首页 > 最新文献

Pulmonary Therapy最新文献

英文 中文
Perspectives on Drug Product Design Among Patients with Lung Cancer in the United Kingdom. 英国肺癌患者对药物产品设计的看法。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-10-28 DOI: 10.1007/s41030-024-00279-7
Joshua R Coulter, Louis Edward Baig, Amy Antipas, Debra Montague, Angela Terry, Sally-Anne Dews, Michaela Ogden-Barker, Colm Doody, Brett Hauber

Introduction: The use of oral anticancer medications has become more prevalent in cancer therapy. This is particularly the case in the management of advanced non-small cell lung cancer (NSCLC). However, when the treatment delivery interaction between the patient and the healthcare provider is removed, the risk of non-adherence increases. Insights into patient preferences can allow drug product formulation scientists to design more patient-centric medications that may promote an increase in adherence which, in turn, may lead to more beneficial health outcomes.

Methods: We conducted an advisory board with patients with NSCLC in the United Kingdom to elicit and understand preferences for drug product attributes related to appearance, instructions, and modality. The advisory board was preceded by a quantitative preference survey that included three object-case best-worst scaling exercises and was followed by administering the same survey to a broader group of patients to confirm the results.

Results: Patients strongly prefer once-daily dosing over more frequent dosing, regardless of the number of pills because taking tablets or capsules multiple times each day can disrupt daily activities. In addition, patients place high importance on surface smoothness because a rough surface implies decreased swallowability. Finally, food restrictions involving directions regarding taking medication with or without food represent difficulties for patients with cancer. Results of the follow-up survey confirmed these results.

Conclusions: Drug developers should attempt to limit the dosing of these medications to once-daily regimens, avoid surface roughness, and develop formulations that can be taken without regard to the timing of meals to the greatest extent possible.

介绍:在癌症治疗中,口服抗癌药物的使用越来越普遍。在晚期非小细胞肺癌(NSCLC)的治疗中尤其如此。然而,如果患者与医疗服务提供者之间的治疗互动被取消,不坚持用药的风险就会增加。了解患者的偏好可以让药物制剂科学家设计出更多以患者为中心的药物,从而提高患者的依从性,进而带来更有益的健康结果:我们与英国的 NSCLC 患者举行了一次咨询会,以了解他们对与外观、说明和方式相关的药物产品属性的偏好。在咨询委员会召开之前,我们进行了一项定量偏好调查,其中包括三个对象-案例-最佳-最差缩放练习,随后我们对更广泛的患者群体进行了同样的调查,以确认结果:结果:与更频繁的服药方式相比,无论药片数量多少,患者都强烈倾向于每天服药一次,因为每天多次服用药片或胶囊会影响日常活动。此外,患者非常重视药片表面的光滑度,因为粗糙的表面意味着吞咽性降低。最后,对于癌症患者来说,食物限制涉及到服药与否的说明,这也是困难所在。后续调查的结果证实了这些结果:药物开发人员应尽量将这些药物的剂量限制在每日一次,避免药物表面粗糙,并尽可能开发出无需考虑进餐时间的配方。
{"title":"Perspectives on Drug Product Design Among Patients with Lung Cancer in the United Kingdom.","authors":"Joshua R Coulter, Louis Edward Baig, Amy Antipas, Debra Montague, Angela Terry, Sally-Anne Dews, Michaela Ogden-Barker, Colm Doody, Brett Hauber","doi":"10.1007/s41030-024-00279-7","DOIUrl":"10.1007/s41030-024-00279-7","url":null,"abstract":"<p><strong>Introduction: </strong>The use of oral anticancer medications has become more prevalent in cancer therapy. This is particularly the case in the management of advanced non-small cell lung cancer (NSCLC). However, when the treatment delivery interaction between the patient and the healthcare provider is removed, the risk of non-adherence increases. Insights into patient preferences can allow drug product formulation scientists to design more patient-centric medications that may promote an increase in adherence which, in turn, may lead to more beneficial health outcomes.</p><p><strong>Methods: </strong>We conducted an advisory board with patients with NSCLC in the United Kingdom to elicit and understand preferences for drug product attributes related to appearance, instructions, and modality. The advisory board was preceded by a quantitative preference survey that included three object-case best-worst scaling exercises and was followed by administering the same survey to a broader group of patients to confirm the results.</p><p><strong>Results: </strong>Patients strongly prefer once-daily dosing over more frequent dosing, regardless of the number of pills because taking tablets or capsules multiple times each day can disrupt daily activities. In addition, patients place high importance on surface smoothness because a rough surface implies decreased swallowability. Finally, food restrictions involving directions regarding taking medication with or without food represent difficulties for patients with cancer. Results of the follow-up survey confirmed these results.</p><p><strong>Conclusions: </strong>Drug developers should attempt to limit the dosing of these medications to once-daily regimens, avoid surface roughness, and develop formulations that can be taken without regard to the timing of meals to the greatest extent possible.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"469-482"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11573933/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142522812","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Survival Outcomes in US Medicare Patients with Non-Cystic Fibrosis Bronchiectasis by Rate of Baseline Exacerbations. 按基线恶化率分列的美国医疗保险非囊性纤维化支气管扩张症患者的生存结果。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-10-10 DOI: 10.1007/s41030-024-00275-x
Joseph Feliciano, Benjamin Lewing, Maitreyee Mohanty, Melanie Lauterio, Sebastian Fucile, Joseph Tkacz, Alan F Barker

Introduction: There are limited real-world data on outcomes in patients with non-cystic fibrosis bronchiectasis (NCFBE). This study assessed clinical characteristics and survival in patients with NCFBE by baseline exacerbation rate.

Methods: Patients with bronchiectasis (≥ 1 inpatient or ≥ 2 outpatient claims with a bronchiectasis diagnosis code, or one outpatient claim with bronchiectasis code and a chest computed tomography scan) were from the 100% Medicare Fee-for-Service database (Jan 2014-Dec 2020). Patients had continuous enrollment ≥ 12 months pre-index (baseline) and post-index (follow-up), with index a random bronchiectasis claim preceded by ≥ 12 months bronchiectasis history. Patients with cystic fibrosis were excluded. Patients were stratified by exacerbations during baseline (0, 1, or ≥ 2). Follow-up exacerbation rate and all-cause mortality were assessed. Controls were identified using a multistep direct matching approach. Time to death from index was estimated by Kaplan-Meier analyses.

Results: Exacerbation analysis included 92,529 patients with NCFBE and 92,529 matched controls. Exacerbations were common (43% had ≥ 1 exacerbation), with patients with more baseline exacerbations more likely to have ≥ 2 exacerbations during follow-up (11.4%, 24.2%, and 46.8% of patients with 0, 1, and ≥ 2 baseline exacerbations, respectively). Survival analysis included 110,298 patients with NCFBE and 110,298 controls. Time to death was shorter in patients with more baseline exacerbations (P < 0.0001). Five-year survival was 55.3%, 62.6%, and 65.4% for patients with ≥ 2, 1, and 0 baseline exacerbations, respectively, compared with 64.1% for controls.

Conclusions: In these patients with NCFBE, exacerbations were common. History of exacerbations was associated with future exacerbations and increased all-cause mortality.

