{"title":"Pancreatic Status Is Not a Risk Factor for Cystic Fibrosis-Related Bone Disease.","authors":"Miri Dotan, Maya Trau, Meir Mei-Zahav, Huda Mussaffi, Yulia Gendler, Hannah Blau, Dario Prais","doi":"10.1002/ppul.71078","DOIUrl":"10.1002/ppul.71078","url":null,"abstract":"<p><strong>Background: </strong>As the life expectancy of people with cystic fibrosis (PwCF) increases, understanding long-term complications, including CF-related bone disease (CFBD), is crucial.</p><p><strong>Objective: </strong>This study aimed to longitudinally characterize CFBD and to compare the bone status of pancreatic sufficient (PS) and pancreatic insufficient (PI) PwCF.</p><p><strong>Methods: </strong>This longitudinal analysis included PwCF older than 8 years of age who had at least one dual-energy X-ray absorptiometry test between 2008 and 2021. Data were collected on serum parameters of bone metabolism, nutritional history, habitual activity, and fractures in addition to other demographic and clinical characteristics.</p><p><strong>Results: </strong>The study included 80 PwCF: 32 (40%) were PS and 48 (60%) PI. Normal dual-energy X-ray absorptiometry results were found in 42 (53%) patients: 16 (50%) in the PS group and 26 (54%) in the PI group (p = 0.72). Three (9%) of the PS group and seven (15%) of the PI group had at least one Z-score below -2 (p = 0.49). The longitudinal bone density decline over a mean of 4.8 years was similar in the two groups. In a logistic regression analysis, pancreatic insufficiency was not found to be a risk factor for CFBD. Female sex was the only significant risk factor for a pathological Z-score.</p><p><strong>Conclusions: </strong>The prevalence and severity of CFBD were not found to correlate with pancreatic sufficiency. The similar prevalence of CFBD between patients with PS and PI suggests that screening, and eventually treatment, should be offered to all PwCF, irrespective of pancreatic status.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71078"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11962574/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143764604","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":"Utility and Outcome of Follow-Up Polysomnography in Patients With Ambulatory Noninvasive Ventilation.","authors":"Athiwat Tripipitsiriwat, Prakarn Tovichien, Neepa Gurbani, Md M Hossain, Narong Simakajornboon","doi":"10.1002/ppul.71103","DOIUrl":"https://doi.org/10.1002/ppul.71103","url":null,"abstract":"<p><strong>Background: </strong>Noninvasive ventilation (NIV) is a cornerstone of respiratory management in patients with various chronic conditions. While a follow-up polysomnography (PSG) is recommended to optimize ventilatory support, its utility and outcome data are limited. This study aimed to describe problems identified during the follow-up PSG and subsequent ventilator setting adjustments in this population.</p><p><strong>Methods: </strong>The follow-up titration PSGs of patients prescribed with ambulatory NIV between January 2022 and January 2024 were retrospectively reviewed. Mixed effects logistic regression models were used to identify factors associated with setting changes.</p><p><strong>Results: </strong>Two hundred ninety-seven PSGs from 106 patients (median age 15.9 years) were included in the analysis, including 216 titration and 81 baseline studies. Fifty percent of patients were diagnosed with neuromuscular diseases. The most common code of NIV was 77.4% on BPAP-ST, followed by PC-SIMV (14.2%) and PCV mode (8.5%). 81.5% of the titration studies resulted in ventilator setting adjustments. The most common problems identified were residual respiratory events, inadequate ventilation and significant leaks. The lower home IPAP setting and higher BMI were associated with increased odds of ventilator setting adjustment, with odds ratio of 0.86 (95% CI: 0.76, 0.98; p-value 0.021) and 1.02 (95% CI: 1.00, 1.03; p-value 0.017), respectively.</p><p><strong>Conclusion: </strong>Follow-up PSG frequently results in ventilator setting adjustments in patients with ambulatory NIV. Patients with lower IPAP setting and higher BMI are more likely to have changes in ventilator setting after PSG. The facility involving the care of ambulatory NIV patients should be familiar with the common problems found in PSGs.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71103"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC12016003/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017112","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}
