High rates of anxiety detected in mothers of children with inconclusive cystic fibrosis screening results.

OBJECTIVE: The purpose was to assess postpartum depression, anxiety, and depression in mothers of children with an inconclusive diagnosis after a positive cystic fibrosis (CF) newborn screening (NBS), known as cystic fibrosis transmembrane conductance regulator (CFTR)-related metabolic syndrome (CRMS) or CF screen positive, inconclusive diagnosis (CFSPID). There is limited information on the prognosis and on the impact of this designation on maternal mental health. METHODS: Mothers of children with CRMS/CFSPID and CF identified by NBS were recruited from two centers in California. Maternal mental health was assessed using measures of depression, anxiety, and a scripted interview. Descriptive statistics and multivariate logistic regression were applied for data reporting. RESULTS: A total of 109 mothers were recruited: CF: 51, CRMS/CFSPID: 58. Mothers from both groups showed higher rates of depression and anxiety symptoms than women in the general population. CRMS/CFSPID and CF mothers had no significant difference on their self-reported symptoms of anxiety and depression after adjusting for potential confounders. Mothers equally reported that their child's diagnosis had a negative impact, and that genetic counseling had a positive impact on their emotional health. CONCLUSIONS: CF and CRMS/CFSPID diagnoses impact maternal mental health similarly. Uncertain prognosis of CRMS/CFSPID likely contributed to the negative mental health impact. Providers should consider conducting mental health screening for every mother of a child with CRMS/CFSPID, in addition to the recommended mental health screening for mothers of children with CF. Genetic counseling has potential to mitigate emotional stress on these families.

Cystic Fibrosis Screen Positive, Inconclusive Diagnosis Genotypes in People with Cystic Fibrosis from the U.S. Patient Registry.

Rationale: Variants within the cystic fibrosis (CF) transmembrane conductance regulator gene, CFTR, that are of unknown significance or are categorized as non-CF causing may be observed in persons with CF. These variants are frequently detected in children with inconclusive newborn screen results and, in some cases, may be associated with a benign presentation in early childhood that progresses to a CF phenotype later in life. Objectives: To analyze data from individuals enrolled in the U.S. Cystic Fibrosis Foundation Patient Registry who have received a diagnosis of CF and who have variants found in a population of children with a CF screen positive, inconclusive diagnosis (CFSPID). Methods: This retrospective review analyzed registry data from individuals with a diagnosis of CF who also harbor one or more variants of interest because of their frequency within a CFSPID population and/or their interpretation as non-CF causing. Three groups were defined by the number of CF-causing variants identified (CF-Cx2, CF-Cx1, and CF-Cx0), which were reported in addition to the variant(s) of interest. Multivariate quantile regression modeling of the outcome for forced expiratory volume in 1 second (FEV1) generated a disease severity score for each person determined by six selected variables. Median scores were calculated for the three groups. Results: Patients carrying one CF-causing variant and at least one variant of interest (CF-Cx1) had higher median disease severity scores compared with those carrying CF-Cx2, suggesting a milder phenotype (P < 0.05). However, there was no statistically significant difference in scores between CF-Cx2 and the two other groups combined (CF-Cx1 and CF-Cx0; P = 0.33). Analysis revealed that the CF-Cx1 and CF-Cx0 groups, when compared with the CF-Cx2 group, had later median diagnoses (8 years vs. newborn; P < 0.0001), lower median sweat chloride (48 mmol/L vs. 94.5 mmol/L; P < 0.0001), lower prevalence of pancreatic insufficiency (29% vs. 78%; P < 0.0001), and higher median FEV1% predicted (95% vs. 87%; P = 0.0002). Conclusions: Individuals with CF who have specific variants frequently identified in children with CFSPID have a similar range of disease severity scores compared with those who have two CF-causing variants, but a milder phenotype overall. Variants that should be given careful scrutiny because of their high prevalence are G576A+R668C, T854T, R75Q, F1052V, R1070W, R31C, and L967S.

Gradual increase in sweat chloride concentration is associated with a higher risk of CRMS/CFSPID to CF reclassification.