导言:有关非囊性纤维化支气管扩张症(NCFBE)患者疗效的实际数据非常有限。本研究根据基线恶化率评估了非囊性纤维化支气管扩张症患者的临床特征和存活率:支气管扩张症患者(≥1次住院或≥2次门诊索赔中包含支气管扩张症诊断代码,或1次门诊索赔中包含支气管扩张症代码和胸部计算机断层扫描)来自100%医疗保险付费服务数据库(2014年1月-2020年12月)。患者在索引前(基线)和索引后(随访)连续注册时间≥ 12 个月,索引为随机支气管扩张索赔,且支气管扩张病史≥ 12 个月。囊性纤维化患者除外。根据基线期间的病情加重情况对患者进行分层(0、1 或≥ 2)。评估随访加重率和全因死亡率。对照组采用多步骤直接匹配法确定。通过 Kaplan-Meier 分析法估算了从发病到死亡的时间:结果:病情加重分析包括 92,529 例 NCFBE 患者和 92,529 例匹配对照。病情恶化很常见(43%的患者病情恶化≥1次),基线病情恶化较多的患者在随访期间病情恶化≥2次的可能性更大(基线病情恶化为0、1和≥2次的患者分别占11.4%、24.2%和46.8%)。生存分析包括 110,298 名 NCFBE 患者和 110,298 名对照组患者。基线病情加重次数越多的患者死亡时间越短(P 结论:基线病情加重次数越多的患者死亡时间越短):在这些 NCFBE 患者中,病情加重很常见。病情加重史与未来病情加重和全因死亡率增加有关。
{"title":"Survival Outcomes in US Medicare Patients with Non-Cystic Fibrosis Bronchiectasis by Rate of Baseline Exacerbations.","authors":"Joseph Feliciano, Benjamin Lewing, Maitreyee Mohanty, Melanie Lauterio, Sebastian Fucile, Joseph Tkacz, Alan F Barker","doi":"10.1007/s41030-024-00275-x","DOIUrl":"10.1007/s41030-024-00275-x","url":null,"abstract":"<p><strong>Introduction: </strong>There are limited real-world data on outcomes in patients with non-cystic fibrosis bronchiectasis (NCFBE). This study assessed clinical characteristics and survival in patients with NCFBE by baseline exacerbation rate.</p><p><strong>Methods: </strong>Patients with bronchiectasis (≥ 1 inpatient or ≥ 2 outpatient claims with a bronchiectasis diagnosis code, or one outpatient claim with bronchiectasis code and a chest computed tomography scan) were from the 100% Medicare Fee-for-Service database (Jan 2014-Dec 2020). Patients had continuous enrollment ≥ 12 months pre-index (baseline) and post-index (follow-up), with index a random bronchiectasis claim preceded by ≥ 12 months bronchiectasis history. Patients with cystic fibrosis were excluded. Patients were stratified by exacerbations during baseline (0, 1, or ≥ 2). Follow-up exacerbation rate and all-cause mortality were assessed. Controls were identified using a multistep direct matching approach. Time to death from index was estimated by Kaplan-Meier analyses.</p><p><strong>Results: </strong>Exacerbation analysis included 92,529 patients with NCFBE and 92,529 matched controls. Exacerbations were common (43% had ≥ 1 exacerbation), with patients with more baseline exacerbations more likely to have ≥ 2 exacerbations during follow-up (11.4%, 24.2%, and 46.8% of patients with 0, 1, and ≥ 2 baseline exacerbations, respectively). Survival analysis included 110,298 patients with NCFBE and 110,298 controls. Time to death was shorter in patients with more baseline exacerbations (P < 0.0001). Five-year survival was 55.3%, 62.6%, and 65.4% for patients with ≥ 2, 1, and 0 baseline exacerbations, respectively, compared with 64.1% for controls.</p><p><strong>Conclusions: </strong>In these patients with NCFBE, exacerbations were common. History of exacerbations was associated with future exacerbations and increased all-cause mortality.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"439-450"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11573945/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142401113","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Is 'Cardiopulmonary' the New 'Cardiometabolic'? Making a Case for Systems Change in COPD. 心肺 "是新的 "心脏代谢 "吗?为慢性阻塞性肺病的系统变革提供依据。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-09-09 DOI: 10.1007/s41030-024-00270-2
Nathaniel M Hawkins, Alan Kaplan, Dennis T Ko, Erika Penz, Mohit Bhutani

Chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD) have a syndemic relationship with shared risk factors and complex interplay between genetic, environmental, socioeconomic, and pathophysiological mechanisms. CVD is among the most common comorbidities in patients with COPD and vice versa. Patients with COPD, irrespective of their disease severity, are at increased risk of CVD morbidity and mortality, driven in part by COPD exacerbations. Despite these known interrelationships, CVD is underestimated and undertreated in patients with COPD. Similarly, COPD is an independent risk-enhancing factor for adverse cardiovascular (CV) events, yet it is not incorporated into current CV risk assessment tools, leading to under-recognition and undertreatment. There is a pressing need for systems change in COPD management to move beyond symptom control towards a comprehensive cardiopulmonary disease paradigm with proactive prevention of exacerbations and adverse cardiopulmonary outcomes and mortality. However, there is a dearth of evidence defining optimal cardiopulmonary care pathways. Fortunately, there is a precedent to support systems-level change in the field of diabetes, which evolved from glycemic control to comprehensive multi-organ risk assessment and management. Key elements included integrated multidisciplinary care, intensive risk factor management, coordination between primary and specialist care, care pathways and protocols, education and self management, and disease-modifying therapies. This commentary article draws parallels between the cardiometabolic and cardiopulmonary paradigms and makes a case for systems change towards multidisciplinary, integrated cardiopulmonary care, using the evolution in diabetes care as a potential framework.

