of lung function from childhood to mid-adulthood. 从童年到成年中期肺功能的不同轨迹。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2023-220436
Xian Zhang, Andrew R Gray, Robert J Hancox
{"title":"Distinct trajectories of lung function from childhood to mid-adulthood.","authors":"Xian Zhang, Andrew R Gray, Robert J Hancox","doi":"10.1136/thorax-2023-220436","DOIUrl":"10.1136/thorax-2023-220436","url":null,"abstract":"<p><strong>Rationale: </strong>Life course trajectories of lung function development and decline influence the risk for lung disease but are poorly documented.</p><p><strong>Objective: </strong>To document lung function trajectories from childhood to mid-adult life.</p><p><strong>Methods: </strong>We modelled forced expiratory volume in 1 s (FEV<sub>1</sub>), forced vital capacity (FVC) and FEV<sub>1</sub>/FVC at ages 9, 11, 13, 15, 18, 21, 26, 32, 38 and 45 years from a population-based cohort using latent profile analysis to identify distinct subgroups of participants with similar lung function trajectories. Regression analyses were used to assess associations between the trajectories, early life factors and postbronchodilator airflow obstruction at age 45.</p><p><strong>Results: </strong>Among 865 participants with ≥6 measures of lung function, we identified 10 distinct FEV<sub>1</sub> trajectories. Most were approximately parallel except for a childhood airway hyper-responsiveness-related persistently low trajectory (3% of study population); two accelerated-decline trajectories, one of which (8%) was associated with smoking and higher adult body mass index (BMI) and a catch-up trajectory (8%). Findings for FEV<sub>1</sub>/FVC trajectories were similar. Nine trajectories were identified for FVC: most were also approximately parallel except for a higher BMI-related accelerated-decline trajectory. The three FEV<sub>1</sub> trajectories leading to the lowest FEV<sub>1</sub> values comprised 19% of the cohort but contributed 55% of airflow obstruction at age 45.</p><p><strong>Conclusions: </strong>Lung function trajectories to mid-adult life are largely established before adolescence, with a few exceptions: a childhood airway hyper-responsiveness-related persistently low trajectory, which starts low and gets worse with age, and accelerated adult decline trajectories associated with smoking and obesity. Adverse trajectories are associated with a high risk of airflow obstruction in mid-adult life.</p>","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"754-761"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140159113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Survival outcomes following urgent lung transplantation in France and the USA. 法国和美国紧急肺移植手术后的存活率。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2023-220847
Arnaud Roussel, Edouard Sage, Pierre-Emmanuel Falcoz, Pascal Alexandre Thomas, Yves Castier, Elie Fadel, Françoise Le Pimpec-Barthes, François Tronc, Jacques Jougon, Philippe Lacoste, Johanna Claustre, Laurent Brouchet, Richard Dorent, Edward Cantu, Michael Harhay, Raphaël Porcher, Pierre Mordant
{"title":"Survival outcomes following urgent lung transplantation in France and the USA.","authors":"Arnaud Roussel, Edouard Sage, Pierre-Emmanuel Falcoz, Pascal Alexandre Thomas, Yves Castier, Elie Fadel, Françoise Le Pimpec-Barthes, François Tronc, Jacques Jougon, Philippe Lacoste, Johanna Claustre, Laurent Brouchet, Richard Dorent, Edward Cantu, Michael Harhay, Raphaël Porcher, Pierre Mordant","doi":"10.1136/thorax-2023-220847","DOIUrl":"10.1136/thorax-2023-220847","url":null,"abstract":"<p><strong>Introduction: </strong>Lung graft allocation can be based on a score (Lung Allocation Score) as in the USA or sequential proposals combined with a discrete priority model as in France. We aimed to analyse the impact of allocation policy on the outcome of urgent lung transplantation (LT).