The ancient Greeks took the entertainment of their crowds very seriously and used drama as a way of investigating the world they lived in. The theatrical re-enactment of suffering and grief in front of a large audience was to foster compassion, as well as a cathartic release of emotion that was restorative through a deeply felt interconnection between actors and audience. When our patients encounter tragedy, we are also drawn into their ordeal and experience it first hand. Such it was with Rose.

A 5-month-old male baby was brought to the emergency room with a one-day history of respiratory distress, wheezing, vomiting, and high fever. Wheezing had been present since birth but had recently become more frequent and intense, occurring even at rest. The baby was born full-term by cesarean delivery and had a medical history of gallbladder agenesis, secundum atrial septal defect (ASD), and cryptorchidism.

ETI treatment reduces inflammatory markers and positive bacterial cultures on BAL in PwCF. These findings suggest that ETI has a greater impact on chronic infection and inflammation than ivacaftor alone. However, airway inflammation persists in a fraction of treated individuals, indicating an ongoing need to optimize other treatments in a subset of patients.

Introduction: Preterm children with bronchopulmonary dysplasia (BPD) frequently require supplemental oxygen in the outpatient setting. In this study, we sought to determine patient characteristics and demographics associated with need for supplemental oxygen at initial hospital discharge, timing to supplemental oxygen liberation, and associations between level of supplemental oxygen and likelihood of respiratory symptoms and acute care usage in the outpatient setting. Methods: A retrospective analysis of subjects with BPD on supplemental oxygen (O 2) was performed. Subjects were recruited from outpatient clinics at Johns Hopkins University and the Children’s Hospital of Philadelphia between 2008 and 2021. Data were obtained by chart review and caregiver questionnaires. Results: Children with BPD receiving > 1 liter of O 2 were more likely to have severe BPD, pulmonary hypertension and be older at initial hospital discharge. Children discharged on higher levels of supplemental O 2 were slower to wean to room air compared to lower O 2 groups (p<0.001). Additionally, weaning off supplemental O 2 in the outpatient setting was delayed in children with gastrostomy tubes and those prescribed inhaled corticosteroids, on public insurance or with lower estimated household incomes. Level of supplemental O 2 at discharge did not influence outpatient acute care usage or respiratory symptoms. Conclusion: BPD severity and level of supplemental oxygen use at discharge did not correlate with subsequent acute care usage or respiratory symptoms in children with BPD. Weaning of O 2 however was significantly associated with socioeconomic status and respiratory medication use, contributing to the variability in O 2 weaning in the outpatient setting.

Interstitial Lung Disease in childhood (chILD) is rare and no longer solely a childhood issue. Many are surviving into adult life. Therefore, many affected with chILD need to transition from paediatric to adult healthcare services. Transition is a significant life event that has the potential to impact on physical and mental health outcomes and across Europe this is a haphazard process for chILD. This qualitative study explores how young people and parents in the United Kingdom experienced transition from paediatric to adult healthcare services for chILD. Participants (n = 7) were recruited from chILD patient organisations and online communities. We focused on the experience of transition and whether there were any information packs or support provided for the transition. The data was analysed by constructivist grounded theory. The study presents a lived experience of transition with themes of lack of transition preparation and planning, challenges of learning how to adapt to adult services and a changing healthcare scene. Due to the complexity of ChILD, parents discussed their need to remain, in part, as an advocate for the young person. Respondents provided recommendations for how transition could be improved along with tips for young people who are new to the transition process, which include educating oneself about the condition and learning medical terminology, being open if there are issues and reaching out for support.

