European Respiratory Journal最新文献

筛选
英文 中文
MK-5475, an inhaled soluble guanylate cyclase stimulator, for treatment of pulmonary arterial hypertension: the INSIGNIA-PAH study. 用于治疗肺动脉高压的吸入式可溶性鸟苷酸环化酶刺激剂 MK-5475:INSIGNIA-PAH 研究。
IF 24.3 1区 医学
European Respiratory Journal Pub Date : 2024-09-10 DOI: 10.1183/13993003.01110-2024
Marc Humbert,Paul M Hassoun,Kelly M Chin,Guillermo Bortman,Mahesh J Patel,Carmen La Rosa,Wei Fu,Maria José Loureiro,Marius M Hoeper
{"title":"MK-5475, an inhaled soluble guanylate cyclase stimulator, for treatment of pulmonary arterial hypertension: the INSIGNIA-PAH study.","authors":"Marc Humbert,Paul M Hassoun,Kelly M Chin,Guillermo Bortman,Mahesh J Patel,Carmen La Rosa,Wei Fu,Maria José Loureiro,Marius M Hoeper","doi":"10.1183/13993003.01110-2024","DOIUrl":"https://doi.org/10.1183/13993003.01110-2024","url":null,"abstract":"BACKGROUNDMK-5475 is an investigational inhaled soluble guanylate cyclase stimulator hypothesised to avoid most side-effects of systemic vasodilation.METHODSThe phase 2 INSIGNIA-PAH (NCT04732221) trial randomised adults with pulmonary arterial hypertension (PAH) on stable background therapy 1:1:1:1 to once-daily dosing with placebo, MK-5475 32 µg, 100 µg or 380 µg via dry powder inhalation for 12 weeks.OBJECTIVESThe objectives were to evaluate pulmonary vascular resistance (PVR; primary), 6-min walk distance (6MWD; secondary), additional selected haemodynamic parameters, and safety and tolerability in participants with PAH.RESULTS168 participants were randomised to placebo (n=41), MK-5475 32 µg (n=42), 100 µg (n=44), and 380 µg (n=41). Median age was 51 years. Most participants were female (73.8%), diagnosed with idiopathic PAH (63.7%), receiving concomitant phosphodiesterase type 5 inhibitors (PDE5i; 93.5%), and treated with double or triple combination therapy (85.1%). At week 12, the placebo-corrected changes in PVR by least-squares means were -9.2% (95% CI -21.3%, 2.9%; p=0.068) with 32 µg, -22.0% (95% CI -33.7%, -10.3%; p<0.001) with 100 µg, and -19.9% (95% CI -33.4%, -6.4%; p=0.002) with 380 µg MK-5475. No treatment differences versus placebo were observed in 6MWD. Treatment-related adverse events and serious adverse events were similar across treatment groups. Three participants died: two on placebo and one on MK-5475 100 µg. One participant had symptomatic hypotension and one had haemoptysis (both on MK-5475 100 µg).CONCLUSIONSIn participants with PAH on stable background therapy, including PDE5i, inhaled MK-5475 reduced PVR and was well tolerated, without evidence of systemic side-effects such as hypotension, suggesting a pulmonary selective pharmacodynamic effect.","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":24.3,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142170493","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
Cathepsin C (dipeptidyl peptidase 1) inhibition in adults with bronchiectasis: AIRLEAF®, a Phase II randomised, double-blind, placebo-controlled, dose-finding study. 成人支气管扩张症患者的胰蛋白酶 C(二肽基肽酶 1)抑制剂:AIRLEAF®,一项随机、双盲、安慰剂对照、剂量测定的 II 期研究。
IF 24.3 1区 医学
European Respiratory Journal Pub Date : 2024-09-10 DOI: 10.1183/13993003.01551-2024
James D Chalmers,Michal Shteinberg,Marcus A Mall,Anne E O'Donnell,Henrik Watz,Abhya Gupta,Edith Frahm,Anastasia Eleftheraki,Johanna Rauch,Sanjay H Chotirmall,April W Armstrong,Peter Eickholz,Naoki Hasegawa,Wiebke Sauter,Pamela J McShane
{"title":"Cathepsin C (dipeptidyl peptidase 1) inhibition in adults with bronchiectasis: AIRLEAF®, a Phase II randomised, double-blind, placebo-controlled, dose-finding study.","authors":"James D Chalmers,Michal Shteinberg,Marcus A Mall,Anne E O'Donnell,Henrik Watz,Abhya Gupta,Edith Frahm,Anastasia Eleftheraki,Johanna Rauch,Sanjay H Chotirmall,April W Armstrong,Peter Eickholz,Naoki Hasegawa,Wiebke Sauter,Pamela J McShane","doi":"10.1183/13993003.01551-2024","DOIUrl":"https://doi.org/10.1183/13993003.01551-2024","url":null,"abstract":"Bronchiectasis is characterised by uncontrolled neutrophil serine protease (NSP) activity. Cathepsin C (CatC; dipeptidyl peptidase 1) activates