Pulmonary Therapy最新文献

{"title":"Impact of Flow Restrictors on Aerosol Delivery of the Respimat® Soft Mist Inhaler.","authors":"Moritz Fleischhauer, Kai Berkenfeld, David Stadermann, Sitaram Velaga, Igor Gonda, Peter Langguth, Herbert Wachtel","doi":"10.1007/s41030-025-00312-3","DOIUrl":"https://doi.org/10.1007/s41030-025-00312-3","url":null,"abstract":"<p><strong>Introduction: </strong>The modification of an inhaler's air flow resistance influences a patient's inhalation flow profile, thereby affecting the exit velocity of an aerosol leaving the Respimat® mouthpiece. A slower inhalation maneuver results in reduced plume velocity and thus a decreased oropharyngeal deposition due to reduced impaction. This could not only lead to fewer unwanted side effects associated with inhaled therapies, but also enhance lung deposition.</p><p><strong>Methods: </strong>Device prototypes with different air flow resistances were designed using custom-made inserts that can be clipped into the Respimat mouthpiece. The consequences on aerosol characteristics, as well as on in vitro deposition, were analyzed. Computational fluid dynamics simulations contributed to a better understanding of the modified aerodynamic conditions.</p><p><strong>Results: </strong>Different insert geometries resulted in modified device resistances. However, an increased flow resistance does not necessarily result in an improved in vitro performance. The flow restrictors critically determine aerosol characteristics such as plume velocity and spray pattern, thereby altering in vitro deposition patterns. Quantitative data on mouth-throat deposition and aerosol characteristics are reported.</p><p><strong>Conclusions: </strong>Integrating flow restrictors into the Respimat mouthpiece offers a promising approach to enhance patient centricity by promoting slower inhalation, thereby reducing the likelihood of suboptimal use. The use of a porous insert acting as a diffuser demonstrated minimal impact on internal airflow dynamics and in vitro deposition, suggesting that such designs can support correct inhalation technique without compromising aerosol performance. By minimizing the influence of patient-dependent factors, this strategy may help standardize the inhalation process and improve therapeutic outcomes. Video Abstract available for this article. Video Abstract (MP4 85313 KB).</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-10","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145034065","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 Adherence and Persistence of Upfront Therapy in Patients with Pulmonary Arterial Hypertension in the United States.","authors":"Teresa De Marco, Carly J Paoli, Nicole S Croteau, Fei Tang, Harrison W Farber","doi":"10.1007/s41030-025-00311-4","DOIUrl":"https://doi.org/10.1007/s41030-025-00311-4","url":null,"abstract":"<p><strong>Introduction: </strong>Pulmonary arterial hypertension (PAH) is a rare, progressive disease resulting from elevated pulmonary arterial pressure leading to right ventricular failure and death. Optimal adherence and persistence to medical therapy are necessary to improve outcomes. The objective of this study was to characterize adherence and persistence to first-line PAH therapies in patients newly initiating treatment.</p><p><strong>Methods: </strong>This retrospective cohort study utilized Komodo Research Database claims data. Adults initiating therapy were identified based on ≥ 1 claim for a phosphodiesterase 5 inhibitor (PDE5i) and/or an endothelin receptor antagonist (ERA) from January 1, 2017, to June 30, 2022 (index date), continuous medical and pharmacy health plan enrollment for ≥ 12 months before and including index, ≥ 1 inpatient or ≥ 2 outpatient claims for pulmonary hypertension/PAH, and ≥ 1 claim for right heart catheterization. Adherence was measured by proportion of days covered (PDC); nonadherence was defined as PDC < 80%. Persistence was defined as time from index to treatment discontinuation (gap in therapy > 60 days). Propensity score matching was utilized 1:1:1 across groups.</p><p><strong>Results: </strong>A total of 9176 patients met the study criteria (6989 PDE5i, 1006 ERA, 1181 dual combination). After matching, each cohort included 714 patients. Median (95% confidence interval) persistence was highest for ERA monotherapy (26.5 [19.0-33.0] months), followed by dual combination therapy (19.8 [16.6-23.4] months) and PDE5i monotherapy (12.9 [10.8-17.4] months)-P = 0.019, dual combination versus ERA; P = 0.026, dual combination versus PDE5i. Nonadherence was highest with dual combination therapy (35.4%), followed by PDE5i monotherapy (17.1%) and ERA monotherapy (11.9%)-P < 0.001, dual combination versus each monotherapy.