Pediatric Pulmonology最新文献

{"title":"Peripheral Muscle Function and Body Composition in People With Cystic Fibrosis on Elexacaftor/Tezacaftor/Ivacaftor: A Cross-Sectional Single-Centre Study.","authors":"Lauren J Clayton, Anthony I Shepherd, Jo Corbett, Mathieu Gruet, Gary Connett, Mark Allenby, Julian Legg, Thomas Daniels, Don S Urquhart, Zoe L Saynor","doi":"10.1002/ppul.71044","DOIUrl":"10.1002/ppul.71044","url":null,"abstract":"<p><strong>Background: </strong>People with cystic fibrosis (pwCF) often have multifactorial peripheral muscle abnormalities attributed to, for example, malnutrition, steroid use, altered redox balance and, potentially, CF-specific intrinsic alterations. Malnutrition in CF now includes an increasing prevalence of overweight and obesity, particularly in those receiving CF transmembrane conductance regulator (CFTR) modulator therapy (CFTRm). We aimed to characterise peripheral muscle function and body composition in pwCF on Elexacaftor/Tezacaftor/Ivacaftor (ETI) CFTRm, compared to healthy controls.</p><p><strong>Methods: </strong>Fifteen pwCF on ETI, and 15 healthy age- and sex-matched controls (CON), underwent whole-body dual-energy X-ray absorptiometry scans, and a comprehensive evaluation of peripheral muscle function. Tests included quadriceps maximal isometric force measurement, an intermittent isometric quadriceps fatiguing protocol, handgrip strength dynamometry, squat jump height assessment, and 1-min sit-to-stand testing.</p><p><strong>Results: </strong>No significant differences in quadriceps maximal isometric force (CON: 181.60 ± 92.90 Nm vs. CF: 146.15 ± 52.48 Nm, p = 0.21, d = 0.47), handgrip strength (CON: 34 ± 15 kg vs. CF: 31 ± 11 kg, p = 0.62, d = 0.18), peripheral muscle endurance, fatigue, or power were observed between the groups. Moreover, no significant differences in whole-body, trunk or limb lean mass, fat-free mass, fat mass, or whole-body bone mineral density were evident.</p><p><strong>Conclusion: </strong>Comparable peripheral muscle mass and function has been demonstrated in pwCF on ETI, albeit a group with good lung function. Research is needed to confirm these findings longitudinally in pwCF, including those with more severe lung disease, who are less physically active, and have less optimal nutrition and exercise support.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71044"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11898567/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143605620","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":"Elexacaftor/Tezacaftor/Ivacaftor Treatment Accessibility and Mental Health: Reducing Anxiety in People With Cystic Fibrosis.","authors":"Burcu Uzunoglu, Merve Selcuk Balci, Mine Kalyoncu, Seyda Karabulut, Neval Metin Cakar, Ceren Ayca Yildiz, Gamze Tastan, Damla Kocaman, Almala Pinar Ergenekon, Yasemin Gökdemir, Ela Erdem Eralp, Fazilet Karakoc, Bülent Karadag","doi":"10.1002/ppul.71037","DOIUrl":"10.1002/ppul.71037","url":null,"abstract":"<p><strong>Background: </strong>Although modulator therapies have proven effective in cystic fibrosis (CF) access is limited due to reimbursement issues in Turkey. We aimed to examine anxiety and depression levels of people with CF (pwCF) and their caregivers according to their access to modulator treatment.</p><p><strong>Methods: </strong>Participants genetically eligible for elexacaftor/tezacaftor/ivacaftor (ETI) were divided into Group 1 (access via court decision, not yet on treatment) and Group 2 (unable to access due to reimbursement issues). Genetically ineligible participants formed Group 3. All pwCF and parents of those under 18 were screened for depression by the Patient Health Questionnaire-9 (PHQ-9) and for anxiety by the Generalized Anxiety Disorder-7 (GAD-7). Surveys for Group 1 patients were conducted just before starting ETI. Binary logistic regression analysis was performed to evaluate the effects of independent variables on anxiety and depression in pwCF and their primary caregivers.</p><p><strong>Results: </strong>A total of 389 pwCF and 285 caregivers were included. Group 3 (ineligible) had the highest depression rate (72.9%, n = 35), while Group 1 (pre-ETI) had the lowest (50.0%, n = 35). Median PHQ-9 scores were significantly lower in Group 1 (p < 0.006). Anxiety rates were higher in Groups 2 and 3 compared to Group 1 (p = 0.011 and p = 0.003, respectively). Access to ETI reduced the odds of anxiety by 67.7% (p = 0.029). Caregiver GAD-7 scores showed a weak negative correlation with pwCF age (r = -0.117).</p><p><strong>Conclusion: </strong>Limited access to modulator therapies is associated with higher depression and anxiety symptoms among pwCF. Addressing these barriers is critical to improving their well-being.