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), with limited guidance regarding prognosis and standardized care. Rates of reclassification from CFSPID to CF vary and risk factors for reclassification are not well established. We investigated whether clinical characteristics are associated with the 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 CF transmembrane conductance regulator (CFTR) functional assessment. Multivariate logistic regression was used to assess the risk of 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 (wtCFTR) function in those who reclassified and 27%-67% of wtCFTR 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 a history of PSA colonization are associated with the risk of reclassification from CFSPID to CF in a population with high IRT and two CFTR variants. A close follow-up to monitor phenotypic changes remains critical in this population. The role of CFTR functional assays in this population requires further exploration.

Microtia Reconstruction and Erector Spinae Block in Children: A Case Series With Chart Review.

OBJECTIVES: Autologous reconstruction of microtia is advantageous due to its inherent biocompatibility and long-term stability, but postoperative pain associated with costal harvest is a significant issue. A well-planned pain management approach is imperative. Our objective is to introduce the novel application of erector spinae block anesthesia in pediatric microtia reconstruction and evaluate its impact on pain scores, use of opioids, and hospital length of stay. STUDY DESIGN: Case series with chart review. SETTING: Patients undergoing stage 1 microtia reconstruction at a tertiary pediatric hospital. METHODS: Data collected included demographics, opioid amounts, Wong-Baker FACES Pain Rating Scale scores, opioid-related side effects, and hospital length of stay. We used generalized estimating equations to examine the effect of erector spinae block use on total opioid use and pain scores and a linear regression model to assess the effect on hospital stay. RESULTS: Forty-seven patients were included: 14 in the erector spinae block group and 33 in the continuous wound pump group. The mean age was 8.3 years (SD, 2; range, 6-13), and 13 (32%) were female. Patients in the erector spinae block group had a 65.44% decrease in adjusted total opioid use (95% CI, -79.72% to -41.10%; P < .0001), a decrease in length of hospital stay (ß = -1.69 [95% CI, -2.11 to -1.26], P < .0001), and no difference in reported pain scores when compared with patients in the continuous wound pump group. CONCLUSIONS: This study demonstrates that early experience with an erector spinae block resulted in decreased opioid use and shorter hospital stay as compared with continuous wound infiltration with local anesthetic.

When CFSPID becomes CF.

There has been a growing number of infants identified as CRMS/CFSPID in countries applying genetic testing as part of cystic fibrosis (CF) newborn screening. Currently there are neither standardized protocols for follow up beyond infancy, nor established predictors to stratify this population as high or low risk of reclassification to CF or CFTR-related disorder. We report a series of 10 children who reclassified, including eight carrying CFTR variants of varying clinical consequence and seven with initial sweat chloride measurements <30 mmol/L. The overall increase in sweat chloride concentration was 5.8 mmol/L/year. Pseudomonas aeruginosa was isolated from respiratory cultures in five subjects, and reclassification was aided by human nasal epithelial cultures in two cases. In this center's experience, 6% of all CRMS/CFSPID referrals reclassified to CF over a 12-year period. The rate of sweat chloride increase, genotype, and CFTR functional assay can potentially be used as prognostic tools in the CRMS/CFSPID population.

Cerebral oxygen saturation and cerebrovascular instability in newborn infants with congenital heart disease compared to healthy controls.

OBJECTIVE: Infants with Congenital Heart Disease (CHD) are at risk for developmental delays, though the mechanisms of brain injury that impair development are unknown. Potential causes could include cerebral hypoxia and cerebrovascular instability. We hypothesized that we would detect significantly reduced cerebral oxygen saturation and greater cerebrovascular instability in CHD infants compared to the healthy controls. METHODS: We performed a secondary analysis on a sample of 43 term infants (28 CHD, 15 healthy controls) that assessed prospectively in temporal cross-section before or at 12 days of age. CHD infants were assessed prior to open-heart surgery. Cerebral oxygen saturation levels were estimated using Near-Infrared Spectroscopy, and cerebrovascular stability was assessed with the response of cerebral oxygen saturation after a postural change (supine to sitting). RESULTS: Cerebral oxygen saturation was 9 points lower in CHD than control infants in both postures (ß = -9.3; 95%CI = -17.68, -1.00; p = 0.028), even after controlling for differences in peripheral oxygen saturation. Cerebrovascular stability was significantly impaired in CHD compared to healthy infants (ß = -2.4; 95%CI = -4.12, -.61; p = 0.008), and in CHD infants with single ventricle compared with biventricular defects (ß = -1.5; 95%CI = -2.95, -0.05; p = 0.04). CONCLUSION: CHD infants had cerebral hypoxia and decreased cerebral oxygen saturation values following a postural change, suggesting cerebrovascular instability. Future longitudinal studies should assess the associations of cerebral hypoxia and cerebrovascular instability with long-term neurodevelopmental outcomes in CHD infants.

