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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。
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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)。
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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》。
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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。
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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。
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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。
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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。
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引用次数: 0
Exploring Clinical Remission in Moderate Asthma - Perspectives from Asia, the Middle East, and South America. 探索中度哮喘的临床缓解--来自亚洲、中东和南美洲的视角。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-09-01 Epub Date: 2024-06-04 DOI: 10.1007/s41030-024-00262-2
Kittipong Maneechotesuwan, Bhumika Aggarwal, Gabriel Garcia, Daniel Tan, Hugo Neffen, Ramon Jason M Javier, Mona Al-Ahmad, Mousa Khadada, Vu Tran Thien Quan, Krittika Teerapuncharoen, Mario Soto Ramos, Gur Levy, Maximilian Plank, Abhay Phansalkar, Peter G Gibson

Introduction: Clinical remission is a relatively new concept in asthma but recent research initiatives suggest it could be an ambitious and achievable therapeutic target for patients with asthma.

Methods: In this modified Delphi study (comprising two online surveys, completed either side of a virtual scientific workshop), the opinions of a panel of respiratory physicians were evaluated to summarize perspective statements on key therapeutic outcomes and criteria for on-treatment clinical remission in patients with moderate asthma. An agreement threshold was pre-defined as agreement by ≥ 75% of participants.

Results: Surveys 1 and 2 were completed by 20 and 18 participants, respectively. Most participants (95%) agreed with the concept of clinical remission in moderate asthma and that this should be a desirable treatment goal (90%). Based on a composite measure of 4-6 desirable therapeutic outcomes, current understanding of clinical remission was considered as 12 months with no exacerbations, no oral corticosteroids, no daytime or night-time asthma symptoms (Asthma Control Test score ≥ 20 or Asthma Control Questionnaire score ≤ 0.75), stable lung function, and no treatment-related adverse events. No agreement was reached on the role of relievers in defining therapeutic outcomes or on the wider use of biomarkers and airway hyperresponsiveness for defining asthma remission in clinical practice.

Conclusions: In line with recent consensus statements from the United States and Europe, there was a high level of agreement on the elements of clinical remission among a panel of respiratory physicians from Asia, the Middle East, and South America. Extension of the concept of clinical remission to patients with moderate asthma was considered aligned with the potential of clinical remission as a goal of therapy.

导言:临床缓解是哮喘病的一个相对较新的概念,但最近的研究表明,临床缓解是哮喘患者可以实现的一个宏伟治疗目标:临床缓解在哮喘病中是一个相对较新的概念,但最近的研究表明,对于哮喘患者来说,临床缓解是一个雄心勃勃且可以实现的治疗目标:在这项经过修改的德尔菲研究(包括两项在线调查,分别在虚拟科学研讨会的两侧完成)中,对呼吸科医生小组的意见进行了评估,以总结中度哮喘患者的关键治疗结果和治疗中临床缓解标准的观点陈述。同意阈值被预先设定为≥75%的参与者同意:分别有 20 名和 18 名参与者完成了调查 1 和 2。大多数参与者(95%)同意中度哮喘临床缓解的概念,并认为这应该是一个理想的治疗目标(90%)。根据 4-6 项理想治疗结果的综合衡量标准,目前对临床缓解的理解是 12 个月内无病情加重、无口服皮质类固醇、无白天或夜间哮喘症状(哮喘控制测试评分≥ 20 或哮喘控制问卷评分≤ 0.75)、肺功能稳定、无治疗相关不良事件。对于缓解剂在确定治疗结果中的作用,以及在临床实践中更广泛地使用生物标志物和气道高反应性来确定哮喘缓解,没有达成一致意见:来自亚洲、中东和南美洲的呼吸内科医生小组就临床缓解的要素达成了高度一致,这与美国和欧洲最近发表的共识声明是一致的。将临床缓解的概念扩展到中度哮喘患者被认为符合临床缓解作为治疗目标的潜力。
{"title":"Exploring Clinical Remission in Moderate Asthma - Perspectives from Asia, the Middle East, and South America.","authors":"Kittipong Maneechotesuwan, Bhumika Aggarwal, Gabriel Garcia, Daniel Tan, Hugo Neffen, Ramon Jason M Javier, Mona Al-Ahmad, Mousa Khadada, Vu Tran Thien Quan, Krittika Teerapuncharoen, Mario Soto Ramos, Gur Levy, Maximilian Plank, Abhay Phansalkar, Peter G Gibson","doi":"10.1007/s41030-024-00262-2","DOIUrl":"10.1007/s41030-024-00262-2","url":null,"abstract":"<p><strong>Introduction: </strong>Clinical remission is a relatively new concept in asthma but recent research initiatives suggest it could be an ambitious and achievable therapeutic target for patients with asthma.</p><p><strong>Methods: </strong>In this modified Delphi study (comprising two online surveys, completed either side of a virtual scientific workshop), the opinions of a panel of respiratory physicians were evaluated to summarize perspective statements on key therapeutic outcomes and criteria for on-treatment clinical remission in patients with moderate asthma. An agreement threshold was pre-defined as agreement by ≥ 75% of participants.</p><p><strong>Results: </strong>Surveys 1 and 2 were completed by 20 and 18 participants, respectively. Most participants (95%) agreed with the concept of clinical remission in moderate asthma and that this should be a desirable treatment goal (90%). Based on a composite measure of 4-6 desirable therapeutic outcomes, current understanding of clinical remission was considered as 12 months with no exacerbations, no oral corticosteroids, no daytime or night-time asthma symptoms (Asthma Control Test score ≥ 20 or Asthma Control Questionnaire score ≤ 0.75), stable lung function, and no treatment-related adverse events. No agreement was reached on the role of relievers in defining therapeutic outcomes or on the wider use of biomarkers and airway hyperresponsiveness for defining asthma remission in clinical practice.</p><p><strong>Conclusions: </strong>In line with recent consensus statements from the United States and Europe, there was a high level of agreement on the elements of clinical remission among a panel of respiratory physicians from Asia, the Middle East, and South America. Extension of the concept of clinical remission to patients with moderate asthma was considered aligned with the potential of clinical remission as a goal of therapy.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"279-295"},"PeriodicalIF":2.3,"publicationDate":"2024-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11339013/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"141238078","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
Qualitative Interviews Exploring Adverse Event Mitigation Strategies in Adults Receiving Amikacin Liposome Inhalation Suspension. 通过定性访谈探索成人吸入阿米卡星脂质体混悬液后的不良事件缓解策略。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-09-01 Epub Date: 2024-07-16 DOI: 10.1007/s41030-024-00263-1
Juzar Ali, Jasmanda Wu, Mariam Hassan, Jui-Hua Tsai, Nancy Touba, Kelly McCarrier, Mark Ballard, Anjan Chatterjee

