European Respiratory Journal最新文献

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Risk stratification and treatment goals in pulmonary arterial hypertension. 肺动脉高压的风险分层和治疗目标。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01323-2024
Fabio Dardi, Athénaïs Boucly, Raymond Benza, Robert Frantz, Valentina Mercurio, Horst Olschewski, Göran Rådegran, Lewis J Rubin, Marius M Hoeper
{"title":"Risk stratification and treatment goals in pulmonary arterial hypertension.","authors":"Fabio Dardi, Athénaïs Boucly, Raymond Benza, Robert Frantz, Valentina Mercurio, Horst Olschewski, Göran Rådegran, Lewis J Rubin, Marius M Hoeper","doi":"10.1183/13993003.01323-2024","DOIUrl":"10.1183/13993003.01323-2024","url":null,"abstract":"<p><p>Risk stratification has gained an increasing role in predicting outcomes and guiding the treatment of patients with pulmonary arterial hypertension (PAH). The most predictive prognostic factors are three noninvasive parameters (World Health Organization functional class, 6-min walk distance and natriuretic peptides) that are included in all currently validated risk stratification tools. However, suffering from limitations mainly related to reduced specificity of PAH severity, these variables may not always be adequate in isolation for guiding individualised treatment decisions. Moreover, with effective combination treatment regimens and emerging PAH therapies, markers associated with pulmonary vascular remodelling are expected to become of increasing relevance in guiding the treatment of patients with PAH. While reaching a low mortality risk, assessed with a validated risk tool, remains an important treatment goal, preliminary data suggest that invasive haemodynamics and cardiac imaging may add incremental value in guiding treatment decisions.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525341/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105951","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
Treatment algorithm for pulmonary arterial hypertension. 肺动脉高压的治疗算法。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01325-2024
Kelly M Chin, Sean P Gaine, Christian Gerges, Zhi-Cheng Jing, Stephen C Mathai, Yuichi Tamura, Vallerie V McLaughlin, Olivier Sitbon
{"title":"Treatment algorithm for pulmonary arterial hypertension.","authors":"Kelly M Chin, Sean P Gaine, Christian Gerges, Zhi-Cheng Jing, Stephen C Mathai, Yuichi Tamura, Vallerie V McLaughlin, Olivier Sitbon","doi":"10.1183/13993003.01325-2024","DOIUrl":"10.1183/13993003.01325-2024","url":null,"abstract":"<p><p>Pulmonary arterial hypertension leads to significant impairment in haemodynamics, right heart function, exercise capacity, quality of life and survival. Current therapies have mechanisms of action involving signalling <i>via</i> one of four pathways: endothelin-1, nitric oxide, prostacyclin and bone morphogenetic protein/activin signalling. Efficacy has generally been greater with therapeutic combinations and with parenteral therapy compared with monotherapy or nonparenteral therapies, and maximal medical therapy is now four-drug therapy. Lung transplantation remains an option for selected patients with an inadequate response to therapies.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525349/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105954","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
Management of pulmonary hypertension in special conditions. 特殊情况下的肺动脉高压管理。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01180-2024
Ioana R Preston, Luke S Howard, David Langleben, Mona Lichtblau, Tomas Pulido, Rogerio Souza, Karen M Olsson
{"title":"Management of pulmonary hypertension in special conditions.","authors":"Ioana R Preston, Luke S Howard, David Langleben, Mona Lichtblau, Tomas Pulido, Rogerio Souza, Karen M Olsson","doi":"10.1183/13993003.01180-2024","DOIUrl":"10.1183/13993003.01180-2024","url":null,"abstract":"<p><p>Care of pulmonary hypertension (PH) patients in special situations requires insightful knowledge of the pathophysiology of the cardiopulmonary system and close interaction with different specialists, depending on the situation. The role of this task force was to gather knowledge about five conditions that PH patients may be faced with. These conditions are 1) perioperative care; 2) management of pregnancy; 3) medication adherence; 4) palliative care; and 5) the influence of climate on PH. Many of these aspects have not been covered by previous World Symposia on Pulmonary Hypertension. All of the above conditions are highly affected by psychological, geographical and socioeconomic factors, and share the need for adequate healthcare provision. The task force identified significant gaps in information and research in these areas. The current recommendations are based on detailed literature search and expert opinion. The task force calls for further studies and research to better understand and address the special circumstances that PH patients may encounter.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525332/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105946","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