</p><p><strong>Conclusions: </strong>Adherence to initial PAH therapy is suboptimal, especially with upfront dual combination therapy. Persistence was highest for ERA monotherapy, followed by dual combination therapy and PDE5i monotherapy. Strategies to improve adherence and persistence are crucial to optimizing outcomes.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":""},"PeriodicalIF":3.0,"publicationDate":"2025-09-06","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145006558","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":"Assessment of Small Airways Function in Eosinophilic Preserved Ratio Impaired Spirometry.","authors":"Tianran Zhou, Hongyu Gao, Xingxing Sun, Jianhua Xu, Hanqing Zhu, Mian He, Wenlan Yang, Jinming Liu, Jian Guo","doi":"10.1007/s41030-025-00309-y","DOIUrl":"10.1007/s41030-025-00309-y","url":null,"abstract":"<p><strong>Introduction: </strong>Preserved ratio impaired spirometry (PRISm) is an important phenotype of pulmonary function in clinical and public health practice. It is possible for some patients to have chronic obstructive pulmonary disease (COPD) at an early stage. At present there is little research on the association of PRISm with type 2 (T2) inflammation biomarkers. The blood eosinophilia and impairment of small airway function in PRISm have not been fully assessed. This study investigated the eosinophilic phenotype in PRISm.</p><p><strong>Methods: </strong>Between January 2019 and September 2022, a retrospective assessment was conducted in a single pulmonary function unit in China. PRISm was defined as forced expiratory volume in 1 s (FEV1)/forced vital capacity (FVC) ≥ 70% and FEV1 < 80% predicted. Two groups were formed among the PRISm participants: eosinophilic PRISm, blood eosinophil count (BEC) ≥ 150/µL; non-eosinophilic PRISm, BEC < 150/µL. Differences were analyzed between eosinophilic and non-eosinophilic PRISm.</p><p><strong>Results: </strong>The study included 313 participants, of whom 135 were assigned to the eosinophilic PRISm group. After adjusting for potential confounders, compared to non-eosinophilic PRISm, eosinophilic PRISm remained correlated with lower natural logarithm (ln) MEF25% predicted (- 0.161 [- 0.267, - 0.054], P = 0.003) and elevated fractional exhaled nitric oxide (FeNO) (10.616 [6.384, 14.849], P < 0.001).</p><p><strong>Conclusion: </strong>Eosinophilic phenotype was common in individuals with PRISm. Compared to participants with non-eosinophilic PRISm, those with eosinophilic PRISm tended to have impaired MEF25% predicted and elevated FeNO.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":"11 3","pages":"461-473"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12414862/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145016130","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":"Obstructive Sleep Apnea and Sleep Disorders in Children with Attention Deficit Hyperactivity Disorder.","authors":"Mai Nguyen-Thi-Phuong, Mai Nguyen-Thi-Thanh, Robert Joel Goldberg, Hoa L Nguyen, An Dao-Thi-Minh, Sy Duong-Quy","doi":"10.1007/s41030-025-00299-x","DOIUrl":"10.1007/s41030-025-00299-x","url":null,"abstract":"<p><strong>Introduction: </strong>Sleep disorders are common yet often underdiagnosed in children with attention deficit/hyperactivity disorder (ADHD). These disturbances can exacerbate ADHD symptoms and negatively affect cognitive, emotional, and behavioral functioning. This study aimed to describe the prevalence of obstructive sleep apnea (OSA) and other sleep disorders in children with ADHD using standardized diagnostic criteria and to identify associated clinical and behavioral factors.</p><p><strong>Methods: </strong>A cross-sectional study was conducted on 629 children aged 6-12 years (mean age: 7.8 ± 1.5 years) who were diagnosed with ADHD. Sleep disturbances were assessed using the Children's Sleep Habits Questionnaire (CSHQ), the Pediatric Sleep Questionnaire (PSQ), and respiratory polygraphy. Sleep disorders were classified on the basis of the International Classification of Sleep Disorders, Third Edition (ICSD-3). Multivariate logistic regression was used to identify associated risk factors.</p><p><strong>Results: </strong>Sleep disorders were diagnosed in 70.0% of children with ADHD. The most common disorders were insomnia (40.2%), OSA (23.4%), parasomnias (27.8%), restless legs syndrome (10.5%), and delayed sleep-wake phase disorder (4.8%). The inattentive ADHD subtype, psychiatric comorbidities, tonsil and adenoid hypertrophy, iron-deficiency anemia, and sleep-related behaviors in children with ADHD were significantly associated with sleep disturbances.