Abigail Strang, Kimberly Renner, Kerry Smallacombe, Dawn Selhorst, Aaron Chidekel
{"title":"Home Monitoring Using Telehealth and Mobile Spirometry Is Feasible, Acceptable, and Accessible in Children With Asthma From Rural and Medically Underserved Areas.","authors":"Abigail Strang, Kimberly Renner, Kerry Smallacombe, Dawn Selhorst, Aaron Chidekel","doi":"10.1002/ppul.71109","DOIUrl":"https://doi.org/10.1002/ppul.71109","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71109"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143993806","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}
Laura Petrarca, Valentina Guida, Raffaella Nenna, Alessandro De Luca, Marina Goldoni, Laura Bernardini, Maria Giulia Conti, Giuseppe Cimino, Enrica Mancino, Laura Masuelli, Piercarlo Poli, Fabio Midulla
{"title":"Genotype-Phenotype Correlation in a Group of Italian Patients With Primary Ciliary Dyskinesia.","authors":"Laura Petrarca, Valentina Guida, Raffaella Nenna, Alessandro De Luca, Marina Goldoni, Laura Bernardini, Maria Giulia Conti, Giuseppe Cimino, Enrica Mancino, Laura Masuelli, Piercarlo Poli, Fabio Midulla","doi":"10.1002/ppul.71057","DOIUrl":"10.1002/ppul.71057","url":null,"abstract":"<p><strong>Introduction: </strong>Primary Ciliary Dyskinesia (PCD) is a rare genetic disorder characterized by abnormalities in the motile cilia. Diagnosis could be hard to make, but genetic analysis could be important for the diagnosis and for defining prognosis.</p><p><strong>Aim of the study: </strong>To evaluate the clinical, ultrastructural, and molecular characteristics of a cohort of PCD subjects.</p><p><strong>Materials and methods: </strong>The study cohort included PCD patients enrolled in two Italian centers. Clinical data were retrospectively collected consulting medical records. All patients underwent nasal brushing and peripheral blood sampling for ultrastructural analysis of motile cilia and genetic testing, respectively.</p><p><strong>Results: </strong>A total of 39 patients with PCD were enrolled (median age 25.5 years, range 2.5-54.3 years). All patients showed common clinical features, which included SIT in 22/39 (56.4%), chronic rhinitis in 31/39 (79.5%), chronic sinusitis in 26/37 (66.7%), chronic cough in 32/39 (82.1%), and neonatal respiratory distress in 46.2% (18/39). The genetic defect was identified in 27/39 patients (69.2%), while a diagnostic ultrastructure was found in 27/35 (77.1%). Assessing genotype-phenotype correlations, subjects with biallelic pathogenic variants in CCDC39 and CCDC40 genes had a significantly lower forced expiratory volume in the first second of exhalation value (p = 0.017) than subjects with pathogenic variants in DNAH5 or in other PCD-related genes.</p><p><strong>Conclusions: </strong>Our study further highlights the high heterogeneity of ultrastructural defects and genetics characterizing patients with PCD, as well as providing additional evidence that patients with biallelic pathogenic variants in CCDC39 or CCDC40 display a worse clinical phenotype than patients with pathogenic variants in other PCD genes.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71057"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143780845","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}
Dharini M Bhammar, Daniel P Wilhite, Temitayo Olojo, Kavya Alagappan, Katerina Jones, Yu-Lun Liu, Tony G Babb
{"title":"Effects of Obesity and Sex on Supine Functional Residual Capacity and Expiratory Flow Limitation in Children.","authors":"Dharini M Bhammar, Daniel P Wilhite, Temitayo Olojo, Kavya Alagappan, Katerina Jones, Yu-Lun Liu, Tony G Babb","doi":"10.1002/ppul.71061","DOIUrl":"https://doi.org/10.1002/ppul.71061","url":null,"abstract":"<p><strong>Introduction: </strong>Childhood obesity is associated with a higher risk of sleep-disordered breathing, but our understanding of the underlying physiologic mechanisms is limited. This study investigated the effects of obesity on lung volumes, expiratory flow limitation (EFL), and respiratory symptoms in the supine position.</p><p><strong>Methods: </strong>Ninety-four children (8-12 years old, n = 58 with obesity and n = 36 without obesity) underwent lung volume and EFL measurements in the seated and supine positions in this repeated-measures cross-sectional study. Children without obesity underwent an additional condition where either a 2.3 kg (n = 26) or 5.0 kg (n = 8) mass was placed on their abdomen to simulate obesity. Ratings of perceived breathlessness (RPB) were recorded.