NSPs during neutrophil maturation. CatC inhibitors can potentially reduce neutrophil-mediated lung damage. This Phase II, randomised, double-blind, placebo-controlled trial (AIRLEAF®; NCT05238675) evaluated efficacy, safety and optimal dosing of BI 1291583, a novel, reversible CatC inhibitor, in adults with bronchiectasis.In total, 322 participants were randomised (2:1:1:2) to receive one of three oral doses of BI 1291583 (1 mg/2.5 mg/5 mg) or placebo for 24 to 48 weeks. A multiple comparison procedure and modelling approach was used to demonstrate a non-flat dose-response curve based on the time to first pulmonary exacerbation up to Week 48. In addition, efficacy of individual BI 1291583 doses was evaluated based on the frequency of exacerbations, severe exacerbations (fatal or leading to hospitalisation and/or intravenous antibiotic administration), lung function and quality of life.A significant dose-dependent benefit of BI 1291583 over placebo was established based on time to first exacerbation (shape: Emax; adjusted p-value: 0.0448). Treatment with BI 1291583 5 mg and 2.5 mg numerically reduced the risk of an exacerbation compared with placebo (hazard ratios: 0.71 and 0.66, 95% CIs 0.48-1.05 and 0.40-1.08; both p>0.05). BI 1291583 2.5 mg showed numerically better efficacy compared with 5 mg across several endpoints; 1 mg was similar to placebo. The safety profile of BI 1291583 was similar to placebo.Treatment with BI 1291583 resulted in a reduction in the risk of experiencing an exacerbation in adults with bronchiectasis.","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":24.3,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142170495","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
Integrating hot topics and implementation of treatable traits in asthma. 整合热点话题,落实哮喘的可治疗特征。
IF 24.3 1区 医学
European Respiratory Journal Pub Date : 2024-09-10 DOI: 10.1183/13993003.00861-2024
Peter G Gibson,Vanessa M McDonald
{"title":"Integrating hot topics and implementation of treatable traits in asthma.","authors":"Peter G Gibson,Vanessa M McDonald","doi":"10.1183/13993003.00861-2024","DOIUrl":"https://doi.org/10.1183/13993003.00861-2024","url":null,"abstract":"People with asthma experience many different problems related to their illness. The number and type of problems differs between patients. This results in asthma being a complex and heterogeneous disorder which mandates a personalised approach to management. These features pose very significant challenges for the effective implementation of evidence-based management. Treatable Traits is a model of care that has been specifically designed to address these issues. Traits are identified in the pulmonary, extra-pulmonary (comorbidity) and risk factor/behavioural domains. Traits are clinically relevant, recognisable with validated trait identification markers, and treatable using evidence-based therapies. The clinician and patient agree on a personalised management plan that addresses the relevant traits, and trials show superiority of this approach with significant improvements in asthma control and quality of life. A number of tools have now been developed to assist the clinician in the implementation of this approach. The success of the treatable traits model of care is now being realised in other disease areas.","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":24.3,"publicationDate":"2024-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142170494","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
Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two F508del alleles. Elexacaftor/Tezacaftor/Ivacaftor疗法对患有囊性纤维化和一个或两个F508del等位基因的儿童肺清除指数和磁共振成像的影响。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 Print Date: 2024-09-01 DOI: 10.1183/13993003.00004-2024
Mirjam Stahl, Martha Dohna, Simon Y Graeber, Olaf Sommerburg, Diane M Renz, Sophia T Pallenberg, Andreas Voskrebenzev, Katharina Schütz, Gesine Hansen, Felix Doellinger, Eva Steinke, Stephanie Thee, Jobst Röhmel, Sandra Barth, Claudia Rückes-Nilges, Julian Berges, Susanne Hämmerling, Mark O Wielpütz, Lutz Naehrlich, Jens Vogel-Claussen, Burkhard Tümmler, Marcus A Mall, Anna-Maria Dittrich