</p><p><strong>Conclusions: </strong>Sleep disorders are highly prevalent and diverse in children with ADHD. Early identification and targeted management of sleep disturbances, particularly OSA and insomnia, are essential to improving sleep quality and optimizing ADHD outcomes. Routine sleep screening should be integrated into clinical ADHD evaluations. Graphical abstract available for this article.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"423-441"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373571/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144576159","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":"The Evolution of the Indwelling Pleural Catheter.","authors":"Abdulla Baguneid, Thisarana Wijayaratne, Avinash Aujayeb, Rakesh Panchal","doi":"10.1007/s41030-025-00300-7","DOIUrl":"10.1007/s41030-025-00300-7","url":null,"abstract":"<p><p>An indwelling pleural catheter (IPC) is a valuable tool in the management of pleural effusions, allowing drainage strategies to be tailored to match patient-centred goals. Previously, IPCs were primarily utilised in malignant pleural effusion (MPE) in the presence of non-expandable lung (NEL) or after the failure of chemical pleurodesis. Several studies have compared IPC to intercostal chest drain (ICD) with talc pleurodesis (TP), as well as different drainage regimens, resulting in a transition of practice. Continued developments have led to novel adjuncts, such as digital drainage, which allow controlled flow rates. The emerging field of intrapleural therapy in MPE is gaining attention as a potential new treatment modality, possibly increasing the scope of IPCs further. This article will provide a narrative review of the role of IPCs and will be based on published evidence to date and highlight the importance of an individualised, patient-centred care approach.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"519-533"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373602/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144333802","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 Practical Approach to Pleural Infection.","authors":"Steven J Smith, Benjamin J Pippard","doi":"10.1007/s41030-025-00308-z","DOIUrl":"10.1007/s41030-025-00308-z","url":null,"abstract":"<p><p>Pleural infection encompasses a spectrum of disease that can present significant challenges in clinical practice. Despite better understanding of the underlying pathophysiology and microbiology, outcomes for patients remain poor. The use of antibiotics and chest tube drainage continue to be the mainstay of treatment, with surgery often reserved for those not responding to initial medical therapy. However, at present, the optimal management strategy for individual patients-including the role of early surgical and/or intrapleural therapy-is not clear. In this article, we provide an overview of the pathophysiology, diagnosis and management of pleural infection, highlighting current concepts and key practice points to aid the reader in caring for this important and often complex group of patients.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"535-551"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373619/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144718352","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":"Real-World Use of MART in Moderate-Severe Asthma: Results from the Italian WAMP Survey among Healthcare Professionals and Patients.","authors":"Fulvio Braido, Matteo Bonini, Walter Castellani, Andrea Claudio Comel, Francesco Paolo Lombardo, Antonio Spanevello, Alessandro Vatrella, Marco Contoli","doi":"10.1007/s41030-025-00310-5","DOIUrl":"10.1007/s41030-025-00310-5","url":null,"abstract":"<p><strong>Introduction: </strong>Moderate-severe asthma affects a significant proportion of patients and poses challenges in symptom control and exacerbation prevention. The preferred track 1 endorsed by the Global Initiative for Asthma (GINA) recommendations offers a single-inhaler approach combining inhaled corticosteroids and formoterol for both maintenance and symptom relief (maintenance and reliever therapy; MART). However, MART's real-world adoption remains suboptimal and concerns regarding its correct implementation persist. \"What About MART Posology\" (WAMP) survey assessed the knowledge and clinical application of MART among Italian healthcare professionals (HCPs) and patients.