Introduction: This study aimed to gain insight from patients with refractory Mycobacterium avium complex lung disease (MAC-LD) into strategies used to manage adverse events (AEs) associated with amikacin liposome inhalation suspension (ALIS).

Methods: We conducted semi-structured interviews with US patients with refractory MAC-LD prescribed ALIS in a real-world setting. Interview transcripts were analyzed and coded to identify patterns in participants' descriptions of their ALIS treatment experiences, including AEs and their disruptiveness, and AE mitigation strategies, including participants' ratings of strategies' effectiveness. Concept saturation was also assessed.

Results: Twenty participants (mean age 48.7 years; 80% women; mean ALIS duration 5.45 months) were interviewed. At the time of the interview, 15 participants (75%) had received ALIS for > 1 month and 13 (65%) were currently receiving ALIS. Participants described 44 unique AE mitigation strategies, which can be categorized into three groups: prepare for treatment; prevent increased emergence of AEs; and persist on treatment by mitigating AEs. Common strategies (reported by ≥ 50% of participants) included use of educational materials from the patient support program, localized management of throat irritation, and symptom management to reduce fatigue. Evidence of concept saturation was observed: no new strategies were identified in the last five interviews, which suggests the sample was robust enough to identify all mitigation strategies likely to be used by the broader patient population.

Conclusions: This real-world study identified a diverse set of potential AE mitigation strategies intended to help individual patients prepare for ALIS treatment, prevent the increased emergence of certain AEs, and mitigate the impact of AEs on treatment persistence. Developing a comprehensive accounting of the types of mitigation strategies in use among patients in real-world settings can inform future investigation of the effectiveness of such strategies, and support evidence-based recommendations for treatment management.

