Patient Characteristics and Caregiver Asthma Knowledge of Children with Well-Controlled and Poorly Controlled Asthma.

Purpose: To describe the socio-demographics and clinical characteristics of children in a pulmonology clinic or admitted to a children's hospital with well-controlled and poorly controlled asthma, and to assess caregiver knowledge of asthma pathogenesis, treatment, and self-management. Patients and Methods: A cohort of 132 children aged 2-18 years and their caregivers seen in a pediatric pulmonology clinic with a diagnosis of asthma (n=112) or admitted to the hospital with a diagnosis of asthma exacerbation (n=20) were invited to participate in a cross-sectional study. Caregivers completed a survey, which healthcare providers then used to tailor asthma education to the patient and caregiver. Two-tail t-tests and Chi-square tests were used to compare demographics and clinical characteristics of children with well-controlled vs poorly controlled asthma. Results: Of 132 children, 111 children in this cohort had poorly controlled asthma (84%). Medicaid insurance was associated with poorly controlled asthma versus well-controlled asthma (63% vs 35% p=0.01). Asthma action plans (AAP) had previously been given to 113 caregivers (86%), but caregivers of children with both well-controlled and poorly controlled asthma still reported misconceptions about asthma pathology and management, such as stopping daily medications when asthma is controlled. Conclusion: This study contributes to the existing evidence that socio-demographics have a significant impact on asthma prevalence and proper management. Our study suggests that caregivers of children with asthma need comprehensive asthma education beyond the AAP focusing on asthma-related misconceptions.

Electrical status epilepticus during sleep in a child with Prader-Willi syndrome: a case report.

Prader-Willi syndrome (PWS) is a rare and complex genomic imprinting disorder caused by an absence of expression of paternal genes from chromosome 15q11.2-q13. Clinical manifestations of PWS depends on age. In early infancy, PWS patients is characterized by hypotonia and failure to thrive. Later in life, they can also exhibit hyperphagia, obesity, short stature, hypogonadism, behavioral issues and cognitive disability. Multiple sleep abnormalities including obstructive and/or central sleep apnea, daytime hypersomnolence, and impaired responses to hypercapnia and hypoxia have been described in patients with PWS. Recent studies also demonstrated an increased risk of seizures in PWS patients. Electrical status epilepticus in sleep (ESES) is an age-limited epilepsy with various seizure types, neurophysiological and motor impairment. The classic electroencephalogram (EEG) pattern of ESES involves continuous epileptic activity at 2-3 Hz occupying greater than 85% of non-rapid eye movement (REM) sleep. Treatment of the ESES syndrome consists of anti-epileptic drugs in routine cases, and corticosteroids, gamma globulins, the ketogenic diet, and surgery in refractory cases. In this project, we describe ESES during polysomnography in a 5-year-old female with PWS and no history of seizure disorder. To the best of our knowledge, this is the first case report on ESES in a PWS patient.

Catathrenia and Treatment With Positive Airway Pressure in the Pediatric Population.

None: Catathrenia, also known as sleep-related groaning, is a relatively rare sleep disorder with characteristics consistent with loud groaning on expiration during sleep. Organic causes of catathrenia are unknown and the decision of whether or not to treat is unclear. Limited research is available concerning appropriate treatment and none of the literature focuses primarily on pediatrics. We report a series of three, male pediatric cases with catathrenia that were treated with continuous positive airway pressure (CPAP) while identifying and comparing clinical symptoms, polysomnogram findings, treatment, and patient response to treatment. Catathrenia may be associated with abnormal nocturnal oxygenation and ventilation and may lead to negative clinical daytime symptoms which may warrant treatment. If catathrenia leads to sleep disruption and negative daytime symptoms, treatment with CPAP should be considered.

Outcomes and Safety of Outpatient Parenteral Antimicrobial Therapy in Select Children with Cystic Fibrosis.

