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Objective Guidelines for methotrexate (MTX) use in rheumatic disease may not be feasible for physicians practicing in the least developed countries. We aimed to understand the experiences of MTX prescribers relating to MTX use for rheumatic disease in African countries to inform the development of culturally and geographically appropriate recommendations. Methods African physicians who self‐identified as MTX prescribers from countries classified as having a low versus a medium or high Human Development Index (L‐HDI versus MH‐HDI) participated in semistructured interviews between August 2016 and September 2017. Interviews were transcribed verbatim, coded thematically, and stratified by HDI. Results Physicians (23 rheumatologists; six internists) from 29 African countries were interviewed (15 L‐HDI; 14 MH‐HDI). Identified barriers to MTX use included inconsistent MTX supply (reported by 87% L‐HDI versus 43% MH‐HDI), compounded by financial restrictions (reported by 93% L‐HDI versus 64% MH‐HDI), patient hesitancy based partly on cultural beliefs and societal roles (reported by 71%), few prescribers (reported by 33%), prevalent infections (especially viral hepatitis, tuberculosis, and human immunodeficiency virus), and both availability and cost of monitoring tests. MTX pretreatment evaluation and starting and maximal doses were similar between L‐HDI countries and MH‐HDI countries. Conclusion The challenges of treating rheumatic disease in African countries include unreliable drug availability and cost, limited subspecialists, and patient beliefs. Adapting recommendations for MTX use in the context of prevalent endemic infections; ensuring safe but feasible MTX monitoring strategies, enhanced access to stable drug supply, and specialized rheumatology care; and improving patient education are key to reducing the burden of rheumatic diseases in L‐HDI countries.

Policy-makers should perform a cost-benefit analysis before initiating a war. This article describes a methodology for such assessment, and applies it post hoc to five military actions undertaken by the United States between 1950 and 2000 (the Korean War, the Vietnam War, the invasion of Grenada, the invasion of Panama, and the First Gulf War). The analysis identifies three broad categories of value: human capital, economic outcomes, and national influence. Different stakeholders (politicians, generals, industry, etc.) may assign different weights to these three categories, so this analysis tabulates each separately, and then, as may sometimes be necessary, monetizes them for unified comparison.

There is increasing evidence that nonalcoholic steatohepatitis (NASH) is a risk factor for hepatocellular carcinoma (HCC) in the absence of cirrhosis, a phenomenon termed noncirrhotic HCC. Early diagnosis of HCC is critical to a favorable prognosis. We tested the hypothesis that hydroxyproline content of liver biopsy samples is diagnostic for HCC in murine models of NASH induced by diet or by diet and chemicals. The training set comprised mice fed a standard diet or a fast-food diet with or without administration of thioacetamide. At harvest, livers from the modified diet cohort exhibited NASH with a subset of NASH livers exhibiting HCC. Hydroxyproline content was measured in liver biopsy samples with tissue in the NASH+HCC cohort sampled from the remote, nontumor parenchyma. Plotting the receiver operating characteristics (ROC) with hydroxyproline as the continuous variable against the absence or presence of HCC yielded an area under ROC of 0.87, a threshold of >0.18 μg hydroxyproline/mg liver and sensitivity of 91% with a specificity of 83.3%. The use of liver hydroxyproline content as a diagnostic for HCC in a test set comprising healthy, NASH and NASH+HCC livers proved 87% accurate.

Abstract Objective Rett syndrome (RTT) is characterized by neurological regression. This pioneering study investigated the effect of age on brain volume reduction by analyzing magnetic resonance imaging findings in participants with RTT, ranging from toddlers to adults. Methods Functional evaluation and neuroimaging were performed. All scans were acquired using a Siemens Tim Trio 3 T scanner with a 32-channel head coil. Results The total intracranial volume and cerebral white matter volume significantly increased with age in the control group compared with that in the RTT group (p

In this paper, a novel ultrawideband phased array is presented based on exponential curve modified dipoles. A tightly-coupled exponential curve modified dipole arm is used to extend the operating bandwidth with a simple coplanar strip structure, which avoids the use of additional complex impedance conversion networks and reduces the processing difficulty and cost. The problematic common-mode resonance is moved out of the required operating bandwidth by the shorting pin. A wide angle impedance matching layer is used to improve beam scanning ability of the array. The proposed antenna array achieves 8.5:1 (2.06-17.6 GHz) bandwidth at broadside, with a scanning range of ±45° from 2.06-17.6 GHz (8.5:1 bandwidth) in the E-plane and ±45° from 2.2-17.6 GHz (8:1 bandwidth) in the H-plane, subject to active VSWR < 3. A $6\times6$ prototype was fabricated and measured with good agreement in measured and simulated results, which verify the ultrawideband performance of proposed array.

