In response to: Spurr R, Ng E, Onchiri FM, Rapha B, Nakatumba-Nabende J, Rosenfeld M, Najjingo I, Stout J, Nantanda R, Ellington LE. Performance and usability of a new mobile application for measuring respiratory rate in young children with acute lower respiratory infections. Pediatr Pulmonol. 2022 Aug 22. doi: 10.1002/ppul.26125.

Clinician perspectives may inform health service strategies to meet optimal nutrition needs for infants with cystic fibrosis (CF). We conducted a qualitative study with CF-specialized dietitians (RDs) and physicians between July to December 2020 to characterize the current state of infant nutrition care delivery and organize input into a conceptual model to inform CF care program strategies. Among 42 participants, 36 completed survey responses and 6 completed interviews; 93% were RDs. Three global themes emerged in the current care model: nutrition management, family-centered connections, and collaborative care delivery. Within nutrition management clinicians emphasized providing education, setting goals, and maintaining adequate follow-up with families. Under family-centered connections clinicians expressed the need to foster relationships with families and link families to resources for assistance to social stressors such as food insecurity. Collaborative care delivery for clinicians interviewed was defined by sharing expertise from across the interdisciplinary team. Based on the timing of this study, clinicians reported compelling examples for various modes of telehealth and home weight monitoring to facilitate and support these domains of nutrition care, including potential advantages for education, supporting family needs, and communication. We integrate these themes to propose a conceptual model for integration of in-person and telehealth activities to enhance quality infant CF nutrition care delivery. Future implementation can refine this model through testing of practical telehealth interventions to optimize nutrition outcomes for infants with CF.

Background. Cystic fibrosis (CF) is an autosomal recessive disorder associated with an increased susceptibility to respiratory infections that cause progressive decline in lung function and lead to lung damage and chronic respiratory failure. To analyze the CF-related mortality trends in the Mexican population during a 22-year period.  We conducted a mortality trend analysis using death certificates data. Trends in CF-specific and in age-specific mortality rates were evaluated using Joinpoint regression analysis. Among subjects ≤40 years, 1184 CF-related deaths were identified. In 1999–2009 median age at death was 7 years compared to 10 years in 2010–2020. Overall mortality rate increased from 0.03 per 100,000 in 1999 to 0.06 per 100,000 in 2020. A decline in mortality rate for patients ≤28 days and an increase in mortality rate in older age groups was observed. Conclusion. The increasing trend in overall mortality, associated with a downward trend in neonatal mortality and an increase in median age at death is conceivably due to enhanced diagnosis, as well as major advances in treatment modalities, leading to higher survival rates. Key words: age at death, cystic fibrosis, epidemiology, low- and middle-income countries, mortality, survival rate.

As the population of ventilator-dependent children (VDC) with tracheostomies due to underlying severe BPD grows, there is an increasing need to shift the care of these children from hospital to home. Transitioning the ventilator-dependent child from the hospital to home is a complex process that requires coordination between the medical team and the family. One crucial step in the process is transitioning from an Intensive care unit (ICU) ventilator to a portable home ventilator (PHV). The Clinical team needs to understand the nuances in transitioning to PHV, including assessing readiness to transition and choosing the optimum settings on an available home ventilator. In recent years, various ventilator modes have been available in PHV that can help achieve synchronous breathing to allow for adequate gas exchange for the infant. This review details some approaches to asses readiness to transition and the process of Transition along with commonly used modes of support available in PHV, as well as the primary and secondary settings in which we should be mindful in supporting a child with chronic respiratory failure in the home setting.

While our survey showed that PwCF felt that the COVID-19 pandemic had impacted their mental health more than ETI therapy, around 9% of survey responders felt that ETI did cause an increase in either anxiety or depression. This finding was similar to the numbers Spoletini and colleagues found in their adult clinic. They also found that a dose reduction of ETI improved or resolved mental health adverse events for most patients. [2] A recently published study by Guimbellot and colleagues studied ivacaftor levels in PwCF who were on ivacaftor monotherapy and showed many patients had levels higher than the published minimum effective concentrations.[6] Thus, there may be a correlation between ETI levels and mental health adverse events that should be further explored in a prospective manner.

