Glenda N. Bendiak

The approach to Pseudomonas aeruginosa in cystic fibrosis.

Pseudomonas aeruginosa continues to be the most common pathogen in cystic fibrosis (CF) lung disease, and chronic infection with mucoid strains is associated with an accelerated decline in lung function. Although multiple factors can potentially explain the susceptibility of CF airways to this organism, their individual relevance is still largely unclear. Prevention of infection remains an important task, and hygiene measures have been successful in reducing cross-infection, but the universal presence of the organism creates an ongoing challenge, and vaccination strategies have not been highly successful to date. Over the last decade treatment strategies have shifted from controlling chronic infection to attempting to eradicate P. aeruginosa in the early stages of infection. Multiple strategies have been shown to be efficacious, but the optimal form and duration of therapy have yet to be defined. Inhaled antibiotics are a key component of maintenance therapy for chronic infection, and the spectrum of available compounds is rapidly expanding. Pulmonary exacerbations can be reduced with this strategy but usually require intravenous antibiotic therapy once they occur. Nonantibiotic approaches to address P. aeruginosa infection are currently being developed and may expand the therapeutic repertoire in the future.

Long-term non-invasive ventilation therapies in children: A scoping review

Long-term non-invasive ventilation (NIV) is a common modality of breathing support used for a range of sleep and respiratory disorders. The aim of this scoping review was to provide a summary of the literature relevant to long-term NIV use in children. We used systematic methodology to identify 11,581 studies with final inclusion of 289. We identified 76 terms referring to NIV; the most common term was NIV (22%). Study design characteristics were most often single center (84%), observational (63%), and retrospective (54%). NIV use was reported for 73 medical conditions with obstructive sleep apnea and spinal muscular atrophy as the most common conditions. Descriptive data, including NIV incidence (61%) and patient characteristics (51%), were most commonly reported. Outcomes from sleep studies were reported in 27% of studies followed by outcomes on reduction in respiratory morbidity in 19%. Adverse events and adherence were reported in 20% and 26% of articles respectively. Authors reported positive conclusions for 73% of articles. Long-term use of NIV has been documented in a large variety of pediatric patient groups with studies of lower methodological quality. While there are considerable data for the most common conditions, there are fewer data to support NIV use for many additional conditions.

Long-term non-invasive ventilation therapies in children: a scoping review protocol

Introduction Non-invasive ventilation (NIV) in children has become an increasingly common modality of breathing support where pressure support is delivered through a mask interface or less commonly through other non-invasive interfaces. At this time, NIV is considered a first-line option for ventilatory support of chronic respiratory insufficiency associated with a range of respiratory and sleep disorders. Previous reviews on the effectiveness, complications and adherence to NIV treatment have lacked systematic methods. The purpose of this scoping review is to provide an overview of the evidence for the use of long-term NIV in children. Methods and analysis We will use previously established scoping methodology. Ten electronic databases will be searched to identify studies in children using NIV for longer than 3 months outside an intensive care setting. Grey literature search will include conference proceedings, thesis and dissertations, unpublished trials, reports from regulatory agencies and manufacturers. Two reviewers will independently screen titles and abstracts for inclusion, followed by full-text screening of potentially relevant articles to determine final inclusion. Data synthesis will be performed at three levels: (1) an analysis of the number, publication type, publication year, and country of publication of the studies; (2) a summary of the study designs, outcomes measures used; (3) a thematic analysis of included studies by subgroups. Ethics and dissemination This study will provide a wide and rigorous overview of the evidence on the use of long-term NIV in children and provide critical information for healthcare professionals and policymakers to better care for this group of children. We will disseminate our findings through conference proceedings and publications, and evaluate the results for further systematic reviews and meta-analyses.

Longitudinal changes in clinical characteristics and outcomes for children using long-term non-invasive ventilation

Objectives To describe longitudinal trends in long-term non-invasive ventilation (NIV) use in children including changes in clinical characteristics, NIV technology, and outcomes. Methods This was a multicenter retrospective cohort of all children started on long-term NIV from 2005 to 2014. All children 0 to 18 years who used NIV continuously for at least 3 months were included. Measures and main outcomes were: 1) Number of children starting NIV; 2) primary medical condition; 3) medical complexity defined by number of comorbidities, surgeries and additional technologies; 4) severity of sleep disordered breathing measured by diagnostic polysomnography; 5) NIV technology and use; 6) reasons for NIV discontinuation including mortality. Data were divided into equal time periods for analysis. Results A total of 622 children were included in the study. Median age at NIV initiation was 7.8 years (range 0–18 years). NIV incidence and prevalence increased five and three-fold over the 10-year period. More children with neurological and cardio-respiratory conditions started NIV over time, from 13% (95%CI, 8%-20%) and 6% (95%CI, 3%-10%) respectively in 2005–2008 to 23% (95%CI, 18%-28%) and 9% (95%CI, 6%-14%, p = 0.008) in 2011–2014. Medical complexity and severity of the sleep-disordered breathing did not change over time. Overall, survival was 95%; mortality rates, however, rose from 3.4 cases (95% CI, 0.5–24.3) to 142.1 (95% CI 80.7–250.3, p<0.001) per 1000 children-years between 2005–2008 and 2011–2014. Mortality rates differed by diagnostic category, with higher rates in children with neurological and cardio-respiratory conditions. Conclusions As demonstrated in other centers, there was a significant increase in NIV prevalence and incidence rate. There was no increase in medical complexity or severity of the breathing abnormalities of children receiving long-term NIV over time. The mortality rate increased over time, maybe attributable to increased use of NIV for children with neurological and cardio-respiratory conditions.

