A large collection of real-world pediatric sleep studies.

Despite being crucial to health and quality of life, sleep-especially pediatric sleep-is not yet well understood. This is exacerbated by lack of access to sufficient pediatric sleep data with clinical annotation. In order to accelerate research on pediatric sleep and its connection to health, we create the Nationwide Children's Hospital (NCH) Sleep DataBank and publish it at Physionet and the National Sleep Research Resource (NSRR), which is a large sleep data common with physiological data, clinical data, and tools for analyses. The NCH Sleep DataBank consists of 3,984 polysomnography studies and over 5.6 million clinical observations on 3,673 unique patients between 2017 and 2019 at NCH. The novelties of this dataset include: (1) large-scale sleep dataset suitable for discovering new insights via data mining, (2) explicit focus on pediatric patients, (3) gathered in a real-world clinical setting, and (4) the accompanying rich set of clinical data. The NCH Sleep DataBank is a valuable resource for advancing automatic sleep scoring and real-time sleep disorder prediction, among many other potential scientific discoveries.

What Are the Factors Affecting Total Sleep Time During Video Polysomnography in Infants?

OBJECTIVE: The aim of the study is to investigate factors affecting total sleep time (TST) during infant polysomnography (PSG) and assess if <4 hours of TST is sufficient for accurate interpretation. STUDY DESIGN: Overall, 242 PSGs performed in 194 infants <6 months of chronological age between March 2013 and December 2015 were reviewed to identify factors that affect TST, including age of infant, location and timing of study, presence of medical complexity, and presence of nasal tubes. A continuum of apnea-hypopnea index (AHI) in relation to TST was reviewed. Data were examined in infants who had TST <4 hours and low AHI. RESULTS: Greater TST (p < 0.001) was noted among infants during nocturnal PSGs, at older chronological and post-menstrual ages, and without medical complexity. The presence of nasogastric/impedance probes reduced TST (p = 0.002). Elevated AHIs were identified even in PSGs with TST <4 hours. Short TST may have affected interpretation and delayed initial management in one infant without any inadvertent complications. CONCLUSION: Clinical factors such as PMA and medical complexity, and potentially modifiable factors such as time of day and location of study appeared to affect TST during infant PSGs. TST < 4 hours can be sufficient to identify high AHI allowing physician interpretation. KEY POINTS: · Less than 4 hours of TST is enough for interpretation of infant polysomnography.. · Shorter TST appears related to infant age, medical complexity, and higher apnea-hypopnea index.. · Modifiable factors seen with higher TST were time of day, environment, and presence of nasal tubes..

Home Oxygen Therapy for Children. An Official American Thoracic Society Clinical Practice Guideline.

BACKGROUND: Home oxygen therapy is often required in children with chronic respiratory conditions. This document provides an evidence-based clinical practice guideline on the implementation, monitoring, and discontinuation of home oxygen therapy for the pediatric population. METHODS: A multidisciplinary panel identified pertinent questions regarding home oxygen therapy in children, conducted systematic reviews of the relevant literature, and applied the Grading of Recommendations, Assessment, Development, and Evaluation approach to rate the quality of evidence and strength of clinical recommendations. RESULTS: After considering the panel's confidence in the estimated effects, the balance of desirable (benefits) and undesirable (harms and burdens) consequences of treatment, patient values and preferences, cost, and feasibility, recommendations were developed for or against home oxygen therapy specific to pediatric lung and pulmonary vascular diseases. CONCLUSIONS: Although home oxygen therapy is commonly required in the care of children, there is a striking lack of empirical evidence regarding implementation, monitoring, and discontinuation of supplemental oxygen therapy. The panel formulated and provided the rationale for clinical recommendations for home oxygen therapy based on scant empirical evidence, expert opinion, and clinical experience to aid clinicians in the management of these complex pediatric patients and identified important areas for future research.

Sedation with Propofol for Bronchoscopy in Cystic Fibrosis Lung Transplant Recipients.

