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Objective: To determine and compare the birth history or postnatal complications of idiopathic toe walking (ITW) and toe walking known to be associated with a medical condition.Study Design: This was a retrospective chart review of parent-reported birth histories of children who presented to a dedicated toe-walking clinic between 2010 and 2014. This cohort comprised children diagnosed with ITW and children with a medical reason for their toe-walking gait. Data were compared with Australian Perinatal statistical normative data.Results: Ninety-five children (60 males, 63%) were diagnosed with ITW, with a mean (SD) age of 5.8 (2.9) years. Children with an ITW gait were found to have greater rates of prematurity (OR 2.4; 95% CI 1.43-4.03), greater rates of admission to a special care nursery or neonatal intensive care unit (OR 1.98; 95% CI 1.23-3.18), and lower birth weights (OR 6.6; 95% CI 3.48-12.5) than the normative population. Children with a medical reason for toe walking (n = 28, 68% males) also had greater rates of prematurity (OR 2.4; 95% CI 0.94-6.09) than the normative population and more instrumented births than the ITW cohort (OR 1.56; 95% CI 0.64-3.77). No association was found between assisted-birth intervention and the ITW cohort compared with the normative population or group with a medical cause for toe walking.Conclusions: ITW gait was associated with greater rates of complications during and after delivery. Such complications have been associated previously as risk factors for neurologic insult affecting motor development. [ABSTRACT FROM AUTHOR]

Purpose Statin selection for the largest hospital formulary in Qatar is not systematic, not comparative, and does not consider the multi-indication nature of statins. There are no reports in the literature of multi-indication−based comparative scoring models of statins or of statin selection criteria weights that are based primarily on local clinicians’ preferences and experiences. This study sought to comparatively evaluate statins for first-line therapy in Qatar, and to quantify the economic impact of this. Methods An evidence-based, multi-indication, multi-criteria pharmacotherapeutic model was developed for the scoring of statins from the perspective of the main health care provider in Qatar. The literature and an expert panel informed the selection criteria of statins. Relative weighting of selection criteria was based on the input of the relevant local clinician population. Statins were comparatively scored based on literature evidence, with those exceeding a defined scoring threshold being recommended for use. Findings With 95% CI and 5% margin of error, the scoring model was successfully developed. Selection criteria comprised 28 subcriteria under the following main criteria: clinical efficacy, best publish evidence and experience, adverse effects, drug interaction, dosing time, and fixed dose combination availability. Outcome measures for multiple indications were related to effects on LDL cholesterol, HDL cholesterol, triglyceride, total cholesterol, and C-reactive protein. Atorvastatin, pravastatin, and rosuvastatin exceeded defined pharmacotherapeutic thresholds. Atorvastatin and pravastatin were recommended as first-line use and rosuvastatin as a nonformulary alternative. It was estimated that this would produce a 17.6% cost savings in statins expenditure. Sensitivity analyses confirmed the robustness of the evaluation’s outcomes against input uncertainties. Implications Incorporating a comparative evaluation of statins in Qatari practices based on a locally developed, transparent, multi-indication, multi-criteria scoring model has the potential to considerably reduce expenditures on statins. Atorvastatin and pravastatin should be the first-line statin therapies in the main Qatari health care provider, with rosuvastatin as an alternative. [ABSTRACT FROM AUTHOR]

Fetal atrioventricular (AV) block is a rare and potentially devastating condition. Most commonly fetal AV block is mediated by maternal lupus antibodies which cause irreversible damage to the AV node. For many fetuses, the only potential intervention is premature delivery and highly invasive pacemaker implantation. However, there exists a small subset of fetuses with non-immune mediated AV block who appear to have far better outcomes, with potential for spontaneous resolution and a return to sinus rhythm. Despite this, it is not clear that prenatal counseling takes this fact into account. We describe a series of three patients with non-immune fetal second-degree AV block with spontaneous resolution prior to delivery, underscoring the need for appropriate prenatal counseling in this scenario. [ABSTRACT FROM AUTHOR]