The Simple Data Link (SDL) framing protocol is uniquely designed to delineate packets in high-speed communications, SDL can identify the boundaries of variable length frames by using length indication and Header Error Correction (HEC) check. In this paper, we derive an HEC hunting algorithm for bit-level and octet-level operations and propose a hardware circuit to simplify the implementation of an SDL framer. By employing only one CRC detector, the proposed framer can reduce the hunting time of packet delineation just like using multiple framers in parallel. To evaluate the performance of SDL in next generation optical networks, the packet loss probability of Optical Burst Switching (OBS) networks is examined. Analytical results reveal that receivers with SDL framers can greatly reduce packet loss probability than those with only pure IP headers. Keywords: Simple Data Link Protocol (SDL); Optical Burst Switching (OBS); CRC; Framer

Abstract Separation and identification of molecules and biomolecules such as nucleic acids, proteins, and polysaccharides from complex fluids are known to be important due to unmet needs in various applications. Generally, many different separation techniques, including chromatography, electrophoresis, and magnetophoresis, have been developed to identify the target molecules precisely. However, these techniques are expensive and time consuming. “Lab‐on‐a‐chip” systems with low cost per device, quick analysis capabilities, and minimal sample consumption seem to be ideal candidates for separating particles, cells, blood samples, and molecules. From this perspective, different microfluidic‐based techniques have been extensively developed in the past two decades to separate samples with different origins. In this review, “lab‐on‐a‐chip” methods by passive, active, and hybrid approaches for the separation of biomolecules developed in the past decade are comprehensively discussed. Due to the wide variety in the field, it will be impossible to cover every facet of the subject. Therefore, this review paper covers passive and active methods generally used for biomolecule separation. Then, an investigation of the combined sophisticated methods is highlighted. The spotlight also will be shined on the elegance of separation successes in recent years, and the remainder of the article explores how these permit the development of novel techniques.

Summary: Background: While vaccines have established utility against COVID-19, phase 3 efficacy studies have generally not comprehensively evaluated protection provided by previous infection or hybrid immunity (previous infection plus vaccination). Individual patient data from US government-supported harmonized vaccine trials provide an unprecedented sample population to address this issue. We characterized the protective efficacy of previous SARS-CoV-2 infection and hybrid immunity against COVID-19 early in the pandemic over three-to six-month follow-up and compared with vaccine-associated protection. Methods: In this post-hoc cross-protocol analysis of the Moderna, AstraZeneca, Janssen, and Novavax COVID-19 vaccine clinical trials, we allocated participants into four groups based on previous-infection status at enrolment and treatment: no previous infection/placebo; previous infection/placebo; no previous infection/vaccine; and previous infection/vaccine. The main outcome was RT-PCR-confirmed COVID-19 >7–15 days (per original protocols) after final study injection. We calculated crude and adjusted efficacy measures. Findings: Previous infection/placebo participants had a 92% decreased risk of future COVID-19 compared to no previous infection/placebo participants (overall hazard ratio [HR] ratio: 0.08; 95% CI: 0.05–0.13). Among single-dose Janssen participants, hybrid immunity conferred greater protection than vaccine alone (HR: 0.03; 95% CI: 0.01–0.10). Too few infections were observed to draw statistical inferences comparing hybrid immunity to vaccine alone for other trials. Vaccination, previous infection, and hybrid immunity all provided near-complete protection against severe disease. Interpretation: Previous infection, any hybrid immunity, and two-dose vaccination all provided substantial protection against symptomatic and severe COVID-19 through the early Delta period. Thus, as a surrogate for natural infection, vaccination remains the safest approach to protection. Funding: National Institutes of Health.