</p><p><strong>Results: </strong>Functional residual capacity (FRC) in the supine when compared with the seated posture decreased more so in children without obesity (43.9 ± 1.0 vs. 34.4 ± 1.0% total lung capacity [TLC]) compared with children with obesity (35.5 ± 0.8 vs. 29.6 ± 0.8% TLC; p = 0.0048 group*posture). EFL in the supine position was present in 17% of children with obesity compared with 6% of children without obesity (p = 0.1218). In children without obesity, mass-loading with 5.0 kg produced further reductions in FRC (seated: 45.0 ± 1.7% TLC; supine: 36.7 ± 1.7% TLC; supine+5.0 kg: 29.4 ± 1.7% TLC; p = 0.0087 seated vs. supine+ and p = 0.0178 supine vs. supine + ) and an increase in RPB (seated: 0.38 ± 0.24; supine: 0.42 ± 0.24; supine+5.0 kg: 1.25 ± 0.27; p = 0.0002 seated vs. supine+ and p = 0.0001 supine vs. supine + ).</p><p><strong>Conclusions: </strong>Children with obesity breathe at significantly lower lung volumes while supine, increasing the risk of EFL. A better physiologic understanding of the mechanical effects of obesity could potentially improve the management of sleep-related symptoms among children with obesity.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71061"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11997637/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144041991","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}
Manvi Bansal, Winston M Manimtim, Amit Agarwal, Stamatia Alexiou, Jessica L Rice, Leif Nelin, Audrey N Miller, Jonathan C Levin, Khanh Lai, Jacob A Kaslow, Lystra P Hayden, Julie L Fierro, Anita Bhandari, Eric D Austin, Brianna Aoyama, Gangaram Akangire, Natalie Villafranco, Nicole Stephenson, Roopa Siddaiah, Robin L McKinney, Melissa A House, Christopher D Baker, Steven H Abman, Sharon A McGrath-Morrow, Joseph M Collaco
{"title":"Outcomes of Ventilator-Dependent Children With Severe Bronchopulmonary Dysplasia and Tracheobronchomalacia.","authors":"Manvi Bansal, Winston M Manimtim, Amit Agarwal, Stamatia Alexiou, Jessica L Rice, Leif Nelin, Audrey N Miller, Jonathan C Levin, Khanh Lai, Jacob A Kaslow, Lystra P Hayden, Julie L Fierro, Anita Bhandari, Eric D Austin, Brianna Aoyama, Gangaram Akangire, Natalie Villafranco, Nicole Stephenson, Roopa Siddaiah, Robin L McKinney, Melissa A House, Christopher D Baker, Steven H Abman, Sharon A McGrath-Morrow, Joseph M Collaco","doi":"10.1002/ppul.71100","DOIUrl":"https://doi.org/10.1002/ppul.71100","url":null,"abstract":"<p><strong>Objectives: </strong>To characterize an observational cohort of ventilator-dependent infants and children with bronchopulmonary dysplasia (BPD) with or without tracheobronchomalacia (TBM) and determine the impact of TBM on the need for ventilator support, liberation from the ventilator and tracheostomy decannulation.</p><p><strong>Methods: </strong>Demographics and clinical outcomes were obtained by retrospective review from 12 centers participating in the outpatient BPD Collaborative registry. The cohort consisted of infants born between 2016 and 2021 who were dependent on invasive mechanical ventilation at home. The respiratory outcomes of those infants with TBM were compared to those who did not have TBM.</p><p><strong>Results: </strong>There were 154 subjects included and about half (48.7%) had documented TBM. Both the TBM and non-TBM groups had similar demographic characteristics and respiratory outcomes. However, the non-TBM were found to have lower mean birth weight (673 vs. 832 grams; p = 0.006), higher likelihood of having Nissen fundoplication (34.2% vs. 12.2%; p = 0.006) and higher use of diuretics (59.2% vs. 37.3%; p = 0.007). Both groups were similar in terms of ventilator requirements, timing of liberation from the ventilator, and rate of decannulation.</p><p><strong>Conclusions: </strong>The presence of TBM in ventilator-dependent infants with BPD did not affect ventilator support needs, liberation from the ventilator and the rate of tracheostomy decannulation. We speculate that the relative contributions of the other components of BPD disease may play critical roles in determining the need for tracheostomy and their ultimate respiratory outcomes. A prospective multicenter study to assess the impact of TBM in severe BPD is urgently needed.