Objectives: The CF Foundation sponsored competitive awards for Mental Health Coordinators (MHCs) from 2016-2018 to implement the international guidelines for mental health screening and treatment in US CF centers. Longitudinal surveys evaluated success in implementing these guidelines using the Consolidated Framework for Implementation Research (CFIR). Methods: MHCs completed annual surveys assessing implementation from Preparation/Basic Implementation (e.g., using recommended screeners) to Full Implementation/Sustainability (e.g., providing evidence-based treatments). Points were assigned to questions through consensus, with higher scores assigned to more complex tasks. Linear regression and mixed effects models were used to: 1) examine differences in centers and MHC characteristics, 2) identify predictors of success, 3) model the longitudinal trajectory of implementation scores. Results: 122 MHCs (88.4% responded): Cohort 1 N=80, Cohort 2 N=30, Cohort 3 N=12. No differences in center characteristics were found. Significant improvements in implementation were observed across centers over time. Years of experience on a CF team was the only significant predictor of success; those with 1-5 years or longer reported the highest implementation scores. Change over time was predicted by >5 years of experience. Conclusions: Implementation of the mental health guidelines was highly successful over time. Funding for MHCs with dedicated time was critical. Longitudinal modeling indicated that CF centers with diverse characteristics could implement them, supported by evidence from the CF Patient Registry showing nearly universal uptake of mental health screening in the US. Years of experience predicted better implementation, suggesting that education and training of MHCs and retention of experienced providers are critical to success.

We are hopeful that CTFR modulation does have a positive impact of bone health, but we have concerns about this study design being used to support the hypothesis.

RATIONALE: Obstructive sleep apnea is highly prevalent in children with asthma, particularly in obese children. The sleep related breathing disorder screening questionnaire has low screening accuracy for obstructive sleep apnea in children with asthma. Our goal was to identify the questions on the sleep related breathing disorder survey associated with obstructive sleep apnea in children with asthma. METHODS: Participants completed the survey, underwent polysomnography and their body mass index z-score was measured. Participants with survey scores above 0.33 were considered high risk for obstructive sleep apnea and those with an apnea hypopnea index ≥ 2 events/hour classified as having obstructive sleep apnea. Logistic regression was used to examine the association of each survey question and obstructive sleep apnea. Positive and negative predictive values were calculated to estimate screening accuracy. RESULTS: The prevalence of obstructive sleep apnea was 40% in our sample (n=136). Loud snoring, morning dry mouth and being overweight were the questions associated with obstructive sleep apnea. A combined model of loud snoring, morning dry mouth and being overweight had positive and negative predictive values of 57.4% and 81.0% respectively, while the composite survey score had positive and negative predictive values of 51.0% and 65.5%. Body mass index z-score had positive and negative predictive values of 76.3% and 72.2%. CONCLUSIONS: The body mass index z-score is useful for obstructive sleep apnea screening in children with asthma and should be applied routinely given its simplicity and concerns that obstructive sleep apnea may contribute to asthma morbidity.

A document by Daniel Penela-Sánchez. Click on the document to view its contents.

Background: Among children with tracheostomies, little is known about how respiratory culture results differ between states with and without acute respiratory infections (ARI), or the overall test performance of respiratory cultures. Objective: To determine the association of respiratory culture organism isolation with diagnosis of ARI in children with tracheostomies, and assess test characteristics of respiratory cultures in the diagnosis of bacterial ARI (bARI). Methods: This single-center, retrospective cohort study included respiratory cultures of children with tracheostomies obtained between 2010-2018. The primary predictor was ARI diagnosis code at the time of culture; the primary outcomes were respiratory culture organism isolation and species identified. Generalized estimating equations were used to assess for association between ARI diagnosis and isolation of any organism while controlling for potential confounders and accounting for within-patient clustering. A multinomial logistic regression equation assessed for association with specific species. Test characteristics were calculated using bARI diagnosis as the reference standard. Results: Among 3,578 respiratory cultures from 533 children (median 4 cultures/child, IQR: 1-9), 25.9% were obtained during ARI and 17.2% had ≥1 organism. Children with ARI diagnosis had higher odds of organism identification (aOR 1.29, 95% CI 1.16–1.44). When controlling for covariates, ARI was associated with isolation of H. influenzae, M. catarrhalis, S. pneumoniae, and S. pyogenes. Test characteristics revealed a 24.3% sensitivity, 85.2% specificity, 36.5% positive predictive value, and 76.3% negative predictive value in screening for bARI. Conclusion: The utility of respiratory culture testing to screen for, diagnose, and direct treatment of ARI in children with tracheostomies is limited.

