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.
Introduction Very preterm birth is associated with lung function impairment later in life, but several aspects have not been studied. We aimed to comprehensively assess lung function at school age in very preterm infants and term controls, with special emphasis on bronchopulmonary dysplasia (BPD), sex and bronchodilator response. Methods At 12 years of age, 136 children born very preterm (85 with and 51 without BPD), and 56 children born at term performed spirometry, body plethysmography, impulse oscillometry, measurement of diffusion capacity and multiple breath washout, before and after bronchodilator inhalation. Results Airway symptoms and a diagnosis of asthma were more common in children born very preterm. These children had more airflow limitation, seen as lower FEV 1 (p<0.001), FEV 1/FVC (p=0.011) and FEF 25-75 (p<0.001), and a higher total and peripheral airway resistance compared to term born controls. There was no difference in total lung capacity, but air trapping and lung clearance index were higher in children born very preterm. Diffusion capacity was lower in children born very preterm, especially in those with a diagnosis of BPD. In most other tests, the differences between preterm-born children with or without BPD were smaller than between children born preterm versus at term. Boys born preterm had more lung function deficits than preterm born girls. In children born very preterm, airway obstruction was to a large extent reversible. Conclusion At 12 years of age, children born very preterm had lower lung function than children born at term in most aspects. Airway obstruction improved markedly after bronchodilator inhalation, and there was only little difference between children with or without BPD.
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.
Objectives: Airway clearance therapy (ACT) is an important component of therapy for cystic fibrosis (CF) but is associated with significant treatment burden. Highly effective CFTR modulator therapy (HEMT) has improved pulmonary function for many people with CF(pwCF). We sought to understand changes in attitudes and practices about ACT in the post-HEMT era. Study design: Surveys of CF community members and CF care team members Methodology: Separate surveys were created for the CF community and CF care providers to evaluate attitudes towards ACT and exercise in the post-HEMT era. We solicited answers from pwCF via the CF Foundation’s Community Voice and from CF care providers via CF Foundation listservs. Surveys were available between July 20 and August 3, 2021. Results: Surveys were completed by 153 community members (parents of children and pwCF) and 192 CF care providers. Belief that exercise can substitute partially for ACT was endorsed similarly by community members (59%) and providers (68%) . After starting HEMT, 36% of parents of children and 51% of adults did fewer ACT treatments including 13% who stopped ACT. Adults reported altering their ACT regimen more than parents of children. Half of providers had changed their ACT recommendations for those on HEMT. Fifty-three percent of respondents had discussed changing ACT with their care team (36% of parents, 58% of pwCF). Conclusions: Providers should be aware that ACT management changes may have been undertaken by pwCF who have pulmonary benefits of HEMT. Treatment burden should be considered in co-management decisions regarding ACT and exercise.
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.
Background Children with chronic lung disease (CLD) of prematurity who require invasive home mechanical ventilation (iHMV) are medically vulnerable and experience high caregiving and healthcare costs. Predictors for duration of iHMV remain unclear, which can make prognostication and decision-making challenging. Methods A retrospective cohort study of children with CLD of prematurity requiring invasive iHMV was conducted from an independent children’s hospital records (2005-2021). The primary outcome was iHMV duration, defined as time from initial discharge home on iHMV until cessation of positive pressure ventilation (day and night). Two new variables were included: corrected tracheostomy age (CTA) (chronological age at discharge minus age at tracheotomy) and level of ventilator support at discharge (minute ventilation per kg per day). Univariable Cox regression was performed with variables of interest compared to iHMV duration. Significant nonlinear factors (P<0.05) were included in the multivariable analysis. Results One-hundred-and-nineteen patients used iHMV primarily for CLD of prematurity. Patient median index hospitalization lasted 12 months (IQR 8.0,14.4). Once home, half of patients were weaned off iHMV by 36.0 months and 90% by 52.2 months. Being Hispanic/Lantix ethnicity (HR 0.14 (95% CI 0.04, 0.53), p<0.01) and having a higher CTA were associated with increased iHMV duration (HR 0.66 (CI 0.43, 0.98), p<0.05). Conclusions Disparity in iHMV duration exists among patients using iHMV after prematurity. Prospective multisite studies that further investigate new analytic variables, such as CTA and level of ventilator support, and address standardization of iHMV care are needed to create more equitable iHMV management strategies.