The incidence and risk factors for venous thromboembolism in adolescent and young adult oncology patients.

BACKGROUND: Venous thromboembolism (VTE) is a known complication among pediatric and adult cancer patients. Adolescent and young adult oncology (AYAO) patients have unique biological and physiological characteristics that make them distinct from other populations. Our objective was to study the VTE incidence, risk factors, and outcomes, which have been understudied in this population. PROCEDURE: A retrospective case-control study was conducted on AYAO participants with new or relapsed cancer and an imaging confirmed VTE from January 2011 to November 2016 at our institution. Eligible AYAO participants without a history of VTE were designated as controls and were randomly selected from our institution's tumor registry. Demographics, medical history, surgeries, central venous catheter (CVC) data, VTE diagnosis and treatment, relapses, and deaths were abstracted. RESULTS: Thirty-five VTE cases and 70 controls were included in this analysis. Eighty percent of cases had leukemia or lymphoma (vs. a solid tumor) compared to 58% of controls. The majority of VTEs (57%) were CVC associated, and more than 70% of cases had more than one CVC placed during their cancer treatment versus 34% of controls. Infection was associated with increased VTE risk (OR = 6.35, 95% CI = 2.30, 17.55, p < .0001). VTE cases had increased cancer relapse (23% vs. 10%) and mortality rates (29% vs. 16%) than controls. CONCLUSION: AYAO participants with a VTE were more likely to have leukemia or lymphoma, more than one CVC or infection. Further studies are needed to identify patients who would benefit from modifiable prevention measures, such as limiting to one CVC, preventing infections, or considering prophylactic anticoagulation for those with a liquid tumor.

Abnormal Lung Clearance Index in Cystic Fibrosis Children with Normal FEV1 and Single-Breath Nitrogen Washout Test.

Single- and multiple-breath washout tests (SBW and MBW) measure ventilation inhomogeneity, but the relationship between them is unclear. Forty-three subjects with cystic fibrosis (CF) and healthy controls (HC) 8-21 years of age were recruited (CF = 30 and HC = 13) and performed nitrogen MBW, vital capacity SBW, spirometry, and plethysmography. Mean phase III slope from SBW (SIII) and lung clearance index (LCI) were significantly different between CF and HC (p = 0.017 and p < 0.0001, respectively). Based on Pearson correlation, SIII and LCI showed strong correlation (ρ = 0.81, p < 0.0001). Both SIII and LCI significantly correlated with spirometry (all p < 0.05). Among CF subjects with normal FEV1 (≥ 80%; n = 17), 76% (n = 13) had normal SIII but abnormal LCI. We conclude that LCI can be abnormal despite normal SIII and FEV1 in CF children. Although LCI and SIII showed strong correlation, our results suggest that LCI is a better test to detect ventilation inhomogeneity in CF children with normal FEV1.

Prospective Evaluation of a Smartphone Otoscope for Home Tympanostomy Tube Surveillance: A Pilot Study.

