Pediatric Pulmonology最新文献

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Upcoming events of interest. 即将发生的有趣事件。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71346
{"title":"Upcoming events of interest.","authors":"","doi":"10.1002/ppul.71346","DOIUrl":"https://doi.org/10.1002/ppul.71346","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71346"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293131","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}
引用次数: 0
Identification and Validation of TFF3 as a Diagnostic Biomarker and Therapeutic Agent for Bronchopulmonary Dysplasia. TFF3作为支气管肺发育不良诊断生物标志物和治疗剂的鉴定和验证。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71320
Zhenzhuang Zou, Li Fu, Jiaying Liu, Yunfei He, Bo Huang, Shaohua Wang
{"title":"Identification and Validation of TFF3 as a Diagnostic Biomarker and Therapeutic Agent for Bronchopulmonary Dysplasia.","authors":"Zhenzhuang Zou, Li Fu, Jiaying Liu, Yunfei He, Bo Huang, Shaohua Wang","doi":"10.1002/ppul.71320","DOIUrl":"https://doi.org/10.1002/ppul.71320","url":null,"abstract":"<p><strong>Background: </strong>Bronchopulmonary dysplasia (BPD) is a common and severe complication in preterm infants, driven by oxidative stress, inflammation, and disrupted alveolar development. Despite advancements in neonatal care, current treatments remain supportive, with limited options targeting the underlying mechanisms of BPD pathogenesis. Emerging evidence suggests that modulating cell death pathways, including apoptosis and pyroptosis, may provide therapeutic benefits. This study aims to investigate the role of TFF3 as a diagnostic biomarker and therapeutic agent for BPD, focusing on its potential to regulate cell death and promote alveolar repair.</p><p><strong>Methods: </strong>Tracheal aspirate samples from BPD and control groups were analyzed using the GSE156028 data set to identify differentially expressed genes (DEGs). Oxidative stress (OS)-related DEGs were prioritized through protein-protein interaction (PPI) network analysis and machine learning algorithms (XGBoost and random forest). Experimental validation was conducted in a hyperoxia-induced BPD rat model and RLE-6TN cells to evaluate TFF3's effects on alveolar development, inflammation, apoptosis, and pyroptosis through histological, immunohistochemical, and molecular assays.</p><p><strong>Results: </strong>TFF3 was identified as a top-ranking biomarker for BPD, with robust diagnostic performance (AUC = 0.709). Hyperoxia-induced BPD rats exhibited reduced TFF3 expression, severe alveolar simplification, and elevated apoptotic and pyroptotic markers. TFF3 treatment significantly improved alveolar structure, reduced mean linear intercept (MLI), and partially restored pulmonary vascular density. In vitro, TFF3 enhanced cell viability, suppressed cleaved caspase-3 and NLRP3 expression, and decreased IL-1β and IL-18 secretion. These results demonstrate that TFF3 mitigates hyperoxia-induced inflammation, apoptosis, and pyroptosis while promoting alveolar and vascular repair.</p><p><strong>Conclusions: </strong>TFF3 serves as a promising biomarker and Therapeutic agent for BPD. By regulating multiple pathological pathways, TFF3 alleviates hyperoxia-induced lung injury and restores alveolar development. These findings provide a foundation for future clinical studies aimed at optimizing TFF3-based therapies for preterm infants with BPD.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71320"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293181","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}
引用次数: 0
Antenatal Late Preterm Infants Exposure to Steroids (ALPINES) and Risk of Respiratory Distress and Hypoglycemia: A Retrospective Chart Review. 产前晚期早产儿暴露于类固醇(ALPINES)和呼吸窘迫和低血糖的风险:回顾性图表回顾。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71336
Naveed Ur Rehman Durrani, Maria Bashir Ibrahim Mohammed, Farhan Sachal Cyprian, Muhammad Naseem Khan