Effect of sotatercept on circulating proteomics in pulmonary arterial hypertension. 索泰特受体对肺动脉高压循环蛋白质组学的影响
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01483-2024
Laurent Savale, Ly Tu, Corinne Normand, Athénaïs Boucly, Olivier Sitbon, David Montani, Karen M Olsson, Da-Hee Park, Jan Fuge, Jan C Kamp, Marc Humbert, Marius M Hoeper, Christophe Guignabert
{"title":"Effect of sotatercept on circulating proteomics in pulmonary arterial hypertension.","authors":"Laurent Savale, Ly Tu, Corinne Normand, Athénaïs Boucly, Olivier Sitbon, David Montani, Karen M Olsson, Da-Hee Park, Jan Fuge, Jan C Kamp, Marc Humbert, Marius M Hoeper, Christophe Guignabert","doi":"10.1183/13993003.01483-2024","DOIUrl":"10.1183/13993003.01483-2024","url":null,"abstract":"","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525346/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142125253","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
Embracing the challenges of neonatal and paediatric pulmonary hypertension. 迎接新生儿和小儿肺动脉高压的挑战。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01345-2024
Dunbar Ivy, Erika B Rosenzweig, Steven H Abman, Maurice Beghetti, Damien Bonnet, Johannes Menno Douwes, Alessandra Manes, Rolf M F Berger
{"title":"Embracing the challenges of neonatal and paediatric pulmonary hypertension.","authors":"Dunbar Ivy, Erika B Rosenzweig, Steven H Abman, Maurice Beghetti, Damien Bonnet, Johannes Menno Douwes, Alessandra Manes, Rolf M F Berger","doi":"10.1183/13993003.01345-2024","DOIUrl":"10.1183/13993003.01345-2024","url":null,"abstract":"<p><p>Paediatric pulmonary arterial hypertension (PAH) shares common features with adult disease, but is associated with several additional disorders and challenges that require unique approaches. This article discusses recent advances, ongoing challenges and distinct approaches for caring for infants and children with PAH, as presented by the paediatric task force of the 7th World Symposium on Pulmonary Hypertension. We provide updates on diagnosing, classifying, risk-stratifying and treating paediatric pulmonary hypertension (PH) and identify critical knowledge gaps. An updated risk stratification tool and treatment algorithm is provided, now also including strategies for patients with associated cardiopulmonary conditions. Treatment of paediatric PH continues to be hindered by the lack of randomised controlled clinical trials. The challenging management of children failing targeted PAH therapy is discussed, including balloon atrial septostomy, lung transplantation and pulmonary-to-systemic shunt (Potts). A novel strategy using a multimodal approach for the management of PAH associated with congenital heart diseases with borderline pulmonary vascular resistance is included. Advances in diagnosing neonatal PH, especially signs and interpretation of PH by echocardiography, are highlighted. A team approach to the rapidly changing physiology of neonatal PH is emphasised. Challenges in drug approval are discussed, particularly the challenges of designing accurate paediatric clinical trials with age-appropriate end-points and adequate enrolment.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525338/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105942","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
Definition, classification and diagnosis of pulmonary hypertension. 肺动脉高压的定义、分类和诊断。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01324-2024
Gabor Kovacs, Sonja Bartolome, Christopher P Denton, Michael A Gatzoulis, Sue Gu, Dinesh Khanna, David Badesch, David Montani
{"title":"Definition, classification and diagnosis of pulmonary hypertension.","authors":"Gabor Kovacs, Sonja Bartolome, Christopher P Denton, Michael A Gatzoulis, Sue Gu, Dinesh Khanna, David Badesch, David Montani","doi":"10.1183/13993003.01324-2024","DOIUrl":"10.1183/13993003.01324-2024","url":null,"abstract":"<p><p>Pulmonary hypertension (PH) is a haemodynamic condition characterised by elevation of mean pulmonary arterial pressure (mPAP) >20 mmHg, assessed by right heart catheterisation. Pulmonary arterial wedge pressure (PAWP) and pulmonary vascular resistance (PVR) distinguish pre-capillary PH (PAWP ≤15 mmHg, PVR >2 Wood Units (WU)), isolated post-capillary PH (PAWP >15 mmHg, PVR ≤2 WU) and combined post- and pre-capillary PH (PAWP >15 mmHg, PVR >2 WU). Exercise PH is a haemodynamic condition describing a normal mPAP at rest with an abnormal increase of mPAP during exercise, defined as a mPAP/cardiac output slope >3 mmHg/L/min between rest and exercise. The core structure of the clinical classification of PH has been retained, including the five major groups. However, some changes are presented herewith, such as the re-introduction of \"long-term responders to calcium channel blockers\" as a subgroup of idiopathic pulmonary arterial hypertension, the addition of subgroups in group 2 PH and the differentiation of group 3 PH subgroups based on pulmonary diseases instead of functional abnormalities. Mitomycin-C and carfilzomib have been added to the list of drugs with \"definite association\" with PAH. For diagnosis of PH, we propose a stepwise approach with the main aim of discerning those patients who need to be referred to a PH centre and who should undergo invasive haemodynamic assessment. In case of high probability of severe pulmonary vascular disease, especially if there are signs of right heart failure, a fast-track referral to a PH centre is recommended at any point during the clinical workup.