Abstract: Objective: To examine the association between parent-reported ability of young children with cerebral palsy (CP) to eat different food textures and gross motor functional abilities. Design: Prospective, longitudinal, representative cohort study. Setting: Community and tertiary pediatric hospital settings. Participants: Children (N=170; 110 boys [65%]) were assessed on 396 occasions (range, 1–4 occasions), including 67 at 1 year 6 months (49 boys), 99 at 2 years (66 boys), 111 at 2 years 6 months (71 boys), and 119 at 3 years (64 boys). Interventions: Not applicable Main Outcome Measures: Gross motor function was determined using the Gross Motor Function Classification System (GMFCS). Parent-reported eating ability was determined using 4 items of the Pediatric Evaluation of Disability Inventory. The association between capability to eat food textures and GMFCS level was examined using generalized estimating equations. Results: The distribution of GMFCS levels at initial presentation was as follows: I, n=62; II, n=32; III, n=24; IV, n=22; and V, n=30. Reported capability to eat cut-up/chunky and “all textures” of table foods decreased significantly as GMFCS level increased. A decreased capability to eat pureed/blended and ground/lumpy foods compared with GMFCS I was significantly associated with GMFCS levels IV and V only. Conclusions: Reported attainment of eating skills was closely associated with GMFCS level in young children with CP across age levels. These results emphasize the need for early oral-motor and feeding screening in young children with CP across gross motor functional abilities. [Copyright &y& Elsevier]

Abstract: Aim: To investigate the relationship between motor ability and early social development in a cohort of preschool children with cerebral palsy (CP). Design: Population-based cohort study. Methods: Participants were 122 children with CP assessed at 18, 24 and 30 months, corrected age (ca). Motor ability was measured by the Gross Motor Function Classification System (GMFCS) with classification assigned by physiotherapists. The sample was representative of a population-based cohort (I=48, 38.4%, II=19, 15.2%, III=17, 13.6%, IV=22, 17.6% and V=19, 15.2%). Social development was measured by the Paediatric Evaluation of Disability Inventory (PEDI) and included capabilities in social interaction, social communication, interactive play and household/community tasks. Results: Cross-sectional analyses indicated a significant relationship between motor ability and social development at 18 months, F(4, 56)=11.44, p <.0001, η 2 =.45, at 24 months, F(4, 79)=15.66, p <.0001, η 2 =.44 and at 30 months, F(4, 76)=16.06, p <.0001, η 2 =.49. A longitudinal analysis with a subset of children (N =24) indicated a significant interaction between age at assessment and GMFCS, F(2, 21)=7.02, p =.005, η 2 =.40. Comparison with community norms indicated that at 18 months corrected age, 44.3% of the cohort was greater than two standard deviations below the mean (>2SD) for social development and a further 27.9% of the cohort was greater than one standard deviation below the mean (>1SD). Interpretation: There is a relationship between motor ability and social development in preschool children with CP. Children with CP may require support for social development in additional to physical interventions, from as early as 18 months. [ABSTRACT FROM AUTHOR]

Objective: To determine the efficacy of electrocardiography (ECG) in detecting left ventricular hypertrophy (LVH) in pediatric hypertension (HT). Study design: Concomitant echocardiograms and electrocardiograms in 108 children with HT were reviewed. Left ventricular mass (LVM), assessed by echocardiography, was used as a basis for a diagnosis of LVH (echo LVH) using accepted pediatric criteria. Using Wilcoxon''s rank-sum test, 14 ECG variables were compared between subjects with and without echo LVH. Spearman correlations were used to examine the linear association between echo LVH and these ECG variables. The sensitivity and specificity of ECG in diagnosing LVH were computed. Results: Of the 108 subjects studied, 35 (32%) met the pediatric criteria for LVH; of these, 8 (7.4%) also met the adult criteria (>51 g/m2.7) for LVH. Mean values for only 5 ECG criteria differed significantly among the groups: RI, SaVR, RaVL, RI+SIII, and SVI+RV6 (P < .05). Significant correlations were found for several ECG criteria and at least 1 measure of LVM, but the magnitudes were modest. Standard ECG criteria predicted LVH with high specificity (>90%) but low sensitivity (<35%). RI >10 mm was identified as demonstrating a modestly improved positive likelihood ratio of ∼3. Conclusions: ECG is not an adequate predictor of LVH for clinical use in HT. [Copyright &y& Elsevier]

Cerebral palsy (CP) is a nonprogressive neurological disorder and the most common physical disability of childhood. There is no cure for CP, but stem cells have the potential to improve brain injury and hence function. This phase 1 clinical trial investigated the safety of the intravenous infusion of full-matched sibling cord blood cells for children with CP aged 1 to 16 years. Preliminary efficacy outcomes were also investigated. Twelve participants received 12/12 HLA-matched sibling cord blood cell infusions. One treatable serious adverse reaction to cryoprotectant was observed, and no adverse reactions occurred beyond 24 h after infusion. Gross motor function measure (GMFM-66) scores did not improve compared with baseline beyond what could be expected from developmental levels, and participants had varied changes in the Quality of Upper Extremity Skills Test (QUEST) and Vineland Adaptive Behavior Scales (VABS-II) scores. In conclusion, matched sibling cord blood cell infusion for children with CP is relatively safe when conducted in an appropriate facility. Australian and New Zealand Clinical Trials Registry (ACTRN12616000403437) and Clinicaltrials.gov (NCT03087110). [ABSTRACT FROM AUTHOR]

