Therapeutic Advances in Respiratory Disease最新文献

{"title":"Antifibrotic therapy combined with pulmonary vasodilator therapy may improve survival in patients with pulmonary fibrosis and pulmonary hypertension: a retrospective cohort study.","authors":"Christian Cardillo, Gerard J Criner, Shameek Gayen","doi":"10.1177/17534666251326743","DOIUrl":"10.1177/17534666251326743","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary fibrosis is a severe, progressive form of interstitial lung disease associated with increased morbidity and mortality. Pulmonary hypertension often accompanies severe pulmonary fibrosis and is also associated with worse outcomes. Antifibrotic therapy and pulmonary vasodilator therapy have demonstrated clinical benefits in pulmonary fibrosis and pulmonary hypertension, respectively. However, the benefit of combined antifibrotic and pulmonary vasodilator therapy in patients with both pulmonary fibrosis and pulmonary hypertension is less established.</p><p><strong>Objectives: </strong>We aimed to determine the effectiveness of a combination pulmonary vasodilator and antifibrotic therapy with regard to transplant-free survival and six-minute walk distance improvement in patients with pulmonary fibrosis and pulmonary hypertension.</p><p><strong>Design: </strong>This was a retrospective cohort study of patients with pulmonary fibrosis (idiopathic pulmonary fibrosis, combined pulmonary fibrosis and emphysema, and other fibrotic interstitial lung disease) and pulmonary hypertension diagnosed via right heart catheterization. Patients received antifibrotic therapy with or without pulmonary vasodilator therapy.</p><p><strong>Methods: </strong>Patients who received combination antifibrotic therapy and pulmonary vasodilator therapy were compared to those prescribed antifibrotic therapy alone. Transplant-free survival and change in six-minute walk distance were compared between the two groups. Multivariable Cox regression was performed to determine predictors of transplant-free survival.</p><p><strong>Results: </strong>Patients who received antifibrotic and pulmonary vasodilator therapy had significantly improved transplant-free survival (log rank <i>p</i> = 0.001). Treatment with antifibrotic and pulmonary vasodilator therapy was significantly and independently associated with reduced risk of death or lung transplantation (HR 0.24, 95% CI 0.06-0.93, <i>p</i> = 0.04). These patients had worse pulmonary hemodynamics than those receiving antifibrotic therapy alone.</p><p><strong>Conclusion: </strong>We found a potential survival benefit when pulmonary vasodilator therapy was given in combination with antifibrotic therapy in patients with pulmonary fibrosis and pulmonary hypertension. This may be reflective of a pulmonary vascular phenotype among those with pulmonary fibrosis and pulmonary hypertension. Further trials are needed to better elucidate which patients benefit from combination therapy.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251326743"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11907552/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143626139","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predicting response to benralizumab in patients with COPD: a plain language summary of publication of the GALATHEA and TERRANOVA studies.","authors":"Gerard J Criner, Dave Singh, Alberto Papi, Maria Jison, Natalya Makulova, Vivian H Shih, Laura Brooks, Peter N Barker, Ubaldo J Martin","doi":"10.1177/17534666241312060","DOIUrl":"10.1177/17534666241312060","url":null,"abstract":"<p><p>Summary<b>What is this summary about?</b>● This is a plain language summary of two articles originally published in <i>The New England Journal of Medicine</i> and <i>The Lancet Respiratory Medicine</i>. These articles presented the results of GALATHEA and TERRANOVA, two clinical studies that took place across 41 countries. ○ GALATHEA and TERRANOVA measured how patients' COPD changed from before their first <b>benralizumab</b> (10, 30, or 100 mg) injection, to after 56 weeks of treatment. ○ In both studies, <b>benralizumab</b> was compared with <b>placebo</b>. ○ To see whether <b>benralizumab</b> treatment would benefit any particular patients included in these studies, researchers carried out an additional analysis following the main studies of GALATHEA and TERRANOVA.