慢性阻塞性肺疾病(COPD)和心血管疾病(CVD)具有共同的风险因素以及遗传、环境、社会经济和病理生理机制之间复杂的相互作用,两者之间存在着一种综合关系。心血管疾病是慢性阻塞性肺病患者最常见的合并症之一,反之亦然。慢性阻塞性肺病患者无论病情严重程度如何,其心血管疾病发病率和死亡率的风险都会增加,部分原因是慢性阻塞性肺病加重。尽管存在这些已知的相互关系,但慢性阻塞性肺病患者的心血管疾病仍被低估,且治疗不足。同样,慢性阻塞性肺病也是不良心血管(CV)事件的一个独立风险增强因素,但它并未被纳入当前的 CV 风险评估工具,从而导致认识不足和治疗不足。慢性阻塞性肺病的治疗亟需系统变革,从症状控制转向全面的心肺疾病模式,积极预防病情恶化、不良心肺结局和死亡率。然而,定义最佳心肺护理路径的证据还很匮乏。幸运的是,糖尿病领域已有支持系统层面变革的先例,即从血糖控制发展到全面的多器官风险评估和管理。其关键要素包括多学科综合护理、强化风险因素管理、初级和专科护理之间的协调、护理路径和协议、教育和自我管理以及疾病调整疗法。这篇评论文章将心血管代谢范例与心肺范例相提并论,并以糖尿病护理的演变为潜在框架,提出了向多学科综合心肺护理系统转变的理由。
{"title":"Is 'Cardiopulmonary' the New 'Cardiometabolic'? Making a Case for Systems Change in COPD.","authors":"Nathaniel M Hawkins, Alan Kaplan, Dennis T Ko, Erika Penz, Mohit Bhutani","doi":"10.1007/s41030-024-00270-2","DOIUrl":"10.1007/s41030-024-00270-2","url":null,"abstract":"<p><p>Chronic obstructive pulmonary disease (COPD) and cardiovascular disease (CVD) have a syndemic relationship with shared risk factors and complex interplay between genetic, environmental, socioeconomic, and pathophysiological mechanisms. CVD is among the most common comorbidities in patients with COPD and vice versa. Patients with COPD, irrespective of their disease severity, are at increased risk of CVD morbidity and mortality, driven in part by COPD exacerbations. Despite these known interrelationships, CVD is underestimated and undertreated in patients with COPD. Similarly, COPD is an independent risk-enhancing factor for adverse cardiovascular (CV) events, yet it is not incorporated into current CV risk assessment tools, leading to under-recognition and undertreatment. There is a pressing need for systems change in COPD management to move beyond symptom control towards a comprehensive cardiopulmonary disease paradigm with proactive prevention of exacerbations and adverse cardiopulmonary outcomes and mortality. However, there is a dearth of evidence defining optimal cardiopulmonary care pathways. Fortunately, there is a precedent to support systems-level change in the field of diabetes, which evolved from glycemic control to comprehensive multi-organ risk assessment and management. Key elements included integrated multidisciplinary care, intensive risk factor management, coordination between primary and specialist care, care pathways and protocols, education and self management, and disease-modifying therapies. This commentary article draws parallels between the cardiometabolic and cardiopulmonary paradigms and makes a case for systems change towards multidisciplinary, integrated cardiopulmonary care, using the evolution in diabetes care as a potential framework.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"363-376"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11573969/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142154890","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis. 关于囊性纤维化患者接受伊伐卡夫托治疗的长期持久性的回顾性纵向登记研究。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-09-12 DOI: 10.1007/s41030-024-00269-9
Christian Merlo, Teja Thorat, Lisa J McGarry, Christina V Scirica, Maral DerSarkissian, Catherine Nguyen, Yuqian M Gu, Aruna Muthukumar, Joe Healy, Jaime L Rubin, M Alan Brookhart

Introduction: Ivacaftor (IVA) has been shown to change the trajectory of cystic fibrosis (CF) disease progression by slowing the rate of lung function decline in clinical studies. Long-term real-world data help to confirm the durability of this response.

Methods: This non-interventional, longitudinal study used data from the US CF Foundation Patient Registry to describe the annualized rate of change in lung function in people with CF receiving IVA. The IVA-treated cohort included people with CF aged ≥ 6 years who had ≥ 1 CF transmembrane conductance regulator (CFTR)-gating mutation and initiated IVA between 31 January 2012 and 31 December 2018. An age-matched comparator cohort included people with CF heterozygous for the F508del-CFTR mutation and a minimal function mutation (R117H excluded) and had not received CFTR modulator therapy. Baseline characteristics were balanced using standardized mortality ratio (SMR) weights computed from estimated propensity scores. The annualized rate of change in percent predicted forced expiratory volume in 1 s (ppFEV1) was estimated over 5 years and used to calculate the relative annualized rate of change in lung function in the IVA-treated versus comparator cohorts.

Results: In the 5-year follow-up period, 548 people were in the IVA-treated and 541 in the comparator cohorts after SMR weighting. The annualized rate of change in ppFEV1 over 5 years was -1.23 (95% CI -1.45, -1.03) and -2.03 (-2.16, -1.90) percentage points in the IVA-treated and comparator cohorts, respectively. There was a 39% reduction (95% CI: 28, 50) in the rate of lung function decline in the IVA-treated versus comparator cohort over 5 years. Findings were generally consistent with those of shorter follow-up periods.

Conclusion: IVA showed a durable clinical benefit by slowing the rate of lung function decline over 5 years. Results support a sustained and consistent impact of IVA on lung function trajectory in people with CF. Word count: 300 (limit: 300 words).

简介在临床研究中,伊伐卡夫托(IVA)通过减缓肺功能下降的速度,改变了囊性纤维化(CF)疾病的发展轨迹。长期实际数据有助于证实这种反应的持久性:这项非干预性纵向研究利用美国CF基金会患者登记处的数据,描述了接受IVA治疗的CF患者肺功能的年变化率。接受IVA治疗的队列包括年龄≥6岁、CF跨膜传导调节器(CFTR)-门控突变≥1个且在2012年1月31日至2018年12月31日期间开始接受IVA治疗的CF患者。年龄匹配的参照队列包括杂合子F508del-CFTR突变和最小功能突变(R117H除外)且未接受过CFTR调节剂治疗的CF患者。根据估计的倾向评分计算出的标准化死亡率 (SMR) 权重平衡了基线特征。我们估算了5年中1 s内用力呼气容积预测值百分比(ppFEV1)的年化变化率,并以此计算了IVA治疗组与对照组的肺功能相对年化变化率:结果:在为期5年的随访中,经过SMR加权后,IVA治疗组有548人,对照组有541人。5年间,IVA治疗组和对照组的ppFEV1年化变化率分别为-1.23(95% CI -1.45, -1.03)和-2.03(-2.16, -1.90)个百分点。5年中,IVA治疗组与对照组相比,肺功能下降率降低了39%(95% CI:28,50)。这些结果与较短随访期的结果基本一致:IVA可在5年内减缓肺功能下降的速度,从而显示出持久的临床益处。结果表明,IVA对CF患者的肺功能轨迹具有持续、一致的影响。字数:300(字数限制:300字)。
{"title":"A Retrospective, Longitudinal Registry Study on the Long-Term Durability of Ivacaftor Treatment in People with Cystic Fibrosis.","authors":"Christian Merlo, Teja Thorat, Lisa J McGarry, Christina V Scirica, Maral DerSarkissian, Catherine Nguyen, Yuqian M Gu, Aruna Muthukumar, Joe Healy, Jaime L Rubin, M Alan Brookhart","doi":"10.1007/s41030-024-00269-9","DOIUrl":"10.1007/s41030-024-00269-9","url":null,"abstract":"<p><strong>Introduction: </strong>Ivacaftor (IVA) has been shown to change the trajectory of cystic fibrosis (CF) disease progression by slowing the rate of lung function decline in clinical studies. Long-term real-world data help to confirm the durability of this response.</p><p><strong>Methods: </strong>This non-interventional, longitudinal study used data from the US CF Foundation Patient Registry to describe the annualized rate of change in lung function in people with CF receiving IVA. The IVA-treated cohort included people with CF aged ≥ 6 years who had ≥ 1 CF transmembrane conductance regulator (CFTR)-gating mutation and initiated IVA between 31 January 2012 and 31 December 2018. An age-matched comparator cohort included people with CF heterozygous for the F508del-CFTR mutation and a minimal function mutation (R117H excluded) and had not received CFTR modulator therapy. Baseline characteristics were balanced using standardized mortality ratio (SMR) weights computed from estimated propensity scores. The annualized rate of change in percent predicted forced expiratory volume in 1 s (ppFEV<sub>1</sub>) was estimated over 5 years and used to calculate the relative annualized rate of change in lung function in the IVA-treated versus comparator cohorts.</p><p><strong>Results: </strong>In the 5-year follow-up period, 548 people were in the IVA-treated and 541 in the comparator cohorts after SMR weighting. The annualized rate of change in ppFEV<sub>1</sub> over 5 years was -1.23 (95% CI -1.45, -1.03) and -2.03 (-2.16, -1.90) percentage points in the IVA-treated and comparator cohorts, respectively. There was a 39% reduction (95% CI: 28, 50) in the rate of lung function decline in the IVA-treated versus comparator cohort over 5 years. Findings were generally consistent with those of shorter follow-up periods.</p><p><strong>Conclusion: </strong>IVA showed a durable clinical benefit by slowing the rate of lung function decline over 5 years. Results support a sustained and consistent impact of IVA on lung function trajectory in people with CF. Word count: 300 (limit: 300 words).</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"483-494"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11573995/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142294087","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Efficacy of High Flow Nasal Cannula in the Treatment of Patients with COVID-19 with Acute Respiratory Distress Syndrome: Results of Single Centre Study in Vietnam. 高流量鼻导管治疗 COVID-19 急性呼吸窘迫综合征患者的疗效:越南单中心研究结果
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-10-18 DOI: 10.1007/s41030-024-00272-0
Sy Duong-Quy, Duc Huynh-Truong-Anh, Tram Tang-Thi-Thao, Thu Nguyen-Ngoc-Phuong, Phung Hoang-Phi-Tuyet, Anh Nguyen-Tuan, Toi Nguyen-Van, Thanh Nguyen-Chi, Thanh Nguyen-Thi-Kim, Tien Nguyen-Quang, Thuy Tran-Ngoc-Anh, Nam Nguyen-Van-Hoai, Mai Do-Thi-Thu, Huong Hoang-Thi-Xuan, Thai Nguyen-Duy, Cong Nguyen-Hai, Tuan Huynh-Anh, Quan Vu-Tran-Thien, Khue Bui-Diem, Giang Nguyen-Mong, Hieu Nguyen-Lan, Giap Vu-Van, Phuong Phan-Thu, Long Nguyen-Viet, Chuong Nguyen-Hong, Sy Dinh-Ngoc, Trong Nguyen-Duc, Dung Truong-Viet, Thu Vo-Pham-Minh, Bao Le-Khac, Duc Nguyen-Hong, Timothy Craig, Vinh Nguyen-Nhu