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71100"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"144017059","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":"Automatic Analysis of Lung Sounds in 3-Year-Old Children.","authors":"Hiroyuki Mochizuki, Takashi Matsushita, Kota Hirai, Fumio Niimura, Hiroyuki Furuya, Yoshiyuki Yamada, Atsushi Uchiyama","doi":"10.1002/ppul.71071","DOIUrl":"10.1002/ppul.71071","url":null,"abstract":"<p><strong>Introduction: </strong>In recent years, with the advent of artificial intelligence, clear progress has been made in the clinical application of lung sound analysis techniques.</p><p><strong>Objective: </strong>Using a new software program to analyze pediatric lung sounds using machine learning (ML), we conducted a lung sound survey study of 139 healthy 3-year-old children.</p><p><strong>Subjects and methods: </strong>All cases were surveyed using the ATS-DLD questionnaire, which mainly included items related to a history of wheezing, diagnosis of asthma, and history of respiratory syncytial virus (RSV) infection, allergies and environment. The characteristics of the lung sounds were examined, along with the results of the questionnaire and lung sound parameters.</p><p><strong>Results: </strong>Children with a history of wheezing showed a higher maximum inspiratory frequency (FAP<sub>0</sub>), lower basal power (PAP<sub>0</sub>) (p < 0.001 and p < 0.001, respectively), and lower RPF<sub>50p</sub> and RPF<sub>75p</sub> (p = 0.003 and p = 0.003, respectively), suggesting the enhancement of the high-pitched region of the lung sound spectrum. A similar tendency was observed in children with a history of asthma or RSV infection. Furthermore, in the group of children with a history of wheezing, those with a history of acute respiratory tract infection (ARI) within 1 week were found to have an enhancement of the high-pitched region relative to those without history of ARI.</p><p><strong>Conclusions: </strong>By utilizing a new analysis software program using ML, we found that 3-year-old children with a history of wheezing or suspected asthma had characteristic lung sounds even when healthy.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71071"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143754090","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}
Laura A Voss, Rebekah J Nevel, Jennifer A Wambach, Lawrence M Nogee, Robin R Deterding, Alicia M Casey, Michael G O'Connor, Daniel I Craven, Jane B Taylor, Gail H Deutsch, Jade B Tam-Williams, Lea C Steffes, Steven K Brennan, Maria T Santiago, Sara C Sadreameli, Andrea F Heras, Michael R Powers, Antonia P Popova, Manvi Bansal, Aaron Hamvas, William A Gower, Fernando Urrego, Lisa R Young
{"title":"Genetic Testing Utilization in the U.S. Registry for Childhood Interstitial and Diffuse Lung Diseases.","authors":"Laura A Voss, Rebekah J Nevel, Jennifer A Wambach, Lawrence M Nogee, Robin R Deterding, Alicia M Casey, Michael G O'Connor, Daniel I Craven, Jane B Taylor, Gail H Deutsch, Jade B Tam-Williams, Lea C Steffes, Steven K Brennan, Maria T Santiago, Sara C Sadreameli, Andrea F Heras, Michael R Powers, Antonia P Popova, Manvi Bansal, Aaron Hamvas, William A Gower, Fernando Urrego, Lisa R Young","doi":"10.1002/ppul.71073","DOIUrl":"10.1002/ppul.71073","url":null,"abstract":"<p><strong>Introduction: </strong>Childhood interstitial and diffuse lung diseases (chILD) comprise a diverse group of rare disorders. Identifying the underlying cause is crucial for treatment, prognosis, and estimating recurrence risk. The objective of this study was to assess the utilization of genetic testing for subjects enrolled in the United States National Registry for ChILD, a multicenter observational study.</p><p><strong>Methods: </strong>Genetic data from participating sites were reviewed and analyzed in relationship to clinical characteristics.</p><p><strong>Results: </strong>Of 609 children enrolled from 22 centers, genetic testing was performed for 55.5% (n = 338). Genetic testing results were positive (diagnostic) for 22.8% (n = 77), negative for 60.7% (n = 205), and uncertain for 16.6% (n = 56). Most testing was performed through gene panels (55.9%), followed by exome sequencing (ES) or whole genome sequencing (WGS) (26.9%), single gene testing (24.6%), and/or chromosomal microarray (11.8%). For participants with positive (diagnostic) genetic testing results, the majority were diagnosed through gene panel (33.8%; n = 26) or single gene testing (32.5%; n = 25). The most common diagnosis confirmed by genetic testing was SFTPC-associated surfactant metabolism dysfunction. Of the 59 subjects with unclassified ILD, only 22% (n = 13) had undergone ES or WGS, 61% (n = 36) had received panel testing, and 27% (n = 16) did not have any genetic testing reported.