Insulin Resistance and Hypertension in Obese Youth With Sleep-Disordered Breathing Treated With Positive Airway Pressure: A Prospective Multicenter Study

STUDY OBJECTIVES There is evidence that cardiometabolic disease associated with obesity and sleep-disordered breathing (SDB) in adults is present in youth. SDB is often treated with positive airway pressure (PAP) in youth with obesity. Our aims were to determine: (1) the prevalence of cardiometabolic disease and (2) whether PAP improves markers of cardiometabolic disease, in youth with obesity and newly diagnosed moderate-severe SDB. METHODS A prospective multicenter cohort study was conducted in youth (8 to 16 years old) with obesity, prescribed PAP therapy for newly diagnosed moderate-severe SDB. Assessments occurred at baseline and at 6 and 12 months. Outcomes included markers of insulin resistance (change in homeostasis model assessment of insulin resistance (HOMA-IR) at 6 months = primary outcome), hypertension (24-hour ambulatory/blood pressure) and inflammation (high-sensitivity C-reactive protein: hs-CRP). RESULTS Twenty-seven participants were enrolled. Of those with baseline testing available, 10/25 (40%) had HOMA-IR above the 97th percentile, 10/23 (44%) were hypertensive, 16/23 (70%) had loss of nocturnal blood pressure dip and hs-CRP was elevated in 16/27 (64%). There were no significant changes over time in markers of metabolic dysfunction or blood pressure, nor between PAP-adherent and non-adherent subgroups. CONCLUSIONS In youth with obesity and SDB, metabolic dysfunction and hypertension were highly prevalent. There were no statistically significant improvements in cardiometabolic markers 1 year after the prescription of PAP therapy, although clinically relevant improvements were seen in insulin resistance and systolic blood pressure load, important predictors of future risk of cardiovascular disease. Larger, longer-term studies are needed to determine whether PAP improves cardiometabolic outcomes in obese youth. COMMENTARY A commentary on this article appears in this issue on page 1025.

Long-Term Impact of Sleep-Disordered Breathing on Quality of Life in Children With Obesity

STUDY OBJECTIVES (1) To determine baseline quality of life (QOL) among children with obesity and newly diagnosed moderate-severe sleep-disordered breathing (SDB) and to compare it to the reported QOL of children with obesity or SDB alone and healthy children. (2) To evaluate QOL change after 1 year. METHODS A prospective multicenter cohort study was conducted in children (8-16 years) with obesity, prescribed positive airway pressure (PAP) therapy for moderate-severe SDB. Outcomes included parent-proxy and self-report total and subscale scores on the PedsQL questionnaire (baseline and 1-year). RESULTS Total PedsQL scores were indicative of impaired QOL in 69% of cases based on parent-report and in 62% on self-report. Parents reported significantly lower QOL in our cohort than that reported in other studies for children with obesity or SDB alone or healthy children, on total PedsQL score and on social and psychosocial subscales. PedsQL total scores for participants were significantly higher (mean difference 7.3 ± 15.3, P = .03) than those reported by parents. Parents reported significant improvements in total PedsQL (mean change 7.29 ± 13.73, P = .04) and social functioning (mean change 17.65 ± 24.69, P = .04) scores after 1 year. No significant differences were found by childrens self-report or by PAP adherence. CONCLUSIONS QOL of children with obesity and SDB is lower than in children with obesity or SDB alone or healthy children. One year later, children reported no significant changes in QOL; parents reported significant improvements in total PedsQL and social functioning scores. PAP adherence did not significantly affect QOL change in this population. COMMENTARY A commentary on this article appears in this issue on page 307.

Lung clearance index is elevated in young children with symptom‐controlled asthma

Pulmonary function testing has been recommended as an adjunct to symptom monitoring for assessment of asthma control. Lung clearance index (LCI) measures ventilation inhomogeneity and is thought to represent changes in the small airways. It has been proposed as a useful early marker of airway disease in asthmatic subjects, and determining it is feasible in preschool children. This study aims to assess whether LCI remains elevated in symptomatically controlled asthmatic children with a history of severe asthma, compared with healthy controls. A secondary aim was to determine whether the results were consistent across the preschool and school‐aged populations.

Association of wheeze with lung function decline in children with sickle cell disease

Emerging evidence suggests that wheezing in children with sickle cell disease (SCD) is associated with higher rates of acute chest syndrome (ACS) and vaso-occlusive crises, independent of a diagnosis of asthma [1, 2]. We investigated the relationships of longitudinal pulmonary function decline with wheeze, asthma and atopy in a cohort of paediatric patients with SCD, using the International Study of Asthma and Allergies in Childhood (ISAAC) questionnaire [3], skin prick testing (SPT) and airway nitric oxide (NO) measurements. Wheeze in children with sickle cell disease is associated with airflow limitation and lung function decline http://ow.ly/oyZQ30fXnQs