NTM-infected CF patients may be at risk of being underdiagnosed or inappropriately treated when relying on culture and susceptibility results from non-specialized laboratories. Since this was a small study with convenience samples, a larger study needs to be carried out. If our findings are confirmed, the drivers should be elucidated for the discrepant results. Given the increasing prevalence of NTM in the population at large and not in the CF community alone[[13]](#ref-0013), elucidating any differences in testing to ensure the correct identification, including subspeciation and antimicrobial susceptibilities should be paramount

Introduction: Bronchopulmonary dysplasia (BPD) is characterized by lung injury with varying degrees of disrupted alveolarization, vascular remodeling, inflammatory cell proliferation, and pulmonary edema. Diuretics are often used to ameliorate the symptoms or progression of BPD. Our primary objective was to use lung ultrasound (LUS) to determine if diuretics decrease pulmonary edema in infants with BPD. The secondary objective was to assess changes in respiratory support during the first week after initiation of diuretics. Methods: Premature infants requiring non-invasive respiratory support and starting diuretic therapy for evolving BPD were compared with a similar group of infants not receiving diuretics (control). For the diuretic group, LUS exams were performed before and on days 1, 3 and 6 after initiation of treatment. For the control group, LUS was performed at equivalent time points. A composite pulmonary edema severity (PES) score of 0 to 5 was calculated based on the total number of B-lines in 6 scanned areas. Respiratory support parameters (FiO2, nasal cannula flow or CPAP) were also recorded. Results: Infants in the diuretic (n=28) and control (n=23) groups were recruited at median corrected gestational ages of 34.2 (33.3-35.9) and 34.0 (33.4-36.3) weeks, respectively ( p=0.82). PES scores, FiO2, and respiratory flow support decreased significantly from day 0 to 6 ( p<.0001, p=0.001, and p=0.01, respectively) in the diuretic group, but not in the control group. Conclusion: Diuretic use is associated with decreased pulmonary edema and improved oxygenation in infants with BPD during the first week of treatment.

Background Cystic Fibrosis (CF) and autism spectrum disorder (ASD) are life-long conditions with intense treatment burdens for patients and families. Patients with a concurrent diagnosis (CF-ASD) experience unique challenges to CF care. This study describes the experiences of our multidisciplinary CF team in caring for patients with CF-ASD and provides insight into provider and parental perspectives on clinical management. Methods This is a three-part IRB-approved study involving 1) retrospective chart review of patients with CF-ASD, 2) qualitative interviews with multi-disciplinary care teams, and 3) qualitative interviews with caregivers of patients with CF-ASD. Challenges in clinical management of this specific cohort were compiled using data from chart review and care team interviews. Strategies to address these challenges were identified and rated by individual families based on relevance and practicality. Results Within our CF center, 12 patients have an official diagnosis of ASD. Median age of patients with CF-ASD was 8.5 years (range 3-20 years), 75% were male, and 83% were on highly effective modulator therapy. Clinical challenges included sensory processing issues, environmental overstimulation, intolerance to procedures and disrupted routines. Potentially impactful strategies include patient-specific coping plans, guided behavioral interventions, parental advocacy, and improved communication between the family and multidisciplinary team. Conclusions Children with CF-ASD face extraordinary challenges beyond the experience of neurotypical children with CF. Increased awareness of this complex dual diagnosis will help providers be sensitive to the unique needs of these patients, to help build consistent and trustworthy relationships with families, and to provide effective clinical care despite limitations.