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11533989/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105941","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
Pulmonary hypertension associated with lung diseases. 与肺部疾病相关的肺动脉高压。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01200-2024
Oksana A Shlobin, Yochai Adir, Joan A Barbera, Vincent Cottin, Sergio Harari, Etienne-Marie Jutant, Joanna Pepke-Zaba, Hossein-Ardeschir Ghofrani, Richard Channick
{"title":"Pulmonary hypertension associated with lung diseases.","authors":"Oksana A Shlobin, Yochai Adir, Joan A Barbera, Vincent Cottin, Sergio Harari, Etienne-Marie Jutant, Joanna Pepke-Zaba, Hossein-Ardeschir Ghofrani, Richard Channick","doi":"10.1183/13993003.01200-2024","DOIUrl":"10.1183/13993003.01200-2024","url":null,"abstract":"<p><p>Pulmonary hypertension (PH) associated with chronic lung disease (CLD) is both common and underrecognised. The presence of PH in the setting of lung disease has been consistently shown to be associated with worse outcomes. Recent epidemiological studies have advanced understanding of the heterogeneity of this patient population and shown that defining both the specific type of CLD as well as the severity of PH (<i>i.e.</i> deeper phenotyping) is necessary to inform natural history and prognosis. A systematic diagnostic approach to screening and confirmation of suspected PH in CLD is recommended. Numerous uncontrolled studies and one phase 3 randomised, controlled trial have suggested a benefit in treating PH in some patients with CLD, specifically those with fibrotic interstitial lung disease (ILD). However, other studies in diseases such as COPD-PH showed adverse outcomes with some therapies. Given the expanding list of approved pharmacological treatments for pulmonary arterial hypertension, developing a treatment algorithm for specific phenotypes of CLD-PH is required. This article will summarise existing data in COPD, ILD and other chronic lung diseases, and provide recommendations for classification of CLD-PH and approach to the diagnosis and management of these challenging patients.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525344/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105950","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
Pathophysiology of the right ventricle and its pulmonary vascular interaction. 右心室及其肺血管相互作用的病理生理学。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01321-2024
Anna R Hemnes, David S Celermajer, Michele D'Alto, Francois Haddad, Paul M Hassoun, Kurt W Prins, Robert Naeije, Anton Vonk Noordegraaf
{"title":"Pathophysiology of the right ventricle and its pulmonary vascular interaction.","authors":"Anna R Hemnes, David S Celermajer, Michele D'Alto, Francois Haddad, Paul M Hassoun, Kurt W Prins, Robert Naeije, Anton Vonk Noordegraaf","doi":"10.1183/13993003.01321-2024","DOIUrl":"10.1183/13993003.01321-2024","url":null,"abstract":"<p><p>The right ventricle and its stress response is perhaps the most important arbiter of survival in patients with pulmonary hypertension of many causes. The physiology of the cardiopulmonary unit and definition of right heart failure proposed in the 2018 World Symposium on Pulmonary Hypertension have proven useful constructs in subsequent years. Here, we review updated knowledge of basic mechanisms that drive right ventricular function in health and disease, and which may be useful for therapeutic intervention in the future. We further contextualise new knowledge on assessment of right ventricular function with a focus on metrics readily available to clinicians and updated understanding of the roles of the right atrium and tricuspid regurgitation. Typical right ventricular phenotypes in relevant forms of pulmonary vascular disease are reviewed and recent studies of pharmacological interventions on chronic right ventricular failure are discussed. Finally, unanswered questions and future directions are proposed.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525331/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105948","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
Pulmonary hypertension associated with left heart disease. 与左心疾病相关的肺动脉高压。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01344-2024
