Pulmonary Therapy最新文献

{"title":"Retrospective Cohort Study of Elderly Users of Single- or Multiple-Inhaler Triple Therapy for the Treatment of Asthma in the USA.","authors":"Russell A Settipane, Guillaume Germain, Francois Laliberté, Malena Mahendran, Annalise Hilts, Mei Sheng Duh, Rosirene Paczkowski, Emmeline Burrows","doi":"10.1007/s41030-024-00285-9","DOIUrl":"https://doi.org/10.1007/s41030-024-00285-9","url":null,"abstract":"<p><strong>Introduction: </strong>Escalation to single- or multiple-inhaler triple therapy (SITT; MITT) is a recommended option for patients with asthma who remain uncontrolled by medium-dose inhaled corticosteroid/long-acting β₂-agonist; however, characterization of elderly users of triple therapy is limited. This real-world cohort study describes demographics and clinical characteristics of elderly patients with asthma with and without comorbid chronic obstructive pulmonary disease (COPD) who are new users of triple therapy, and asthma treatment patterns preceding triple therapy initiation.</p><p><strong>Methods: </strong>This retrospective cohort study used administrative claims data from the Optum Clinformatics Data Mart database. Eligible patients were ≥ 65 years of age with asthma or with asthma and comorbid COPD who initiated either triple therapy with single-inhaler fluticasone furoate/umeclidinium/vilanterol (FF/UMEC/VI; 100/62.5/25 μg) or MITT between September 18, 2017 and September 30, 2020. Demographics, clinical characteristics, healthcare resource utilization, healthcare costs, and asthma treatment patterns were described in the 12-month period before triple therapy initiation (baseline period).</p><p><strong>Results: </strong>In total, 15,557 patients were included. Among FF/UMEC/VI initiators with asthma (N = 635) mean age was 73.3 years and 66.6% were female. During the baseline period, > 75% of patients used controller therapy, > 92% used rescue medications, 27.9% experienced ≥ 1 asthma-related exacerbation, with mean annual exacerbation rate of 0.42, and mean all-cause healthcare costs were $23,407. Patients with asthma initiating MITT and patients with asthma and comorbid COPD initiating FF/UMEC/VI or MITT had similar characteristics, healthcare resource utilization, healthcare costs, and asthma treatment patterns to FF/UMEC/VI initiators with asthma.</p><p><strong>Conclusions: </strong>Triple therapy is often initiated following use of other asthma controller medications in real-world practice. Substantial rescue medication use and high disease and economic burden among this elderly patient population suggest that their asthma was not adequately controlled prior to triple therapy initiation. This retrospective study provides an early profile of elderly patients with asthma in the USA.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-01-27","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143047319","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}

{"title":"Prevalence of Obstructive Sleep Apnea in Vietnamese Children with Attention Deficit Hyperactivity Disorder (ADHD).","authors":"Mai Nguyen-Thi-Phuong, Mai Nguyen-Thi-Thanh, Thuy Nguyen-Thi-Dieu, Sy Duong-Quy","doi":"10.1007/s41030-024-00286-8","DOIUrl":"https://doi.org/10.1007/s41030-024-00286-8","url":null,"abstract":"<p><strong>Introduction: </strong>Obstructive sleep apnea (OSA) is a common sleep-related breathing disorder among children with attention deficit hyperactivity disorder (ADHD). This study aims to determine the prevalence of OSA in children with ADHD, compare the differences in clinical characteristics between children with ADHD-OSA and those without OSA (ADHD-nonOSA), and to identify the correlation between OSA and ADHD in children.</p><p><strong>Methods: </strong>This cross-sectional descriptive study was conducted on 524 children with ADHD, aged 6-12 years, at the Vietnam National Children's Hospital from October 2022 to September 2023. Respiratory polygraphy was used to determine the prevalence of OSA in this study population; clinical data of children with ADHD-OSA and ADHD-nonOSA were collected and analyzed in each group. The severity of ADHD symptoms was measured by the Vanderbilt ADHD Parent Diagnosis Rating Scale (VADPRS questionnaire), and the severity of OSA was defined by Apnea-Hypopnea Index (AHI).</p><p><strong>Results: </strong>The prevalence of OSA in children with ADHD was 23.3%, with the majority of moderate-to-severe OSA. Significant differences were observed in sleep onset time, total sleep duration, and various sleep-related behaviors, such as bedtime resistance and difficulty waking up in the morning, between children with ADHD-OSA and ADHD-nonOSA (p < 0.001, p < 0.001, p < 0.05, and p < 0.005, respectively). The severity levels of inattention, hyperactivity, and behavioral disorders were significantly higher in children with moderate-to-severe OSA compared to those with mild OSA (p < 0.005, p < 0.005, and p < 0.001, respectively). There were significant correlations between AHI with inattention scores (r = 0.677; p < 0.0001), hyperactivity scores (r = 0.438; p = 0.05), behavioral disorder scores (r = 0.342; p < 0.05), and anxiety/depression scores (r = 0.357; p < 0.05) measured by VADPRS questionnaire.</p><p><strong>Conclusions: </strong>OSA is a common sleep-related breathing disorder in children with ADHD, which might exacerbate ADHD symptoms. Thus, screening of OSA in children with ADHD is essential during the management of ADHD in this population.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-01-14","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142979790","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}