简介:本研究旨在从难治性复合分枝杆菌肺病(MAC-LD)患者处了解与阿米卡星脂质体吸入悬浮剂(ALIS)相关的不良事件(AEs)的处理策略:我们在真实世界环境中对开具 ALIS 处方的美国难治性 MAC-LD 患者进行了半结构化访谈。我们对访谈记录进行了分析和编码,以确定参与者对其 ALIS 治疗经历(包括 AE 及其破坏性)和 AE 缓解策略(包括参与者对策略有效性的评价)的描述模式。此外,还对概念饱和度进行了评估:20 名参与者(平均年龄 48.7 岁;80% 为女性;ALIS 平均持续时间为 5.45 个月)接受了访谈。在接受访谈时,15 名参与者(75%)已接受 ALIS 超过 1 个月,13 名参与者(65%)目前正在接受 ALIS。参与者描述了 44 种独特的 AE 缓解策略,可分为三类:为治疗做好准备;防止出现更多 AE;通过缓解 AE 坚持治疗。常见的策略(≥50% 的参与者报告过)包括使用患者支持计划提供的教育材料、对咽喉刺激进行局部处理以及减轻疲劳的症状处理。研究观察到了概念饱和的证据:在最后五次访谈中没有发现新的策略,这表明样本足够强大,能够识别更广泛的患者群体可能使用的所有缓解策略:这项真实世界研究发现了一系列不同的潜在AE缓解策略,旨在帮助患者为ALIS治疗做好准备,防止某些AE的增加,并减轻AE对治疗持续性的影响。全面了解患者在真实世界环境中使用的缓解策略类型,可为今后调查此类策略的有效性提供依据,并为基于证据的治疗管理建议提供支持。
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引用次数: 0
The Role of Medical Thoracoscopy with Talc Poudrage in Spontaneous, Iatrogenic, and Traumatic Pneumothorax: A Prolonged Experience of a Tertiary Care Center. 胸腔镜联合滑石粉填塞术在自发性、先天性和创伤性气胸中的作用:一家三级医疗中心的长期经验。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-09-01 Epub Date: 2024-08-10 DOI: 10.1007/s41030-024-00268-w
Alberto Fantin, Nadia Castaldo, Ernesto Crisafulli, Giulia Sartori, Avinash Aujayeb, Paolo Vailati, Giuseppe Morana, Filippo Patrucco, Maria de Martino, Miriam Isola, Vincenzo Patruno

Introduction: Medical thoracoscopy is a minimally invasive and safe procedure mostly performed for unexplained exudative pleural effusions but may be considered for pneumothorax (PNX).

Methods: This retrospective study included participants affected by PNX who underwent medical thoracoscopy with talc poudrage at a single academic hospital from 2008 to 2021. The primary endpoint was the observation of complete radiographical lung re-expansion and absence of air supply from the chest drain within 7 days of medical thoracoscopy. The secondary endpoint was achieving no recurrence of ipsilateral PNX at 24 months post-discharge.

Results: A total of 95 patients affected by primary spontaneous PNX (PSP), secondary spontaneous PNX (SSP), iatrogenic, and traumatic PNX were enrolled. An additional procedure was required by 17.89% of patients, and only one patient with SSP required subsequent surgery. Recurrence of PNX occurred on the same side within 24 months after discharge in 9.47% of patients, with a median time to recurrence of 13.5 months. The PSP group was significantly more likely to achieve the primary endpoint. Pleural morphology was significantly associated with reaching the primary endpoint, while receiving a cumulative dose of talc greater than or equal to 4 g during hospitalization was associated with a lower risk of meeting it. Receiving a cumulative dose of talc greater than or equal to 4 g led in all cases to the achievement of the secondary endpoint. Patients with iatrogenic and traumatic PNX had an excellent prognosis in both the short- and long-term evaluation.

Conclusion: Medical thoracoscopy is an effective procedure for treating PNX in the acute setting in selected cases while preventing long-term relapses. Large prospective clinical studies are needed to support and better define the role of medical thoracoscopy in current clinical practice.

简介:医学胸腔镜是一种微创、安全的手术,主要用于治疗不明原因的渗出性胸腔积液,也可用于治疗气胸:内科胸腔镜检查是一种微创、安全的手术,主要用于不明原因的渗出性胸腔积液,但也可考虑用于气胸(PNX):这项回顾性研究纳入了 2008 年至 2021 年期间在一家学术医院接受内科胸腔镜和滑石粉包扎的 PNX 患者。主要终点是观察到内科胸腔镜手术后 7 天内肺部完全放射学再扩张和胸腔引流管无供气。次要终点是出院后 24 个月同侧 PNX 不再复发:共有 95 名原发性自发性 PNX(PSP)、继发性自发性 PNX(SSP)、先天性和外伤性 PNX 患者入选。17.89%的患者需要进行额外手术,只有一名 SSP 患者需要进行后续手术。9.47%的患者在出院后24个月内在同一侧PNX复发,中位复发时间为13.5个月。PSP组达到主要终点的可能性明显更高。胸膜形态与达到主要终点显著相关,而住院期间接受大于或等于4克的滑石粉累积剂量与达到主要终点的风险较低相关。在所有病例中,接受大于或等于4克滑石粉的累积剂量都会导致达到次要终点。在短期和长期评估中,先天性和外伤性PNX患者的预后良好:结论:内科胸腔镜手术是一种有效的方法,可在急性期治疗特定病例的 PNX,同时预防长期复发。需要进行大规模的前瞻性临床研究,以支持和更好地确定医用胸腔镜在当前临床实践中的作用。
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引用次数: 0
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Pulmonary Therapy
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