</p><p><strong>Methods: </strong>WAMP was a cross-sectional, web-based survey conducted among 1000 Italian HCPs and 400 patients with moderate-severe asthma. HCPs answered questions regarding treatment preferences, adherence to GINA recommendations and MART implementation. Patients reported on their therapeutic regimens, inhaler use, and adherence behaviors.</p><p><strong>Results: </strong>Most HCPs demonstrated awareness of GINA recommendations. Pulmonologists (73.6%) and allergists (62.0%) reported favoring track 1, while general practitioners (GPs) showed greater variability (55.1%). Most of HCPs reported the use of inhaled corticosteroids (ICS)-formoterol, according to the MART approach, to manage moderate-severe asthma. GPs reported that approximately 45.5% of moderate-severe patients with asthma treated with ICS-formoterol inhaled therapy were also prescribed short-acting β2-agonists (SABA). Among patients, ICS-formoterol was the most reported regimen (59.7%), despite only 21.6% adhered to the MART approach correctly. Triple therapy was preferred for patients with recurrent exacerbations, yet its adoption was lower than expected.</p><p><strong>Conclusions: </strong>The WAMP survey suggests a strong awareness of GINA track 1 among Italian HCPs. MART was widely implemented, particularly by specialists; patient data supported these findings. Gaps in education on MART's dual function persist though. Targeted training for HCPs and improved patient education are essential to optimize asthma management and adherence to evidence-based strategies.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"475-489"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373597/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144785148","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":"Electronic Nicotine Delivery Systems (ENDS): Implications for the Clinician.","authors":"Jean-Guillaume Starnini, Giulio Natalello, Federico Nigroli, Chiara Diana, Elena Bargagli, Andrea Sisto Melani","doi":"10.1007/s41030-025-00305-2","DOIUrl":"10.1007/s41030-025-00305-2","url":null,"abstract":"<p><p>The evidence that tobacco cigarettes are harmful to the health of smokers led to the introduction of electronic nicotine delivery systems (ENDS) as a safer alternative. ENDS, which include electronic cigarettes (e-cigs) and heated tobacco products (battery-operated devices that heat a liquid and produce an aerosol), are portable, cheap, easy-to-use, self-powered devices, and resemble tobacco cigarettes. After an overview of the toxicological, clinical, and epidemiological implications associated with the increasingly widespread use of ENDS, this narrative paper evaluates their role as a smoking cessation aid. Randomized controlled trials show that e-cigs can help in achieving cigarette smoking cessation, but their role in real life is still debated. There is no clear association in current smokers between the prevalence of e-cig use and overall quit rates. Although ENDS are not Food and Drug Administration (FDA)- and European Medicines Agency (EMA)-approved for quitting, they are one of the most widely utilized pharmacological support devices for smoking cessation. Physicians should ask for ENDS use and amount at each visit, be able to advise on how to manage ENDS as an aid for quitting, encourage vapers not to continue their use indefinitely, and explain how to stop ENDS.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"387-404"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373594/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144626972","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":"Study Design and Rationale for the PHINDER Study: Pulmonary Hypertension Screening in Patients with Interstitial Lung Disease for Earlier Detection.","authors":"Tejaswini Kulkarni, David A Zisman, Oksana A Shlobin, David G Kiely, Maral DerSarkissian, Eric Shen, Kevin M Maher, Meredith Broderick, Mary Beth Scholand","doi":"10.1007/s41030-025-00307-0","DOIUrl":"10.1007/s41030-025-00307-0","url":null,"abstract":"<p><strong>Introduction: </strong>A common complication of interstitial lung disease (ILD) is pulmonary hypertension (PH), which is associated with increased morbidity and mortality and worsened quality of life. In ILD, evaluating for PH is recommended prior to lung transplantation. However, this is not standardized or routinely performed in earlier stages of ILD, and guidelines lack an evidence-based approach for PH screening in this population. Furthermore, right-heart catheterization (RHC) access can be limited in many settings. The objective of PHINDER (Pulmonary Hypertension Screening in Patients with Interstitial Lung Disease for Earlier Detection) is to prospectively develop screening strategies for PH in patients with ILD.