Episodic weakness is typically associated with a group of disorders so called periodic paralyses. Their major causes are mutation of ion channels, and have rarely been linked to mitochondrial disorders. We report a 20-year-old man with episodic weakness and axonal sensorimotor neuropathy since the age of 10 years. Analysis of the next generation sequencing data of the entire mitochondrial genome extracted from the blood revealed a homoplasmic m.9185T > C variant in MT-ATP6. Acetazolamide may be responsive for episodic weakness, and supplements with l-carnitine with coenzyme-Q10 seem to be beneficial as well. To the best of our knowledge, this is the first report in Taiwan which reveals episodic weakness and sensorimotor polyneuropathy as a unique phenotype of MT-ATP6 mutations.

Abstract The current decline of mammals worldwide makes quantitative population assessments crucial, especially for range‐restricted and threatened species. However, robust abundance estimations are challenging for elusive or otherwise difficult to detect species. Alternative metrics requiring only presence/absence data, that is, occupancy, are possible but calibration with independent density estimates should be foreseen, although rarely performed. Here, we calibrated density estimates from acoustic surveys to occupancy estimates from camera‐trapping detections to derive the abundance of the endangered Sanje mangabey (Cercocebus sanjei) across its entire range in the Udzungwa Mountains of Tanzania. We found marked occupancy–density relationships for the two forest blocks where this primate occurs and used them to derive spatially explicit density estimates. Occupancy increased in montane forest zones at mid‐elevation but decreased slightly with proximity to forest borders. We predicted an average density (±SE) of 0.26 ± 0.05 groups/km2 in the national park and 0.24 ± 0.06 in the nature reserve. Accordingly, and given the much larger area of the reserve, the average predicted individual abundance was 1555 ± 325 and 2471 ± 571 in the national park and nature reserve, respectively. We found higher density and abundance in the nature reserve compared with previous studies. Given the past disturbance and poorer protection in the nature reserve relative to the national park, our results instill optimism for the status of the species, although occupancy analysis highlighted the potential vulnerability of this primate to human disturbance. Our approach appears valuable for spatially explicit density estimations of elusive species, and provides robust assessments of vulnerability and identification of priority areas for conservation of threatened populations.

Patients with Gaucher disease type 3 (GD3), especially those with GBA p.L444P homozygous mutation, often suffer from complications including lymphadenopathy even under regular enzyme replacement therapy (ERT). In order to improve their outcome, we administrated eliglustat, a substrate reduction therapy (SRT), in combination with ERT to four patients, age ranged 9–18 years, for two years. The results revealed that patients' plasma glucosylsphingosine (lyso-GL1) level and chitotriosidase activity both decreased after adding eliglustat. In three patients who completed follow-up MRI scanning, sizes of lymph nodes all decreased. No severe adverse events were attributed to eliglustat. Therefore, our data suggest that a combined SRT and ERT treatment may improve the ERT-resistant symptoms in patients with GD3.

Abstract Individuals with Rett syndrome (RTT) commonly demonstrate Parkinsonian features and dystonia at teen age; however, the pathological reason remains unclear. Abnormal iron accumulation in deep gray matter were reported in some Parkinsonian-related disorders. In this study, we investigated the iron accumulation in deep gray matter of RTT and its correlation with dystonia severity. We recruited 18 RTT-diagnosed participants with MECP2 mutations, from age 4 to 28, and 28 age-gender matched controls and investigated the iron accumulation by susceptibility weighted image (SWI) in substantia nigra (SN), globus pallidus (GP), putamen, caudate nucleus, and thalamus. Pearson’s correlation was applied for the relation between iron accumulation and dystonia severity. In RTT, the severity of dystonia scales showed significant increase in subjects older than 10 years, and the contrast ratios of SWI also showed significant differences in putamen, caudate nucleus and the average values of SN, putamen, and GP between RTT and controls. The age demonstrated moderate to high negative correlations with contrast ratios. The dystonia scales were correlated with the average contrast ratio of SN, putamen and GP, indicating iron accumulation in dopaminergic system and related grey matter. As the first SWI study for RTT individuals, we found increased iron deposition in dopaminergic system and related grey matter, which may partly explain the gradually increased dystonia.