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666241312060"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11905023/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606364","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Pulmonary nontuberculous mycobacterial infections among women with cystic fibrosis and non-cystic fibrosis bronchiectasis.","authors":"Jane E Gross, Morgan C Jones, Ashley Buige, D Rebecca Prevots, Shannon Kasperbauer","doi":"10.1177/17534666251323181","DOIUrl":"10.1177/17534666251323181","url":null,"abstract":"<p><p>Nontuberculous mycobacteria (NTM) are ubiquitous, opportunistic pathogens that can cause lung disease in people with non-cystic fibrosis bronchiectasis (NCFB) and cystic fibrosis (CF). The incidence of NTM pulmonary infections and lung disease has continued to increase worldwide over the last decade among both groups. Notably, women with NCFB NTM pulmonary disease (NTM-PD) bear a disproportionate burden with NTM rates increasing in this population as well as having consistently higher incidence of NTM-PD compared to men. In contrast, among people with CF, an overall increased risk among women has not been observed. In the United States, the majority of people with CF are taking highly effective cystic fibrosis transmembrane conductance regulator (CFTR) modulators, and these numbers are increasing worldwide. The long-term impact of CFTR modulator medications on NTM infections is not entirely understood. Guidelines for the screening, diagnosis, and management of NTM-PD exist for people with NCFB and CF, but do not consider unique implications relevant to women. This review highlights aspects of NTM-PD among women with NCFB and CF, including the epidemiology of NTM infection, special considerations for treatment, and unmet research needs relevant to women with NTM-PD.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251323181"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11898043/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606366","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"A novel decision tree algorithm model based on chest CT parameters to predict the risk of recurrence and metastasis in surgically resected stage I synchronous multiple primary lung cancer.","authors":"Shuangjiang Li, Guona Chen, Wenbiao Zhang, Huiyun Ma, Baocong Liu, Li Xu, Qiong Li","doi":"10.1177/17534666251325443","DOIUrl":"10.1177/17534666251325443","url":null,"abstract":"<p><strong>Background: </strong>Chest computed tomography (CT) may provide evidence to forecast unexpected recurrence and metastasis following radical surgery for stage I synchronous multiple primary lung cancer (SMPLC).</p><p><strong>Objective: </strong>This study aims to develop and validate a novel CT-based multi-parametric decision tree algorithm (CT-DTA) model capable of accurate risk assessment.</p><p><strong>Design: </strong>A multicenter retrospective cohort study.</p><p><strong>Methods: </strong>There were 209 patients with pathological stage I SMPLC from three tertiary centers included. We initially screened all of the CT-derived imaging parameters in the training cohort (130 patients from Center A) and then selected those showing statistical significance to construct a DTA model. The discriminative strength of the CT-DTA model for postoperative recurrence and metastasis was then validated in the validation cohort (79 patients from Centers B and C). Moreover, the performance of the CT-DTA model was further evaluated across different subgroups of the entire cohort.</p><p><strong>Results: </strong>Five key imaging parameters measured on chest thin-section CT, including consolidation tumor ratio (CTR), long-axis diameter of the lesion, number of pure solid nodules, presence of spiculation and pleural indentation, constituted a CT-DTA model with nine leaf nodes, and CTR was the leading risk contributor of them. The CT-DTA model achieved a satisfactory predictive accuracy indicated by an area under the curve of more than 0.80 in both the training cohort and validation cohort. Meanwhile, this CT-DTA model was also exhaustively demonstrated to play as the only independent risk factor for postoperative recurrence and metastasis. Its promising predictive performance still remained stable across nearly all of the subgroups stratified by clinicopathological characteristics.</p><p><strong>Conclusion: </strong>This CT-DTA model could serve as a noninvasive, user-friendly, and practicable risk prediction tool to aid treatment decision-making in operable stage I SMPLC.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251325443"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11907625/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143626138","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Sleep and breathing in children with Joubert syndrome and a review of other rare congenital hindbrain malformations.","authors":"Jia-Der Ju-Wang, Jennifer C Dempsey, Cristian Zhang, Daniel Doherty, Manisha Witmans, Mary Anne Tablizo, Maida Lynn Chen","doi":"10.1177/17534666241308405","DOIUrl":"https://doi.org/10.1177/17534666241308405","url":null,"abstract":"<p><strong>Background: </strong>Joubert syndrome (JS) is an autosomal recessive disorder with a distinctive mid-hindbrain malformation known as the \"molar tooth sign\" which involves the breathing control center and its connections with other structures. Literature has reported significant respiratory abnormalities which included hyperpnea interspersed with apneic episodes during wakefulness. Larger-scale studies looking at polysomnographic findings or subjective reports of sleep problems in this population have not yet been published.