Introduction: Most hospitalized patients required invasive or non-invasive ventilation and High Flow Nasal Cannula (HFNC). Therefore, this study was conducted to describe the characteristics of patients with severe Coronavirus Disease-2019 (COVID-19) treated by HFNC and its effectiveness for reducing the rate of intubated-mechanical ventilation in the Intensive Care Unit (ICU) of Phu Chanh COVID-19 Department-Binh Duong General Hospital.

Methods: It was a cross-sectional and descriptive study. All severe patients with COVID-19 with acute respiratory failure eligible for the study were included. Patient characteristics, clinical symptoms, laboratory results, and treatment methods were collected for analysis; parameters and data related to HFNC treatment and follow-up were analysed.

Results: 80 patients, aged of 49.7 ± 16.6 years, were treated with HFNC at admission in ICU. 14 patients had type 2 diabetes (17.5%), 3 patients had chronic respiratory disease (3.8%), 19 patients had high blood pressure (23.8%), and 5 patients with other comorbidities (7.4%). The majority of patients with severe COVID-19 had typical symptoms of COVID-19 such as shortness of breath (97.5%), intensive tired (81.3%), cough (73.7%), anosmia (48.3%), ageusia (41.3%), and fever (26.3%). The results of arterial blood gases demonstrated severe hypoxia under optimal conventional oxygen therapy (PaO2 = 52.5 ± 17.4 mmHg). Respiratory rate, SpO2, PaO2 were significantly improved after using HFNC at 1st day, 3rd day and 7th day (P < 0.05; P < 0.05; P < 0.01; respectively). Receiver operating characteristics (ROC) index was significantly increased after treating with HFNC vs before HFNC treatment (4.79 ± 1.86, 5.53 ± 2.39, and 7.41 ± 4.24 vs 2.97 ± 0.39; P < 0.05, P < 0.05 and P < 0.01, respectively). 54 (67.5%) patients were success with HFNC treatment and 26 (32.5%) patients with HFNC failure needed to treat with Continuous Positive Airway Pressure (CPAP) (13 patients; 50%) or intubated ventilation (13 patients; 50%).

Conclusion: HFNC therapy could be considered as a useful and effective alternative treatment for patients with acute respiratory failure. HFNC might help to delay the intubated ventilation for patients with respiratory failure and to minimise the risk of invasive ventilation complications and mortality. However, it is crucial to closely monitor the evolution of patient's respiratory status and responsiveness of HFNC treatment to avoid unintended delay of intubation-mechanical ventilation.

Trial registration: An independent ethics committee approved the study (The Ethics Committee of Binh Duong General Hospital; No. HDDD-BVDK BINH DUONG 9.2021), which was performed in accordance with the Declaration of Helsinki, Guidelines for Good Clinical Practice.