Abstract The Project Baseline Health Study (PBHS) was launched to map human health through a comprehensive understanding of both the health of an individual and how it relates to the broader population. The study will contribute to the creation of a biomedical information system that accounts for the highly complex interplay of biological, behavioral, environmental, and social systems. The PBHS is a prospective, multicenter, longitudinal cohort study that aims to enroll thousands of participants with diverse backgrounds who are representative of the entire health spectrum. Enrolled participants will be evaluated serially using clinical, molecular, imaging, sensor, self-reported, behavioral, psychological, environmental, and other health-related measurements. An initial deeply phenotyped cohort will inform the development of a large, expanded virtual cohort. The PBHS will contribute to precision health and medicine by integrating state of the art testing, longitudinal monitoring and participant engagement, and by contributing to the development of an improved platform for data sharing and analysis.

Background: Inborn errors of bile acid metabolism (IEBAM) cause rare but treatable genetic disorders that can present as neonatal cholestasis or neurological diseases. Without timely primary bile acid treatment, patients may develop liver failure early in life. This study aimed to analyze the types and treatment outcomes of IEBAM in Taiwanese infants and document the allele frequency of CYP7B1 hot spot mutations in the population. Methods: Urine samples from patients with infantile intrahepatic cholestasis and suspected IEBAM were subjected to urinary bile acid analysis by gas chromatography-mass spectrometry (GC/MS). Genetic diagnoses were made using direct sequencing or next-generation sequencing. We also tested healthy control subjects for a probable hot spot point mutation of CYP7B1. Results: Among the 75 patients with infantile intrahepatic cholestasis tested during 2000 –2016, three had ∆4-3-oxosteroid 5β-reductase deficiency with AKR1D1 mutations, and three had oxysterol-7α-hydroxylase deficiency with CYP7B1 mutation. Two patients with ∆4-3-oxosteroid 5β-reductase deficiency were successfully treated with cholic acid. The three unrelated infants with oxysterol 7α-hydroxylase deficiencies had the same p.R112X homozygous CYP7B1 mutation. Two had mild renal or neurological involvement. Among 608 healthy control subjects, the allele frequency of the heterozygous mutation for p.R112X was 2/1216 (0.16%). The only surviving patient with oxysterol 7α-hydroxylase deficiency recovered from liver failure after chenodeoxycholic acid (CDCA) treatment beginning at 3 months of age. Conclusion: Distinct types of IEBAM disease were found in the Taiwanese population. Patients with early diagnosis and early treatment had a favorable outcome. IEBAM prevalence rates may be higher than expected due to the presence of heterozygous mutations in the general population. Key Words: chenodeoxycholic acid, cholic acid, inborn errors of bile acid metabolism, neonatal cholestasis, oxysterol 7α-hydroxylase deficiency

Background/Purpose: The study aim to investigate the correlation between diffusion-weighted magnetic resonance imaging (DW-MRI) and transient elastography (TE) liver fibrosis findings in children with cholestatic liver diseases, and the utility of TE findings to predict cholestatic complications in children. Methods: This cross-sectional study enrolled 36 cholestatic children (21 boys and 15 girls). All study subjects underwent TE and DW-MRI studies to assess liver stiffness. All study subjects were followed prospectively, and their cholestatic complications were analyzed. The optimum cut-off TE value for the prediction of cholestatic complications was determined by receiver operating characteristic (ROC) analysis. Results: A significant negative correlation between liver stiffness measurements (LSMs) and right-liver-to-psoas apparent diffusion coefficient ratios (LTPARs) was found in the study cohort (correlation coefficient = −0.52, p = 0.001). An LSM cut-off > 8.6 kPa was optimal for predicting complications of cholestasis in 6 months of this cohort (p 8.6 kPa was significantly predictive of cholestatic complications in 6 months (hazard ratio = 4.89; 95% CI = 1.41–16.97; p = 0.01). Conclusion: TE and DW-MRI findings showed a similar ability to predict liver fibrosis in cholestatic children. The LSMs measured by TE are predictive of the occurrence of cholestatic complications in 6 months in children with cholestatic liver diseases. Keywords: Liver fibrosis, Diffusion-weighted magnetic resonance imaging, Transient elastography, Liver stiffness measurement

In the mouse, the osteoblast-derived hormone Lipocalin-2 (LCN2) suppresses food intake and acts as a satiety signal. We show here that meal challenges increase serum LCN2 levels in persons with normal or overweight, but not in individuals with obesity. Postprandial LCN2 serum levels correlate inversely with hunger sensation in challenged subjects. We further show through brain PET scans of monkeys injected with radiolabeled recombinant human LCN2 (rh-LCN2) and autoradiography in baboon, macaque, and human brain sections, that LCN2 crosses the blood-brain barrier and localizes to the hypothalamus in primates. In addition, daily treatment of lean monkeys with rh-LCN2 decreases food intake by 21%, without overt side effects. These studies demonstrate the biology of LCN2 as a satiety factor and indicator and anorexigenic signal in primates. Failure to stimulate postprandial LCN2 in individuals with obesity may contribute to metabolic dysregulation, suggesting that LCN2 may be a novel target for obesity treatment.