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71037"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11892081/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143586628","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 Rare Cause of Diffuse Alveolar Hemorrhage in Children: COPA Syndrome.","authors":"Ayyüce Ünlü, Şule Selin Akyan Soydaş, Satı Özkan Tabakçı, Işıl Bilgiç, Meltem Kürtül Çakar, Gamze Akça Dinç, Hande Yetişgin, Çelebi Yıldırım, Muhammet Ali Çetin, Gökçen Dilşa Tuğcu, Dilber Ademhan Tural, Sanem Eryılmaz Polat, Güzin Cinel","doi":"10.1002/ppul.71050","DOIUrl":"https://doi.org/10.1002/ppul.71050","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71050"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606145","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":"Outdoor Air Pollution and Its Effects on Lung Health.","authors":"Monika Gappa","doi":"10.1002/ppul.27433","DOIUrl":"10.1002/ppul.27433","url":null,"abstract":"<p><p>This paper summarizes key factors regarding outdoor pollution and its effects on paediatric respiratory health.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"S109-S110"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142807554","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":"Immunomodulatory therapy by macrolides: More than killing bugs.","authors":"Bruce K Rubin","doi":"10.1002/ppul.27251","DOIUrl":"10.1002/ppul.27251","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"S22-S23"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142909956","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":"Expanding the Impact of New Cystic Fibrosis Therapies in Low- and Middle-Income Countries.","authors":"Eitan Kerem","doi":"10.1002/ppul.27362","DOIUrl":"10.1002/ppul.27362","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis (CF) primarily affects Caucasian populations, with the highest prevalence in countries like Ireland, the UK, Australia, and Canada. Despite significant improvements in survival, pulmonary insufficiency remains the leading cause of death. Factors such as nutrition, chronic Pseudomonas aeruginosa (PsA) infection, genotype, pancreatic status, and cystic fibrosis-related diabetes affect pulmonary function across age groups.</p><p><strong>Objective: </strong>This review examines disparities in CF care and outcomes between high-income countries (HICs) and low-income countries (LICs), focusing on the impact of CFTR modulators like Elexacaftor/Tezacaftor/Ivacaftor (ETI) and challenges in accessing care in LICs.</p><p><strong>Methods: </strong>Data from the European CF Society Patient Registry and studies on CF outcomes across regions were reviewed to assess survival trends, pulmonary function, and infection rates among people with CF (pwCF). The effects of CFTR modulator therapies, particularly for F508del carriers, were also evaluated.</p><p><strong>Results: </strong>In HICs, improvements in survival rates and pulmonary function have been noted, especially with the use of CFTR modulators like ETI. However, in LICs, challenges like limited access to therapies, delayed diagnosis, poor nutrition, and high PsA infection rates lead to poorer outcomes. In regions with fewer F508del carriers, access to care and medications is further limited, exacerbating disparities.</p><p><strong>Conclusion: </strong>Although CF treatment advancements have improved outcomes in many pwCF, these benefits are not evenly distributed globally. Efforts to improve CF care in LICs, such as increasing awareness, ensuring access to therapies, and establishing specialized clinics, are essential to bridging this gap.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":" ","pages":"S90-S91"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11921072/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"142771264","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":"What Time Is It? Circadian Rhythm Abnormalities in a Patient With Spinal Muscular Atrophy.","authors":"Kelly Gardner, Kevin Gipson, Lawrence Epstein, T Bernard Kinane, Benjamin A Nelson","doi":"10.1002/ppul.71053","DOIUrl":"https://doi.org/10.1002/ppul.71053","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71053"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143658147","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":"Survey Health Data on Allergic Rhinitis (AR) and Asthmatic Symptoms and Blood Samples (IgE Sensitizations) in Preschool Children: An Observational Study.","authors":"Susanne Kutzora, Jonas Mehrl, Jonas Huß, Jeroen Buters, Joana Candeias, Renate Effner, Lana Hendrowarsito, Alisa Weinberger, Caroline Quartucci, Caroline Herr, Stefanie Heinze","doi":"10.1002/ppul.71030","DOIUrl":"https://doi.org/10.1002/ppul.71030","url":null,"abstract":"<p><strong>Background: </strong>Aim of the study was to investigate the association of IgE sensitizations in serum and self-reported allergic rhinitis (AR) and asthmatic symptoms.