{"title":"Antenatal Late Preterm Infants Exposure to Steroids (ALPINES) and Risk of Respiratory Distress and Hypoglycemia: A Retrospective Chart Review.","authors":"Naveed Ur Rehman Durrani, Maria Bashir Ibrahim Mohammed, Farhan Sachal Cyprian, Muhammad Naseem Khan","doi":"10.1002/ppul.71336","DOIUrl":"https://doi.org/10.1002/ppul.71336","url":null,"abstract":"<p><strong>Introduction: </strong>There is a need for a better understanding of the use of antenatal steroids (ANS) in late preterm infants due to their efficacy and safety profile from existing evidence.</p><p><strong>Aims: </strong>To identify the real-world impact of ANS on short-term outcomes in late preterm infants at a quaternary care setting in Qatar.</p><p><strong>Methods: </strong>Retrospective chart review of 654 late preterm infants delivered at Sidra Medicine between 1st January 2018 and 29th February 2024. After excluding criteria, infants were categorized based on their exposure to ANS. We utilized regression models to analyze the relationship between ANS exposure and primary and secondary outcomes, including respiratory distress, Noninvasive respiratory support, hypoglycemia, and length of NICU stay, respectively, while adjusting for relevant maternal and infant characteristics.</p><p><strong>Results: </strong>Our findings reveal no significant protective effect of any ANS dosage against respiratory distress (adjusted odd ratio (aOR) 0.78; 95% CI: 0.46-1.32; p = 0.357). Conversely, ANS exposure was associated with an increased risk of hypoglycemia (aOR 1.22; 95% CI: 0.81-1.84; p = 0.350) and prolonged stay in NICU (incidence rate ratio [IRR] 1.90; 95% CI: 1.74-2.08; p < 0.001).</p><p><strong>Conclusion: </strong>Our study findings suggest a lack of protection against respiratory distress in late preterm infants with any ANS, highlighting the need for better understanding and adopting an individualized approach.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71336"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293383","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}
引用次数: 0
Large Airway and Lung Findings in Symptomatic Children and Young Adults With Down Syndrome. 有症状的唐氏综合症儿童和青年的大气道和肺的发现。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71339
Emily DeBoer, Kristine Wolter-Warmerdam, Francis Hickey, Jason P Weinman
{"title":"Large Airway and Lung Findings in Symptomatic Children and Young Adults With Down Syndrome.","authors":"Emily DeBoer, Kristine Wolter-Warmerdam, Francis Hickey, Jason P Weinman","doi":"10.1002/ppul.71339","DOIUrl":"https://doi.org/10.1002/ppul.71339","url":null,"abstract":"<p><strong>Background and objectives: </strong>Epidemiology studies report pulmonary disease, lung infection, and pneumonia as the largest causes of morbidity and mortality in individuals with Down syndrome (DS), but how significant airway and lung diagnoses contribute to morbidity remains unknown. Our goal was to evaluate the presence and presentation of structural lung abnormalities from chest computed tomography (CT) scans in this population.</p><p><strong>Methods: </strong>Retrospective review of chest CT scans performed clinically between 2012 and 2023 merged with clinical data was conducted on individuals with DS (birth-22 years; n = 80) treated at one institution. Chi-square and Fisher's exact tests evaluated associations between large airway and lung findings and co-occurring diagnoses.</p><p><strong>Results: </strong>Overall, 85.0% (n = 68/80) of patients in this cohort of children with DS (average age = 7.95 years, SD = 6.69) had abnormal CT results: large airway abnormalities only (n = 20, 25.0%), abnormal lung findings only (n = 28, 35.0%), both large airway and lung abnormalities (n = 11, 13.8%), and other abnormal results without large airway and lung abnormalities (n = 9, 11.2%). The most common large airway abnormality was tracheal compression from vascular ring or other aberrant vasculature (16.3%). Common abnormal lung findings were peribronchial thickening (22.5%), cystic abnormality/lucencies, and architectural distortion (18.8%). Children with lung abnormalities were more likely to have a history of obstructive sleep apnea and higher incidence of chronic lung disease, aspiration, respiratory distress syndrome at birth, and pulmonary hypertension.</p><p><strong>Conclusions: </strong>There was a high rate of abnormalities on chest CT in children with DS who required clinical CT scanning. The moderate association between pulmonary diagnoses and lung findings increases concern that structural lung disease is related to pulmonary morbidity.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71339"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293149","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}