Abstract Influenza virus uses a unique mechanism to initiate viral transcription named cap-snatching. The PB2 subunit of the viral heterotrimeric RNA polymerase binds the cap structure of cellular pre-mRNA to promote its cleavage by the PA subunit. The resulting 11–13 capped oligomer is used by the PB1 polymerase subunit to initiate transcription of viral proteins. VX-787 is an inhibitor of the influenza A virus pre-mRNA cap-binding protein PB2. This clinical stage compound was shown to bind the minimal cap-binding domain of PB2 to inhibit the cap-snatching machinery. However, the binding of this molecule in the context of an extended form of the PB2 subunit has remained elusive. Here we generated a collection of PB2 truncations to identify a PB2 protein representative of its structure in the viral heterotrimeric protein. We present the crystal structure of VX-787 bound to a PB2 construct that recapitulates VX-787's biological antiviral activity in vitro. This co-structure reveals more extensive interactions than previously identified and provides insight into the observed resistance profile, affinity, binding kinetics, and conformational rearrangements induced by VX-787.

OBJECTIVES/GOALS: In March 2019, Duke updated recruitment guidelines and adopted an “Engagement” policy that requires patients to ‘opt-out’ of communications regarding potential research participation. This created an opportunity to evaluate recruitment for ongoing clinical studies pre and post implementation. METHODS/STUDY POPULATION: Implementation of the new policy required new training for study teams, modification to recruitment plans, and expansion of ongoing efforts to improve direct-to-patient messaging through EPIC/MyChart tools. The impact of this new policy on overall recruitment was monitored and characterized both prior to and after implementation of the policy. Customized MyChart messages have been generated for over 22 studies, with a total of 41,386 messages sent to potential participants. RESULTS/ANTICIPATED RESULTS: Only a small number of study teams have modified their recruitment plans with transition to the new policy. This may be related to lack of understanding about policy implementation, potential recruitment opportunities, required training, resource limitations, etc. However, our case study, TMIST, had an 48% improvement in average enrollment within the first 2 months of implementation, and an almost 40% improvement in recruitment efficiency. Since becoming an “opt-out” institution, 11 study teams have implemented direct-to-patient recruitment via the MyChart patient portal. One unintended consequence we’ve noted is several different study invitations to potential participants within some patient populations. DISCUSSION/SIGNIFICANCE OF IMPACT: The new policy allows study teams to engage in direct-to-patient outreach, leading to an increase in enrollment for some studies. Incorporation of direct-to-patient messaging strategies can be a cost and resource saving measure to improve recruitment. The need to recruit similar populations demonstrated that strategic, thoughtful approaches are needed.

To clarify the relationship between neuroimaging findings, neuropsychiatric comorbidities, and epilepsy in patients with tuberous sclerosis complex (TSC) in Taiwan. Methods: Medical records from 32 patients with TSC were retrospectively reviewed, including mutational analysis, neuroimaging findings, electroencephalogram findings, and neuropsychiatric comorbidities. Results: Of these patients, six (18.75%) were diagnosed to have autism spectrum disorders (ASD), and 10 (31.25%) were diagnosed to have attention-deficit–hyperactivity disorder. In the latter patients, there were no differences in the regional distribution of tuber burden. In addition to a high prevalence of cystic-like tubers, tubers in insular and temporal areas were associated with ASD. Nonsense mutations in the TSC2 gene group had a correlation with autistic behavior. In 26 (81.25%) patients with a history of epilepsy, infantile spasms and partial seizures were the predominant type of epilepsy. Most of them developed seizures prior to age 1 year. Conclusion: ASD is a common comorbidity in TSC. Cortical tubers in the temporal lobe and insular area were associated with ASD. The presence of cystic-like tubers on magnetic resonance imaging may also offer a structural marker for ASD in TSC.