INTRODUCTION: Flexible fiberoptic bronchoscopy (FFB) plays an important role in the surveillance of cystic fibrosis (CF) patients after lung transplantation (LTx). With rapid onset and clearance, propofol provides a safe and efficient method for sedation during FFB, yet sedation requirements for CF patients are not well described. OBJECTIVES: Due to pharmacokinetic differences for other classes of drugs in CF patients, this study was performed to examine propofol requirements for sedation during bronchoscopy in lung transplant recipients with CF. METHODS: A single-center retrospective cohort study was performed to examine propofol sedation requirements during outpatient surveillance. FFB procedures with transbronchial biopsy (TBB) in post-LTx recipients between 2009 and 2014 were conducted. RESULTS: A total of 40 FFB procedures with TBB were performed 20 CF (11 females), 20 non-CF (11 females). Mean (± SD) age was 25.6 ± 9.2 (range 13-42) years and 22.2 ± 10.8 (range 11-39) years for the CF and non-CF groups, respectively. Propofol requirements were significantly higher in the CF patients compared to the non-CF patients. Mean (± SD) propofol dose for CF patients was 334 ± 86 versus 214 ± 88 mg for non-CF patients (p < 0.001). Mean (± SD) propofol dose per weight (mg/kg) was 6.5 ± 2.1 for CF patients versus 3.8 ± 1.6 for non-CF patients (p < 0.001). CONCLUSIONS: Compared to a non-CF cohort, CF lung transplant recipients required higher dosages of propofol for sedation during FFB with TBB.

Adult survivor with repaired complete pentalogy of Cantrell.

Pentalogy of Cantrell is a very rare condition with very high mortality. We present an adult survivor with a classic pentad who underwent sequential surgical repairs as a neonate, child, and young adult. He required home mechanical ventilation for the first two years of life and subsequently needed noninvasive nocturnal ventilation as an adult.

Polysomnographic differences associated with pulmonary hypertension in patients with advanced lung disease due to cystic fibrosis.

BACKGROUND: Pulmonary hypertension (PH) commonly occurs in patients with cystic fibrosis (CF), but there is no current data regarding alterations of sleep in patients with PH. METHODS: A single-center, retrospective review was performed in patients with advanced lung disease due to CF who completed both nocturnal polysomnography and right heart catheterization (RHC) from January 2010 to June 2013. For statistical analysis, two-tailed unpaired t tests and Pearson correlation coefficient analysis were performed after normal distribution was confirmed. RESULTS: A total of 18 consecutive CF patients were enrolled with RHC identifying PH in 56 % (10/18) of patients. The PH group had significantly lower mean sleep efficiency (72 ± 4 vs. 87 ± 3 %, p = 0.01), significantly higher ETCO(2) levels (54.5 ± 2.2 vs. 43.8 ± 3.0 mmHg, p = 0.01) on capnography, and significantly lower PO(2) (53.8 ± 3.1 vs. 65.5 ± 3.9 mmHg, p = 0.03) on capillary blood gas. Correlations with poor sleep efficiency included mean PAP (r = - 0.55, p = 0.01), systolic PAP (r = -0.5, p = 0.03), ETCO(2) (r = - 0.53, p = 0.02), and PO(2)) (r = 0.62, p = 0.01); ETCO(2) with systolic PAP (r = 0.47, p = 0.04) and PCO(2) (r = - 0.57, p = 0.01); and PO(2) to 6-min walk distance (r = 0.55, p = 0.02). CONCLUSIONS: We found significant differences in sleep efficiency and gas exchange associated with PH in CF patients with advanced lung disease.

Pseudomonas aeruginosa in children with cystic fibrosis diagnosed through newborn screening: assessment of clinic exposures and microbial genotypes.

