Pulmonary Therapy最新文献

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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
Pulmonary Therapy 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
{"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
Pulmonary Therapy 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
{"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
Pulmonary Therapy 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
{"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
Pulmonary Therapy 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
{"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
Pulmonary Therapy 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
{"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
Pulmonary Therapy 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
{"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
Pulmonary Therapy Pub Date : 2024-12-01 Epub Date: 2024-11-08 DOI: 10.1007/s41030-024-00277-9
Alan G Kaplan
{"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
Pulmonary Therapy 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
{"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
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
Pulmonary Therapy 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
{"title":"A Retrospective Study Evaluating Asthma Control in Patients on Fluticasone Propionate/Salmeterol Proactive Regular Dosing with a History of Uncontrolled Asthma.","authors":"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","doi":"10.1007/s41030-024-00278-8","DOIUrl":"https://doi.org/10.1007/s41030-024-00278-8","url":null,"abstract":"<p><strong>Introduction: </strong>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).</p><p><strong>Methods: </strong>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.</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>This study highlights the effectiveness of the FP/SAL PRD treatment approach in patients with uncontrolled asthma on a PRN treatment regimen.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142626891","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
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
Pulmonary Therapy 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
{"title":"Long-Term Safety and Efficacy of Macitentan in Inoperable Chronic Thromboembolic Pulmonary Hypertension: Results from MERIT and its Open-Label Extension.","authors":"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","doi":"10.1007/s41030-024-00276-w","DOIUrl":"https://doi.org/10.1007/s41030-024-00276-w","url":null,"abstract":"<p><strong>Introduction: </strong>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.</p><p><strong>Methods: </strong>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).</p><p><strong>Results: </strong>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.</p><p><strong>Conclusion: </strong>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.</p><p><strong>Trial registration: </strong>ClinicalTrials.gov Identifiers: NCT02021292; NCT02060721.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-11-09","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142626902","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
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