</p><p><strong>Conclusion: </strong>The utilization of genetic testing has been variable in infants and children enrolled in the ChILD Registry. Additional efforts are needed to develop genetic testing recommendations for children with suspected ILD. Furthermore, there is opportunity for broader utilization of ES/WGS and genetic discovery for children with lung disease of unclear etiology.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71073"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"https://www.ncbi.nlm.nih.gov/pmc/articles/PMC11960725/pdf/","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143754096","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":"Cost-Effectiveness Analysis of Omalizumab Combined With Standard of Care in Treating Moderate to Severe Asthma Children in China.","authors":"Wang Cao, Yuncui Yu, Yue Xiao, Alec Morton, Huijie Huang, Jing Wei, Peng Guo","doi":"10.1002/ppul.71066","DOIUrl":"10.1002/ppul.71066","url":null,"abstract":"<p><strong>Objectives: </strong>It has been recognized that adding omalizumab to standard of care (SOC) is a promising treatment for pediatric moderate to severe asthma patients in the Chinese setting, as shown by a previous multicenter retrospective study. Nevertheless, there is as yet no concrete evidence of its economic advantages. Several international studies conducted in different countries have shown varying results. The study is designed to better demonstrate the economic impact of omalizumab for Chinese children with asthma.</p><p><strong>Methods: </strong>Based on consultation with physicians, a three-state Markov model was developed. The progress of asthma is modeling using the states \"no symptoms,\" \"secondary control, no asthmatic attack\" and \"asthmatic attack.\" To better reflect clinical treatment, exacerbation state was further divided into \"exacerbation with outpatient service\" and \"exacerbation with hospitalization.\" To understand the effects of treatment, the response rate of omalizumab was also modeled. The modeling cycle length was set at 1 week over a 5-year time horizon. Clinical effect and transition probabilities were sourced from published literature, while cost data were primarily obtained from local databases. Cost-effectiveness analysis was assessed from the health system perspective. One-way sensitivity analyses and scenario analysis were performed to ensure the robustness of the results. The willingness-to-pay (WTP) threshold was set at 1 GDP/capital, as recommended by the national guidelines.</p><p><strong>Results: </strong>It was shown that adding omalizumab to SOC treatment would increase both the total cost and quality-adjusted life years (QALYs) over 5 years, with an incremental cost-effectiveness ratio (ICER) of CNY 111,644.77/QALY, which is higher than the WTP threshold. Among the parameter variations, the two factors that altered the results were the proportion of hospitalizations in SOC therapy during asthma exacerbation and the weekly cost of omalizumab with full dose, which were both negatively correlated with the ICER. Scenario analysis, simulating the Medicare reimbursement conditions, reduced the ICER to an acceptable level.</p><p><strong>Conclusions: </strong>Adding omalizumab to SOC treatment in pediatric moderate to severe asthma patients would be a cost-effective choice under the current medical insurance reimbursement policy in China.</p>","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71066"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143731218","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":"Uncovering the Physiologic Basis of Improved Oxygenation during Prone Positioning in Preterm Infants.","authors":"R Sindelar, H Ehrhardt","doi":"10.1002/ppul.71086","DOIUrl":"https://doi.org/10.1002/ppul.71086","url":null,"abstract":"","PeriodicalId":19932,"journal":{"name":"Pediatric Pulmonology","volume":"60 4","pages":"e71086"},"PeriodicalIF":2.7,"publicationDate":"2025-04-01","publicationTypes":"Journal Article","fieldsOfStudy":null,"isOpenAccess":false,"openAccessPdf":"","citationCount":null,"resultStr":null,"platform":"Semanticscholar","paperid":"143795886","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}