{"title":"Impact of elexacaftor/tezacaftor/ivacaftor therapy on lung clearance index and magnetic resonance imaging in children with cystic fibrosis and one or two <i>F508del</i> alleles.","authors":"Mirjam Stahl, Martha Dohna, Simon Y Graeber, Olaf Sommerburg, Diane M Renz, Sophia T Pallenberg, Andreas Voskrebenzev, Katharina Schütz, Gesine Hansen, Felix Doellinger, Eva Steinke, Stephanie Thee, Jobst Röhmel, Sandra Barth, Claudia Rückes-Nilges, Julian Berges, Susanne Hämmerling, Mark O Wielpütz, Lutz Naehrlich, Jens Vogel-Claussen, Burkhard Tümmler, Marcus A Mall, Anna-Maria Dittrich","doi":"10.1183/13993003.00004-2024","DOIUrl":"10.1183/13993003.00004-2024","url":null,"abstract":"<p><strong>Background: </strong>We recently demonstrated that elexacaftor/tezacaftor/ivacaftor (ETI) improves the lung clearance index (LCI) and abnormalities in lung morphology detected by magnetic resonance imaging (MRI) in adolescent and adult patients with cystic fibrosis (CF). However, real-world data on the effect of ETI on these sensitive outcomes of lung structure and function in school-age children with CF have not been reported. The aim of this study was therefore to examine the effect of ETI on the LCI and the lung MRI score in children aged 6-11 years with CF and one or two <i>F508del</i> alleles.</p><p><strong>Methods: </strong>This prospective, observational, multicentre, post-approval study assessed the longitudinal LCI up to 12 months and the lung MRI score before and 3 months after initiation of ETI.</p><p><strong>Results: </strong>A total of 107 children with CF including 40 heterozygous for <i>F508del</i> and a minimal function mutation (F/MF) and 67 homozygous for <i>F508del</i> (F/F) were enrolled in this study. Treatment with ETI improved the median (interquartile range (IQR)) LCI in F/MF (-1.0 (-2.0- -0.1); p<0.01) and F/F children (-0.8 (-1.9- -0.2); p<0.001) from 3 months onwards. Further, ETI improved the median (IQR) MRI global score in F/MF (-4.0 (-9.0-0.0); p<0.01) and F/F children (-3.5 (-7.3- -0.8); p<0.001).</p><p><strong>Conclusions: </strong>ETI improves early abnormalities in lung ventilation and morphology in school-age children with CF and at least one <i>F508del</i> allele in a real-world setting. Our results support early initiation of ETI to reduce or even prevent lung disease progression in school-age children with CF.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":16.6,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11375515/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141431702","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
Genetic predisposition to high BMI increases risk of early life respiratory infections and episodes of severe wheeze and asthma. 高体重指数的遗传易感性会增加生命早期呼吸道感染以及严重喘息和哮喘发作的风险。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 Print Date: 2024-09-01 DOI: 10.1183/13993003.00169-2024
Signe Kjeldgaard Jensen, Casper-Emil Tingskov Pedersen, Kasper Fischer-Rasmussen, Mathias Elsner Melgaard, Nicklas Brustad, Julie Nyholm Kyvsgaard, Nilo Vahman, Ann-Marie Malby Schoos, Jakob Stokholm, Bo Chawes, Anders Eliasen, Klaus Bønnelykke
{"title":"Genetic predisposition to high BMI increases risk of early life respiratory infections and episodes of severe wheeze and asthma.","authors":"Signe Kjeldgaard Jensen, Casper-Emil Tingskov Pedersen, Kasper Fischer-Rasmussen, Mathias Elsner Melgaard, Nicklas Brustad, Julie Nyholm Kyvsgaard, Nilo Vahman, Ann-Marie Malby Schoos, Jakob Stokholm, Bo Chawes, Anders Eliasen, Klaus Bønnelykke","doi":"10.1183/13993003.00169-2024","DOIUrl":"10.1183/13993003.00169-2024","url":null,"abstract":"<p><strong>Background: </strong>High body mass index (BMI) is an established risk factor for asthma, but the underlying mechanisms remain unclear.</p><p><strong>Objective: </strong>To increase understanding of the BMI-asthma relationship by studying the association between genetic predisposition to higher BMI and asthma, infections and other asthma traits during childhood.