Background: Cystic fibrosis (CF) is an autosomal recessive disease affecting multiple organ systems, including the liver, leading to cystic fibrosis-related liver disease (CFLD). It was noted that CFLD in Egyptian children with CF is more common than in non-Egyptian people with CF (pwCF). The present study aimed to determine the incidence of CFLD and the potential risk factors for developing CFLD in Egyptian children. The correlation between CFLD and the various genotypes prevalent in Egyptian CF children and the comparison of CFLD in Egyptian and non-Egyptian CF patients will be done. Methods: The current cross-sectional study included 50 CF cases from Ain Sham University’s Pediatric Pulmonology Clinic in Children‘s Hospital, Cairo, Egypt. The sweat chloride test and genetic studies were done at the time of diagnosis. All patients’ caregivers provided informed consent. Additionally, all subjects underwent detailed history taking, laboratory investigations, clinical assessment, and pelviabdominal ultrasound for evaluation of hepatic involvement. Results: Male sex, severe genetic mutation (class I and II), long duration, and early onset of the disease were independent risk factors for CFLD development. In addition, pancreatic insufficiency, as well as meconium ileus history, were predictors of CFLD. Diabetes mellitus and severe lung disease were proven to significantly elevate the risk of CFLD development. Conclusion: CFLD is not uncommon in Egyptian CF patients as one-third of the patients were found to have liver affection. CF patients with multiple risk factors are at increased risk of developing liver disease.
Objective: There has been a rise in the prenatal detection of right sided aortic arch (RAA). When associated with a left-sided arterial duct (LD), this forms a vascular ring encircling the trachea. Infants may have symptoms or signs suggestive of trache-oesophageal compression but many are asymptomatic. The objective of this study was to investigate the relationship between symptoms and severity of tracheo-bronchial compression assessed by bronchoscopy. Methods: Retrospective review of all cases of prenatally diagnosed RAA-LD in the absence of associated congenital heart disease at Evelina London Children’s Hospital and Kings College Hospital over a 4-year period between April 2015-2019. Clinical records, fetal echocardiograms and free-breathing flexible bronchoscopy (FB) data were reviewed. Results: 112 cases of isolated RAA-LD were identified of whom 82 cases (73%) underwent FB. FB was performed median age of 11 months (range 1-36 months), no complications occurred. Aberrant left subclavian artery (ALSA) was present in 86% (96/112) and mirror image branching (MIB) in 13% (15/112). 34/112 (30%) reported symptoms during follow up. 36/77 (47%) with ALSA who underwent FB showed moderate-severe compression mostly at distal tracheal and carinal level of whom 38% had parent-reported symptoms. Moderate-severe compression was seen in 3/5 (60%) with MIB mostly at mid tracheal level of whom 3 were symptomatic but only 2 with compression. In total 36% (18/50) of investigated asymptomatic patients showed moderate to severe compression. Respiratory symptoms were poorly predictive of moderate-severe compression (positive predictive value 66%). Conclusion: The absence of symptoms did not exclude significant tracheal compression. The anatomical effect of the vascular ring is underappreciated when symptoms alone are used as a marker of tracheal compression.
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.
Introduction: Congenital diaphragmatic hernia (CDH) is known with high mortality rates and significant pulmonary morbidities. The objective of this study was to describe the histopathological findings of necropsy and clinical manifestations in CDH patients to find the clinicopathological correlations. Methods: We reviewed the postmortem findings with associated clinical characteristics retrospectively in 8 CDH cases from 2017 to2022 July. Results: Of the eight cases, one was bilateral congenital eventration diagnosed by autopsy. Severe pulmonary hypertension with a right to left shunting of large patent ductus arteriosus (PDA) obtained from echocardiogram were most common. And the average time of survive was 46 (8-624) h. According to the autopsy reports, the major pathological lung changes were diffuse alveolar damages (congestion and hemorrhage) and hyaline membrane formation. Notably, although the lung volume was significantly reduced, pulmonary structural dysplasia was not observed at all, presented normal lung development (50%); bilateral (25%) or ipsilateral (25%). Lobulated deformities were accompanied in three (37.5%) cases. All patients exhibited large PDA and a patent foramen ovale with increased right ventricle (RV) volume, and the myocardial fibers were slightly congested and swollen. Pulmonary vessels showed mild to moderate arterial media thickening. Lung hypoplasia and diffuse lung damages reduced gas exchange, meanwhile the PDA and PH caused RV failure, contributed a clinical picture of organ dysfunction, which lead to death. Conclusions: Pulmonary structures have certain heterogeneity in CDH. The arteries’ pathological changes are not consistent with clinical diagnosis. And the adverse outcome may should be due to the cardiopulmonary vicious cycle.
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.