</p><p><strong>Methods: </strong>US United Network for Organ Sharing (UNOS) and French Cristal databases were retrospectively reviewed to analyse LT performed between 2007 and 2017. We analysed the mortality risk of urgent LT by fitting Cox models and adjusted Restricted Mean Survival Time. We then compared the outcome after urgent LT in the UNOS and Cristal groups using a propensity score matching.</p><p><strong>Results: </strong>After exclusion of patients with chronic obstructive pulmonary disease/emphysema and redo LT, 3775 and 12 561 patients underwent urgent LT and non-urgent LT in the USA while 600 and 2071 patients underwent urgent LT and non-urgent LT in France. In univariate analysis, urgent LT was associated with an HR for death of 1.24 (95% CI 1.05 to 1.48) in the Cristal group and 1.12 (95% CI 1.05 to 1.19) in the UNOS group. In multivariate analysis, the effect of urgent LT was attenuated and no longer statistically significant in the Cristal database (HR 1.1 (95% CI 0.91 to 1.33)) while it remained constant and statistically significant in the UNOS database (HR 1.12 (95% CI 1.05 to 1.2)). Survival comparison of urgent LT patients between the two countries was significantly different in favour of the UNOS group (1-year survival rates 84.1% (80.9%-87.3%) vs 75.4% (71.8%-79.1%) and 3-year survival rates 66.3% (61.9%-71.1%) vs 62.7% (58.5%-67.1%), respectively).</p><p><strong>Conclusion: </strong>Urgent LT is associated with adverse outcome in the USA and in France with a better prognosis in the US score-based system taking post-transplant survival into account. This difference between two healthcare systems is multifactorial.</p>","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"745-753"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141071230","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Bilateral mediastinal cysts with müllerian differentiation. 双侧纵隔囊肿伴有苗勒氏分化。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2024-221708
Nuria Domedel Puig, Marta Cufí Quintana, Vanessa Escobedo Rodriguez, Manuela Iglesias Sentís, Miguel Gallego
{"title":"Bilateral mediastinal cysts with müllerian differentiation.","authors":"Nuria Domedel Puig, Marta Cufí Quintana, Vanessa Escobedo Rodriguez, Manuela Iglesias Sentís, Miguel Gallego","doi":"10.1136/thorax-2024-221708","DOIUrl":"10.1136/thorax-2024-221708","url":null,"abstract":"","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"796-797"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141180802","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Human mesenchymal stromal cells inhibit Mycobacterium avium replication in clinically relevant models of lung infection. 在临床相关的肺部感染模型中,人间质基质细胞可抑制分枝杆菌的复制。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2023-220819
Timothy D Shaw, Anna D Krasnodembskaya, Gunnar N Schroeder, Declan F Doherty, Johnatas Dutra Silva, Shikha M Tandel, Yue Su, David Butler, Rebecca J Ingram, Cecilia M O'Kane
{"title":"Human mesenchymal stromal cells inhibit <i>Mycobacterium avium</i> replication in clinically relevant models of lung infection.","authors":"Timothy D Shaw, Anna D Krasnodembskaya, Gunnar N Schroeder, Declan F Doherty, Johnatas Dutra Silva, Shikha M Tandel, Yue Su, David Butler, Rebecca J Ingram, Cecilia M O'Kane","doi":"10.1136/thorax-2023-220819","DOIUrl":"10.1136/thorax-2023-220819","url":null,"abstract":"<p><strong>Introduction: </strong>Novel therapeutic strategies are urgently needed for <i>Mycobacterium avium</i> complex pulmonary disease (MAC-PD). Human mesenchymal stromal cells (MSCs) can directly inhibit MAC growth, but their effect on intracellular bacilli is unknown. We investigated the ability of human MSCs to reduce bacterial replication and inflammation in MAC-infected macrophages and in a murine model of MAC-PD.</p><p><strong>Methods: </strong>Human monocyte-derived macrophages (MDMs) were infected with <i>M. avium</i> Chester strain and treated with human bone marrow-derived MSCs. Intracellular and extracellular colony-forming units (CFUs) were counted at 72 hours. Six-week-old female balb/c mice were infected by nebulisation of <i>M. avium</i> Chester. Mice were treated with 1×10<sup>6</sup> intravenous human MSCs or saline control at 21 and 28 days post-infection. Lungs, liver and spleen were harvested 42 days post-infection for bacterial counts. Cytokines were quantified by ELISA.