Background: Pulmonary exacerbations (PExs) are common in individuals with cystic fibrosis (CF). Data regarding outcomes of outpatient parenteral antimicrobial therapy (OPAT) in children are sparse. Methods: Retrospective data of PEx episodes treated in the hospital versus OPAT collected. Children ≤18 years were included. Outcome measures included FEV1, FVC, FEF25-75%P, time to the next PEx, and weight gain. Results: Eighty-three subjects with 290 PEx events were eligible. The hospital group had 242 and the OPAT group had 48 PEx events. The median age was 13.1 years for the OPAT and 13.4 years for the hospital group. Medicaid coverage was higher in the hospital group (82.2%) versus OPAT group (48.9%, P < 0.0001). The hospital group had lower FEV1%P on admission [72%P (interquartile range [IQR] = 59.7 and 84) versus 80%P (IQR = 70.7 and 89); P = 0.001] and at the end of treatment [86%P (IQR = 72 and 96.7) versus 92%P (IQR = 82 and 101); P = 0.003] in comparison with OPAT group. FEV1%P improved more in the hospital group, [12%P (IQR = 4 and 20)] versus in the OPAT group [8%P (IQR = 2 and 22.5); (P = 0.41)] but did not quite reach a statistically significant level. The hospital intravenous (IV) group gained more weight (P < 0.0001). There was no difference between the 2 groups in time to the first PEx (P = 0.47) and adverse events. Conclusion: OPAT was safe and comparable with hospital therapy in a select group of children with CF. Hospital IV should be considered for sicker children and families with limited resources.

Impact of Degree of Obesity on Sleep, Quality of Life, and Depression in Youth.

OBJECTIVE: Obese youth are more likely to report difficulties with sleep, depression, and quality of life (QOL). This study aims to characterize sleep problems, QOL, and symptoms of depression by degree of obesity. METHOD: The cross-sectional study was conducted in a specialized obesity clinic. Obese youth and their caregivers (N = 150) were evaluated with the Child Sleep Habits Questionnaire and Pediatric Quality of Life. Youth completed the Children's Depression Inventory. Regression models and correlations were calculated. RESULTS: Degree of obesity was predictive of increased sleep difficulties and decreased QOL scores. Children's Depression Inventory scores showed that children with more symptoms of depression had more sleep problems, and these were not associated with the degree of obesity. Adolescents with more difficulties sleeping also reported more symptoms of depression and lower QOL. CONCLUSIONS: Degree of obesity negatively affected QOL and sleep variables. Patients with greater sleeping difficulties reported more symptoms of depression.

Peripheral quantitative computed tomography detects differences at the radius in prepubertal children with cystic fibrosis compared to healthy controls.

INTRODUCTION: In 2015, 11.9% of people with cystic fibrosis (CF) in the United States had osteopenia, 5.1% osteoporosis, and 0.3% experienced a fracture. Screening for CF-related bone disease starts in childhood, and dual energy x-ray absorptiometry (DXA) is the recommended method. It is unknown whether peripheral quantitative computed tomography (pQCT) can detect deficits earlier than DXA. This study compared pQCT and DXA scans in a group of pre-pubertal children with CF and healthy controls. METHODS: This was a cross-sectional study of children at Tanner stage 1. A pQCT scan of the radius at proximal and distal sites was performed plus a total body DXA scan. Serum C-reactive protein, interleukin-6 and tumor necrosis factor-alpha were also measured. RESULTS: A total of 34 subjects completed the study; 14 with CF and 20 controls. At the distal radius, pQCT showed a lower total bone mineral density (BMD) Z-score for the CF group (P = 0.01 and P = 0.03 for 2 different reference databases) compared to controls. At the proximal site, the polar strength-strain index was lower in the CF group (P = 0.017). Finally, the total body BMD Z-score by DXA was lower in the CF group, although it did not meet the definition of reduced bone density (P = 0.004). Biomarkers of inflammation were not different. CONCLUSIONS: In this group of pre-pubertal children with CF, measures of bone strength and density by both pQCT and DXA were reduced compared to healthy controls.

Delayed Appearance of Cutaneous Lesions of Cutaneovisceral Angiomatosis (CAT) Leading to Misdiagnosis of Immune Thrombocytopenia.