AIMS To determine the growth rate of renal masses (RMs) under active surveillance (AS), and to describe the clinical outcome of AS patients. Materials and Methods We conducted a retrospective review of an AS database to obtain demographics, radiological and pathologic characteristics and RM size of patients. RMs were followed at 6-12 month intervals for ≥1 year with computed tomography (CT), magnetic resonance imaging (MRI), or renal ultrasound. Kaplan-Meier analysis determined the annual likelihood of intervention. RMs were divided into 3 radiographic subcategories (solid, cystic, and angiomyolipoma). A linear regression model determined RM growth rates. Results 131 RMs in 114 patients were included. Median age, Charlson Comorbidity Index score and mean follow-up were 69.1 years, 4.0 and 4.2±2.6 years, respectively. Maximal tumor diameter (MTD) at diagnosis was 2.1±1.3 cm. 49 RMs exhibited negative or zero net growth. Mean MTD growth rate for all RMs was 0.72±3.2 (95% CI: 0.16-1.28) mm/year. When stratified by MTD at diagnosis, mean RM growth rates were 0.84, 0.84, 0.44, 0.74 and 0.71 mm/year for RMs

Myoclonic astatic epilepsy (MAE) is characterized by multiple seizure types, which are often refractory. Although vagus nerve stimulation (VNS) is an alternative treatment for medically intractable seizures, its exact mechanism of action remains unclear. Herein, we report the case of a 4-year-old boy with intractable MAE who has been in a seizure-free status for 2 years and 3 months since 6 months after the implantation of a vagus nerve stimulator (Model 103, Cyberonics, Inc., Houston, TX). Various test results 6 months after VNS were compared with those before VNS. Results of an electroencephalograph revealed disappearance of epileptiform discharges and an increased beta–gamma spectrum rhythm. The brain diffusion-tensor imaging showed an increased ratio of fraction anisotropy in the right fimbria–fornix, indicating improved diffusion of the white matter tract, and 18F-fluorodeoxyglucose positron emission tomography revealed globally improved cerebral glucose metabolism. His cognitive and social–emotional performances also improved at 2 years after VNS. To the best of our knowledge, this is the first report to describe the effects of VNS on fimbria–fornix and glucose metabolism in MAE.

Esophageal variceal hemorrhaging is potentially life threatening for long-term survivors of biliary atresia. We evaluated the feasibility of less-invasive parameters for predicting the presence of clinically significant esophageal variceal bleeding in biliary atresia patients. Methods: A total of 30 patients aged 108–5314 days (median = 285 days) with biliary atresia underwent a magnetic resonance examination with fast spin-echo T2-weighted imaging and spin-echo, T1-weighted images with fat saturation after use of a contrast medium on a 1.5-tesla scanner. The splenic length-platelet ratio was divided by the each patient's body height (m) to produce the corrected splenic length-platelet ratios. In addition, the splenic volume index-to-platelet count ratio was divided by the patient's body weight (kg) to produce a corrected ratio. Results: The corrected splenic length-platelet ratio was more significantly increased in 21 patients with esophageal variceal bleeding (Group A) than in nine patients without variceal bleeding [(Group B) 0.98 ± 0.64 vs. 0.44 ± 0.18, p

Surveillance and control of tuberculous infection in pediatric patients, especially in those with a contact history, is important to prevent tuberculous infection in the general population. Totally 26 patients, younger than 14 years of age, who had a diagnosis of pulmonary Mycobacterium tuberculosis (TB), underwent both chest radiographs and computed tomography (CT), which were retrospectively reviewed and compared with those of 20 patients with community-acquired bacterial pneumonia (CABP). TB patients were commonly afebrile and had less cavitating lesions or pleural fluid than CABP patients had. Focal or sub-segmental lung opacities suggested the diagnosis of TB than of CABP. Chest CT could also help to identify enlarged, calcified, necrotic mediastinal lymph nodes, which are less frequently found in CABP and frequently obscured by thymic shadows on chest radiographs of children. Low-dose CT for children or infants suspected to have pulmonary TB infection could help to make the decision of further antibiotic treatment.

