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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。
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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。
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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。
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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 Retrospective Study Evaluating Asthma Control in Patients on Fluticasone Propionate/Salmeterol Proactive Regular Dosing with a History of Uncontrolled Asthma. 一项回顾性研究,评估使用丙酸氟替卡松/沙美特罗主动定期给药且有哮喘失控史的患者的哮喘控制情况。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-11-09 DOI: 10.1007/s41030-024-00278-8
Ahmad Izuanuddin Ismail, Irfhan Ali Hyder Ali, Chee Kuan Wong, Andrea Yu-Lin Ban, Fatimah Mz Zahrah, Li Khen Lem, Zamzurina Abu Bakar, Arvindran Alaga, Azza Omar, Azlina Samsudin, Siew Li Lai, Alap Gandhi

Introduction: The MERIT study in Malaysia is a real-world retrospective, observational, multicenter study that evaluated asthma control in patients with uncontrolled asthma who were switched from as-needed (pro re nata [PRN]) budesonide/formoterol or inhaled corticosteroid (ICS) whenever a short-acting beta-agonist (SABA) was taken, to proactive regular dosing of fluticasone propionate/salmeterol (FP/SAL PRD).

Methods: Data from the medical records of patients who were stepped up to FP/SAL PRD were extracted retrospectively at baseline and follow-up (between 3 and 6 months after stepping up to FP/SAL PRD). The primary endpoint was the percentage of patients with improvement in asthma control assessed via the Asthma Control Test (ACT). Secondary endpoints included safety and the percentage of patients with moderate and severe exacerbations. Additionally, patient-reported use of reliever medication, systemic corticosteroids, emergency department visits, or hospitalization was also analyzed.

Results: One hundred twenty patients with uncontrolled asthma who were stepped up to FP/SAL PRD were enrolled in the study. Of these, 76 (63.3%) patients were on prior budesonide/formoterol PRN, and 44 (36.7%) were on prior ICS with SABA PRN treatment. After stepping up to FP/SAL PRD with a mean follow-up of 5.8 months, 110 (91.7%) patients achieved asthma control at the follow-up visit (p < 0.001). Similar improvements were observed regardless of prior PRN regimen. A statistically significant improvement was observed in the mean ACT score at the follow-up visit (p < 0.0001). The proportion of patients with moderate and severe exacerbations was also reduced after stepping up to FP/SAL PRD, with no adverse events reported. Over 80% of patients reported a decrease in the use of systemic corticosteroids, visits to the emergency department, or hospitalization.

Conclusion: This study highlights the effectiveness of the FP/SAL PRD treatment approach in patients with uncontrolled asthma on a PRN treatment regimen.

简介:马来西亚的 MERIT 研究是一项真实世界的回顾性、观察性、多中心研究,该研究评估了未得到控制的哮喘患者的哮喘控制情况,这些患者从按需服用(pro re nata [PRN])布地奈德/福莫特罗或吸入性皮质类固醇(ICS)转为主动定期服用丙酸氟替卡松/沙美特罗(FP/SAL PRD):回顾性提取了被升级为氟替卡松丙酸盐/沙美特罗(FP/SAL PRD)患者的基线和随访(升级为FP/SAL PRD后的3至6个月)病历数据。主要终点是通过哮喘控制测试(ACT)评估哮喘控制有所改善的患者比例。次要终点包括安全性以及中度和重度病情恶化患者的比例。此外,还分析了患者报告的缓解药物、全身皮质类固醇、急诊就诊或住院治疗的使用情况:该研究共纳入了 120 名未受控制的哮喘患者,他们都已接受 FP/SAL PRD 治疗。其中,76 例(63.3%)患者曾接受布地奈德/福莫特罗 PRN 治疗,44 例(36.7%)患者曾接受 ICS 与 SABA PRN 治疗。在接受 FP/SAL PRD 治疗后,平均随访时间为 5.8 个月,有 110 名(91.7%)患者在随访时哮喘得到了控制(p 结论:该研究强调了 FP/SAL PRD 治疗的有效性:本研究强调了 FP/SAL PRD 治疗方法对接受 PRN 治疗方案的未控制哮喘患者的有效性。
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引用次数: 0
Long-Term Safety and Efficacy of Macitentan in Inoperable Chronic Thromboembolic Pulmonary Hypertension: Results from MERIT and its Open-Label Extension. 马西替坦治疗无法手术的慢性血栓栓塞性肺动脉高压的长期安全性和有效性:MERIT 及其开放标签扩展研究的结果。
IF 2.3 Q2 RESPIRATORY SYSTEM Pub Date : 2024-11-09 DOI: 10.1007/s41030-024-00276-w
Nick H Kim, Andrea M D'Armini, Luke S Howard, David P Jenkins, Zhi-Cheng Jing, Eckhard Mayer, Liliya Chamitava, Gabriela Lack, Hany Rofael, Maria Solonets, Hossein-Ardeschir Ghofrani

Introduction: Evidence for use of pulmonary arterial hypertension targeted-therapies in patients with chronic thromboembolic pulmonary hypertension (CTEPH) is limited. In MERIT-1, the endothelin receptor antagonist macitentan improved hemodynamic and functional parameters versus placebo in patients with inoperable CTEPH over a 24-week double-blind (DB) period. Its open-label (OL) extension study (MERIT-2) provides long-term safety/efficacy data.