Rationale: Cystic Fibrosis (CF) newborn screening (NBS) algorithms in the USA vary by state. Differences in CF NBS algorithms could potentially affect the detection rate of CF newborns and lead to disparities in CF diagnosis amongst different racial and ethnic groups. Objectives: Generate a database of CF NBS algorithms in the USA and identify processes that may potentially lead to missed diagnoses or lead to health care disparities. Methods: We sent an online survey to state and regional CF and NBS leaders about the type and threshold of immunoreactive trypsinogen (IRT) cutoff used and methods used for CFTR gene variant analysis. Follow-up by email and phone was done to ensure a response from every state, clarify responses, and resolve discordances . Results: There was wide variation in the NBS algorithms employed by different states. Approximately half the states use a floating IRT cutoff and half use a fixed IRT cutoff. CFTR variant analysis also varied widely, with 2 states analyzing only for the F508del variant and 4 states incorporating CFTR gene sequencing. The other states used CFTR variant panels ranging from 23 to 365 CFTR variants. Conclusions: CF NBS algorithms vary widely amongst the different states in the USA, which affects the ability of CF NBS to diagnose newborn infants with CF consistently and uniformly across the country and potentially may miss more infants with CF from minority populations. Our results identify an important area for quality improvement in CF NBS.

Although COP is idiopathic by definition, it is important to investigate each diagnosed case for potential causes, such as iatrogenic from radiation or known causative medications [1], connective tissue diseases, inflammatory bowel disease, malignancies, history of lung transplant or bone marrow graft. OP may present weeks to months before other signs of connective tissue disorders and therefore the patient should undergo serologic testing for these diseases. [6] In the case presented, the specific etiology was unyielding and so remains cryptogenic in nature. This patient’s symptoms quickly improved following the use of steroids and tolerated tapering off completely without relapse one year out from initial diagnosis.

Background: Whether Lung ultrasound (LUS) can be used for pathogenic diagnosis is still controversial. This was conducted to test the accuracy and reliability of ultrasound in the diagnosis of pneumonia and to clarify whether ultrasound has diagnostic value for the etiology. Methods: A total of 135 neonatal pneumonia patients with an identified pathogen and 50 newborns with normal lungs in the newborn intensive care unit of 10 tertiary hospitals in China were enrolled. The study ran from November 2020 to December 2021. The infants were divided into various groups according to pathogens, the time of infection, the gestational age, the severity of the disease. The distribution of pleural line abnormalities, pulmonary edema, and pulmonary consolidation, as well as the incidence of air bronchogram and pleural effusion based on LUS, were compared between the above groups and between the pneumonia and healthy control groups. Results: There were significant differences in pulmonary consolidation. The sensitivity and specificity of the diagnosis of severe pneumonia based on the extent of pulmonary consolidation were 83.3% and 85.2%, respectively. The area under the receiver operating characteristic curve for the identification of mild or severe pneumonia based on the distribution of pulmonary consolidation was 0.776. Conclusion: Lung ultrasound has good performance in differentiating the severity of neonatal pneumonia, but cannot be used for pathogenic diagnosis.

Introduction: E-cigarette, or vaping, product use-associated lung injury (EVALI) results from inhaling the aerosol of e-cigarettes and has similar clinical features to coronavirus disease 2019 (COVID-19). EVALI case counts since the declaration of the COVID-19 pandemic is unknown. Methods: A retrospective electronic health record chart review of adolescents hospitalized at one institution with EVALI was conducted. Clinical characteristics and hospital course of patients hospitalized during the pandemic were compared to those pre-pandemic. Results: The clinical presentation of adolescents hospitalized prior-to (n=19) and during the COVID-19 pandemic (n=22) were similar with respect to constitutional, respiratory, and gastrointestinal symptoms. All patients hospitalized during the pandemic were tested for COVID-19 at least once. Only one patient had a positive SARS-CoV-2 RT-PCR test result. 31 out of 39 patients treated with corticosteroids had clinical improvement within 24 hours (79%). Patients hospitalized during the pandemic had a shorter median length of stay (5 vs 7 days, p<0.01), and were less often discharged with home oxygen (1 vs 6 patients, p=0.04). Pulmonary function tests improved pre-to post-corticosteroid treatment and post-corticosteroid to follow-up. Conclusions: Eliciting a history of vaping in adolescents presenting with constitutional, respiratory, and gastrointestinal symptoms is important to identify EVALI cases, which have continued throughout the COVID-19 pandemic. A shorter length of stay with less need for mechanical ventilation and home oxygen in adolescents hospitalized during the pandemic may reflect increased familiarity with EVALI characteristics. Corticosteroids led to clinical and pulmonary function improvement.