</p><p><strong>Methods: </strong>Between 2015 and 2018, parents of 1190 children in Günzburg, Germany participated in the study by completing a questionnaire. Settings were the Health-Monitoring-Units (HMU) during the obligatory school entry examinations and a medical examination for all German children at the age of 60-64 months (U9). Random serum samples from 340 children were analyzed for IgE-sensitizations. For possible associations of sensitization and six health outcomes (symptoms of AR, dry cough at night, wheeze, asthma diagnosis, physician-diagnosed asthma, and physician-diagnosed AR), a regression analysis was performed.</p><p><strong>Results: </strong>The parents of 1190 children completed the questionnaire. Parental asthma and physician-diagnosed asthma (OR 4.79; 95%-CI [2.16-10.65]) and underweight at birth/preterm delivery showed the highest associations with asthma based on the German ISAAC definition (OR 3.58; 95%-CI [1.77-7.22]). Out of 340 children blood samples 118 children were sensitized against at least one allergen (airborne pollen allergens, non-seasonal airborne allergens, or food allergens). Sensitized children reported more frequently from symptoms of AR, wheeze, and asthma based on the German ISAAC definition than children without sensitization.</p><p><strong>Conclusion: </strong>This observational study identified the prevalence of AR, asthma and existing sensitization among the participating children. Parental asthma and underweight at birth were identified as risk factors for allergic symptoms and asthma.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71030"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143573643","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":"Dornase Alfa Instillation via Flexible Fiberoptic Bronchoscopy to Treat Atelectasis in Children.","authors":"Birce Sunman, Ebru Yalcin, Raziye Atan, Halime Nayir Buyuksahin, Ismail Guzelkas, Didem Alboga, Meltem Akgul Erdal, Ipek Demir, Burcu Capraz Yavuz, Nagehan Emiralioglu, Deniz Dogru, Uğur Özçelik, Nural Kiper","doi":"10.1002/ppul.71049","DOIUrl":"https://doi.org/10.1002/ppul.71049","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71049"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606149","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":"Impact of Elexacaftor/Tezacaftor/Ivacaftor on Microbiology and Antibiotic Utilization in People With Cystic Fibrosis.","authors":"Cindy L Williams, Joanna Billings, Harriet McGowan, Rebecca McDevitt, Charles R Esther, Cameron J McKinzie, William S Wilson, Charissa W Kam","doi":"10.1002/ppul.71038","DOIUrl":"https://doi.org/10.1002/ppul.71038","url":null,"abstract":"<p><strong>Background: </strong>Cystic fibrosis (CF) is a multisystem disease characterized by persistent lung infection. Treatment with elexacaftor/tezacaftor/ivacaftor (ETI) improves respiratory-related quality of life and reduces rates of infection and antibiotic treatments. Reduced antibiotic use may alter bacterial drug resistance patterns.</p><p><strong>Methods: </strong>This was a single center, retrospective, observational study analyzing respiratory cultures obtained from people with CF (pwCF) before and after starting ETI therapy. Antibiotic courses and culture data, including susceptibilities, were obtained from the electronic medical record.</p><p><strong>Results: </strong>There were 312 pwCF on ETI included, with an average age at ETI initiation of 20.9 ± 12.0 years and an average length of time on ETI 2.48 ± 0.69 years. Compared to the pre-ETI period, pwCF post-ETI had reductions in the number of antibiotic courses per year (2.5 to 0.7, p < 0.001), antibiotics utilized per course (1.4 to 1.0, p < 0.001), and percentage of courses including intravenous antibiotics (59% to 38%, p < 0.001). The fraction of pwCF with at least one culture positive for Pseudomonas aeruginosa, Burkholderia species, or Stenotrophomonas maltophilia decreased after ETI initiation, though changes were not significant for Staphylococcus aureus. Antibacterial resistance patterns were similar for most antibiotics in pre- and post-ETI periods, with P. aeruginosa exhibiting more resistance to fluoroquinolones post-ETI. Individuals with resistant organisms pre-ETI were less likely to clear the pathogen post-ETI.</p><p><strong>Conclusion: </strong>Treatment with ETI significantly decreased antibiotic utilization and the prevalence of gram-negative organisms. Although fewer antibiotics were used, antibiotic resistance remained unchanged or even increased post-ETI due largely to the greater persistence of resistant organisms.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 3","pages":"e71038"},"PeriodicalIF":2.7,"publicationDate":"2025-03-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143606151","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}