OBJECTIVES: Recent technological advances have led to the capability of performing high resolution imaging of the tympanic membrane. Smartphone technologies and applications have provided the opportunity to capture digital images and easily share them. The smartphone otoscope device was developed as a simple system that can convert a smartphone into a digital otoscope. This device has the prospective ability to improve physician-patient communication and assist with the diagnosis and management of ear disease. Our objective was to evaluate the feasibility and physician/parental satisfaction using the Cellscope® smartphone attachment for at home tympanostomy tube monitoring. METHODS: Children between 6 months and 15 years of age at an urban tertiary children's hospital that were scheduled for bilateral tympanostomy tube insertion or underwent bilateral tympanostomy tube surgery were prospectively enrolled in the study. Comparisons were made between parental home-recorded videos and findings during in-office otoscopy. Two independent otolaryngologists reviewed the videos and concordance between inter-rater agreements was calculated. Acceptability and use questionnaires were administered to physicians and parents. RESULTS: There was good intra-rater agreement between traditional otoscopy and video-otoscopy for tube extruding, tube blocked and tube extruded with at least 80% agreement (P < .05) and excellent inter-rater agreement between physicians for nearly all tube variables (P < .0001) There was a high degree of satisfaction with this mode of surveillance. Parents and physicians agreed that the CellScope® smartphone was easy to use, helpful with the occurrence of acute events, and appeared to improve quality of care. CONCLUSIONS: The CellScope® smartphone is feasible for use in tympanostomy tube surveillance. Use of the device may allow otolaryngologists to easily follow a child's tympanostomy tube remotely over time and offer greater parental satisfaction.

Retrospective analysis of inpatient polysomnogram characteristics and discharge outcomes in infants with bronchopulmonary dysplasia requiring home oxygen therapy.

RATIONALE: Little is known about the polysomnogram (PSG) characteristics in infants with bronchopulmonary dysplasia (BPD), especially severe BPD, who do not need home ventilatory support but are at increased risk for chronic hypoxia and are vulnerable to its effects. OBJECTIVE: This study aims to assess PSG characteristics and change in discharge outcomes in premature infants with BPD who required oxygen therapy at discharge. METHODS: This is a retrospective chart review of premature infants with BPD who were admitted to a quaternary newborn and infant intensive care unit from January 1, 2012 to December 31, 2015 and who underwent polysomnography before discharge. MEASUREMENTS AND MAIN RESULTS: Data from 127 patients were analyzed. The median gestational age of our patients was 26 weeks and 1 day (interquartile range [IQR]: 24.71, 28.86). The majority of the patients had moderate-to-severe BPD. The median obstructive apnea-hypopnea index was 5.3 events/h (IQR: 2.2, 10.1). The median oxygen desaturation index was 15.7 events/h (IQR: 4.7, 35). Nadir oxygen saturation measured by pulse oximeter was 81% (IQR: 76-86) and the arousal/awakening index was 21.9 (IQR: 13.3-30.9). No statistically significant difference was noted between severe and nonsevere BPD groups for PSG characteristics. However, average end-tidal CO2 was significantly higher in the severe BPD group (p = .0438). Infants in the severe BPD group were intubated longer than infants with nonsevere BPD (p = .0082). The corrected gestational age (CGA) at the time of discharge (CGA-PSG) and PSG (CGA-DC) was higher in severe BPD patients but not statistically different. The majority of premature infants who underwent a PSG were discharged home with oxygen, and 69% required a titration of their level of support based on results from the PSG. CONCLUSION: Our results highlight the presence of abnormal PSG characteristics in BPD patients, as early as 43 weeks CGA. These findings have not been previously described in this patient population prior to initial discharge from the hospital. A severe BPD phenotype tends to be associated with higher respiratory morbidity compared with a nonsevere BPD phenotype for the comparable CGA. PSG, when available, may be helpful for individualizing and streamlining treatment in preparation for discharge home and mitigating the effects of intermittent hypoxic episodes.

Anesthesia for pediatric patients with anti-NMDA receptor encephalitis: A retrospective case series.