Methods: MERIT-2 (NCT02060721) was a multicenter, single-arm, OL, phase 2 extension study of MERIT-1. Patients completing MERIT-1 were eligible to receive 10 mg macitentan once-daily in MERIT-2. Safety and efficacy (6-min walk distance [6MWD] and change in World Health Organization functional class [WHO FC]) were assessed in all patients in MERIT-2 regardless of treatment received in DB (All patients MERIT-2 OL macitentan 10 mg group) and the subgroup of patients receiving DB macitentan in MERIT-1 (Long-term [DB/OL] macitentan 10 mg subgroup).

Results: Of the 80 patients randomized in MERIT-1, 76 entered MERIT-2 (All patients MERIT-2 OL macitentan 10 mg group): 40 who received DB macitentan (DB-macitentan patients) and 36 DB placebo (DB-placebo patients). Median (interquartile range) macitentan exposure in the All patients MERIT-2 OL macitentan 10 mg group was 45.5 (26.0, 66.1) months. During the OL period, treatment-emergent adverse events (AE) were reported in 72 (94.7%) patients; most frequent were worsening of pulmonary hypertension (19.7%), decreased hemoglobin (18.4%) and upper respiratory tract infection (15.8%). Fourteen (18.4%) patients died; none were assessed as macitentan-related. At Month 6 post-OL baseline, mean (standard deviation) change in 6MWD was - 0.4 m (43.62) for DB-macitentan patients and 10.7 m (45.63) for DB-placebo patients; the majority had unchanged (83.3%) or improved (12.5%) WHO FC. Safety/efficacy analyses were consistent in the Long-term (DB/OL) macitentan 10 mg subgroup.

Conclusion: These analyses provide long-term safety/efficacy data in patients with inoperable CTEPH treated with macitentan. No unexpected safety findings occurred; reported AEs were consistent with the known safety profile of macitentan. At 6 months post-OL baseline, DB-placebo patients modestly improved 6MWD; DB-macitentan patients maintained improvements observed in MERIT-1. WHO FC was largely unchanged.

Trial registration: ClinicalTrials.gov Identifiers: NCT02021292; NCT02060721.

简介:在慢性血栓栓塞性肺动脉高压(CTEPH)患者中使用肺动脉高压靶向疗法的证据有限。在MERIT-1研究中,内皮素受体拮抗剂马西替坦与安慰剂相比,能在24周的双盲(DB)期间改善无法手术的CTEPH患者的血液动力学和功能参数。其开放标签(OL)延伸研究(MERIT-2)提供了长期安全性/有效性数据:MERIT-2(NCT02060721)是MERIT-1的一项多中心、单臂、OL、2期扩展研究。完成MERIT-1的患者有资格在MERIT-2中接受10毫克马西替坦,每日一次。对MERIT-2的所有患者(所有患者MERIT-2 OL马西替坦10毫克组)和MERIT-1中接受DB马西替坦治疗的患者亚组(长期[DB/OL]马西替坦10毫克亚组)的安全性和疗效(6分钟步行距离[6MWD]和世界卫生组织功能分级[WHO FC]的变化)进行了评估,无论患者接受的是哪种DB治疗(所有患者MERIT-2 OL马西替坦10毫克组):在MERIT-1随机抽取的80名患者中,76人进入MERIT-2(所有患者MERIT-2 OL马西替坦10毫克组):40名患者接受了DB马西替坦治疗(DB-马西替坦患者),36名患者接受了DB安慰剂治疗(DB-安慰剂患者)。所有患者 MERIT-2 OL 马西替坦 10 毫克组的马西替坦暴露中位数(四分位数间距)为 45.5 (26.0, 66.1) 个月。在OL期间,72名患者(94.7%)报告了治疗突发不良事件(AE);最常见的不良事件是肺动脉高压恶化(19.7%)、血红蛋白下降(18.4%)和上呼吸道感染(15.8%)。14名患者(18.4%)死亡,经评估,无一例外与马西替坦有关。OL基线后第6个月,DB-马西替坦患者6MWD的平均(标准差)变化为-0.4米(43.62),DB-安慰剂患者为10.7米(45.63);大多数患者的WHO FC没有变化(83.3%)或有所改善(12.5%)。长期(DB/OL)马基替坦 10 mg 亚组的安全性/有效性分析结果一致:这些分析提供了使用马西替坦治疗无法手术的CTEPH患者的长期安全性/有效性数据。没有出现意外的安全性结果;报告的AE与已知的马西替坦安全性特征一致。在OL基线后6个月,DB-安慰剂患者的6MWD略有改善;DB-马西替坦患者保持了MERIT-1中观察到的改善。WHO FC基本保持不变:试验注册:ClinicalTrials.gov Identifiers:NCT02021292;NCT02060721。
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引用次数: 0
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Pulmonary Therapy
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