{"title":"Overlap Syndrome (COPD and OSA): A Treatable Trait for Triple Treatment?","authors":"Athanasios Voulgaris, Alexandros Kalkanis, Paschalis Steiropoulos","doi":"10.1007/s41030-024-00282-y","DOIUrl":"https://doi.org/10.1007/s41030-024-00282-y","url":null,"abstract":"<p><p>The coexistence of chronic obstructive pulmonary disease (COPD) and obstructive sleep apnea (OSA) in the same patient is referred to as overlap syndrome (OS). Patients with OS suffer more frequently from cardiovascular disease (CVD) and carry a higher risk of COPD-related exacerbations than patients with COPD alone, especially when OSA is left untreated. Based on recent evidence, triple therapy, namely inhaled corticosteroid/long-acting muscarinic antagonist/long-acting beta-agonist (ICS-LABA-LAMA), is a treatment strategy in COPD patients with a history of exacerbations and/or CVD comorbidity. While several studies have previously focused on the role of triple therapy in patients with COPD, none of these has examined the potential benefits of this treatment in patients with COPD and concomitant OSA. Moreover, it is unknown whether patients with OS should be treated with triple therapy starting from their initial assessment, since they represent a population at risk for future exacerbations, in comparison to patients with COPD alone. In this commentary, we discuss these issues and highlight the need for further studies regarding the role of triple therapy in outcomes for patients with OS.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2025-01-02","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142915490","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}

{"title":"Real-World Effectiveness of Bebtelovimab Versus Nirmatrelvir/Ritonavir in Outpatients with COVID-19.","authors":"Christopher G Rowan, Russell M Nichols, Neil Dhopeshwarkar, Jennifer M Alyea, Baojin Zhu, Sengwee Toh, K Arnold Chan, Elsie L Grace","doi":"10.1007/s41030-024-00284-w","DOIUrl":"https://doi.org/10.1007/s41030-024-00284-w","url":null,"abstract":"<p><strong>Introduction: </strong>This real-world study assessed the effectiveness of bebtelovimab (BEB) versus nirmatrelvir/ritonavir (NR) among outpatients with COVID-19 during the Omicron variant era.</p><p><strong>Methods: </strong>We conducted a cohort study evaluating patients treated with BEB or NR from February to August 2022 (study period). Follow-up began the day after treatment and continued for 30 days. Cohorts were constructed using de-identified electronic health record data from TriNetX Dataworks USA. The study assessed 30-day all-cause hospitalization or death (composite) using the risk difference (RD) and 95% confidence interval (95% CI).</p><p><strong>Results: </strong>Unmatched cohorts included 12,920 BEB- and 70,741 NR-treated patients. After exact matching on key baseline covariates (age > 65 years, immunocompromised, recent emergency department [ED] visit, and COVID-19 vaccination) and high-dimensional propensity score matching (1:1) on a broader set of covariates, 5827 patients were included in each cohort. BEB-treated patients were older and had more comorbidities compared to NR-treated patients prior to matching. After matching, baseline characteristics were well balanced. The cumulative incidence of the primary outcome (hospitalization or death) was 2.0% and 1.8% for BEB and NR, respectively (RD 0.2%; 95% CI - 0.3%, 0.7%). The upper bound of the RD 95% CI (0.7%) excluded the noninferiority margin (1.795%), demonstrating that BEB was not inferior to NR. The RDs of the secondary outcomes were (BEB vs NR): hospitalization (RD 0.1%; 95% CI - 0.4%, 0.6%); ED visit (RD 0.5%; 95% CI - 0.3%, 1.3%); and death (RD 0.09%; 95% CI - 0.003%, 0.2%). Results from subgroup, sensitivity, and linked analyses (EHR + claims + mortality data) were consistent with the main results.</p><p><strong>Conclusion: </strong>Treatment with BEB was not inferior to NR with respect to 30-day all-cause hospitalization or death. The risk of secondary outcomes was not different for patients treated with BEB compared to NR.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-25","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142897145","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}