简介大多数住院患者需要有创或无创通气和高流量鼻导管(HFNC)。因此,本研究旨在描述在 Phu Chanh COVID-19 部门--平阳综合医院重症监护室(ICU)接受高流量鼻导管治疗的重症冠状病毒病-2019(COVID-19)患者的特征及其对降低插管-机械通气率的有效性:这是一项横断面描述性研究。方法:这是一项横断面描述性研究,纳入了所有符合研究条件的 COVID-19 急性呼吸衰竭重症患者。收集患者特征、临床症状、实验室结果和治疗方法进行分析;分析与 HFNC 治疗和随访相关的参数和数据:80名患者(年龄为49.7±16.6岁)在入住重症监护室时接受了HFNC治疗。14名患者患有2型糖尿病(17.5%),3名患者患有慢性呼吸系统疾病(3.8%),19名患者患有高血压(23.8%),5名患者患有其他合并症(7.4%)。大多数重症 COVID-19 患者都有 COVID-19 的典型症状,如气短(97.5%)、极度疲倦(81.3%)、咳嗽(73.7%)、无嗅(48.3%)、衰老(41.3%)和发热(26.3%)。动脉血气结果显示,在最佳常规氧疗条件下(PaO2 = 52.5 ± 17.4 mmHg)缺氧严重。使用 HFNC 后,呼吸频率、SpO2、PaO2 在第 1 天、第 3 天和第 7 天均有明显改善(P 结论:HFNC 可被视为一种有效的治疗方法:对于急性呼吸衰竭患者,HFNC疗法可被视为一种有用且有效的替代疗法。HFNC 可能有助于推迟呼吸衰竭患者的插管通气时间,并将有创通气并发症和死亡风险降至最低。然而,密切监测患者呼吸状况的变化和对 HFNC 治疗的反应至关重要,以避免意外延迟插管-机械通气:独立伦理委员会批准了该研究(平阳总医院伦理委员会;编号:HDD-BVDK BINH DUONG 9.2021),该研究符合《赫尔辛基宣言》和《良好临床实践指南》。
{"title":"Efficacy of High Flow Nasal Cannula in the Treatment of Patients with COVID-19 with Acute Respiratory Distress Syndrome: Results of Single Centre Study in Vietnam.","authors":"Sy Duong-Quy, Duc Huynh-Truong-Anh, Tram Tang-Thi-Thao, Thu Nguyen-Ngoc-Phuong, Phung Hoang-Phi-Tuyet, Anh Nguyen-Tuan, Toi Nguyen-Van, Thanh Nguyen-Chi, Thanh Nguyen-Thi-Kim, Tien Nguyen-Quang, Thuy Tran-Ngoc-Anh, Nam Nguyen-Van-Hoai, Mai Do-Thi-Thu, Huong Hoang-Thi-Xuan, Thai Nguyen-Duy, Cong Nguyen-Hai, Tuan Huynh-Anh, Quan Vu-Tran-Thien, Khue Bui-Diem, Giang Nguyen-Mong, Hieu Nguyen-Lan, Giap Vu-Van, Phuong Phan-Thu, Long Nguyen-Viet, Chuong Nguyen-Hong, Sy Dinh-Ngoc, Trong Nguyen-Duc, Dung Truong-Viet, Thu Vo-Pham-Minh, Bao Le-Khac, Duc Nguyen-Hong, Timothy Craig, Vinh Nguyen-Nhu","doi":"10.1007/s41030-024-00272-0","DOIUrl":"10.1007/s41030-024-00272-0","url":null,"abstract":"<p><strong>Introduction: </strong>Most hospitalized patients required invasive or non-invasive ventilation and High Flow Nasal Cannula (HFNC). Therefore, this study was conducted to describe the characteristics of patients with severe Coronavirus Disease-2019 (COVID-19) treated by HFNC and its effectiveness for reducing the rate of intubated-mechanical ventilation in the Intensive Care Unit (ICU) of Phu Chanh COVID-19 Department-Binh Duong General Hospital.</p><p><strong>Methods: </strong>It was a cross-sectional and descriptive study. All severe patients with COVID-19 with acute respiratory failure eligible for the study were included. Patient characteristics, clinical symptoms, laboratory results, and treatment methods were collected for analysis; parameters and data related to HFNC treatment and follow-up were analysed.</p><p><strong>Results: </strong>80 patients, aged of 49.7 ± 16.6 years, were treated with HFNC at admission in ICU. 14 patients had type 2 diabetes (17.5%), 3 patients had chronic respiratory disease (3.8%), 19 patients had high blood pressure (23.8%), and 5 patients with other comorbidities (7.4%). The majority of patients with severe COVID-19 had typical symptoms of COVID-19 such as shortness of breath (97.5%), intensive tired (81.3%), cough (73.7%), anosmia (48.3%), ageusia (41.3%), and fever (26.3%). The results of arterial blood gases demonstrated severe hypoxia under optimal conventional oxygen therapy (PaO<sub>2</sub> = 52.5 ± 17.4 mmHg). Respiratory rate, SpO2, PaO2 were significantly improved after using HFNC at 1st day, 3rd day and 7th day (P < 0.05; P < 0.05; P < 0.01; respectively). Receiver operating characteristics (ROC) index was significantly increased after treating with HFNC vs before HFNC treatment (4.79 ± 1.86, 5.53 ± 2.39, and 7.41 ± 4.24 vs 2.97 ± 0.39; P < 0.05, P < 0.05 and P < 0.01, respectively). 54 (67.5%) patients were success with HFNC treatment and 26 (32.5%) patients with HFNC failure needed to treat with Continuous Positive Airway Pressure (CPAP) (13 patients; 50%) or intubated ventilation (13 patients; 50%).</p><p><strong>Conclusion: </strong>HFNC therapy could be considered as a useful and effective alternative treatment for patients with acute respiratory failure. HFNC might help to delay the intubated ventilation for patients with respiratory failure and to minimise the risk of invasive ventilation complications and mortality. However, it is crucial to closely monitor the evolution of patient's respiratory status and responsiveness of HFNC treatment to avoid unintended delay of intubation-mechanical ventilation.</p><p><strong>Trial registration: </strong>An independent ethics committee approved the study (The Ethics Committee of Binh Duong General Hospital; No. HDDD-BVDK BINH DUONG 9.2021), which was performed in accordance with the Declaration of Helsinki, Guidelines for Good Clinical Practice.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"451-468"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11574233/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142473398","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Patient Profile-Based Management with Nintedanib in Patients with Idiopathic Pulmonary Fibrosis. 在特发性肺纤维化患者中使用 Nintedanib 进行基于患者特征的管理
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-09-28 DOI: 10.1007/s41030-024-00271-1
Vinod K Viswanathan, Aloke G Ghoshal, Anant Mohan, Ketaki Patil, Chaitanya Bhargave, Sanjay Choudhari, Suyog Mehta

A severe and progressive interstitial lung disease (ILD) known as idiopathic pulmonary fibrosis (IPF) has an unknown etiology with poorly defined mechanisms of development. Among the currently prescribed pharmacological interventions for IPF, nintedanib demonstrates the ability to decelerate the deterioration of lung function and yield positive clinical outcomes. Multiple randomized placebo-controlled trials have confirmed the efficacy and acceptable safety profile of nintedanib. Real-world evidence studies also support the use of nintedanib in IPF, being an efficient and well-tolerated treatment option. It has the potential to stabilize the disease progression in patients with ILD. Patients with IPF frequently have comorbidities like diabetes and hypertension, which can exacerbate the course of disease, reduce quality of life, and decrease treatment adherence. For well-informed decision-making, it is important for healthcare professionals to recognize the position of nintedanib therapy in IPF with comorbidities. The gastrointestinal adverse effects, notably diarrhea, dominate the nintedanib safety profile. These can be effectively controlled by closely monitoring side effects, administering anti-diarrheal and anti-emetic drugs, reducing the nintedanib dose, and discontinuing it in case of severe symptoms with an option to reintroduce the treatment after side effects subside. Symptomatic interventions and monitoring of liver enzymes may reduce the occurrence of permanent treatment discontinuations.

特发性肺纤维化(IPF)是一种严重的进行性间质性肺病(ILD),病因不明,发病机制不清。在目前治疗 IPF 的药物干预中,宁替达尼(nintedanib)能够减缓肺功能的恶化,并产生积极的临床疗效。多项随机安慰剂对照试验证实了宁替尼的疗效和可接受的安全性。真实世界的证据研究也支持在 IPF 中使用宁替达尼,它是一种高效且耐受性良好的治疗选择。它有可能稳定 ILD 患者的疾病进展。IPF 患者常合并糖尿病和高血压等疾病,这可能会加重病程、降低生活质量并降低治疗依从性。为了做出明智的决策,医护人员必须认识到宁替尼治疗合并症的 IPF 的重要性。胃肠道不良反应,尤其是腹泻,是宁替尼安全性的主要表现。通过密切监测副作用、服用止泻和止吐药物、减少宁替达尼的剂量以及在症状严重时停药,并在副作用缓解后重新开始治疗,可以有效控制这些不良反应。对症干预和监测肝酶可减少永久性停药的发生。
{"title":"Patient Profile-Based Management with Nintedanib in Patients with Idiopathic Pulmonary Fibrosis.","authors":"Vinod K Viswanathan, Aloke G Ghoshal, Anant Mohan, Ketaki Patil, Chaitanya Bhargave, Sanjay Choudhari, Suyog Mehta","doi":"10.1007/s41030-024-00271-1","DOIUrl":"10.1007/s41030-024-00271-1","url":null,"abstract":"<p><p>A severe and progressive interstitial lung disease (ILD) known as idiopathic pulmonary fibrosis (IPF) has an unknown etiology with poorly defined mechanisms of development. Among the currently prescribed pharmacological interventions for IPF, nintedanib demonstrates the ability to decelerate the deterioration of lung function and yield positive clinical outcomes. Multiple randomized placebo-controlled trials have confirmed the efficacy and acceptable safety profile of nintedanib. Real-world evidence studies also support the use of nintedanib in IPF, being an efficient and well-tolerated treatment option. It has the potential to stabilize the disease progression in patients with ILD. Patients with IPF frequently have comorbidities like diabetes and hypertension, which can exacerbate the course of disease, reduce quality of life, and decrease treatment adherence. For well-informed decision-making, it is important for healthcare professionals to recognize the position of nintedanib therapy in IPF with comorbidities. The gastrointestinal adverse effects, notably diarrhea, dominate the nintedanib safety profile. These can be effectively controlled by closely monitoring side effects, administering anti-diarrheal and anti-emetic drugs, reducing the nintedanib dose, and discontinuing it in case of severe symptoms with an option to reintroduce the treatment after side effects subside. Symptomatic interventions and monitoring of liver enzymes may reduce the occurrence of permanent treatment discontinuations.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"377-409"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11573957/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142352768","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Do Antidepressants Worsen COPD Outcomes in Depressed Patients with COPD? 抗抑郁药是否会恶化慢性阻塞性肺疾病抑郁患者的预后?
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-11-08 DOI: 10.1007/s41030-024-00277-9
Alan G Kaplan