</p><p><strong>Objectives: </strong>The primary objectives were (1) compare a large group of children with JS and their unaffected siblings for caregiver-reported sleep difficulties. Secondary objectives were (1) present new polysomnography (PSG) data on our JS cohort; (2) review sleep disordered breathing (SDB) in other rare congenital hindbrain anatomic abnormalities.</p><p><strong>Design: </strong>We conducted a cross-sectional study on a cohort of 109 families affected by JS.</p><p><strong>Methods: </strong>Pediatric Sleep Questionnaire (PSQ) and the Children's Sleep Habits Questionnaire (CSHQ) along with general medical health information focused on respiratory and sleep problems were mailed to all patients and families. Caregivers were asked to complete the survey for both children with JS and unaffected siblings, if any. Baseline diagnostic PSG was retrospectively reviewed for those with available studies, and the sleep parameters were compared to a referent cohort.</p><p><strong>Results: </strong>Study participants with JS were older than their unaffected siblings (<i>p</i> = 0.02). Genetic mutations were available for 41 out of 118 individuals, with the most common mutation being MKS3 (31.4%). Patients with JS had higher scores in the PSQ compared to their unaffected siblings (<i>p</i> < 0.001). PSG data showed severe SDB with apnea-hypopnea index (AHI) of 23 ± 15 events/h in patients with JS. Events were primarily obstructive (obstructive AHI 18 ± 15 events/h vs central AHI 4 ± 4 events/h). Abnormal sleep architecture with increased arousal indices, decreased efficiency, and more time awake and in light sleep or wakefulness when compared to the referent data.</p><p><strong>Conclusion: </strong>SDB is common and severe in patients with JS, and the significantly greater obstructive component reported in this cohort makes it necessary to perform complete PSG studies to address or prevent clinical manifestations in this at-risk population. PSQ could represent a viable method to screen for SDB in JS.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666241308405"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11748088/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143056043","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Protective effects of statins on pulmonary function in patients with persistent hyperlipidemia: a retrospective cohort study.","authors":"Hsiao-Chin Shen, Che-Hao Tseng, Yi-Hsuan Lin, Hsiao-Yun Yeh, Hung-Cheng Tsai, Shiao-Ya Hong, Tzu-Hao Li, Chien-Wei Su, Diahn-Warng Perng, Ying-Ying Yang, Ming-Chih Hou","doi":"10.1177/17534666251320875","DOIUrl":"10.1177/17534666251320875","url":null,"abstract":"<p><strong>Background: </strong>Pulmonary function tests offer crucial parameters for evaluating lung health and predicting clinical outcomes. Hyperlipidemia, a prevalent metabolic disorder, has been linked to declining pulmonary function. Statins are an essential therapy for lowering lipid levels in hyperlipidemia.</p><p><strong>Objectives: </strong>This study aims to investigate the therapeutic potential of statins in mitigating the decline in pulmonary function.</p><p><strong>Design: </strong>This is a retrospective cohort study.</p><p><strong>Methods: </strong>Out of 8286 patients who underwent spirometry testing from January 2018 to December 2020, 492 patients were included in the final analysis. The relationship between statin usage, dosage, along with other biometric indices and spirometry parameters were evaluated. Multivariate logistic regression analyses were employed to assess the association between statin use and the decline in pulmonary function.</p><p><strong>Results: </strong>In patients with persistent hyperlipidemia, the use of statins was associated with a higher predicted percentage of forced expiratory volume in 1 second (FEV1) compared to non-users (84.0% vs 78.0%, <i>p</i> = 0.015). Logistic regression models further revealed that statin use independently prevented FEV1 decline, irrespective of dosage (adjusted OR 0.036, 95% CI: 0.002-0.618 in lower statins dose group and adjusted OR 0.170, 95% CI: 0.019-1.552 in higher statins dose group).</p><p><strong>Conclusion: </strong>The findings suggested that statin usage, regardless of dosage, independently mitigated the decline in pulmonary function among patients with persistent hyperlipidemia. Early initiation of statin therapy may hold promise for individuals experiencing hyperlipidemia and declining pulmonary function.