Background/Objective: About 20% of biliary atresia (BA) survivors have attacks of esophageal variceal bleeding. We propose a method to evaluate the risk of esophageal variceal bleeding (EVB) using noninvasive indices by multislice computed tomography (CT). Methods: We reviewed 31 potential living-related liver recipients aged 99–5314 days (mean, 1474 days) who underwent CT examinations using a 64-slice multislice CT scanner. Of the 31 patients, 19 patients (Group A) with fecal occult blood had EVB on esophagogastroduodenoscopy; the rest belonged to Group B. Splenic diameters (mm) were divided by body heights (m) and platelet counts (1000/mm3) to produce standardized ratios of transverse splenic length/body height/platelet count (SLHPR). The transverse diameters of paraesophageal veins (PVs) and perigastric veins (PGVs) were measured adjacent to the lower thoracic esophagus and within the lesser sac, respectively. Results: According to a receiver operating characteristic curve analysis, the SLHPRs (r=0.833), transverse PV (r=0.957), and PGV (r=0.987) diameters were better predictors of EVB than demographic and laboratory variables. However, the transverse diameters of PGVs and PVs were the most accurate predictors of the EVB. Conclusion: For candidates awaiting liver transplantation, screening by noninvasive SLHPR and the transverse diameters of PGVs and PVs by CT may help to identify BA patients with a high risk of EVB.

Congenital cystic adenomatoid malformation (CCAM) and bronchopulmonary sequestration (BPS) are major embryonic pulmonary developmental anomalies. Early surgical excision is becoming an increasingly common option. We investigated the clinical features and management of patients with CCAM and BPS at the National Taiwan University Hospital. Methods: We conducted a retrospective review of neonates diagnosed with CCAM and/or BPS at the Hospital from July 1995 to January 2008. Prenatal examination, postnatal presentation, management and patient outcome were analyzed. We also propose a concise algorithm for the practical management of these conditions. Results: Sixteen patients were recruited including eight (50%) with CCAM, five (31%) with BPS and three (19%) with mixed-type lesions (CCAM with BPS). Thirteen (81%) patients were diagnosed antenatally at a median gestational age of 20 weeks. Eleven (69%) patients underwent surgical resection before 6 months of age because of respiratory distress or repeated pulmonary infection. There were no surgery-related complications among the seven patients who underwent early surgery within 1 month of age. Five (31%) patients remained asymptomatic and did not undergo surgery. All patients survived with no limitations to daily activity during follow-up periods of 1–8 years. Conclusion: The high proportion of mixed-type lesions suggests that CCAM and BPS may share the same developmental ancestry. Early surgical resection within 1 month of age is safe in symptomatic patients.

Primary intestinal lymphangiectasia is a rare disease of children, which is characterized by chronic diarrhea and complicated with malnutrition, including fat-soluble vitamin deficiency. We report a girl aged 4 years and 8 months who was diagnosed with the disease by endoscopic duodenal biopsy at 8 months of age. She presented initially with chronic diarrhea at 4 months of age. Generalized edema with hypoalbuminemia frequently occurred despite regular albumin supplements. Multiple vitamins initially were not supplied regularly. Episodes of tetany caused by hypocalcemia developed 4 years after the diagnosis of intestinal lymphangiectasia. Imaging study (long-bone X-ray and dual-energy X-ray absorptiometry) revealed low bone density. Complicated vitamin D deficiency [low serum 25-hydroxy vitamin D concentration (< 12.48 mmol/L, the detection limit)] and secondary hyperparathyroidism were confirmed via blood testing. Vitamin D supplementation for 3 months improved her bone density, secondary hyperparathyroidism and frequent tetany. Vitamin D status should be monitored in patients with intestinal lymphangiectasia.

Pneumomediastinum usually occurs after assisted ventilation. Spontaneous pneumomediastinum without preceding assisted ventilation, and presenting as a huge cystic mass in a term newborn infant has rarely been reported. We report a term baby who developed respiratory distress at 2 days of age. A huge cystic tumor of the thymus or a congenital mediastinal cystic tumor was initially suggested by chest radiography and computed tomography. Follow-up chest film revealed a “spinnaker sail sign” which is a typical radiographic presentation of pneumomediastinum.

We reported two cases of recurrent meningitis and both of them had Mondini dysplasia, which provides a link between the brain and inner ear and is associated with cerebrospinal fluid, otorrhea/rhinorrhea, hearing impairment, and recurrent meningitis. Patients who have hearing impairment and recurrent meningitis should be evaluated for the possibility of this congenital dysplasia, and early diagnosis and prompt surgical intervention may prevent further episodes.