{"title":"High Interleukin (IL)-6 is Associated with Lower Lung Function and Increased Likelihood of Metabolic Dysfunction in Asthma.","authors":"Dionne Adair, AmirBehzad Bagheri, Matheos Yosef, Shokoufeh Khalatbari, Toby Lewis, Arjun Mohan, Njira Lugogo","doi":"10.1007/s41030-024-00281-z","DOIUrl":"https://doi.org/10.1007/s41030-024-00281-z","url":null,"abstract":"<p><strong>Introduction: </strong>Asthma is a complex condition characterized by airway inflammation. Interleukin-6 (IL-6) plays a significant role in asthma pathogenesis through its effects on T cells and its association with pro-inflammatory responses. Both lung and circulating IL-6 levels are elevated in asthma. IL-6 is positively associated with disease severity, frequent exacerbations, and impaired lung function, all of which can be observed clinically. We developed an IL-6 cut-off model to examine the association between high IL-6, race, high body mass index (BMI), metabolic disease, and asthma severity as assessed by reduced lung function.</p><p><strong>Methods: </strong>This study utilized the Coronary Artery Risk Development in Young Adults (CARDIA) database, comprised of 5115 adults, to investigate the relationship between IL-6 levels, asthma, race, and metabolic dysfunction. A \"healthy\" subset of 427 patients was used to compute the IL-6 cut-off. IL-6 levels within detection limits (0.15-12 pg/mL) were analyzed. The IL-6 cut-off was determined using the 95th percentile of log-transformed IL-6 values for lean (BMI < 25) and healthy individuals. Specific cut-offs were established for racial groups. Statistical analyses involved comparing patient characteristics between high and low IL-6 groups, regression analyses, and assessment of factors influencing lung function changes.</p><p><strong>Results: </strong>Using an IL-6 cut-off of 4.979 pg/mL, the cohort was divided into high and low IL-6 groups. High IL-6 correlated with Black race, higher BMI, hypertension, and markers of metabolic dysfunction, e.g., elevated HbA1c, C-reactive protein (CRP), and reduced lung function. Multivariable analysis linked high IL-6 with male gender, high BMI, Black race, HbA1c, CRP, and inversely with lung function and total cholesterol. Obesity showed a consistent positive association with elevated IL-6, regardless of the presence or absence of asthma. Patients with asthma and high IL-6 were more likely to be Black and showed increased CRP. Lung function was lowest in non-lean, high IL-6 patients with asthma, with similar trends in non-lean (BMI ≥ 25) patients without asthma.</p><p><strong>Conclusion: </strong>This study underscores the significant association between IL-6, asthma, obesity, and metabolic dysfunction. Elevated IL-6 correlates with asthma severity, particularly in individuals with obesity. Future research should explore anti-IL-6 therapies for specific phenotypes, such as obesity-related asthma. These findings advance our understanding of asthma and the role of IL-6 in its pathogenesis.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-23","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142877910","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}

{"title":"Εosinophilic Chronic Obstructive Pulmonary Disease. What Do We Know So Far?","authors":"Konstantinos Bartziokas, Andriana I Papaioannou, Nikoletta Rovina, Georgia Papaiakovou, Stelios Loukides, Paschalis Steiropoulos","doi":"10.1007/s41030-024-00280-0","DOIUrl":"https://doi.org/10.1007/s41030-024-00280-0","url":null,"abstract":"<p><p>The latest advances in asthma treatment have highlighted the significance of eosinophilia and the possible role of some pro-eosinophilic mediators, like interleukins (IL) IL-5, IL-4/IL-13, and IL-33 in the disease's pathogenesis. Considering that a subgroup of patients with chronic obstructive pulmonary disease (COPD) may have blood eosinophilia akin to that seen in asthma, numerous studies in the last decade have suggested that eosinophilic COPD is a separate entity. While the exact role of blood eosinophils in the pathophysiology of COPD remains unclear, eosinophilia seems to increase the effectiveness of corticosteroid therapy. Currently, monoclonal antibodies targeting the interleukins (IL-5, IL-4, IL-13, and IL-33) or their receptors are being investigated in patients with COPD belonging in T2-high endotype. This review focuses on the mechanisms of eosinophilia in COPD, the effects of eosinophilia on disease outcome, and examines the most recent data on the use of peripheral blood eosinophilia in treating patients with COPD. Finally, we emphasize the current implication of monoclonal antibodies in COPD in the context of eosinophilic airway inflammation.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":2.3,"publicationDate":"2024-12-11","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142807206","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}

{"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}

{"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}

{"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}

{"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 (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.</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 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.</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}