</p><p><strong>Results: </strong>MSCs reduced intracellular bacteria in MDMs over 72 hours (median 35% reduction, p=0.027). MSC treatment increased extracellular concentrations of prostaglandin E2 (PGE2) (median 10.1-fold rise, p=0.002) and reduced tumour necrosis factor-α (median 28% reduction, p=0.025). Blocking MSC PGE2 production by cyclo-oxygenase-2 (COX-2) inhibition with celecoxib abrogated the antimicrobial effect, while this was restored by adding exogenous PGE2. MSC-treated mice had lower pulmonary CFUs (median 18% reduction, p=0.012), but no significant change in spleen or liver CFUs compared with controls.</p><p><strong>Conclusion: </strong>MSCs can modulate inflammation and reduce intracellular <i>M. avium</i> growth in human macrophages via COX-2/PGE2 signalling and inhibit pulmonary bacterial replication in a murine model of chronic MAC-PD.</p>","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"778-787"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287638/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140176607","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases. 适应性多干预试验平台,改善纤维化间质性肺病的患者护理。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2023-221148
Leticia Kawano-Dourado, Tejaswini Kulkarni, Christopher J Ryerson, Pilar Rivera-Ortega, Bruno Guedes Baldi, Nazia Chaudhuri, Manuela Funke-Chambour, Anna-Maria Hoffmann-Vold, Kerri A Johannson, Yet Hong Khor, Sydney B Montesi, Lucilla Piccari, Helmut Prosch, María Molina-Molina, Jacobo Sellares Torres, Iazsmin Bauer-Ventura, Sujeet Rajan, Joseph Jacob, Duncan Richards, Lisa G Spencer, Barbara Wendelberger, Tom Jensen, Melanie Quintana, Michael Kreuter, Anthony C Gordon, Fernando J Martinez, Naftali Kaminski, Victoria Cornelius, Roger Lewis, Wendy Adams, Gisli Jenkins
{"title":"Adaptive multi-interventional trial platform to improve patient care for fibrotic interstitial lung diseases.","authors":"Leticia Kawano-Dourado, Tejaswini Kulkarni, Christopher J Ryerson, Pilar Rivera-Ortega, Bruno Guedes Baldi, Nazia Chaudhuri, Manuela Funke-Chambour, Anna-Maria Hoffmann-Vold, Kerri A Johannson, Yet Hong Khor, Sydney B Montesi, Lucilla Piccari, Helmut Prosch, María Molina-Molina, Jacobo Sellares Torres, Iazsmin Bauer-Ventura, Sujeet Rajan, Joseph Jacob, Duncan Richards, Lisa G Spencer, Barbara Wendelberger, Tom Jensen, Melanie Quintana, Michael Kreuter, Anthony C Gordon, Fernando J Martinez, Naftali Kaminski, Victoria Cornelius, Roger Lewis, Wendy Adams, Gisli Jenkins","doi":"10.1136/thorax-2023-221148","DOIUrl":"10.1136/thorax-2023-221148","url":null,"abstract":"<p><strong>Background: </strong>Fibrotic interstitial lung diseases (fILDs) are a heterogeneous group of lung diseases associated with significant morbidity and mortality. Despite a large increase in the number of clinical trials in the last 10 years, current regulatory-approved management approaches are limited to two therapies that prevent the progression of fibrosis. The drug development pipeline is long and there is an urgent need to accelerate this process. This manuscript introduces the concept and design of an innovative research approach to drug development in fILD: a global Randomised Embedded Multifactorial Adaptive Platform in fILD (REMAP-ILD).</p><p><strong>Methods: </strong>Description of the REMAP-ILD concept and design: the specific terminology, design characteristics (multifactorial, adaptive features, statistical approach), target population, interventions, outcomes, mission and values, and organisational structure.</p><p><strong>Results: </strong>The target population will be adult patients with fILD, and the primary outcome will be a disease progression model incorporating forced vital capacity and mortality over 12 months. Responsive adaptive randomisation, prespecified thresholds for success and futility will be used to assess the effectiveness and safety of interventions. REMAP-ILD embraces the core values of diversity, equity, and inclusion for patients and researchers, and prioritises an open-science approach to data sharing and dissemination of results.