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251320875"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11848893/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143484115","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Predictors for spontaneous pleurodesis in patients with indwelling pleural catheters for malignant pleural effusion: a safety net hospital experience.","authors":"Saad Farooq, Sabiha Armin, Jordan E Killingsworth, Akriti Agrawal, Adishwar Rao, Rosa M Estrada-Y-Martin, Sujith V Cherian","doi":"10.1177/17534666251318844","DOIUrl":"10.1177/17534666251318844","url":null,"abstract":"<p><strong>Background: </strong>Malignant pleural effusion (MPE) affects approximately 150,000 patients in the United States each year and usually signifies advanced-stage cancer. The optimal treatment remains a challenge but indwelling pleural catheters (IPC) offer several advantages and may help achieve spontaneous pleurodesis (SP) in some patients.</p><p><strong>Objectives: </strong>We aim to investigate the predictors of SP among patients with MPE, particularly in a resource-limited community-based safety net hospital.</p><p><strong>Design: </strong>This is a retrospective cohort study done at a community-based safety net hospital.</p><p><strong>Methods: </strong>Adults diagnosed with or suspected of having MPE between January 2015 and December 2023 who underwent IPC placement were included. Data was collected retrospectively from December 2023 to June 2024. Data encompassed demographics, imaging, post-procedural complications, pleural fluid analysis, oncology treatment history, and utilization of medical thoracoscopy without chemical pleurodesis (MTWCP) for diagnosis.</p><p><strong>Results: </strong>A total of 173 patients underwent IPC insertion. Most of our patients were women (64.2%), and Latin American (65.9%), with a mean age of 55.3 years. The most common type of primary cancer was breast (28.9%) followed by lung (23.1%) and lymphoma (6.9%). Pleural fluid characteristics such as glucose, eosinophils, Lactate Dehydrogenase (LDH), and protein concentration were not significantly associated with SP. Most patients had low Eastern Cooperative Oncology Group scores of 0-2 (64.6%) and low LENT (Lactate Dehydrogenase (L), Eastern Cooperative Oncology Group (E) Performance Score, Neutrophil-to-Lymphocyte Ratio (N), and Tumor type (T) score) scores of 0-4 (59%). Lower scores (better functional status) were significantly associated with SP. Post-IPC chemotherapy and/or radiotherapy and immunotherapy were significantly associated with SP, adjusted odds ratio (OR) 7.295 (95% CI: 3.05-17.4, <i>p</i> = 0.001) and adjusted OR 6.261 (95% CI: 2.73-14.36, <i>p</i> = 0.001) respectively. MTWCP was also a predictor of SP with an adjusted OR of 4.031 (95% CI: 1.452-11.19, <i>p</i> = 0.007).</p><p><strong>Conclusion: </strong>Our study is the first to assess predictors of SP in a resource-limited safety net hospital representing under-represented and underserved patients. We identify several factors associated with higher rates of SP such as higher functional status, MTWCP, chemotherapy, immunotherapy, and radiation post-IPC placement. The study findings can help clinicians consider IPC placement and guide them regarding the duration and possible complications of IPC. MTWCP appears to improve the success of SP. Further studies are needed to assess these findings further.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251318844"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11831654/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143433829","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cohort multiple randomized controlled trial in pediatric asthma to assess the long- and short-term effects of eHealth interventions: protocol of the CIRCUS study.","authors":"Tamara Ruuls, Romi Sprengers, Vera Hengeveld, Boony Thio, Monique Tabak, Deborah Zagers, Job van der Palen, Mattiènne van der Kamp","doi":"10.1177/17534666251323192","DOIUrl":"10.1177/17534666251323192","url":null,"abstract":"<p><strong>Background: </strong>Asthma is one of childhood's most prevalent chronic conditions significantly impacting the quality of life. Current asthma management lacks real-time, objective, and longitudinal monitoring reflected by a high prevalence of uncontrolled asthma. Long-term home monitoring promises to establish new clinical endpoints for timely anticipation. In addition, integrating eHealth interventions holds promise for timely and appropriate medical anticipation for controlling symptoms and preventing asthma exacerbations.</p><p><strong>Objectives: </strong>This study aims to provide a pragmatic study design for gaining insight into longitudinal monitoring, assessing, and comparing eHealth interventions' short- and long-term effects on improving pediatric asthma care.</p><p><strong>Design: </strong>The CIRCUS study design is a cohort multiple randomized controlled trial (cmRCT) with a dynamic cohort of 300 pediatric asthma patients.</p><p><strong>Methods: </strong>The study gathers observational and patient-reported measurements at set moments including patient characteristics, healthcare utilization, and asthma, clinical, and environmental outcomes. Participants are randomly appointed to the intervention or control group. The effects of the eHealth interventions are assessed and compared to the control group, deploying the CIRCUS outcomes. The participants continue in the CIRCUS cohort after completing the intervention and its follow-up.