INTRODUCTION: Anti-N-methyl-D-aspartate receptor encephalitis is caused by auto-antibodies that target the N-methyl-D-aspartate receptor. Autonomic instability is a hallmark of the disease. The objective of this case series is to examine how anesthesia affects pediatric patients with this disease. METHODS: We performed a retrospective chart review of 28 records in 17 patients who underwent anesthesia. Our primary outcomes were hemodynamic changes during the perioperative period. Heart rate, systolic and diastolic blood pressures, respiratory rate, and oxygen saturation comprise our endpoints. A subgroup of patients, who underwent imaging with anesthesia, was then compared to controls. RESULTS: In anti-N-methyl-D-aspartate receptor encephalitis cases, there were significant percent changes from baseline in heart rate; median = -14.3%, 95% CI (-19.3, -9.0), p < .01 at 30 min and -15.7%, (-21.1, -9.8), p < .01 at 60 min; in systolic blood pressure, -19.4%, (-23.7, -14.8) at 30 min, p < .01, and -14.8%, (-19.7, -9.5) at 60 min, p < .01; in diastolic blood pressure, -41.9%, (-46.9, -36.3), p < .01 at 30 min, and -37.5%, (-43.4, -30.9), p < .01 at 60 min. When compared to controls, there were no significant differences between the two groups across time of anesthesia (baseline to 60 min) in heart rate (p = .24), systolic blood pressure (p = .30), and diastolic blood pressure (p = .11). No patients experienced hemodynamic lability under anesthesia. One patient, with severe symptoms, died within 24 h of anesthesia. CONCLUSION: Although pediatric patients with anti-N-methyl-D-aspartate receptor encephalitis experienced vital sign changes with anesthesia, they were not clinically significant and they behaved similarly to controls. Disease severity may be a risk factor for perioperative complications.

Image-based ß-adrenergic sweat rate assay captures minimal cystic fibrosis transmembrane conductance regulator function.

BACKGROUND: There is a need to prognosticate the severity of cystic fibrosis (CF) detected by newborn screening (NBS) by early assessment of CF transmembrane conductance regulator (CFTR) protein function. We introduce novel instrumentation and protocol for evaluating CFTR activity as reflected by ß-adrenergically stimulated sweat secretion. METHODS: A pixilated image sensor detects sweat rates. Compounds necessary for maximum sweat gland stimulation are applied by iontophoresis, replacing ID injections. Results are compared to a validated ß-adrenergic assay that measures sweat secretion by evaporation (evaporimetry). RESULTS: Ten healthy controls (HC), 6 heterozygous (carriers), 5 with CFTR-related metabolic syndrome (CRMS)/CF screen-positive, inconclusive diagnosis (CFSPID), and 12 CF individuals completed testing. All individuals with minimal and residual function CFTR mutations had low ratios of ß-adrenergically stimulated sweat rate to cholinergically stimulated sweat rate (ß/chol) as measured by either assay. CONCLUSIONS: ß-Adrenergic assays quantitate CFTR dysfunction in the secretory pathway of sweat glands in CF and CRMS/CFSPID populations. This novel image-sensor and iontophoresis protocol detect CFTR function with minimal and residual function and is a feasible test for young children because it is insensible to movement and it decreases the number of injections. It may also assist to distinguish between CF and CRMS/CFSPID diagnosis.

Abnormal Lung Clearance Index in Cystic Fibrosis Screen Positive, Inconclusive Diagnosis (CFSPID) Children with Otherwise Normal FEV1.

In preschool children with cystic fibrosis (CF), lung clearance index (LCI) is a sensitive test to detect early lung disease. Some children with CF screen positive, inconclusive diagnosis (CFSPID) may in time develop clinical features of CF. LCI has not been studied in CFSPID children. LCI and spirometry were performed in preschool age children with CF, CFSPID, and non-CF healthy controls (HCs) during two visits. Fifty-four preschool age children (HC n = 18, CFSPID n = 17, and CF n = 19) were tested. Mean LCI from the CFSPID group was not statistically different from HC (p = 0.49), but significantly different when compared to CF (p = 0.04). LCI was abnormal in 2 CFSPID children who carried potentially deleterious CFTR variants. Mean forced expiratory volume in 1 s (FEV1) was not statistically different between CFSPID and CF (p = 0.26). LCI can potentially detect early lung disease in CFSPID individuals as part of assessing their risk for reclassification to CF diagnosis.

Bacteremia in nonneutropenic pediatric oncology patients with central venous catheters in the ED.