</p><p><strong>Conclusion: </strong>By using an innovative and efficient adaptive multi-interventional trial platform design, we aim to accelerate and improve care for patients with fILD. Through worldwide collaboration, novel analytical methodology and pragmatic trial delivery, REMAP-ILD aims to overcome major limitations associated with conventional randomised controlled trial approaches to rapidly improve the care of people living with fILD.</p>","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"788-795"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11287572/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"140050440","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Reinforcing the benefits of children's physical activity on lung health. 加强儿童体育锻炼对肺部健康的益处。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2024-221493
Gang Wang, Erik Melén
{"title":"Reinforcing the benefits of children's physical activity on lung health.","authors":"Gang Wang, Erik Melén","doi":"10.1136/thorax-2024-221493","DOIUrl":"10.1136/thorax-2024-221493","url":null,"abstract":"","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"703-704"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141088812","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Improvements of the shape and strength of the diaphragm after endoscopic lung volume reduction. 内窥镜肺容积缩小术后横膈膜形状和强度的改善。
IF 9 1区 医学
Thorax Pub Date : 2024-07-16 DOI: 10.1136/thorax-2024-221375
Olivier Taton, Pierre Alain Gevenois, Alain Van Muylem, Benjamin Bondue, Sébastien Van Laethem, Dimitri Leduc
{"title":"Improvements of the shape and strength of the diaphragm after endoscopic lung volume reduction.","authors":"Olivier Taton, Pierre Alain Gevenois, Alain Van Muylem, Benjamin Bondue, Sébastien Van Laethem, Dimitri Leduc","doi":"10.1136/thorax-2024-221375","DOIUrl":"10.1136/thorax-2024-221375","url":null,"abstract":"<p><strong>Rationale: </strong>Endoscopic lung volume reduction improves lung function, quality of life and exercise capacity in severe emphysema patients. However, its effect on the diaphragm function is not well understood. We hypothesised that endoscopic lung volume reduction increases its strength by modifying its shape.</p><p><strong>Objectives: </strong>To investigate changes in both diaphragm shape and strength induced by the insertion of endobronchial valves.</p><p><strong>Methods: </strong>In 19 patients, both the diaphragm shape and strength were investigated respectively by 3D Slicer software applied on CT scans acquired at functional residual capacity and by transdiaphragmatic pressure measurements by bilateral magnetic stimulation of the phrenic nerves before and 3 months after unilateral valves insertion.</p><p><strong>Measurements and main results: </strong>After lung volume reduction (median (IQR), 434 mL (-597 to -156], p<0.0001), diaphragm strength increased (transdiaphragmatic pressure: 3 cmH<sub>2</sub>O (2.3 to 4.2), p<0.0001). On the treated side, this increase was associated with an increase in the coronal (16 mm (13 to 24), p<0.0001) and sagittal (26 mm (21 to 30), p<0.0001) lengths as well as in the area of the zone of apposition (62 cm<sup>2</sup> (3 to 100), p<0.0001) with a decrease in the coronal (8 mm (-12 to -4), p<0.0001) and sagittal (9 mm (-18 to -2), p=0.0029) radii of curvature.</p><p><strong>Conclusions: </strong>Endoscopic lung volume reduction modifies the diaphragm shape by increasing its length and its zone of apposition and by decreasing its radius of curvature on the treated side, resulting in an increase in its strength.</p><p><strong>Trial registration number: </strong>NCT05799352.</p>","PeriodicalId":23284,"journal":{"name":"Thorax","volume":" ","pages":"711-717"},"PeriodicalIF":9.0,"publicationDate":"2024-07-16","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141447182","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Journal club 期刊俱乐部