</p><p><strong>Methods: </strong>Data were obtained from the two ongoing Copenhagen Prospective Studies on Asthma in Childhood (COPSAC) mother-child cohorts. Polygenic risk scores for adult BMI were calculated for each child. Replication was done in the large-scale register-based Integrative Psychiatric Research (iPSYCH) cohort using data on hospitalisation for asthma and infections.</p><p><strong>Results: </strong>In the COPSAC cohorts (n=974), the adult BMI polygenic risk score was significantly associated with lower respiratory tract infections (incidence rate ratio (IRR) 1.20, 95% CI 1.08-1.33, false discovery rate p-value (pFDR)=0.005) at age 0-3 years and episodes of severe wheeze (IRR 1.30, 95% CI 1.06-1.60, pFDR=0.04) at age 0-6 years. Lower respiratory tract infections partly mediated the association between the adult BMI polygenic risk score and severe wheeze (proportion mediated: 0.59, 95% CI 0.28-2.24, p-value associated with the average causal mediation effect (pACME)=2e<sup>-16</sup>). In contrast, these associations were not mediated through the child's current BMI and the polygenic risk score was not associated with an asthma diagnosis or reduced lung function up to age 18 years. The associations were replicated in iPSYCH (n=114 283), where the adult BMI polygenic risk score significantly increased the risk of hospitalisations for lower respiratory tract infections and wheeze or asthma throughout childhood to age 18 years.</p><p><strong>Conclusion: </strong>Children with genetic predisposition to higher BMI had increased risk of lower respiratory tract infections and severe wheeze, independent of the child's current BMI. These results shed further light on the complex relationship between body mass BMI and asthma.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":16.6,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141175262","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
Phenotypes in pulmonary hypertension. 肺动脉高压的表型。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 Print Date: 2024-09-01 DOI: 10.1183/13993003.01633-2023
Jason Weatherald, Anna R Hemnes, Bradley A Maron, Lisa M Mielniczuk, Christian Gerges, Laura C Price, Marius M Hoeper, Marc Humbert
{"title":"Phenotypes in pulmonary hypertension.","authors":"Jason Weatherald, Anna R Hemnes, Bradley A Maron, Lisa M Mielniczuk, Christian Gerges, Laura C Price, Marius M Hoeper, Marc Humbert","doi":"10.1183/13993003.01633-2023","DOIUrl":"10.1183/13993003.01633-2023","url":null,"abstract":"<p><p>The clinical classification of pulmonary hypertension (PH) has guided diagnosis and treatment of patients with PH for several decades. Discoveries relating to underlying mechanisms, pathobiology and responses to treatments for PH have informed the evolution in this clinical classification to describe the heterogeneity in PH phenotypes. In more recent years, advances in imaging, computational science and multi-omic approaches have yielded new insights into potential phenotypes and sub-phenotypes within the existing clinical classification. Identification of novel phenotypes in pulmonary arterial hypertension (PAH) with unique molecular profiles, for example, could lead to new precision therapies. Recent phenotyping studies have also identified groups of patients with PAH that more closely resemble patients with left heart disease (group 2 PH) and lung disease (group 3 PH), which has important prognostic and therapeutic implications. Within group 2 and group 3 PH, novel phenotypes have emerged that reflect a persistent and severe pulmonary vasculopathy that is associated with worse prognosis but still distinct from PAH. In group 4 PH (chronic thromboembolic pulmonary disease) and sarcoidosis (group 5 PH), the current approach to patient phenotyping integrates clinical, haemodynamic and imaging characteristics to guide treatment but applications of multi-omic approaches to sub-phenotyping in these areas are sparse. The next iterations of the PH clinical classification are likely to reflect several emerging PH phenotypes and improve the next generation of prognostication tools and clinical trial design, and improve treatment selection in clinical practice.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":16.6,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141534016","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