OBJECTIVE: To examine clinical characteristics associated with bacteremia in febrile nonneutropenic pediatric oncology patients with central venous catheters (CVCs) in the emergency department (ED). BACKGROUND: Fever is the primary reason pediatric oncology patients present to the ED. The literature states that 0.9% to 39% of febrile nonneutropenic oncology patients are bacteremic, yet few studies have investigated infectious risk factors in this population. METHODS: This was a retrospective cohort study in a pediatric ED, reviewing medical records from 2002 to 2014. Inclusion criteria were patients with cancer, temperature at least 38°C, presence of a CVC, absolute neutrophil count greater than 500 cells/µL, and age less than 22 years. Exclusion criteria were repeat ED visits within 72 hours, bloodwork results not reported by the laboratory, and patients without oncologic history documented at the study hospital. The primary outcome measure is a positive blood culture (+BC). Other variables include age, sex, CVC type, cancer diagnosis, absolute neutrophil count, vital signs, upper respiratory infection (URI) symptoms, and amount of intravenous (IV) normal saline (NS) administered in the ED. Data were analyzed using descriptive statistics and a multiple logistic regression model. RESULTS: A total of 1322 ED visits were sampled, with 534 enrolled, and 39 visits had +BC (7.3%). Variables associated with an increased risk of +BC included the following: absence of URI symptoms (odds ratio [OR], 2.30; 95% CI, 1.13-4.69), neuroblastoma (OR, 3.65; 95% CI, 1.47-9.09), "other" cancer diagnosis (OR, 4.56; 95% CI, 1.93-10.76), tunneled externalized CVC (OR, 5.04; 95% CI, 2.25-11.28), and receiving at least 20 mL/kg IV NS (OR, 2.34; 95% CI, 1.2-4.55). The results of a multiple logistic regression model also showed these variables to be associated with +BC. CONCLUSION: The absence of URI symptoms, presence of an externalized CVC, neuroblastoma or other cancer diagnosis, and receiving at least 20 mL/kg IV NS in the ED are associated with increased risk of bacteremia in nonneutropenic pediatric oncology patients with a CVC.

Pulmonary function abnormalities in childhood cancer survivors treated with bleomycin.

BACKGROUND: Bleomycin is associated with pulmonary toxic side effects including pneumonitis and pulmonary fibrosis. We evaluated the prevalence of long-term pulmonary function abnormalities in children receiving bleomycin therapy in the context of current chemotherapeutic regimens. METHODS: A retrospective review of patients who received bleomycin between January 1999 and December 2011 was conducted. Abnormalities in the most recent pulmonary function test (PFT) at least 1 year after diagnosis were analyzed. RESULTS: Two-hundred and seven patients had received bleomycin. The results of PFT performed at least 1 year from diagnosis were available for 80 patients. Median time of follow up was 3.9 years (range 1.1-11.76 years). Median cumulative dose of bleomycin was 65 IU/m(2) (range 10-120). The most common diagnoses were Hodgkin lymphoma and germ cell tumor. At least one pulmonary function abnormality was present in 42 (52.5%) patients. When classified in groups, 22.5% patients had obstructive lung disease, 7.5% had restrictive lung disease, 28.8% had hyperinflation and 14% of patients had non-uniform distribution of ventilation. Non-Hispanic patients (OR 2.81) and children younger than 8 years (OR 4.14) had higher odds of having an abnormal PFT parameter. Very few patients had pulmonary symptoms. CONCLUSIONS: More than half the patients who received bleomycin had subclinical pulmonary dysfunction as evidenced by abnormalities in pulmonary function tests, although the incidence of clinical symptoms was low.

A longitudinal assessment of lipids in youth with type 1 diabetes.

BACKGROUND: Type 1 diabetes and dyslipidemia are known risk factors for cardiovascular disease (CVD), but the relationship between lipid levels in youth with type 1 diabetes and future CVD remains unknown. OBJECTIVE: To characterize lipid levels and CVD risk factors over time in youth with type 1 diabetes. SUBJECTS: The study included adolescents with type 1 diabetes (12-25 yr) with a minimum of 3-yr follow-up. METHODS: A longitudinal prospective, observational study of 46 youth with type 1 diabetes was performed. Fasting lipid profiles, A1C, and body mass index (BMI) were measured every 6 months for at least 3 yr (median 4.2 yr). Low-density lipoprotein (LDL)-cholesterol, total cholesterol (TC), and triglycerides (TG) were divided into categorical variables. RESULTS: At baseline, median age was 14.3 yr, mean diabetes duration was 6.4 ± 3.8 yr, mean A1C was 8.1 ± 1.0%, and median BMI z-score was 0.92. Fifty percent of subjects had LDL levels ≤ 100 mg/dL (≤ 2.6 mmol/L) at study onset. After adjusting for confounding factors, increasing BMI z-score [ß = 0.2, 95% confidence interval (CI ) = 0.03-0.38, p = 0.03] and increasing A1C (ß = 0.18, 95% CI = 0.08-0.29, p = 0.001) were associated with increasing LDL category over time. Non-Hispanic ethnicity (ß = 0.45, 95% CI = 0.12-0.79, p = 0.008) and family history of stroke (ß = 0.38, 95% CI = 0.04-0.72, p = 0.03) were also associated with increasing LDL category. Age, diabetes duration, and tobacco exposure were not related to change in LDL. Increasing A1C was associated with increases in TG (ß = 18.1, 95% CI = 2.3-33.9, p = 0.03), TC (ß = 20.3, 95% CI = 9.0-31.5, p < 0.0001), and LDL (ß = 13.4, 95% CI = 3.17- 23.6, p = 0.01). CONCLUSION: Glycemic control and BMI are modifiable risk factors for dyslipidemia in youth with type 1 diabetes.