The coexistence of depression with chronic obstructive pulmonary disease (COPD) has been associated with poorer outcomes. Studies have questioned the safety of antidepressants in patients with COPD. This review shows the potential relationships and the possible mechanisms and gives us good warnings on how to approach this problem. Treatment should be both non-pharmacological and pharmacological, but importantly tailored to the individual patient.

抑郁症与慢性阻塞性肺病(COPD)并存与较差的预后有关。有研究对慢性阻塞性肺病患者服用抗抑郁药的安全性提出了质疑。这篇综述展示了潜在的关系和可能的机制,并就如何解决这一问题向我们提出了很好的警示。治疗应既包括非药物治疗,也包括药物治疗,但重要的是要因人而异。
{"title":"Do Antidepressants Worsen COPD Outcomes in Depressed Patients with COPD?","authors":"Alan G Kaplan","doi":"10.1007/s41030-024-00277-9","DOIUrl":"10.1007/s41030-024-00277-9","url":null,"abstract":"<p><p>The coexistence of depression with chronic obstructive pulmonary disease (COPD) has been associated with poorer outcomes. Studies have questioned the safety of antidepressants in patients with COPD. This review shows the potential relationships and the possible mechanisms and gives us good warnings on how to approach this problem. Treatment should be both non-pharmacological and pharmacological, but importantly tailored to the individual patient.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"411-426"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11574234/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142626899","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators. 国家囊性纤维化患者登记处报告质量的比较:确定新型 CFTR 调节剂使用情况的意义。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-12-01 Epub Date: 2024-09-27 DOI: 10.1007/s41030-024-00274-y
Owen W Tomlinson, Philip Mitchelmore, Craig A Williams

Introduction: Advances in development of cystic fibrosis transmembrane conductance regulator modulator (CFTRm) therapies mean that now people who are heterozygous (instead of having to be homozygous) for the common F508del variant can benefit from these therapies. Recent economic estimates suggest only approximately 15% of the global population have CFTRm access, yet it is unknown how prevalence of F508del and economic factors may affect this availability.

Methods: Data related to prevalence of cystic fibrosis (CF), CFTRm usage, and prevalence of F508del in 10 countries were extracted from publicly accessible registry reports from 2021. National gross domestic product (GDP) was obtained via open access World Bank data. Descriptive statistics and correlation coefficients assessed relationships.

Results: Notable discrepancies were noted in the equity of availability of data between national registries-only four countries reported number of patients eligible for CFTRm. Registry data represented 70,694 patients, with 42,858 found to be using CFTRm (60.6%). Prevalence of CFTRm usage ranged from 1.8% to 76.7% and prevalence of F508del ranged from 35.2% to 94.4%. The correlation between prevalence of CFTRm usage and F508del is positive (r = 0.56, p = 0.10), and the correlation between CFTRm usage and GDP (per capita) was also positive, and significant (r = 0.72, p = 0.02).

Conclusion: Both F508del prevalence and GDP are associated with variable CFTRm usage rates, although a predominant reason is unclear as a result of poor consistency in registry reporting. Urgent action is needed to create uniform reporting of registry data and increase availability of novel CFTRm therapies to the global CF population.

导言:囊性纤维化跨膜传导调节器(CFTRm)疗法的研发取得了进展,这意味着现在常见的 F508del 变异杂合子(而非必须是同种杂合子)患者也能从这些疗法中获益。最近的经济估算表明,全球仅有约 15% 的人可以使用 CFTRm,但 F508del 的流行率和经济因素会如何影响 CFTRm 的可用性尚不得而知:从 2021 年可公开获取的登记报告中提取了 10 个国家的囊性纤维化(CF)患病率、CFTRm 使用率和 F508del 患病率的相关数据。国家国内生产总值(GDP)通过开放访问的世界银行数据获得。描述性统计和相关系数评估了两者之间的关系:结果表明:各国登记处数据的可用性存在明显差异,只有四个国家报告了符合 CFTRm 治疗条件的患者人数。登记数据代表了 70,694 名患者,其中 42,858 人使用了 CFTRm(60.6%)。CFTRm的使用率从1.8%到76.7%不等,F508del的使用率从35.2%到94.4%不等。CFTRm使用率与F508del之间呈正相关(r = 0.56,p = 0.10),CFTRm使用率与GDP(人均)之间也呈正相关,且差异显著(r = 0.72,p = 0.02):结论:F508del发病率和国内生产总值都与不同的CFTRm使用率有关,但由于登记报告的一致性较差,主要原因尚不清楚。需要采取紧急措施统一登记数据报告,并增加全球 CF 患者对新型 CFTRm 疗法的使用。
{"title":"Comparison of Reporting Quality in National Cystic Fibrosis Patient Registries: Implications for Identifying Use of Novel CFTR Modulators.","authors":"Owen W Tomlinson, Philip Mitchelmore, Craig A Williams","doi":"10.1007/s41030-024-00274-y","DOIUrl":"10.1007/s41030-024-00274-y","url":null,"abstract":"<p><strong>Introduction: </strong>Advances in development of cystic fibrosis transmembrane conductance regulator modulator (CFTRm) therapies mean that now people who are heterozygous (instead of having to be homozygous) for the common F508del variant can benefit from these therapies. Recent economic estimates suggest only approximately 15% of the global population have CFTRm access, yet it is unknown how prevalence of F508del and economic factors may affect this availability.</p><p><strong>Methods: </strong>Data related to prevalence of cystic fibrosis (CF), CFTRm usage, and prevalence of F508del in 10 countries were extracted from publicly accessible registry reports from 2021. National gross domestic product (GDP) was obtained via open access World Bank data. Descriptive statistics and correlation coefficients assessed relationships.</p><p><strong>Results: </strong>Notable discrepancies were noted in the equity of availability of data between national registries-only four countries reported number of patients eligible for CFTRm. Registry data represented 70,694 patients, with 42,858 found to be using CFTRm (60.6%). Prevalence of CFTRm usage ranged from 1.8% to 76.7% and prevalence of F508del ranged from 35.2% to 94.4%. The correlation between prevalence of CFTRm usage and F508del is positive (r = 0.56, p = 0.10), and the correlation between CFTRm usage and GDP (per capita) was also positive, and significant (r = 0.72, p = 0.02).</p><p><strong>Conclusion: </strong>Both F508del prevalence and GDP are associated with variable CFTRm usage rates, although a predominant reason is unclear as a result of poor consistency in registry reporting. Urgent action is needed to create uniform reporting of registry data and increase availability of novel CFTRm therapies to the global CF population.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"427-438"},"PeriodicalIF":2.3,"publicationDate":"2024-12-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11574223/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142352767","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
Expert Panel Consensus Recommendations for Allergic Rhinitis in Patients with Asthma in India. 印度哮喘患者过敏性鼻炎的专家小组共识建议。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-10-17 DOI: 10.1007/s41030-024-00273-z
R Narasimhan, Sitesh Roy, Meghanadh Koralla, P K Thomas, M Ilambarathi, S Balamurugan, M Harish, R Sabarinath, Gaurav Medikeri, Partha Bose, V R Pattabhiraman, M K Rajasekar, A R Gayathri, T Dhanasekar, V Nandagopal, G Gananathan, S K Ravichandran, M N Shankar, Aniruddha Majumder, Shelley Shamim, Meenesh Juvekar, Vijay K Singh, T Mohankumar, S Prasanna Kumar, Debraj Jash, Salil Bendre, Suhail Neliyathodi, Sunil Janardanan Unnithan, Archana Karadkhele