Bradley A Maron, Guillermo Bortman, Teresa De Marco, Jessica H Huston, Irene M Lang, Stephan H Rosenkranz, Jean-Luc Vachiéry, Ryan J Tedford
{"title":"Pulmonary hypertension associated with left heart disease.","authors":"Bradley A Maron, Guillermo Bortman, Teresa De Marco, Jessica H Huston, Irene M Lang, Stephan H Rosenkranz, Jean-Luc Vachiéry, Ryan J Tedford","doi":"10.1183/13993003.01344-2024","DOIUrl":"10.1183/13993003.01344-2024","url":null,"abstract":"<p><p>Left heart disease (LHD) is the most common cause of pulmonary hypertension (PH), which may be classified further as isolated post-capillary (ipcPH) or combined post- and pre-capillary PH (cpcPH). The 7th World Symposium on Pulmonary Hypertension PH-LHD task force reviewed newly reported randomised clinical trials and contemplated novel opportunities for improving outcome. Results from major randomised clinical trials reinforced prior recommendations against the use of pulmonary arterial hypertension therapy in PH-LHD outside of clinical trials, and suggested possible harm. Greater focus on phenotyping was viewed as one general strategy by which to ultimately improve clinical outcomes. This is potentially achievable by individualising ipcPH <i>versus</i> cpcPH diagnosis for patients with pulmonary arterial wedge pressure within a diagnostic grey zone (12-18 mmHg), and through a newly developed PH-LHD staging system. In this model, PH accompanies LHD across four stages (A=at risk, B=structural heart disease, C=symptomatic heart disease, D=advanced), with each stage characterised by progression in clinical characteristics, haemodynamics and potential therapeutic strategies. Along these lines, the task force proposed disaggregating PH-LHD to emphasise specific subtypes for which PH prevalence, pathophysiology and treatment are unique. This includes re-interpreting mitral and aortic valve stenosis through a contemporary lens, and focusing on PH within the hypertrophic cardiomyopathy and amyloid cardiomyopathy clinical spectra. Furthermore, appreciating LHD in the profile of PH patients with chronic lung disease and chronic thromboembolic pulmonary disease is essential. However, engaging LHD patients in clinical research more broadly is likely to require novel methodologies such as pragmatic trials and may benefit from next-generation analytics to interpret results.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525340/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105949","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
Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension. 肺动脉高压的临床试验设计、终点和新兴疗法。
IF 16.6 1区 医学
European Respiratory Journal Pub Date : 2024-10-31 Print Date: 2024-10-01 DOI: 10.1183/13993003.01205-2024
Jason Weatherald, Thomas R Fleming, Martin R Wilkins, Thomas M Cascino, Mitchell A Psotka, Roham Zamanian, Werner Seeger, Nazzareno Galiè, Mardi Gomberg-Maitland
{"title":"Clinical trial design, end-points, and emerging therapies in pulmonary arterial hypertension.","authors":"Jason Weatherald, Thomas R Fleming, Martin R Wilkins, Thomas M Cascino, Mitchell A Psotka, Roham Zamanian, Werner Seeger, Nazzareno Galiè, Mardi Gomberg-Maitland","doi":"10.1183/13993003.01205-2024","DOIUrl":"10.1183/13993003.01205-2024","url":null,"abstract":"<p><p>Clinical trials in pulmonary arterial hypertension (PAH) have led to the approval of several effective treatments that improve symptoms, exercise capacity and clinical outcomes. In phase 3 clinical trials, primary end-points must reflect how a patient \"feels, functions or survives\". In a rare disease like PAH, with an ever-growing number of treatment options and numerous candidate therapies being studied, future clinical trials are now faced with challenges related to sample size requirements, efficiency and demonstration of incremental benefit on traditional end-points in patients receiving background therapy with multiple drugs. Novel clinical trial end-points, innovative trial designs and statistical approaches and new technologies may be potential solutions to tackle the challenges facing future PAH trials, but these must be acceptable to patients and regulatory bodies while preserving methodological rigour. In this World Symposium on Pulmonary Hypertension task force article, we address emerging trial end-points and designs, biomarkers and surrogate end-point validation, the concept of disease modification, challenges and opportunities to address diversity and representativeness, and the use of new technologies such as artificial intelligence in PAH clinical trials.</p>","PeriodicalId":12265,"journal":{"name":"European Respiratory Journal","volume":" ","pages":""},"PeriodicalIF":16.6,"publicationDate":"2024-10-31","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11525337/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142105940","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
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