Outcome analysis of neonates with congenital diaphragmatic hernia treated with venovenous vs venoarterial extracorporeal membrane oxygenation.

PURPOSE: Venoarterial extracorporeal membrane oxygenation (ECMO) (VA) is used more commonly in neonates with congenital diaphragmatic hernia (CDH) than venovenous ECMO (VV). We hypothesized that VV may result in comparable outcomes in infants with CDH requiring ECMO. METHODS: We retrospectively analyzed the Extracorporeal Life Support Organization (ELSO) database (1991-2006). Multivariate logistic regression analyses were used to compare VV- and VA-associated mortality. RESULTS: Four thousand one hundred fifteen neonates required ECMO, with an overall mortality rate of 49.6%. Venoarterial ECMO was used in 82% and VV in 18% of neonates. Pre-ECMO inotrope use and complications were equivalent between VA and VV. The mortality rate for VA and VV was 50% and 46%, respectively. After adjusting for birth weight, gestational age, prenatal diagnosis, ethnicity, Apgar scores, pH less than 7.20, Paco(2) greater than 50, requiring high-frequency ventilation, and year of ECMO, there was no difference in mortality between VV vs VA. Renal complications and on-ECMO inotrope use were more common with VV, whereas neurologic complications were more common with VA. The conversion rate from VV to VA was 18%; conversion was associated with a 56% mortality rate. CONCLUSION: The short-term outcomes of VV and VA are comparable. Patients with CDH who fail VV may be predisposed to a worse outcome. Nevertheless, VV offers equal benefit to patients with CDH requiring ECMO while preserving the native carotid.

State-based analysis of necrotizing enterocolitis outcomes.

BACKGROUND: Necrotizing enterocolitis (NEC) is a devastating disease of the newborn. We hypothesized that patient and institution level factors lead to NEC-related outcome disparities. METHODS: We analyzed the California Office of Statewide Health Planning and Development database for the years 1999-2004. We selected NEC-specific ICD-9-CM diagnosis and procedure codes. Mortality rate was the primary outcome measure, and length of stay was used a secondary end-point. We stratified the data by birth weight, gender, race/ethnicity, treatment, median household income, insurance status, admission type (inborn or outborn), and NICU levels. RESULTS: We identified 3328 infants with NEC (incidence of 1 per 1000 live births). Overall mortality within the NEC cohort was 12.5% (13.4 deaths per 100,000 live births). Male or Hispanic neonates were less likely to survive. Socioeconomic factors, including insurance status and parental median household income, were not predictors of mortality. Neonates treated surgically had a greater mortality rate compared with ones treated nonsurgically. PDA was present in 32% of patients with NEC, and these neonates were more likely to undergo gastrointestinal surgery. The odds of NEC-associated mortality in level IIIC units were significantly greater than any other NICU level. Admission type (inborn versus outborn) was not associated with increased mortality. CONCLUSIONS: Disparities in NEC outcomes are multifactorial with patient- and treatment-specific factors contributing significantly to the unfavorable outcomes. These data suggest that advances in prediction modeling, prevention, and treatment algorithms are needed for clinicians and state health planners to positively impact this costly neonatal condition.