Rationale: Patients with congenital central hypoventilation syndrome (CCHS) require long-term ventilation to ensure gas exchange and to prevent deleterious consequences for neurocognitive development. Two ventilation modes may be used for these patients depending on their tolerance, one invasive by tracheostomy and the other noninvasive (NIV). For patients who have undergone a tracheostomy, transition to NIV is possible when they meet predefined criteria. Identifying the conditions favorable for weaning from a tracheostomy it critical for the success of the process. Objective: The aim of the study was to share our experience of decannulation in a reference center; we hereby describe the modality of ventilation and its effect on nocturnal gas exchange before and after tracheostomy removal. Methods: Retrospective observational study at Robert Debré Hospital over the past 10 years. The modalities of decannulation and transcutaneous carbon dioxide recordings or polysomnographies before and after decannulation were collected. Results: Sixteen patients underwent decannulation following a specific procedure for transition from invasive to NIV. All decannulations were successful. The median age at decannulation was 12.6 [9.7; 15.0] years. Nocturnal gas exchange was not significantly different before and after decannulation, while expiratory positive airway pressure and inspiratory time increased significantly. An oronasal interface was chosen in two out of three patients. The mean duration of hospital stay for decannulation was 4.0 [3.0; 6.0] days. Conclusion: Our study underlines that decannulation and transition to NIV are achievable in CCHS children using a well-defined procedure. Patient preparation is crucial to the success of the process.

To our knowledge, this is the first reported case of successful transbronchial biopsy via RB in a 5-year-old patient. The sample revealed a potential diagnosis, but more importantly, helped rule out an active infectious process and avoided an open lung biopsy which was being planned and would have otherwise been necessary to establish a diagnosis. We believe this technology can be used to increase the diagnostic yield of flexible bronchoscopy, particularly in the immunocompromised population as there is already evidence supporting image-guided bronchoscopy over conventional bronchoscopy in this population [(4)](#ref-0004). A notable limitation to RB is the size of the available bronchoscopes for robotic platforms (4.5 and 3.5 mm OD). In children, this may limit the capacity to sample more peripheral lesions via fine needle aspiration, as more lung tissue (relative to chest size) may be injured. In our case, the catheter tip was never as close to the lesion as it has been described in adults [(2)](#ref-0002). As the field of pediatric interventional pulmonology continues to develop, RB will likely be one of several options available for safe and high-yield diagnostic procedures.

Pulmonary artery sling is a rare childhood vascualr ring that is frequently associated with tracheal stensois. Consequently, neonates may present with a crictal airway obstruction if tehre is long segemt tarcheal stensosi and complete rings. Rapid diagnosis of this cardiac vascular malfomation and extent airway involvement is essentail as longterm outcoem can be excellent follwoing surgicla repair. In this review we focus on airway invetsigation and management for this challenging congential condition.

The current system for disclosing financial conflicts of interest (COIs) can be traced back to the “Uniform requirements for manuscripts submitted to medical journals,” published in 1997. Meanwhile, new molecular and genetic therapies are transforming clinical medicine and these therapies have radically altered the financial landscape of drug development. The average price of new drugs has risen sharply, and in niche areas like rare diseases, the cost of new therapies can be stratospheric. Now, even rare or “orphan” diseases are funded by private investment. With high profitability, there is a new pathway for drug development involving unprecedented ties between industry and academia. The potential for COIs has greatly expanded and decisions that were once science-driven have become profit-driven. As a result, the risk that marginally effective, ineffective and even hazardous treatments will make their way to patients has greatly increased. In this editorial, we propose a system built on process-based COIs. This new system would trace the developmental journey of a drug from the lab to patients. All financial ties that institutions and individuals have to the drug would be disclosed at each developmental step. The goal would be to create a healthy skepticism and debate over the studies behind new drugs, restoring a scientific focus to the assessment of drug efficacy. When society’s limited resources are used to pay for drugs whose excessive cost primarily benefits investors and corporations, it derails scientific objectivity, harms patients, and threatens the financial stability of our health care systems.