Cutaneovisceral angiomatosis with thrombocytopenia (CAT), also called multifocal lymphangioendotheliomatosis with thrombocytopenia (MLT), is a rare and newly described vascular malformation. Skin manifestations and thrombocytopenia are the hallmark of CAT/MLT, and visceral lesions are described. We report an infant with pulmonary hemorrhage, thrombocytopenia, and antiplatelet antibodies. There was no cutaneous involvement and the child was initially diagnosed with immune thrombocytopenia. Poor response to immune thrombocytopenia-directed therapy raised suspicion for an alternative diagnosis, and the ultimate diagnosis of CAT/MLT was made by lung tissue sampling. Unexpectedly, 2 years after resolution of pulmonary lesions and thrombocytopenia, the child developed typical cutaneous lesions.

Clinical characteristics and epidemiology of methicillin-resistant Staphylococcus aureus (MRSA) in children with cystic fibrosis from a center with a high MRSA prevalence.

BACKGROUND: We describe the clinical characteristics and epidemiology of methicillin-resistant Staphylococcus aureus (MRSA) in children with cystic fibrosis (CF) from the U.S. CF center with the highest MRSA prevalence. METHODS: Medical records of children with CF were retrospectively reviewed from 1997-2009. MRSA clinical isolates from 2007-2009 were analyzed by polymerase chain reaction and pulsed field gel electrophoresis. RESULTS: The prevalence of MRSA was 1% in 1997 and 49% in 2009. Fifty-five children (26%) had persistent MRSA infection. Sixty-eight percent of MRSA isolates were hospital-associated (HA) MRSA, of which 52% were pulsed-field type USA 100. Ninety-three percent of HA MRSA isolates were clindamycin resistant. Twelve children acquired MRSA before 1 year of age, 83% of whom were hospitalized prior to acquisition of MRSA. Ten of 11 sibling pairs carried indistinguishable MRSA strains. Children with persistent MRSA were hospitalized more often (P = .01), required inhaled medications more frequently (P = .01), and had higher rates of Pseudomonas aeruginosa coinfection (P < .001). CONCLUSION: MRSA prevalence in children with CF is increasing, and most children are infected with HA MRSA. Exposure to health care facilities and gastrointestinal surgeries may facilitate early acquisition of MRSA. Siblings carry indistinguishable MRSA strains, indicating household transmission of MRSA. Children with persistent MRSA had worse pulmonary morbidity. Coinfection with MRSA and P aeruginosa is likely associated with further increased pulmonary morbidity.

Transition of respiratory technology dependent patients from pediatric to adult pulmonology care.

OBJECTIVE: Recent advances in medicine have allowed children with chronic life-threatening disorders to survive longer than ever before with the use of complex medical device technology (e.g., mechanical ventilation, dialysis, etc.). The care of children with chronic pulmonary disorders and respiratory-technology dependence is often complex, involving a high level of ongoing interaction between caregivers and the health care team. Unmanaged, non-standardized transition of respiratory technology dependent (RTD) patients to adult care potentially increases the risk of adverse outcomes. Pediatric Pulmonary programs at US children's hospitals were surveyed to ascertain whether a standardized process is utilized for transitioning RTD patients from pediatric to adult subspecialty pulmonology care. METHODOLOGY: Pediatric pulmonology programs with Accreditation Council for Graduate Medical Education certification were invited to participate in an electronic survey inquiring about practices and processes used to transition RTD patients from pediatric to adult pulmonology. RESULTS: The majority of respondents, 78.1% (25/32), reported that they do not utilize a standard protocol for transition while 41.4% (12/29) have no process in place. No program surveyed uses a designated transition leader. Referral to an adult pulmonologist within the same health system occurs more frequently than referral to private practice. Forty-three percent are not satisfied with involvement from the adult pulmonology care team. Coordination of care with other specialty services such as adult otolaryngology is provided by 31% of respondents. Of respondents, 13.8% assessed "readiness to transition" to adult pulmonary for RTD patients. Pediatric pulmonary providers are not satisfied with their current practices or involvement from the adult team, and only 24% track the transition process until the first visit with the adult pulmonologist. CONCLUSION: The survey results highlight a lack of standardized transition programs at US children's hospitals for the transfer of RTD patients from a pediatric to an adult care setting. Improvement in the standardized management of transitions of complex RTD patients from pediatric to adult care may decrease the risk for adverse health outcomes and the stresses associated with changing the health care setting.