IF 1 1区 医学
Thorax Pub Date : 2024-07-01 DOI: 10.1136/thorax-2024-221967
Timothy J Davies
{"title":"Journal club","authors":"Timothy J Davies","doi":"10.1136/thorax-2024-221967","DOIUrl":"https://doi.org/10.1136/thorax-2024-221967","url":null,"abstract":"In 2015 the WHO set ambitious targets to tackle the global epidemic of Mycobacterium Tuberculosis (TB), including increased detection, reduced mortality, and decreased economic burden of disease, however, the WHO update from September 2023 reports that none are on track for their 2025 milestones (https://reliefweb.int/report/world/global-tuberculosis-report-2023). This situation is not new- globally all three of the 2020 milestones were missed despite some country specific successes. However, the pandemic has had a major impact, stalling, or outright reversing pre-pandemic progress. For example, overall incidence estimates had declined year-on-year between 2010 and 2020 from 11.4 million (95% CI, 8.94,14.10) to 10 million (95% CI, 9.40,10.70). However, in 2021 and 2022, global incidence estimates increased to 10.3 million (95% CI, 9.64,11.00) and 10.6 million (95% CI, 9.87,11.40) respectively. Similar trends were seen in mortality, although the initial increase was in 2020 with 1.37 million estimated deaths compared with 1.32 million in 2019, with the number returning back to pre-pandemic levels (1.30 million, 95% CI 1.18 to 1.43) in 2022. The timing discrepancy reflects the delayed onset of the disease post infection, compared with the more immediate effects of reduced access to treatment. Shockingly, they estimate that 500 000 excess TB deaths occurred compared with if pre-pandemic trends had been maintained. As it stands, the total reduction in incidence rate and TB deaths in 2022 compared with 2015 were 8.7% and 19% respectively, a long …","PeriodicalId":23284,"journal":{"name":"Thorax","volume":"34 1","pages":""},"PeriodicalIF":10.0,"publicationDate":"2024-07-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141319845","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis 伊伐卡夫多对囊性纤维化患者死亡率和健康状况的长期影响
IF 1 1区 医学
Thorax Pub Date : 2024-06-27 DOI: 10.1136/thorax-2023-220558
Christian A Merlo, Teja Thorat, Maral DerSarkissian, Lisa J McGarry, Catherine Nguyen, Yuqian M Gu, Joe Healy, Jaime L Rubin, M Alan Brookhart
{"title":"Long-term impact of ivacaftor on mortality rate and health outcomes in people with cystic fibrosis","authors":"Christian A Merlo, Teja Thorat, Maral DerSarkissian, Lisa J McGarry, Catherine Nguyen, Yuqian M Gu, Joe Healy, Jaime L Rubin, M Alan Brookhart","doi":"10.1136/thorax-2023-220558","DOIUrl":"https://doi.org/10.1136/thorax-2023-220558","url":null,"abstract":"Background Ivacaftor (IVA) has been shown to improve lung function and other clinical outcomes in people with cystic fibrosis (CF). A decade of real-world IVA availability has enabled the examination of long-term outcomes with this treatment. This retrospective, longitudinal cohort study investigated the impact of IVA on mortality rate and health outcomes among people with CF in the US. Methods Data from the US CF Foundation Patient Registry from January 2010 to December 2019 were analysed. The IVA-treated cohort included people with a CF transmembrane conductance regulator ( CFTR ) gating mutation (excluding R117H ); age-matched comparator cohort included people with a F508del and a minimal function CFTR mutation who had no prior CFTR modulator treatment. Baseline characteristics were balanced between cohorts using standardised mortality ratio weighting generated from propensity scores. Outcomes of interest were overall survival, lung transplant, percent predicted forced expiratory volume in 1 s (ppFEV1), body mass index (BMI), pulmonary exacerbations (PEx), outpatient visits and hospitalisations. Findings Over a maximum follow-up of 7.9 years, the IVA-treated cohort (N=736) had lower rates