BACKGROUND: Chronic pulmonary infection with Pseudomonas aeruginosa (PA) is responsible for significant morbidity and mortality in cystic fibrosis (CF). Because of the limited studies evaluating early exposure and the progression of genetic variability of PA, our goal was to assess PA in young children with CF followed in two clinic types. METHODS: A total of 39 infants with CF diagnosed through newborn screening were randomly assigned to either a segregated (PA-free) or mixed (PA-positive) clinic at two different CF centers, one of which replaced an older, mixed clinic where nosocomial acquisition was suspected. Oropharyngeal (OP) swab cultures were examined with subsequent genotyping to characterize the strains of PA isolated. RESULTS: We found that 13/21 segregated clinic patients and 14/18 mixed clinic patients showed positive PA, with median acquisition ages of 3.3 and 2.2 years, respectively (P = 0.57). The median time to PA acquisition, however, was significantly longer in the new clinic with proper hygiene precautions compared to an old site (5.0 years vs. 1.7 years, P < 0.001). The majority of subjects isolated a single genotype of PA or AP-PCR types during the study period with eight subjects clearing the isolate after only one positive culture. The development of chronic colonization yielded the predominance of a single major genotype or AP-PCR type. CONCLUSIONS: Segregation of infants and young children with CF in PA-negative or PA-positive clinics did not alter the time to first PA isolation in this randomized assessment of facilities with hygienic precautions. During the early infection period where PA is first isolated in young children with CF, patients cleared different PA strains until a predominant strain established permanent colonization.

Ibuprofen therapy and nasal polyposis in cystic fibrosis patients.

OBJECTIVE: To assess the effects on nasal polyposis from high-dose ibuprofen therapy used in children with cystic fibrosis (CF) pulmonary disease. DESIGN: Retrospective case series. MAIN OUTCOME MEASURE: Presence or absence of nasal polyps. RESULTS: Twenty-two patients treated with high-dose ibuprofen therapy to benefit pulmonary function were identified from 235 patients with CF. Sinonasal disease was present in 19 patients, of whom 12 had nasal polyposis. All 12 patients had observed absence of nasal polyps at some point during their ibuprofen course. Nasal polyps were present in five patients during ibuprofen therapy, and all resolved with increased ibuprofen doses. Polyps occurred in six of eight patients after ibuprofen therapy ceased. Five of the 12 patients required endoscopic sinus surgery for polyposis. CONCLUSION: High-dose ibuprofen therapy chronically administered at appropriate weight-based dosing is a possible treatment option for children and young adults with CF polyposis. More testing is indicated.

Evidence on improved outcomes with early diagnosis of cystic fibrosis through neonatal screening: enough is enough!

OBJECTIVE: To generate and examine evidence in support of diagnosing cystic fibrosis (CF) early through newborn screening (NBS). STUDY DESIGN: Using a randomized controlled trial with unique unblinding/surveillance, we evaluated patients with CF receiving similar treatment after assignment to an early diagnosis (screened) group or to a control group. Outcomes studied at diagnosis and longitudinally included measures of nutritional status and lung disease. RESULTS: Assessment of patients with CF without meconium ileus who had pancreatic insufficiency revealed marked differences in age and condition at diagnosis--screened patients had significantly better length/height, weight, and head circumference. Follow-up evaluation for 16 years showed that height and weight differences persisted long term. Although screened patients had better chest x-ray scores at diagnosis, our trial suggests that the effects of confounders such as Pseudomonas aeruginosa infections led to deterioration of their scores after 10 years, but there were no significant differences between the 2 CF/pancreatic insufficiency subgroups. CONCLUSIONS: Early diagnosis of CF and aggressive nutritional management can prevent malnutrition and growth failure. Although CF NBS provides a potential opportunity for better pulmonary outcomes, it appears that other factors can predominate over time in pulmonary prognosis. Overall, the Wisconsin trial is positive and provides enough evidence for routine CF NBS.

Preventing early, prolonged vitamin E deficiency: an opportunity for better cognitive outcomes via early diagnosis through neonatal screening.