Characteristics and surgical and clinical outcomes of severely obese children with obstructive sleep apnea.

STUDY OBJECTIVES: To describe characteristics and surgical and clinical outcomes of obese children with obstructive sleep apnea (OSA). METHODS: At our institution from 2000 to 2010, 143 obese children with an overnight polysomnography (OPSG) diagnosis of OSA, excluding children with comorbidities, were identified. Relationships between demographics, clinical findings, and the severity of OSA were assessed. Presurgery and postsurgery OPSG indices were compared. We defined cure as an apneahypopnea index (AHI) < 1.5/h on the postsurgery OPSG, and we compared the cure rates of different surgeries. RESULTS: A total of 143 children, median age 12.4 y (interquartile range [IQR] 9.6-14.9) and BMI z-scores 2.8 (IQR 2.6-2.9), were included. Seventy-eight (55%) (Median age 12 y [IQR 9-15]) underwent surgery: 1 had tonsillectomy; 1 tonsillectomy + uvulopharyngopalatoplasty (UPPP); 23 adenotonsillectomy (AT); 27 AT + UPPP; 11 adenoidectomy + UPPP; 8 UPPP; and 7 AT + turbinate trim ± tongue base suspension. Overall, surgery cured 19 children (26%), but AHI improved in the majority of children (p = 0.001). Similarly, the arousal index, PETCO2, and SpO2 nadir improved significantly (p < 0.002, p = 0.019, p < 0.001, respectively). AHI improved significantly in children with mild-to-moderate OSA in comparison to severe OSA (p < 0.001). Children with enlarged tonsils and no history of prior surgery benefitted more often from surgery (p < 0.004 and p = 0.002, respectively). AT was the only surgery reducing the AHI significantly (p = 0.008). Children did not lose weight despite intervention. Adherence with PAP was poor. CONCLUSIONS: Surgery improved OPSG indices in the majority of obese children with OSA.

Protein is an important but undervalued macronutrient in the nutritional care of patients with cystic fibrosis.

PURPOSE OF REVIEW: Achieving an optimal nutritional status in patients with cystic fibrosis (CF) is important to maintain better pulmonary function, physical performance, and to prolong survival. So far, nutrition care in CF has mainly focused on fat intake and achieving energy balance, but there is increasing information on the clinical importance of muscle maintenance and protein intake in CF. RECENT FINDINGS: Low muscle mass is consistently associated with pulmonary decline, bone mineral loss, and poor survival in CF. High energy and fat intake contributes to an increased prevalence of overweight and obesity in CF. Obesity per se is not related to better lung function in CF and has negative metabolic and clinical consequences, particularly when muscle wasting is present. Protein digestion capacity is severely impaired in CF, but high protein intake is needed and essential amino acid mixtures appear highly anabolic even in those with severe muscle loss. SUMMARY: Body composition assessment and achieving protein balance in the routine care in CF is important to prevent muscle loss and further improve the clinical and overall outcome of these patients. New approaches are needed to optimize the interaction between high essential amino-acid-rich protein intake and pancreatic enzyme regimen in CF. The optimal level of protein intake needs to be assessed in clinically stable CF patients as well as in those recovering from an acute exacerbation.

Assessment and treatment of obstructive sleep-disordered breathing.

Obstructive sleep-disordered breathing (OSDB) is a condition that affects 1% to 3% of the pediatric population. These disorders are difficult to diagnosis and left untreated may be serious, including not only medical comorbidities but also cognitive, academic, behavioral, and emotional sequelae. This article is designed to bring awareness of the severity and prevalence to family physicians and pediatricians. It reviews detailed information concerning OSDB, including the predisposing factors, assessment of presenting features, and treatment.

Predictors and outcome of low initial forced expiratory volume in 1 second measurement in children with cystic fibrosis.