引用次数: 0
Exhaled Carbon Dioxide Monitoring Using Instantaneous CO2 Mode During High-Frequency Oscillatory Ventilation in Very Low Birth Weight Infants. 极低出生体重儿高频振荡通气过程中瞬时CO2模式的呼出二氧化碳监测。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71323
Yusuke Nakazawa, Kazuto Ueda, Yoshihiro Tanahashi, Toshiki Aoki, Yoshiaki Sato
{"title":"Exhaled Carbon Dioxide Monitoring Using Instantaneous CO<sub>2</sub> Mode During High-Frequency Oscillatory Ventilation in Very Low Birth Weight Infants.","authors":"Yusuke Nakazawa, Kazuto Ueda, Yoshihiro Tanahashi, Toshiki Aoki, Yoshiaki Sato","doi":"10.1002/ppul.71323","DOIUrl":"10.1002/ppul.71323","url":null,"abstract":"<p><strong>Background: </strong>High-frequency oscillatory ventilation (HFOV) presents challenges for noninvasive CO<sub>2</sub> monitoring due to its rapid oscillations. This study examines the feasibility of instantaneous CO<sub>2</sub> partial pressure (INST CO<sub>2</sub>) monitoring during HFOV with a capnometer equipped with a mainstream CO<sub>2</sub> sensor.</p><p><strong>Methods: </strong>This study included seven neonates receiving HFOV in the neonatal intensive care unit (NICU) at Shizuoka Children's Hospital. INST CO<sub>2</sub> values were recorded over 3- and 5-min intervals surrounding each blood gas sampling to determine the maximum INST CO<sub>2</sub> value (INST CO<sub>2</sub> MX). The primary outcome was the correlation between INST CO<sub>2</sub> MX and arterial partial pressure of carbon dioxide (PCO<sub>2</sub>).</p><p><strong>Results: </strong>A total of 216 paired INST CO<sub>2</sub> MX and PCO<sub>2</sub> values were analyzed. The coefficient of determination (R<sup>2</sup>) was 0.571 for INST CO<sub>2</sub> MX (3 min) and 0.579 for INST CO<sub>2</sub> MX (5 min). Univariable and multivariable analyses revealed that a lower fraction of inspired oxygen (FiO<sub>2</sub>), reduced minute ventilation, and increased postnatal age were associated with improved concordance between INST CO<sub>2</sub> MX and PCO<sub>2</sub>.</p><p><strong>Conclusion: </strong>INST CO<sub>2</sub> MX demonstrated a strong correlation with PCO<sub>2</sub> in neonates undergoing HFOV. This novel, noninvasive monitoring approach may serve as a valuable tool for neonatal respiratory management. Future research is needed to validate these findings in broader patient populations and across various ventilatory settings.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71323"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12522078/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293190","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}
引用次数: 0
Can We Enhance Long Term Respiratory Outcomes of Neonatal Pulmonary Diseases? 我们能提高新生儿肺部疾病的长期呼吸预后吗?
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71341
Amir Kugelman
{"title":"Can We Enhance Long Term Respiratory Outcomes of Neonatal Pulmonary Diseases?","authors":"Amir Kugelman","doi":"10.1002/ppul.71341","DOIUrl":"https://doi.org/10.1002/ppul.71341","url":null,"abstract":"<p><p>Infants born prematurely and infants born with neonatal pulmonary diseases are at risk for long term abnormal lung functions. In this review we will discuss efforts to enhance long term respiratory outcomes in premature infants, including the use of tracheostomy in those with severe bronchopulmonary dysplasia (BPD). We will further discuss long term respiratory abnormalities in newborns with congenital diaphragmatic hernia (CDH), representing infants with lung hypoplasia.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71341"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346577","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}
引用次数: 0
Development of a Simplified Arabic Questionnaire of Health-Related Quality of Life for Children With Cystic Fibrosis. 囊性纤维化儿童健康相关生活质量简易阿拉伯语问卷的编制
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71332
Heba A Ali, Neven S Baiome, Yasmin A Salem