OBJECTIVE: To evaluate whether early diagnosis of cystic fibrosis (CF) through newborn screening (NBS) and early vitamin E status are associated with cognitive function. STUDY DESIGN: We assessed cognitive function for 71 children without meconium ileus (ages 7.3-16.9 years) enrolled in the screened (S) or control (C) group of the Wisconsin CF Neonatal Screening Project. The Test of Cognitive Skills, 2nd edition generated the cognitive skills index (CSI; mean = 100, SD = 16). Vitamin E deficiency at diagnosis was defined as plasma alpha-tocopherol (alpha-T) below 300 microg/dL (<300E). Primary analyses evaluated CSI scores across the 4 levels of group (S or C) by using alpha-T status (<300E or >300E) with analysis of covariance. RESULTS: After adjusting for covariates, CSI in the C<300E group was significantly lower than each of the other groups (C>300E, S<300E, and S>300E; P < .05). The highest proportion of CSI scores >84 occurred in the C<300E group (41%). Patients in this group also had the lowest mean head circumference z-scores at diagnosis. CONCLUSIONS: Our results show that prolonged alpha-T deficiency in infancy is associated with lower subsequent cognitive performance. Thus, diagnosis via NBS may benefit the cognitive development of children with CF, particularly in those prone to vitamin E deficiency during infancy.

Quality of life of children with cystic fibrosis.

OBJECTIVE: To review health-related quality of life (QOL) and associated issues and to describe a study investigating "Child Health Questionnaire" (CHQ) scores in relationship to newborn screening (NBS) for cystic fibrosis (CF) and markers of disease severity. METHODS: A total of 36 patients from 10-15.5 years old who were enrolled in the screened or control group of the Wisconsin CF Neonatal Screening Project completed the CHQ. Scale scores comprised the dependent variables. Independent variables included study group and measures of disease severity. Analyses included Fisher's exact, 2-sample Wilcoxon, and t tests. RESULTS: QOL did not differ significantly between the screened and control groups for any of the scales. None of the comparisons of CHQ scale scores across measures of disease severity were significant in this small sample, but the CHQ and power were limiting. CONCLUSIONS: Our results did not demonstrate a benefit of CF NBS on QOL; however, the CHQ may not be adequately sensitive to QOL in children with CF with disease severity comparable to our sample. The Cystic Fibrosis Questionnaire, a recently validated CF-specific QOL measure for pediatric samples, is likely to provide a more informative evaluation of the effects of CF NBS on patients' QOL.

Longitudinal development of mucoid Pseudomonas aeruginosa infection and lung disease progression in children with cystic fibrosis.

CONTEXT: Although Pseudomonas aeruginosa is the most common virulent respiratory pathogen in cystic fibrosis (CF), the longitudinal development of P aeruginosa infection and its effect on antibody responses and lung disease progression in children with CF remain unclear. OBJECTIVE: To prospectively examine the epidemiology of P aeruginosa infection and its impact on CF pulmonary morbidity. DESIGN, SETTING, AND PATIENTS: We prospectively evaluated 56 CF patients at 2 CF centers in Madison and Milwaukee, Wis, from birth up to age 16 years between April 15, 1985, and April 15, 2004, with diagnoses made through the Wisconsin CF Neonatal Screening Project. MAIN OUTCOME MEASURES: Timing of nonmucoid P aeruginosa and mucoid P aeruginosa acquisition was assessed by first positive result. Longitudinal development from no P aeruginosa to nonmucoid P aeruginosa and from nonmucoid P aeruginosa to mucoid P aeruginosa was examined. Outcome measurements included antibody titers, respiratory symptoms, quantitative chest radiography, and pulmonary function tests. RESULTS: Sixteen patients (29%) acquired nonmucoid P aeruginosa in the first 6 months of life. The age-specific prevalence of mucoid P aeruginosa increased markedly from age 4 to 16 years. Nonmucoid and mucoid P aeruginosa were acquired at median ages of 1.0 and 13.0 years, respectively. In contrast with the short transition time from no P aeruginosa to nonmucoid P aeruginosa, the transition time from nonmucoid to mucoid P aeruginosa was relatively long (median, 10.9 years) and could be slightly extended by brief/low anti-P aeruginosa antibiotic treatment. Antibody titers increased with both transitions, but the deterioration in cough scores, chest radiograph scores, and pulmonary function correlated best with transition from nonmucoid to mucoid P aeruginosa. CONCLUSIONS: Early prevention and detection of nonmucoid and mucoid P aeruginosa are critical because of early acquisition and prevalence. There is a window of opportunity for suppression and possible eradication (by aggressive anti-P aeruginosa treatment) of initial nonmucoid P aeruginosa. Mucoid P aeruginosa plays a much greater role in CF lung disease progression than nonmucoid P aeruginosa. Antibody titers, cough scores, and chest radiographs are early signs of nonmucoid P aeruginosa and especially mucoid P aeruginosa stages.