Introduction: Children with a history of bronchopulmonary dysplasia (BPD) may have lower physical activity levels, but evidence to date is based on self-report. This study compared physical activity levels between children born extremely preterm with and without history of BPD, and examined their associations with pulmonary magnetic resonance imaging (MRI) and pulmonary function test (PFT) indices. Methods: This multi-centre cross-sectional study included children aged 7-9 years born extremely preterm, with and without BPD. Children wore a pedometer for one week, then completed the Physical Activity Questionnaire (PAQ), pulmonary MRI, and PFT. Spearman correlations and multivariable linear regression modelling were performed. Results: Of 45 children, 28 had a history of moderate-severe BPD. There were no differences in any physical activity outcomes by BPD status. Higher average daily step count and higher average daily moderate-vigorous physical activity (MVPA) were each correlated with greater forced vital capacity (r=0.41 and 0.58), greater MRI lung proton density at full expiration (r=0.42 and 0.49), and lower lung clearance index (r=-0.50 and -0.41). After adjusting for MRI total proton density and BPD status, a 5% increase in forced expiratory volume at one second was associated with 738 (95%CI: 208, 1268) more steps per day and 0.1 (0.0, 0.2) more hours of MVPA, respectively. Conclusion: School-aged children born extremely preterm have similar physical activity levels to their peers, regardless of history of BPD. MRI and PFT measures suggestive of gas trapping and/or airflow obstruction are associated with lower physical activity levels.

Background: Infants with Bronchopulmonary Dysplasia (BPD) are often prescribed diuretics before the neonatal intensive care unit (NICU) discharge. It is unknown whether outpatient medication weaning strategies affect duration of home oxygen therapy. Methods: This was a secondary cohort analysis of infants born <32 weeks gestational age with BPD from 2015-2018 discharged from our NICU or regional NICUs, referred to our pulmonary clinic for home oxygen management. We compared three groups: those discharged with no diuretics, diuretics actively weaned (dose decreased) and diuretics passively weaned (dose not adjusted). Results: Out of 125 infants, 116 were included in the analysis. Forty-five infants were discharged without diuretics; 52 infants were discharged with diuretics that were actively weaned; 19 infants were discharged with diuretics that were passively weaned. Infants who were passively weaned spent the most time on home oxygen (median 28 weeks, IQR 16-52; p=0.011); there were no differences in home oxygen duration in infants actively weaned (median 13 weeks, IQR 10-26) versus not on diuretics (median 22 weeks, IQR 12-30, p=0.285). Multivariable adjustment for other illness characteristics associated with duration of home oxygen did not change this finding. Conclusions: Active weaning of diuretics did not prolong duration of home oxygen, in the setting of a standardized clinical guideline for weaning home oxygen in infants with BPD. These data can serve as baseline information to implement and test standardized strategies for outpatient medication management.

Objectives: Universal implementation of cystic fibrosis (CF) newborn screening (NBS) has led to the diagnostic dilemma of infants with CF screen positive, inconclusive diagnosis (CFSPID), for which there is limited guidance regarding prognosis and standardized care. Rates of reclassification from CFSPID to CF vary and risk factors for reclassification are unknown. We investigated whether clinical characteristics are associated with risk of reclassification from CFSPID to a CF diagnosis. Methods: Children with a positive CF NBS were recruited from two sites in California. Retrospective, longitudinal, and cross-sectional data were collected. A subset of subjects had nasal epithelial cells collected for CFTR functional assessment. Multivariate logistic regression was used to assess the risk of CFSPID-to-CF reclassification. Results: A total of 112 children completed the study (CF=53, CFSPID=59). Phenotypic characteristics between groups showed differences in pancreatic insufficiency prevalence, immunoreactive trypsinogen (IRT) levels, and Pseudomonas aeruginosa (PSA) colonization. Spirometry measures were not different between groups. Nasal epithelial cells from 10 subjects showed 7-30% of wild type (WT)-CFTR function in those who reclassified and 27-67% of WT-CFTR function in those who retained the CFSPID designation. Modeling revealed that increasing sweat chloride concentration (sw[Cl -]) and PSA colonization were independent risk factors for reclassification to CF. Conclusion: Increasing sw[Cl -] and history of PSA colonization are associated with risk of reclassification from CFSPID to CF in a population with high IRT and two CFTR variants. Close follow-up to monitor phenotypic changes remains critical in this population. The role of CFTR functional assays in this population requires further exploration.