Addressing the recognised challenges and inequalities in providing high quality health care for rare diseases such as children’s interstitial lung disease (chILD) requires collaboration across institutional, geographical, discipline, and system boundaries. The Children’s Interstitial Lung Disease Respiratory Network of Australia and New Zealand (chILDRANZ) is an example of a clinical network that brings together multidisciplinary health professionals for collaboration, peer learning, and advocacy with the goal of improving the diagnosis and management of this group of rare and ultra-rare conditions. This narrative review explores the multifaceted benefits arising from social learning spaces within rare disease clinical networks by applying the Value Creation Framework. The operation of the chILDRANZ network is used as an example across the framework to highlight how value is generated, realised, and transferred within such collaborative clinical and research networks. The community of clinical practice formed in the chILDRANZ multidisciplinary clinical peer support meetings provides a strong example of social learning that engages with the uncertainty inherent in rare disease diagnosis and management and pays attention to generate new knowledge and best practice to make a difference for children and families living with chILD. This review underscores international calls for further investment in, and support of, collaborative expert clinical networks for rare disease.

Objective: SARS-COV-2 pandemic had a profound impact on acute bronchiolitis epidemiology, especially in RSV diffusion and the burden of disease with implications on the management of prophylaxis and health resources. We aimed to compare clinical and epidemiological characteristics of bronchiolitis before and during the SARS-CoV-2 pandemic.   Methods: We conducted an observational study involving children aged 0-2 years with bronchiolitis admitted to a tertiary children’s hospital during the last 5 years. Demographic, clinical, microbiological, and outcome data were collected. Comparison between pre- and post-pandemic period, RSV positive versus non-RSV patients, and SARS-CoV-2 positive vs negative patients were carried out.    Results: A total of 647 patients admitted for bronchiolitis were included (264 female,40.8%, median age 78 days). Molecular diagnostic tests were performed in 617 patients (95.4%) with RSV detected in 62.5% of patients. SARS-CoV-2 was found in 3.9% of hospitalized bronchiolitis (3.9%). We observed a progressive increase in bronchiolitis admissions and a statistically significant increase over the years in the need for respiratory support. Conversely, this was not true for mechanical ventilation, duration of respiratory support, ICU admission, and length of stay. During the pandemic period children with prematurity increased, although only 1 child had an indication for prophylaxis. Discussion: We confirm the stronger impact of bronchiolitis in the 2021-22 season, which is likely explained by the higher prevalence of RSV and the immunity debt theory. However, our findings were conflicting in terms of worsening clinical severity. The increase of children with prematurity and the inter-seasonal spread of RSV highlight the importance of epidemiological surveillance systems that monitor RSV circulation.

Right and left ventricular dysfunction are commonly encountered in pulmonary disorders. Pulmonary hypertension exerts pressure load on the right ventricle, subsequent RV-LV interactions through shared septum, pericardium and blood supply lead to LV dysfunction. The early detection and treatment of pulmonary hypertension is crucial to improve outcomes of pulmonary disorders. New advanced echocardiographic techniques such as pulmonary artery circumferential strain and pulmonary artery blood speckle tracking might help in replacing invasive hemodynamics, for early detection of pulmonary vasculopathy in pulmonary disorders.

Background: Uniformity and compliance with clinical practice guidelines (CPGs) for use of palivizumab in preventing severe RSV infection in Australian high-risk infants remain unclear. Methods: An online survey was conducted across the Australian and New Zealand Neonatal Network (ANZNN) to determine clinical practices around palivizumab. A literature search was also performed to identify and compare national and international guidelines. Results: Sixty-five of 422 ANZNN members completed the survey. Respondents included 61 senior medical staff of consultants/staff specialists (78%) and four nursing staff (6%). Infants most likely to be recommended palivizumab included preterm infants born <29 weeks gestational age (GA) (30%), children with chronic lung diseases (CLD) born <32 weeks GA (40%), and with hemodynamically significant heart disease (35%). Many respondents (53%) stated that CPGs for palivizumab were developed locally. Twenty guidelines (10 international and 10 domestic) were obtained in total; 16(80%) recommended palivizumab use in preterm infants, 16(80%) recommended use in infants with CLD, 17(85%) congenital heart disease (CHD) and six (30%) bronchopulmonary dysplasia (BPD). Eight (40%) guidelines provided specific recommendations for immunocompromised infants. Canada, Western Australia, and American Academy of Paediatrics provided recommendations for Indigenous children. Frequency and dosage of palivizumab was universal across all CPGs. None of the international guidelines obtained were from low- or middle-income countries. Conclusions: Standardisation of CPGs may improve clinical decision making around use of palivizumab in high-risk infants.