of mortality (hazard ratio [HR] (95% CI): 0.22 (0.09 to 0.45)), lung transplant (HR: 0.11 (95% CI 0.02 to 0.28)), PEx (rate ratio: 0.49 (95% CI 0.42 to 0.55)) and all-cause hospitalisations (rate ratio: 0.50 (95% CI 0.43 to 0.56)) as well as better lung function (mean difference in ppFEV1: 8.46 (95% CI 7.34 to 9.75)) and higher BMI/BMI z -scores (mean difference 1.20 (95% CI 0.92 to 1.71) kg/m2 and 0.27 (95% CI 0.25 to 0.40), respectively) than the comparator cohort (N=733). Interpretation Our analysis suggests that IVA provides sustained clinical benefits in people with CF over a follow-up period of approximately 8 years. These findings reinforce the existing real-world evidence that IVA can slow disease progression and decrease the healthcare burden of CF over the long term. Data are available on reasonable request. The data supporting the findings of this study are available from the US CFFPR at <https://www.cff.org/researchers/patient-registry-data-requests>. The US CFFPR collects and manages its own data and maintains processes for researchers to request summarised data. Restrictions may apply to the availability of these data, which were used under the licence agreement for this study.","PeriodicalId":23284,"journal":{"name":"Thorax","volume":"100 1","pages":""},"PeriodicalIF":10.0,"publicationDate":"2024-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141462461","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Sleep-disordered breathing in children and adults with intellectual disability: mind the gap! 智障儿童和成人的睡眠呼吸障碍:注意差距!
IF 1 1区 医学
Thorax Pub Date : 2024-06-27 DOI: 10.1136/thorax-2023-220032
Renata L Riha, Ankur Singh, Elizabeth A Hill, Hazel Evans, David O'Regan
{"title":"Sleep-disordered breathing in children and adults with intellectual disability: mind the gap!","authors":"Renata L Riha, Ankur Singh, Elizabeth A Hill, Hazel Evans, David O'Regan","doi":"10.1136/thorax-2023-220032","DOIUrl":"https://doi.org/10.1136/thorax-2023-220032","url":null,"abstract":"Background In adults and children with intellectual disability (ID), sleep -disordered breathing (SDB) is thought to be common. However, large epidemiological studies are lacking, and there are few studies on optimal methods of investigation and even fewer randomised, controlled intervention trials of treatment. Method Peer-reviewed publications from various databases were examined in line with search terms relevant to ID and SDB spanning the years 200-2024. Results Findings suggest that, due to comorbid conditions, children and adults with ID may experience both an increased risk of SDB, as well as lower frequency of diagnosis. SDB can compromise the emotional, physical and mental health of individuals with ID. Appropriate treatment when tolerated leads to an improvement in health and well-being and several studies emphasized the importance of consistent follow-up of people with ID - something that is not universally occurring during childhood, in the transition to adulthood and during adulthood itself. As the most frequently occurring form of ID worldwide, we use Down syndrome as a specific example of how diagnosing and treating SDB can lead to improved outcomes. Conclusions This review highlights the importance of identifying SDB in this heterogenous population, recognising the multi-faceted, deleterious consequences of untreated SDB in people with ID, and presents some strategies that can be harnessed to improve diagnosis and management. Until further ID-specific research is available, we urge flexibility in the approach to people with ID and SDB based in guidelines and standard practice developed for the typically developing population.","PeriodicalId":23284,"journal":{"name":"Thorax","volume":"29 1","pages":""},"PeriodicalIF":10.0,"publicationDate":"2024-06-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141462526","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":1,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
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