Longitudinal pulmonary status of cystic fibrosis children with meconium ileus.

Although meconium ileus (MI) is the earliest manifestation of cystic fibrosis (CF), and is associated with poorer growth, the longitudinal pulmonary progression of CF children with MI is not clear. To test the hypothesis that MI is associated with worse pulmonary outcomes, we prospectively compared from diagnosis to 12 years of age 32 CF children with MI to 50 CF children without MI who were diagnosed during early infancy through neonatal screening. Pulmonary outcome measures included respiratory symptoms, respiratory infections, pathogens, antibiotic usage, hospitalizations, quantitative chest radiology, spirometry, and lung volume determinations. Obstructive lung disease was defined as percent predicted spirometry values below the lower limits of normal. Longitudinal analyses revealed no significant differences in cough, wheezing, respiratory infections, prevalence of and median times to acquisition of Pseudomonas aeruginosa or Staphylococcus aureus, antibiotic usage, and chest radiograph scores between the two groups. However, MI children showed significantly worse forced expiratory volume in 1 sec (FEV(1)), forced vital capacity (FVC), forced expiratory flow between 25-75% of FVC (FEF(25-75)), % predicted FEV(1), % predicted FEF(25-75), and total lung capacity (TLC). These differences were particularly apparent beginning at age 8-10 years. MI children also had higher rates of and shorter median times to obstructive lung disease. Subgroup analyses showed MI children treated surgically and those treated medically had similar pulmonary outcomes. In conclusion, MI children have worse lung function and more obstructive lung disease than those without MI. Such abnormalities are accompanied by reduced lung volume. MI is a distinct CF phenotype with more severe pulmonary dysfunction.

Cognitive function of children with cystic fibrosis: deleterious effect of early malnutrition.

OBJECTIVE: Patients who have cystic fibrosis (CF) and experience delayed diagnosis by traditional methods have greater nutritional insult compared with peers diagnosed via neonatal screening. The objective of this study was to evaluate cognitive function in children with CF and the influence of both early diagnosis through neonatal screening and the potential effect of early malnutrition. METHODS: Cognitive assessment data were obtained for 89 CF patients (aged 7.3-17 years) during routine clinic visits. Patients had been enrolled in either the screened (N = 42) or traditional diagnosis (control) group (N = 47) of the Wisconsin CF Neonatal Screening Project. The Test of Cognitive Skills, Second Edition was administered to generate the Cognitive Skills Index (CSI) and cognitive factor scores (Verbal, Nonverbal, and Memory). RESULTS: Cognitive scores in the overall study population were similar to normative data (CSI mean [standard deviation]: 102.5 [16.6]; 95% confidence interval: 99.1-105.9). The mean (standard deviation) CSI scores for the screened and control groups were 104.4 (14.4) and 99.8 (18.5), respectively. Significantly lower cognitive scores correlated with indicators of malnutrition and unfavorable family factors such as single parents, lower socioeconomic status, and less parental education. Our analyses revealed lower cognitive scores in patients with low plasma alpha-tocopherol (alpha-T) levels at diagnosis. In addition, patients in the control group who also had vitamin E deficiency at diagnosis (alpha-T < 300 microg/dl) showed significantly lower CSI scores in comparison with alpha-T-sufficient control subjects and both deficient and sufficient alpha-T subsets of screened patients. CONCLUSION: Results suggest that prevention of prolonged malnutrition by early diagnosis and nutritional therapy, particularly minimizing the duration of vitamin E deficiency, is associated with better cognitive functioning in children with CF.

Longitudinal evaluation of bronchopulmonary disease in children with cystic fibrosis.