Allergic rhinitis and asthma are commonly coexisting conditions, significantly impacting patient health and quality of life. Despite their interrelation, diagnosing allergic rhinitis in patients with asthma remains challenging, leading to underdiagnosis and suboptimal management. The expert consensus engaged a modified Delphi method involving 29 experts including pulmonologists, ear, nose, and throat surgeons, and allergologists. Through group discussions, consensus statements were developed regarding the epidemiology, diagnosis, and management of allergic rhinitis and asthma. Final consensus statements were formulated based on the experts' collective clinical judgment and experience. This expert consensus provides updated recommendations tailored to the Indian context, addressing the gaps in existing research and clinical practice. By promoting a systematic and evidence-based approach to diagnosis and management, this consensus aims to support clinicians in effectively identifying and treating allergic rhinitis in patients with asthma, thereby improving overall disease management and patient well-being.

过敏性鼻炎和哮喘通常同时存在,严重影响患者的健康和生活质量。尽管过敏性鼻炎和哮喘相互关联,但诊断哮喘患者的过敏性鼻炎仍具有挑战性,导致诊断不足和治疗效果不佳。专家共识采用了改良的德尔菲法,共有 29 位专家参与,包括肺科专家、耳鼻喉外科医生和过敏学专家。通过小组讨论,就过敏性鼻炎和哮喘的流行病学、诊断和管理达成了共识。最终的共识声明是根据专家们的集体临床判断和经验制定的。这份专家共识提供了适合印度国情的最新建议,弥补了现有研究和临床实践中的不足。通过推广系统化的循证诊断和管理方法,本共识旨在支持临床医生有效识别和治疗哮喘患者的过敏性鼻炎,从而改善整体疾病管理和患者福祉。
{"title":"Expert Panel Consensus Recommendations for Allergic Rhinitis in Patients with Asthma in India.","authors":"R Narasimhan, Sitesh Roy, Meghanadh Koralla, P K Thomas, M Ilambarathi, S Balamurugan, M Harish, R Sabarinath, Gaurav Medikeri, Partha Bose, V R Pattabhiraman, M K Rajasekar, A R Gayathri, T Dhanasekar, V Nandagopal, G Gananathan, S K Ravichandran, M N Shankar, Aniruddha Majumder, Shelley Shamim, Meenesh Juvekar, Vijay K Singh, T Mohankumar, S Prasanna Kumar, Debraj Jash, Salil Bendre, Suhail Neliyathodi, Sunil Janardanan Unnithan, Archana Karadkhele","doi":"10.1007/s41030-024-00273-z","DOIUrl":"10.1007/s41030-024-00273-z","url":null,"abstract":"<p><p>Allergic rhinitis and asthma are commonly coexisting conditions, significantly impacting patient health and quality of life. Despite their interrelation, diagnosing allergic rhinitis in patients with asthma remains challenging, leading to underdiagnosis and suboptimal management. The expert consensus engaged a modified Delphi method involving 29 experts including pulmonologists, ear, nose, and throat surgeons, and allergologists. Through group discussions, consensus statements were developed regarding the epidemiology, diagnosis, and management of allergic rhinitis and asthma. Final consensus statements were formulated based on the experts' collective clinical judgment and experience. This expert consensus provides updated recommendations tailored to the Indian context, addressing the gaps in existing research and clinical practice. By promoting a systematic and evidence-based approach to diagnosis and management, this consensus aims to support clinicians in effectively identifying and treating allergic rhinitis in patients with asthma, thereby improving overall disease management and patient well-being.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-10-17","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142473399","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
A PrOsPective Cohort Study on Interstitial Lung Disease-Associated Pulmonary Hypertension with a ParticulaR Focus on the Subset with Pulmonary Arterial Hypertension Features (POPLAR Study). 间质性肺病相关性肺动脉高压的队列研究,重点关注具有肺动脉高压特征的人群(POPLAR 研究)。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-09-01 Epub Date: 2024-06-24 DOI: 10.1007/s41030-024-00264-0
Ichizo Tsujino, Kazuki Kitahara, Junichi Omura, Toshiyuki Iwahori, Satoshi Konno

Introduction: The pathogenesis and clinical profiles of patients with pulmonary hypertension (PH) associated with interstitial lung disease (ILD-PH) are poorly understood. Whether and to what extent pulmonary arterial hypertension (PAH)-specific therapy improves hemodynamic and outcome in ILD-PH are also unknown.

Study objective: This study aims to clarify the characteristics, clinical course and response to PAH-specific therapy of ILD and/or PH by enrolling three unique subsets: PAH, ILD-PH, and ILD.

Methods: The proposed study is a retrospective and prospective, multi-centre, observational cohort study of patients treated at any of three university hospitals in the Hokkaido region of Japan who have any one of the following: PAH; ILD-PH with or without PAH features; or ILD without PH. We aim to enrol 250 patients in total. For the retrospective observation period, data obtained after 1 January 2010, will be analysed, and the prospective observation period will be 1 year. We will compare the clinical data of patients with ILD-PH with those of patients with PAH and those of patients with ILD without PH in the real-world clinical setting. In addition, within the cohort of patients with ILD-PH, we will explore the subset with "ILD-PH with PAH features" and compare the response to PAH-specific therapy with that of PAH. The primary outcome will be the change in pulmonary vascular resistance from first treatment to follow-up in patients with PAH and ILD-PH with PAH features (excluding ILD-PH without PAH feature and ILD-no-PH for the primary outcome). The exploratory outcomes will include analyses of PH-associated biomarkers, right ventricular function and patient-reported outcomes.

Results: This is a protocol article and the results will be presented after data collection is completed.

Conclusion: The POPLAR study will provide data that help better understand the pathophysiology of ILD-PH and improve the quality of life and outcome of patients with PH and/or ILD.

Trial registration: Japan Registry of Clinical Trials: jRCT1010230018.