OBJECTIVE: To identify the characteristics of children with cystic fibrosis with low initial forced expiratory volume in 1 second (FEV1) % predicted and to investigate their outcome. STUDY DESIGN: Patients were categorized into low or high initial FEV1 groups using cluster analysis. Comparisons of the demographic and clinical data were performed between the 2 groups. RESULTS: From 122 children, 21 clustered into the low and 101 into the high FEV1 group. The mean FEV1 was 69% ± 12% predicted for the low and 95% ± 12% predicted for the high FEV1 group (P < .001). The low FEV1 group had lower body mass index percentiles (P = .003), were hospitalized more frequently (P = .001), and had been on dornase alfa longer (P = .006). Low FEV1 group had more patients with positive cultures for Pseudomonas aeruginosa (P = .002) and Stenotrophomonas maltophilia (P < .001) and had more total number of cultures positive for mucoid P. aeruginosa (P = .009) and methicillin resistant Staphylococcus aureus + P. aeruginosa (P = .005). The low FEV1 group continued to have low FEV1 measurements, their FEV1 declined slower, required more hospitalizations per year (P = .01), and had more cultures for mucoid (P = .003) and nonmucoid P. aeruginosa (P = .02) ± methicillin resistant S. aureus (P = .002) in comparison with the high FEV1 group. Poor adherence was associated with lower initial FEV1 values in females, and early, rapid decline of FEV1 in males. CONCLUSIONS: Some children with cystic fibrosis may present with poor lung function early in life and continue to have subnormal lung function associated with reduced body mass index, more frequent hospitalization, and higher rates of infection. Such children may benefit from careful evaluation and close follow-up.

Role of a respiratory therapist in improving adherence to positive airway pressure treatment in a pediatric sleep apnea clinic.

BACKGROUND: Many pediatric patients need positive airway pressure (PAP) for treatment of obstructive sleep-disordered breathing. Adherence to PAP (defined as percent of nights with PAP use of > 4 h) is often poor and not sustained long-term. With any chronic disease, education has been shown to help with patient outcomes. Education of patients and parents regarding PAP can be provided by different healthcare professionals. There is no published literature assessing the role of respiratory therapists (RTs) in improving adherence to PAP in children. We hypothesized that the addition of RT visits to a PAP clinic would improve PAP adherence. METHODS: RT services for PAP patients were introduced in a multidisciplinary pediatric sleep clinic in May 2006. We identified children who had been followed in clinic, and had adherence download information before and after introduction of RT services. We collected demographic, polysomnography, and CPAP adherence data at clinic visits. RESULTS: Forty-six subjects met criteria for inclusion. The mean ± SD age was 14.9 ± 6 y. The mean ± SD apnea-hypopnea index was 26.7 ± 30 events/h. Other than the addition of the RT intervention, all subjects continued to receive the same clinical services as before. Subjects were divided into 3 groups, based on baseline adherence: 0% use, use for 1-50% of nights, and use for > 50% of nights. There was a statistically significant improvement in PAP adherence in the subjects with baseline use of 0% and 1-50%, but no improvement in those with > 50% use at baseline. There was no significant change in PAP use at subsequent RT visits. CONCLUSIONS: Utilization at clinic visits of an RT trained in the use of PAP improved adherence in pediatric subjects with obstructive sleep-disordered breathing when their baseline PAP adherence was < 50%.

Stimulated nitric oxide production and arginine deficiency in children with cystic fibrosis with nutritional failure.

OBJECTIVE: To determine whether upregulated whole body de novo arginine synthesis and protein breakdown are present as a compensatory mechanism to meet the increased demand for arginine and nitric oxide (NO) production in pediatric patients with cystic fibrosis (CF) and nutritional failure. STUDY DESIGN: In 16 children with CF, studied at the end of antibiotic treatment for a pulmonary exacerbation, and 17 healthy controls, whole body arginine, citrulline (Cit), and protein turnover were assessed by stable isotope methodology and de novo arginine synthesis, arginine clearance, NO synthesis, protein synthesis and breakdown, and net protein balance were calculated. The plasma isotopic enrichments and amino acid concentrations were measured by liquid chromatography-tandem mass spectrometry. RESULTS: Increased arginine clearance was found in patients with CF (P < .001), whereas whole body NO production rate and plasma arginine levels were not different. Whole body arginine production (P < .001), de novo arginine synthesis, and protein breakdown and synthesis (P < .05) were increased in patients with CF, but net protein balance was comparable. Patients with CF with nutritional failure (n = 7) had significantly higher NO production (P < .05), de novo arginine synthesis, Cit production (P < .001), and plasma Cit concentration (P < .05) and lower plasma arginine concentration (P < .05) than those without nutritional failure (n = 9). CONCLUSIONS: Nutritional failure in CF is associated with increased NO production. However, up-regulation of de novo arginine synthesis and Cit production was not sufficient to meet the increased arginine needs leading to arginine deficiency.