{"title":"Development of a Simplified Arabic Questionnaire of Health-Related Quality of Life for Children With Cystic Fibrosis.","authors":"Heba A Ali, Neven S Baiome, Yasmin A Salem","doi":"10.1002/ppul.71332","DOIUrl":"https://doi.org/10.1002/ppul.71332","url":null,"abstract":"<p><strong>Background: </strong>The measurement of the quality of life (QoL) of cystic fibrosis (CF) children in the Arabic world is of substantial importance. However, the relative lack of disease-specific Arabic QoL questionnaires may represent a major challenge.</p><p><strong>Objectives: </strong>To develop a simple questionnaire that could be used for assessing and monitoring the level of QoL in CF children, particularly in Arabic-speaking populations.</p><p><strong>Patients and methods: </strong>This was a cross-sectional study involving 50 children and adolescent patients with CF and their caregivers who completed 2 self-administered questionnaires on health-related quality of life (HRQOL); the Arabic simplified CF Questionnaire-Child version for children aged more than 8 years, and the Parent version for children aged from 2 to 8 years. The psychometric properties of the questionnaires were evaluated. Children's data and clinical severity scoring were also collected.</p><p><strong>Results: </strong>The CF QoL questionnaires (child and parent versions) exhibited good internal consistency and reliability, with Cronbach's α values being (α = 0.91, 0.871). Item-item and item-total correlation analyses showed adequate construct validity (0.31-0.86) and (0.277-0.682). Furthermore, patients aged 2-8 years scored significantly better than those aged more than 8 years for the total HRQOL score (p < 0.001).</p><p><strong>Conclusions: </strong>The simplified CF QoL questionnaires are reliable and valid measures of HRQOL for CF children, especially in Arabic-speaking populations. The younger children with CF showed satisfactory health-related QoL levels compared to the older ones. These findings support the important role of a careful psychological assessment of CF children as a part of monitoring the disease progression.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71332"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145346580","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}
引用次数: 0
Objectively Monitored Sleep in School-Age Children With Cystic Fibrosis and Their Parents. 囊性纤维化学龄儿童及其父母的客观睡眠监测。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-10-01 DOI: 10.1002/ppul.71325
Andrea L Fidler, Casey Lawless, James Peugh, Dean W Beebe, Silvia Delgado, Robin S Everhart, David A Fedele
{"title":"Objectively Monitored Sleep in School-Age Children With Cystic Fibrosis and Their Parents.","authors":"Andrea L Fidler, Casey Lawless, James Peugh, Dean W Beebe, Silvia Delgado, Robin S Everhart, David A Fedele","doi":"10.1002/ppul.71325","DOIUrl":"10.1002/ppul.71325","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 10","pages":"e71325"},"PeriodicalIF":2.3,"publicationDate":"2025-10-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12522017/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145293167","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}
引用次数: 0
Postexercise Airway Responses by Spirometry and Oscillometry in Nonathlete and Athlete Adolescents. 非运动员和运动员青少年运动后气道反应的肺活量测定和振荡测定。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-09-01 DOI: 10.1002/ppul.71296
Karin Ersson, Kjell Alving, Margareta Emtner, Christer Janson, Henrik Johansson, Andrei Malinovschi
{"title":"Postexercise Airway Responses by Spirometry and Oscillometry in Nonathlete and Athlete Adolescents.","authors":"Karin Ersson, Kjell Alving, Margareta Emtner, Christer Janson, Henrik Johansson, Andrei Malinovschi","doi":"10.1002/ppul.71296","DOIUrl":"10.1002/ppul.71296","url":null,"abstract":"<p><strong>Background: </strong>Exercise-induced bronchoconstriction (EIB) is common among adolescents and athletes. While typically assessed with spirometry, oscillometry may offer complementary insights. This cross-sectional study examined how fractional exhaled nitric oxide (FeNO) and symptoms relate to postexercise airway responses assessed via spirometry and oscillometry, and whether these associations differ between nonathletes and athletes.</p><p><strong>Methods: </strong>Subsamples from two adolescent cohorts (N = 241; 143 nonathletes, 98 athletes) completed questionnaires, FeNO measurements, and an EIB test using spirometry, by change in forced expiratory volume in 1 s (∆FEV<sub>1</sub>), and oscillometry, by change in resistance and reactance at 5 Hz (∆R<sub>5</sub>, ∆X<sub>5</sub>). Correlations were assessed using Spearman's rank; logistic regression evaluated associations between elevated FeNO and EIB; linear regression explored links between symptoms and postexercise airway responses.