</p><p><strong>Results: </strong>This study was ethically approved by the Medical Research Ethics Committee (NL85668.100.23) on February 15th, 2024.</p><p><strong>Discussion: </strong>The CIRCUS study can provide a rich and unique dataset that can improve insight into risk factors of asthma exacerbations and yield new clinical endpoints. Furthermore, the effects of eHealth interventions can be assessed and compared with each other both short- and long-term. In addition, patient groups within the patient population can be discerned to tailor eHealth interventions to personalized needs on improving asthma management.</p><p><strong>Conclusion: </strong>In conclusion, CIRCUS can provide valuable clinical data to discern risk factors for asthma exacerbations, identify and compare effective scalable eHealth solutions, and improve pediatric asthma care.<b><i>Trial registration</i>:</b> The protocol is registered at ClinicalTrials.gov (NCT06278662).</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251323192"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11898028/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606361","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Cystic fibrosis: new challenges and perspectives beyond elexacaftor/tezacaftor/ivacaftor.","authors":"Vito Terlizzi, Miquéias Lopes-Pacheco","doi":"10.1177/17534666251323194","DOIUrl":"10.1177/17534666251323194","url":null,"abstract":"<p><p>Over the past decade, major clinical advances have been made in the healthcare and therapeutic development for cystic fibrosis (CF), a lethal genetic disease caused by mutations in the gene encoding the CF transmembrane conductance regulator (CFTR) protein. CFTR modulators represent innovative treatments that directly target the primary defects in the mutated CFTR protein and have demonstrated significant clinical benefits for many people with CF (pwCF) who are eligible for these treatments. In particular, the triple combination therapy composed of elexacaftor, tezacaftor, and ivacaftor (ETI) has changed the CF therapeutic landscape by significantly improving lung function, quality of life, and predicted survival rates. Here, we provided a comprehensive summary of the impact of ETI on clinical outcomes and the need for further research on long-term efficacy, side effects, pregnancy, possible drug-drug interactions, and extra-pulmonary manifestations. Moreover, a significant number of pwCF are unresponsive to these drugs or cannot afford their high costs. We, therefore, discussed health inequity issues and alternative therapeutic strategies under development aiming to obtain effective therapies for all pwCF.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666251323194"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143754185","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}

{"title":"Potential of phosphodiesterase 4B inhibition in the treatment of progressive pulmonary fibrosis.","authors":"Rebecca Keith, Anoop M Nambiar","doi":"10.1177/17534666241309795","DOIUrl":"10.1177/17534666241309795","url":null,"abstract":"<p><p>Idiopathic pulmonary fibrosis (IPF) is often regarded as the archetypal progressive fibrosing interstitial lung disease (ILD). The term \"progressive pulmonary fibrosis\" (PPF) generally describes progressive lung fibrosis in an individual with an ILD other than IPF. Both IPF and PPF are associated with loss of lung function, worsening dyspnea and quality of life, and premature death. Current treatments slow the decline in lung function but have side effects that may deter the initiation or continuation of treatment. There remains a high unmet need for additional therapies that can be used alone or in combination with current therapies to preserve lung function in patients with IPF and PPF. Phosphodiesterase-4 (PDE4) is an enzyme involved in the regulation of inflammatory processes. Pre-clinical studies have shown that preferential inhibition of PDE4B has anti-inflammatory and antifibrotic effects and a lower potential for gastrointestinal adverse events than pan-PDE4 inhibition. The preferential PDE4B inhibitor nerandomilast demonstrated efficacy in preserving lung function in a phase II trial in patients with IPF and is under investigation in phase III trials as a treatment for IPF and PPF.</p>","PeriodicalId":22884,"journal":{"name":"Therapeutic Advances in Respiratory Disease","volume":"19 ","pages":"17534666241309795"},"PeriodicalIF":3.3,"publicationDate":"2025-01-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11694302/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142915713","PeriodicalName":null,"FirstCategoryId":null,"ListUrlMain":null,"RegionNum":3,"RegionCategory":"医学","ArticlePicture":[],"TitleCN":null,"AbstractTextCN":null,"PMCID":"OA","EPubDate":null,"PubModel":null,"JCR":null,"JCRName":null,"Score":null,"Total":0}