Host-microbial interactions differ with age of asthma onset. 宿主与微生物之间的相互作用随哮喘发病年龄的不同而不同。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 Print Date: 2024-09-01 DOI: 10.1183/13993003.00428-2024
Ali Versi, Adnan Azim, Fransiskus Xaverius Ivan, Mahmoud I Abdel-Aziz, Stewart Bates, John Riley, Anke H Maitland-Van Der Zee, Sven-Erik Dahlen, Ratko Djukanovic, Sanjay H Chotirmall, Peter Howarth, Nazanin Zounemat Kermani, Kian Fan Chung, Ian M Adcock
{"title":"Host-microbial interactions differ with age of asthma onset.","authors":"Ali Versi, Adnan Azim, Fransiskus Xaverius Ivan, Mahmoud I Abdel-Aziz, Stewart Bates, John Riley, Anke H Maitland-Van Der Zee, Sven-Erik Dahlen, Ratko Djukanovic, Sanjay H Chotirmall, Peter Howarth, Nazanin Zounemat Kermani, Kian Fan Chung, Ian M Adcock","doi":"10.1183/13993003.00428-2024","DOIUrl":"10.1183/13993003.00428-2024","url":null,"abstract":"","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":16.6,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11375513/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142079834","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
Sleep disordered breathing in patients with acute myocardial infarction: finding the perfect window for saving the heart 急性心肌梗死患者的睡眠呼吸障碍:寻找挽救心脏的最佳窗口
IF 24.3 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 DOI: 10.1183/13993003.01395-2024
Christine Eulenburg, Claire Arnaud, Renaud Tamisier
{"title":"Sleep disordered breathing in patients with acute myocardial infarction: finding the perfect window for saving the heart","authors":"Christine Eulenburg, Claire Arnaud, Renaud Tamisier","doi":"10.1183/13993003.01395-2024","DOIUrl":"https://doi.org/10.1183/13993003.01395-2024","url":null,"abstract":"<sec><st>Extract</st>\u0000<p>Sleep disordered breathing (SDB) encompasses two different conditions, obstructive sleep apnoea (OSA), which results from upper airway collapse during sleep, and central sleep apnoea (CSA), which is due to an altered ventilatory drive from the central nervous system [1, 2]. Both conditions stand out for interruptions of ventilation, lasting at least 10&nbsp;s, that repeat all along sleep duration producing intermittent hypoxia (IH). IH and the induced hypoxic burden are the main stimuli involved in the cardiovascular pathophysiology of SDB [3, 4].</p>\u0000</sec>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":24.3,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142138308","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
European Respiratory Society statement on preschool wheezing disorders: updated definitions, knowledge gaps and proposed future research directions. 欧洲呼吸学会关于学龄前喘息障碍的声明:最新定义、知识差距和未来研究方向建议。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 Print Date: 2024-09-01 DOI: 10.1183/13993003.00624-2024
Heidi Makrinioti, Valentina Fainardi, Klaus Bonnelykke, Adnan Custovic, Lisa Cicutto, Courtney Coleman, Thomas Eiwegger, Claudia Kuehni, Alexander Moeller, Eva Pedersen, Marielle Pijnenburg, Hilary Pinnock, Sarath Ranganathan, Thomy Tonia, Padmaja Subbarao, Sejal Saglani
{"title":"European Respiratory Society statement on preschool wheezing disorders: updated definitions, knowledge gaps and proposed future research directions.","authors":"Heidi Makrinioti, Valentina Fainardi, Klaus Bonnelykke, Adnan Custovic, Lisa Cicutto, Courtney Coleman, Thomas Eiwegger, Claudia Kuehni, Alexander Moeller, Eva Pedersen, Marielle Pijnenburg, Hilary Pinnock, Sarath Ranganathan, Thomy Tonia, Padmaja Subbarao, Sejal Saglani","doi":"10.1183/13993003.00624-2024","DOIUrl":"10.1183/13993003.00624-2024","url":null,"abstract":"<p><p>Since the publication of the European Respiratory Society (ERS) task force reports on the management of preschool wheezing in 2008 and 2014, a large body