</p><p><strong>Results: </strong>EIB was detected in 70 participants via spirometry (∆FEV<sub>1</sub> ≤ -10%), 81 via oscillometry (∆R<sub>5</sub> ≥ 25%), and 37 by both. Among nonathletes, FeNO was weakly correlated with ∆FEV<sub>1</sub> (r<sub>s</sub> = -0.17, p = 0.04), ∆R<sub>5</sub> (0.35, p < 0.001), and ∆X<sub>5</sub> (-0.25, p = 0.005). Elevated FeNO (> 20 parts per billion) was associated with EIB by ∆FEV<sub>1</sub> (aOR 2.54, 95% CI: 1.05-6.12) and ∆R<sub>5</sub> (aOR 3.05, 95% CI: 1.18-7.9). Respiratory symptoms also related to postexercise airway responses in nonathletes. In contrast, no such associations were observed in athletes.</p><p><strong>Conclusion: </strong>In nonathletes, elevated FeNO and symptoms can indicate who needs EIB testing. These indicators were less predictive in athletes, emphasizing the value of objective assessment. Oscillometry was complementary to spirometry to assess EIB and a combination of methods might be more informative.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 9","pages":"e71296"},"PeriodicalIF":2.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12445329/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145081292","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}
引用次数: 0
Elexacaftor/Tezacaftor/Ivacaftor Effect on Bone Density and Body Composition: A Retrospective Analysis. elexaftor / tezactor /Ivacaftor对骨密度和体成分的影响:回顾性分析。
IF 2.3 3区 医学
Pediatric Pulmonology Pub Date : 2025-09-01 DOI: 10.1002/ppul.71280
Susanne Ursula Trost, Tasma Harindhanavudhi, Qi Wang, Anvitha Ankireddypalli, Azmi Simrah, Sreekant Avula, Amir Moheet
{"title":"Elexacaftor/Tezacaftor/Ivacaftor Effect on Bone Density and Body Composition: A Retrospective Analysis.","authors":"Susanne Ursula Trost, Tasma Harindhanavudhi, Qi Wang, Anvitha Ankireddypalli, Azmi Simrah, Sreekant Avula, Amir Moheet","doi":"10.1002/ppul.71280","DOIUrl":"10.1002/ppul.71280","url":null,"abstract":"<p><strong>Background: </strong>The approval of cystic fibrosis transmembrane conductance regulator modulators elexacaftor/tezacaftor/ivacaftor (ETI), has significantly improved pulmonary function for people with cystic fibrosis (pwCF). However, the effects on CF-related bone disease and body composition remain unclear.</p><p><strong>Methods: </strong>This retrospective real-world study examined adults with CF who received ETI treatment. Bone density and body composition were measured via dual-energy X-ray absorptiometry (DXA) 1.8 (SD 0.7) years before (preDXA) and 1.5 (SD 0.4) years after ETI initiation (postDXA1). In a subgroup, measurements were also available 4 (SD 0.6) years after initiation (postDXA2).</p><p><strong>Results: </strong>The study included 74 pwCF, of whom 42% were female, with an average age of 38.9 (SD 9.3) years at ETI initiation. Bone density decreased significantly at the spine (p = 0.02), left hip (p < 0.01), and right hip (p < 0.01) from pre- to postDXA1. No significant change in bone density was observed when comparing postDXA1 to postDXA2. Body composition analysis revealed significant increases in weight in both females (p < 0.01) and males (p < 0.01), as well as increases in fat mass in females (p < 0.01) and males (p < 0.01), without significant changes in lean mass (p = 0.4).</p><p><strong>Conclusions: </strong>Bone density declined significantly during the study period, with the most pronounced decline occurring in the first 1.5 years after ETI initiation. However, this decline appeared to stabilize in participants with longer follow-up at 4 years post-ETI treatment, suggesting potential bone-protective effects with prolonged therapy. Body weight and fat mass increased early after ETI initiation, suggesting metabolic changes accompanying CFTR modulation.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 9","pages":"e71280"},"PeriodicalIF":2.3,"publicationDate":"2025-09-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12418912/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"145023968","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}
引用次数: 0
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