Children with cystic fibrosis (CF) develop bronchopulmonary disease at variable ages. Determining the epidemiology of chronic lung disease and quantifying its severity, however, have been difficult in infants and young children. As part of the Wisconsin CF Neonatal Screening Project, we were presented with an ideal opportunity to assess longitudinally the evolution of symptoms, signs, and quantitative measures of CF respiratory disease. After newborn screening test results led to early recognition, 64 patients diagnosed at a median age of 6.71 weeks were enrolled and studied systematically at a median age of 11.3 years to obtain clinical information, chest radiographs, and pulmonary function tests. Our observations revealed that a frequent cough by history is evident by 10.5 months of age in half the patients. Quantitative chest radiology (CXR scoring) demonstrated that potentially irreversible abnormalities are present in half the children by 2 years. The severity of Wisconsin and Brasfield CXR scores increased in association with respiratory infections. Longitudinal progression of Wisconsin CXR scores was related to age (P < 0.001), pancreatic insufficiency (P = 0.005), and respiratory secretion cultures positive for Staphylococus aureas (P = 0.039). In contrast, serial spirometry showed limited sensitivity, as did lung volume determinations; neither was satisfactory as repeated measures with acceptable quality control until after 7 years of age. Time to event analyses revealed that half the patients had % predicted FEF(25-75) and FEV(1)/FVC values greater than 80% until 10.7 and 9.9 years, respectively. We conclude that of the methods evaluated, quantitative chest radiology is currently the best procedure for frequent assessment of bronchopulmonary disease in CF, and that radiographic progression is evident in approximately 85% of patients by 5 years of age. Our results also suggest that bronchiectasis and other radiographic evidence of chronic infection are apparent prior to airways obstruction in young CF patients.

Bronchopulmonary disease in children with cystic fibrosis after early or delayed diagnosis.

Although early diagnosis of cystic fibrosis (CF) can lead to nutritional benefits, there has been uncertainty about pulmonary outcomes. Using a randomized controlled trial with unique unblinding/surveillance, we evaluated patients with CF who received similar treatment after being assigned to an early diagnosis (screened) group or to a standard diagnosis (control) group. When the youngest patient was 7 years of age, we compared outcomes using pulmonary function data and quantitative chest radiology. In the screened group (56 patients), diagnosis was made at a younger age of 12.4 weeks, compared with the diagnosis in control group (47 control patients) at the age of 95.8 weeks, but included a significantly greater proportion of patients with deltaF508 genotypes and pancreatic insufficiency. The first chest radiograph showed significantly fewer abnormalities in the screened group; but, over time, the two groups converged, and after 10 years of age the screened patients showed worse chest X-ray scores associated with earlier acquisition of Pseudomonas aeruginosa. No differences were detected in any measure of pulmonary dysfunction, which was generally mild in each group. Although CF neonatal screening provides a potential opportunity for better pulmonary outcomes, it appears that respiratory infections and pancreatic status are the dominant factors in pulmonary prognosis.

Electrogastrography in children with cystic fibrosis.

Patients with cystic fibrosis often develop upper gastrointestinal symptoms, which may be due to abnormal gastric motor function. The aim of the study is to determine the characteristics of gastric electrical activity in patients with cystic fibrosis and to compare electrogastrography patterns in symptomatic and asymptomatic patients. Electrogastrography was recorded in 14 symptomatic and 8 asymptomatic children with CF. Both 30-min baseline and 30-min postprandial recordings was obtained. Dominant frequency cycles per minute, rhythm index, and power in decibels were obtained for the fasting and postprandial periods. The percentage of normal gastric waves was not affected by the meal and was significantly low in symptomatic and asymptomatic cystic fibrosis patients. Tachygastria was the most frequent dysrhythmia in both groups. Decreased postprandial power was seen in three symptomatic patients and one patient had no change. The percentage of normal gastric slow waves was low in symptomatic and asymptomatic cystic fibrosis patients. Tachygastria was the most frequent dysrhythmia. Decreased postprandial power was seen only in symptomatic patients.