of evidence has accumulated suggesting that the clinical phenotypes that were proposed (episodic (viral) wheezing and multiple-trigger wheezing) do not relate to underlying airway pathology and may not help determine response to treatment. Specifically, using clinical phenotypes alone may no longer be appropriate, and new approaches that can be used to inform clinical care are needed for future research. This ERS task force reviewed the literature published after 2008 related to preschool wheezing and has suggested that the criteria used to define wheezing disorders in preschool children should include age of diagnosis (0 to <6 years), confirmation of wheezing on at least one occasion, and more than one episode of wheezing ever. Furthermore, diagnosis and management may be improved by identifying treatable traits, including inflammatory biomarkers (blood eosinophils, aeroallergen sensitisation) associated with type-2 immunity and differential response to inhaled corticosteroids, lung function parameters and airway infection. However, more comprehensive use of biomarkers/treatable traits in predicting the response to treatment requires prospective validation. There is evidence that specific genetic traits may help guide management, but these must be adequately tested. In addition, the task force identified an absence of caregiver-reported outcomes, caregiver/self-management options and features that should prompt specialist referral for this age group. Priorities for future research include a focus on identifying 1) mechanisms driving preschool wheezing; 2) biomarkers of treatable traits and efficacy of interventions in those without allergic sensitisation/eosinophilia; 3) the need to include both objective outcomes and caregiver-reported outcomes in clinical trials; 4) the need for a suitable action plan for children with preschool wheezing; and 5) a definition of severe/difficult-to-treat preschool wheezing.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":16.6,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141283465","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
The younger, the better: lessons learned from real-world studies on CFTR modulators in young children 越小越好:从幼儿 CFTR 调节剂的实际研究中汲取的经验教训
IF 24.3 1区 医学
European Respiratory Journal Pub Date : 2024-09-05 DOI: 10.1183/13993003.01178-2024
Philippe Reix, Guillaume Chassagnon
{"title":"The younger, the better: lessons learned from real-world studies on CFTR modulators in young children","authors":"Philippe Reix, Guillaume Chassagnon","doi":"10.1183/13993003.01178-2024","DOIUrl":"https://doi.org/10.1183/13993003.01178-2024","url":null,"abstract":"<sec><st>Extract</st>\u0000<p>Prevention of cystic fibrosis (CF) lung disease by the early use of triple combination elexacaftor/tezacaftor/ivacaftor (ETI) will be key in the future standard of care of young children with CF [1, 2]. Almost every paediatrician agrees with this assumption, and evidence has been provided by clinical trials that it slows disease progression in school-aged children with CF [3, 4] and has a great potential to limit bronchiectasis development [2]. As patients enrolled in clinical trials are often highly selected, translation of data obtained from these clinical trials to the \"real world\" can be limited, to the extent that children commencing highly effective modulator therapy may differ somewhat from the trial participants in whom the benefits of ETI were proven.</p>\u0000</sec>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":null,"pages":null},"PeriodicalIF":24.3,"publicationDate":"2024-09-05","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142138310","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
0
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
提示
您的信息不完整,为了账户安全,请先补充。
现在去补充
×
提示
您因"违规操作"
具体请查看互助需知
我知道了
×
提示
确定
请完成安全验证×
相关产品
×
本文献相关产品
联系我们:info@booksci.cn Book学术提供免费学术资源搜索服务,方便国内外学者检索中英文文献。致力于提供最便捷和优质的服务体验。 Copyright © 2023 布克学术 All rights reserved.
京ICP备2023020795号-1
ghs 京公网安备 11010802042870号
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术官方微信