Background. The COVID-19 pandemic has spread across the world, leading to government measures associated with a negative impact on mental health. The aim of this study was to evaluate the impact of COVID-19 on depression, anxiety and resilience in Dutch people with cystic fibrosis (PwCF) or primary ciliary dyskinesia (PwPCD) and their caregivers during the pandemic. Methods. Adolescents (12-17 years), adults and caregivers of children (0-17 years) with CF or PCD completed questionnaires on depression (PHQ-9), anxiety (GAD-7) and resilience (BRS) between September 2020 and February 2021. The psychosocial impact of COVID-19 was measured by the Exposure and Family Impact Survey (CEFIS) Part 2. Mixed model analyses compared PHQ-9 and GAD-7 results to participants’ pre-pandemic scores. Results. 110 participants (10 PwCF, 31 PwPCD, 52 CF caregivers, 17 PCD caregivers) completed questionnaires during the pandemic. Pre-pandemic outcomes were available for 87 participants. The prevalence of symptoms of depression and anxiety (PHQ-9 or GAD-7 scores ≥5) in PwCF and PwPCD and their caregivers before and during the pandemic was high, with an increase in depression in PwCF (2.75; 95%-CI: 0.82 to 4.68) and increase in anxiety in CF caregivers (1.03; 0.09 to 1.96) during the pandemic. Resilience was within the normal range for all groups, CEFIS scores corresponded to a low to normal impact. Conclusion. PwCF and PwPCD and their caregivers were at high risk of increased depression and anxiety symptoms both before and during the pandemic, which emphasizes the importance of mental health screening and psychological care in CF and PCD.

Background and aims: We aimed to analyze the correlation of urinary with serum NT-proBNP concentrations in acute bronchiolitis and its association with the severity of the disease.  Material and Methods: A pilot observational study conducted between 1st October and 31st March 2022, including acute bronchiolitis cases who attended our institution. Serum and urinary NT-proBNP concentrations were determined using the Alere NT-proBNP assay in time-matched urine and blood samples. We explored the linear relationship between both concentrations and compared clinical outcomes indicative of severe acute bronchiolitis between groups of raised and normal urinary NT-proBNP.  Results: 17 infants (median age 68 (36-91) days) with 36 time-matched samples were included. The urinary and serum concentrations of NT-proBNP were significantly correlated with (r=0.867 & R-squared coefficient=0.751; p<0.001). The log-10-transformed urinary NT-proBNP concentrations were higher at the time of hospital admission in those infants that required PICU admission with ventilatory support compared with those without this outcome (1.85 (1.16-2.44) pg/mg vs 0.63 (0.45-0.84) pg/mg); p<0.001); and resulted positively and strongly correlated with the duration of the ventilatory support (rho=0.76; p<0.001) and the LOS hospitalization (rho=0.84; p<0.001) Conclusion: The measurement of urinary NT-proBNP concentrations could be a reliable surrogate for serum NT-proBNP levels highlighting the potential value of the urinary NT-proBNP as a non-invasive tool to assess severity in acute bronchiolitis.

In conclusion, our data suggest that subjects with genotype 5T;TG12/VVCC likely have a very low risk of progressing to CF, as compared to those with F508del/5T;TG12. 4 This observation could lead to differentiate follow up in presence of at least one 5T;TG12. Knowing these data is crucial to offer a useful counseling for CRMS/CFSPID infants and for non‐CF adults with CBAVD alone. Anyway further data are needed to evaluate the outcomes after a longer follow up.