Dietary essential amino acids are highly anabolic in pediatric patients with cystic fibrosis.

BACKGROUND: Current nutritional approaches have been partially successful in Cystic Fibrosis (CF). Essential amino acids mixtures with high Leucine levels (EAA) have anabolic properties in catabolic conditions, however data in CF are lacking. METHODS: On two days according a randomized crossover design, 15 pediatric CF patients ingested 6.7 g EAA versus mixture of total amino acids as present in whey. Whole body protein and Arginine metabolism (as EAA lack Arginine) were assessed by stable isotope methodology. RESULTS: Protein synthesis (P<0.05) but not protein breakdown was higher after EAA and 70% higher values for net anabolism (P<0.001)were found both in patients with and without nutritional failure. Arginine turnover was lower (P<0.001) and de novo Arginine synthesis tended lower (P=0.09) after EAA. Nitric oxide synthesis was not different. CONCLUSIONS: CF patients are highly responsive to EAA intake independent of their nutritional status. Addition of Arginine to the EAA mixture may be warranted in CF.

A survey of nutrition practices for patients with cystic fibrosis.

BACKGROUND: Cystic fibrosis (CF) affects approximately 30,000 people in the United States and is characterized by pulmonary disease and poor nutrition. Optimal nutrition is associated with better pulmonary function, and aggressive nutrition interventions have played a major role in increased survival. Even though the CF Foundation (CFF) has published treatment guidelines for certain areas of nutrition care, there may still be variability in other areas. Thus, the purpose of this study was to survey current practices among CF dietitians. MATERIALS AND METHODS: A 25-item web-based survey was developed and sent to nutrition professionals via the CFF nutrition listserv. Responses were anonymous. Areas of practice surveyed included nutrition care for infants, pancreatic enzyme replacement, vitamins and bone health, and miscellaneous practices. Descriptive statistics were used to report the results. RESULTS: Among the 76 respondents (21% response rate), there was general agreement regarding nutrition practices for infants and pancreatic enzyme replacement. Responses varied regarding enzyme administration with gastrostomy tube feeds and in practices related to bone health. Of miscellaneous practices, there was agreement regarding zinc supplementation and appetite stimulants. However, opinions varied regarding concern for coronary artery disease with the CF high-fat diet. Practices related to use of acid blockers also varied. CONCLUSION: The results of this survey demonstrate good agreement among nutrition professionals in some areas and variability in others for the nutrition management of patients with CF. Areas of variability point to the need for further research and continued additions to the guidelines and consensus statements.

Outcomes of children treated with tracheostomy and positive-pressure ventilation at home.

BACKGROUND: Long-term outcomes for children who survive on tracheostomy and positive-pressure ventilation (TPPV) at home are not well known. METHODS: A retrospective review of 20 years of clinical data at a single institution was performed. Outcome measures included 5-year survival, decannulation rate, and neurocognition. RESULTS: A total of 91 children were categorized under neuromotor dysfunction (52%), chronic lung disease (29%), and congenital anomalies (20%). The 5-year survival rates for these categories were 89% (95% confidence interval [CI] = 80%-99%), 76% (95% CI = 57%-100%), and 94% (95% CI = 83%-100%), respectively. Overall, the 5-year decannulation rate was 25% (95% CI = 14%-35%), with children with chronic lung disease having the highest rate (51%). It was found that 14% were extremely delayed in neurocognition. CONCLUSION: Most children on TPPV at home survive beyond 5 years, and a significant number are decannulated. Primary care physicians and communities should be prepared to accommodate the increasing number of children on TPPV at home.