</p><p><strong>Methods: </strong>PHINDER is a prospective, non-interventional study that will enroll approximately 200 patients with ILD treated in a variety of settings in the United States (community centers, academic institutions, etc.). Patients must be diagnosed with ILD by high-resolution computed tomography (HRCT) and must not have a previously reported mean pulmonary arterial pressure (mPAP) > 20 mmHg. To enrich the population for PH, patients must meet additional criteria on Pulmonary Function Tests, HRCT, signs/symptoms, 6-min walk test, or echocardiography. Patients will undergo a variety of routine ILD clinical assessments. Lastly, patients receive a RHC to assess for PH, defined as mPAP > 20 mmHg with pulmonary arterial wedge pressure ≤ 15 mmHg and a pulmonary vascular resistance > 2 Wood Units. All treatment decisions are at the discretion of the provider and not influenced by study participation.</p><p><strong>Planned outcomes: </strong>Following study completion, statistical tools will be used to derive a practical model for a screening algorithm using the variables identified in the study as most predictive of PH in patients with ILD.</p><p><strong>Conclusions: </strong>Using a previously developed list of clinical assessments from PH and ILD experts, the PHINDER study aims to be the first prospectively enrolled study to evaluate prognostic screening strategies that can be used to develop an algorithm to predict the risk of PH in patients with ILD.</p><p><strong>Trail registration: </strong>NCT05776225.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"491-501"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373579/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144708578","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":"Impact of CFTR Modulators on Longitudinal Cystic Fibrosis Survival and Mortality: Review and Secondary Analysis.","authors":"Jaime L Rubin, Craig McKinnon, Gabriel Ghizzi Pedra, Devon A Morgan, Kimberly Zweig, Theodore G Liou","doi":"10.1007/s41030-025-00303-4","DOIUrl":"10.1007/s41030-025-00303-4","url":null,"abstract":"<p><strong>Introduction: </strong>Cystic fibrosis (CF) transmembrane conductance regulator modulators (CFTRm) have transformed CF care, shifting treatment from only managing symptoms to also addressing the underlying defects that cause CF. CFTRm first entered clinical practice in 2012 and was followed by additional CFTRm combinations-including the approval of elexacaftor/tezacaftor/ivacaftor (ELX/TEZ/IVA) in 2019-which treats most CF genotypes.</p><p><strong>Methods: </strong>We identified peer-reviewed literature for a narrative review (January 1990 to January 2025) describing longitudinal trends in CF survival and age of death and assessing the influence of CFTRm, particularly ELX/TEZ/IVA. To supplement the existing literature, a secondary analysis of historical, longitudinal trends in the United States CF Foundation Patient Registry (U.S. CFFPR, 1990-2023) was conducted using recent available data.</p><p><strong>Results: </strong>Quantitative data from published studies show that the median age of survival and death increased over time but with varying magnitudes across regions. Most cohort and registry-based studies were conducted in settings where CFTRm were not yet widely available, limiting the evaluation of CFTRm effects on survival trends over time. In the secondary U.S. CFFPR analysis, the median survival age increased from 29.0 years in 1990 to 38.6 years in 2012 prior to the introduction of CFTRm and to 68.0 years in 2023, demonstrating substantial improvement following the introduction of CFTRm. Linear regression analyses showed gains in median survival age increased from 0.48 years per year prior to CFTRm to 4.79 years per year after approval of ELX/TEZ/IVA in 2019.</p><p><strong>Conclusions: </strong>Study results provide initial evidence of the impact of CFTRm to meaningfully improve survival. Longer-term follow-up data across geographies will provide a deeper understanding of the full impact of CFTRm on predicted CF survival and mortality.</p>","PeriodicalId":20919,"journal":{"name":"Pulmonary Therapy","volume":" ","pages":"365-386"},"PeriodicalIF":3.0,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12373600/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144619858","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}