导言:人们对肺动脉高压(PAH)伴间质性肺病(ILD-PH)患者的发病机制和临床特征知之甚少。肺动脉高压(PAH)特异性治疗是否以及在多大程度上能改善 ILD-PH 患者的血液动力学和预后也不得而知:本研究旨在通过纳入三个独特的子集,阐明 ILD 和/或 PH 的特征、临床过程以及对 PAH 特异性治疗的反应:研究目的:本研究旨在通过纳入三个独特的子集:PAH、ILD-PH 和 ILD,明确 PAH 特异性治疗的临床过程和反应:拟议的研究是一项回顾性和前瞻性、多中心、观察性队列研究,研究对象是在日本北海道地区三家大学医院中接受治疗的患者,这些患者患有以下任何一种疾病:PAH;具有或不具有 PAH 特征的 ILD-PH;或不具有 PH 特征的 ILD。我们的目标是招募 250 名患者。回顾性观察期将分析 2010 年 1 月 1 日之后获得的数据,前瞻性观察期为 1 年。我们将把 ILD-PH 患者的临床数据与 PAH 患者和无 PH 的 ILD 患者的临床数据进行比较。此外,在 ILD-PH 患者队列中,我们还将探索 "具有 PAH 特征的 ILD-PH 患者 "子集,并比较 PAH 患者与 PAH 患者对 PAH 特异性治疗的反应。主要结果将是 PAH 患者和具有 PAH 特征的 ILD-PH 患者从首次治疗到随访期间肺血管阻力的变化(主要结果不包括无 PAH 特征的 ILD-PH 和 ILD-no-PH)。探索性结果将包括 PH 相关生物标志物、右心室功能和患者报告结果的分析:本文为协议文章,结果将在数据收集完成后公布:POPLAR研究将提供数据,帮助更好地了解ILD-PH的病理生理学,改善PH和/或ILD患者的生活质量和预后:试验注册:日本临床试验注册中心:jRCT1010230018。
{"title":"A PrOsPective Cohort Study on Interstitial Lung Disease-Associated Pulmonary Hypertension with a ParticulaR Focus on the Subset with Pulmonary Arterial Hypertension Features (POPLAR Study).","authors":"Ichizo Tsujino, Kazuki Kitahara, Junichi Omura, Toshiyuki Iwahori, Satoshi Konno","doi":"10.1007/s41030-024-00264-0","DOIUrl":"10.1007/s41030-024-00264-0","url":null,"abstract":"<p><strong>Introduction: </strong>The pathogenesis and clinical profiles of patients with pulmonary hypertension (PH) associated with interstitial lung disease (ILD-PH) are poorly understood. Whether and to what extent pulmonary arterial hypertension (PAH)-specific therapy improves hemodynamic and outcome in ILD-PH are also unknown.</p><p><strong>Study objective: </strong>This study aims to clarify the characteristics, clinical course and response to PAH-specific therapy of ILD and/or PH by enrolling three unique subsets: PAH, ILD-PH, and ILD.</p><p><strong>Methods: </strong>The proposed study is a retrospective and prospective, multi-centre, observational cohort study of patients treated at any of three university hospitals in the Hokkaido region of Japan who have any one of the following: PAH; ILD-PH with or without PAH features; or ILD without PH. We aim to enrol 250 patients in total. For the retrospective observation period, data obtained after 1 January 2010, will be analysed, and the prospective observation period will be 1 year. We will compare the clinical data of patients with ILD-PH with those of patients with PAH and those of patients with ILD without PH in the real-world clinical setting. In addition, within the cohort of patients with ILD-PH, we will explore the subset with \"ILD-PH with PAH features\" and compare the response to PAH-specific therapy with that of PAH. The primary outcome will be the change in pulmonary vascular resistance from first treatment to follow-up in patients with PAH and ILD-PH with PAH features (excluding ILD-PH without PAH feature and ILD-no-PH for the primary outcome). The exploratory outcomes will include analyses of PH-associated biomarkers, right ventricular function and patient-reported outcomes.</p><p><strong>Results: </strong>This is a protocol article and the results will be presented after data collection is completed.</p><p><strong>Conclusion: </strong>The POPLAR study will provide data that help better understand the pathophysiology of ILD-PH and improve the quality of life and outcome of patients with PH and/or ILD.</p><p><strong>Trial registration: </strong>Japan Registry of Clinical Trials: jRCT1010230018.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"297-313"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11339191/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141443205","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":0,"RegionCategory":"","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}
引用次数: 0
期刊
Pulmonary Therapy
全部 Acc. Chem. Res. ACS Applied Bio Materials ACS Appl. Electron. Mater. ACS Appl. Energy Mater. ACS Appl. Mater. Interfaces ACS Appl. Nano Mater. ACS Appl. Polym. Mater. ACS BIOMATER-SCI ENG ACS Catal. ACS Cent. Sci. ACS Chem. Biol. ACS Chemical Health & Safety ACS Chem. Neurosci. ACS Comb. Sci. ACS Earth Space Chem. ACS Energy Lett. ACS Infect. Dis. ACS Macro Lett. ACS Mater. Lett. ACS Med. Chem. Lett. ACS Nano ACS Omega ACS Photonics ACS Sens. ACS Sustainable Chem. Eng. ACS Synth. Biol. Anal. Chem. BIOCHEMISTRY-US Bioconjugate Chem. BIOMACROMOLECULES Chem. Res. Toxicol. Chem. Rev. Chem. Mater. CRYST GROWTH DES ENERG FUEL Environ. Sci. Technol. Environ. Sci. Technol. Lett. Eur. J. Inorg. Chem. IND ENG CHEM RES Inorg. Chem. J. Agric. Food. Chem. J. Chem. Eng. Data J. Chem. Educ. J. Chem. Inf. Model. J. Chem. Theory Comput. J. Med. Chem. J. Nat. Prod. J PROTEOME RES J. Am. Chem. Soc. LANGMUIR MACROMOLECULES Mol. Pharmaceutics Nano Lett. Org. Lett. ORG PROCESS RES DEV ORGANOMETALLICS J. Org. Chem. J. Phys. Chem. J. Phys. Chem. A J. Phys. Chem. B J. Phys. Chem. C J. Phys. Chem. Lett. Analyst Anal. Methods Biomater. Sci. Catal. Sci. Technol. Chem. Commun. Chem. Soc. Rev. CHEM EDUC RES PRACT CRYSTENGCOMM Dalton Trans. Energy Environ. Sci. ENVIRON SCI-NANO ENVIRON SCI-PROC IMP ENVIRON SCI-WAT RES Faraday Discuss. Food Funct. Green Chem. Inorg. Chem. Front. Integr. Biol. J. Anal. At. Spectrom. J. Mater. Chem. A J. Mater. Chem. B J. Mater. Chem. C Lab Chip Mater. Chem. Front. Mater. Horiz. MEDCHEMCOMM Metallomics Mol. Biosyst. Mol. Syst. Des. Eng. Nanoscale Nanoscale Horiz. Nat. Prod. Rep. New J. Chem. Org. Biomol. Chem. Org. Chem. Front. PHOTOCH PHOTOBIO SCI PCCP Polym. Chem.
×
引用
GB/T 7714-2015
复制
MLA
复制
APA
复制
导出至
BibTeX EndNote RefMan NoteFirst NoteExpress
×
0
微信
客服QQ
Book学术公众号 扫码关注我们
反馈
×
意见反馈
请填写您的意见或建议
请填写您的手机或邮箱
×
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
现在去查看 取消
×
提示
确定
Book学术官方微信
Book学术文献互助
Book学术文献互助群
群 号:481959085
Book学术
文献互助 智能选刊 最新文献 互助须知 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 Book学术 All rights reserved.
ghs 京公网安备 11010802042870号 京ICP备2023020795号-1