Cobalamin and its metabolites play a crucial role in DNA synthesis and cellular energy metabolism. Disorders of cobalamin metabolism are rare, autosomal recessive, conditions that present with neurological dysfunction of varying severity. We report a child with cobalamin E defect presenting in early infancy with vertical nystagmus, developmental delay, deceleration in head growth, status epilepticus and leukoencephalopathy, with only mild haematological abnormalities. Resolution of seizures and subsequent improvement in development and head growth was observed following early treatment with parenteral hydroxocobalamin, betaine, folate and methionine, emphasising the importance of early diagnosis and treatment in these conditions. [ABSTRACT FROM AUTHOR]

• Resurfacing is significant in reducing crash frequency and severity levels. • As the increase in skid resistance is found to improve safety by severity, therefore, priority should be provided in intersections maintenance program to ensure continued acceptable levels of skid resistance. • Pavement surface condition parameters, roughness and rutting, need to be considered in either intervention criteria or treatment selection to ensure that they are reduced to acceptable levels for users' comfort and safety. • To account for the repeated observations , Generalised Estimating Equation (GEE) with Negative Binomial (NB) regression is an appropriate model in predicting crash frequency. • Road users' safety at signalised intersections are affected by pavement surface condition and geometric characteristics. This study investigated the effects of pavement surface condition and other control factors on casualty crashes at signalized intersections. It involved conducting a before and after study for road surface condition and situational factors. It also included assessing the effects of geometric characteristics on safety performance of signalized intersections post resurfacing to control for the effect of pavement surface condition. Pavement surface condition included roughness, rutting, and skid resistance. The control factors included traffic volume, light and surface moisture condition, and speed limit. The geometric characteristics included approach width, number of lanes, intersection depth, presence of median, presence of shared lane, and presence of bus stop. To account for the repeated observations of the effect of light and surface moisture conditions in four occasions (day-dry, day-wet, night-dry and night-wet) Generalized Estimating Equation (GEE) with Negative Binomial (NB) and log link function was applied. For each signalized intersection in the sample, condition data are collected for the year before and after the year of surface treatment. Crash data, however, are collected for a minimum of three and maximum of five years before and after treatment years. The results show that before treatment, light condition, road surface moisture condition, and skid resistance interaction with traffic volume are the significant contributors to crash occurrence. For after treatment; light condition, road surface moisture condition, their interaction product, and roughness interaction with light condition, surface moisture condition, and traffic volume are the significant contributors. The geometric variables that were found to have significant effects on crash frequency post resurfacing were approach width interactions with presence of shared lane, bus stop, or median. The findings confirm that resurfacing is significant in reducing crash frequency and severity levels. Practical Applications: The study findings would help for better understanding of how geometric characteristics can be improved to reduce crash occurrence. [ABSTRACT FROM AUTHOR]

Background: Intracardiac foreign bodies have been described in the literature, however, they are rare entities, particularly in pediatric patients. We present a case of a teenage boy diagnosed with perimyocarditis who was found to have an unexpected underlying etiology: an unknowingly swallowed sewing pin.Case Report: A 17-year-old boy presented to the Emergency Department with 3 days of chest pain suggestive of perimyocarditis, in the absence of prodromal symptoms or trauma. Electrocardiogram at the time of presentation demonstrated diffuse ST-segment elevation consistent with perimyocarditis. A chest radiograph was significant for a linear density in the anterior mid chest, concerning for foreign body. Chest computed tomography confirmed the presence of a 3.5-cm linear metallic foreign body within the right ventricle. WHY SHOULD AN EMERGENCY PHYSICIAN BE AWARE OF THIS?: This case demonstrates the need to consider alternative etiologies for perimyocarditis, especially in the absence of the typical prodromal symptoms. In addition, it highlights the potential devastating complications of foreign body ingestion and challenges the paradigm that ingested sharp linear foreign bodies < 5 cm in length rarely cause problems. [ABSTRACT FROM AUTHOR]

Purpose The formulary inclusion of proton pump inhibitors (PPIs) in the government hospital health services in Qatar is not comparative or restricted. Requests to include a PPI in the formulary are typically accepted if evidence of efficacy and tolerability is presented. There are no literature reports of a PPI scoring model that is based on comparatively weighted multiple indications and no reports of PPI selection in Qatar or the Middle East. This study aims to compare first-line use of the PPIs that exist in Qatar. The economic effect of the study recommendations was also quantified. Methods A comparative, evidence-based multicriteria decision analysis (MCDA) model was constructed to follow the multiple indications and pharmacotherapeutic criteria of PPIs. Literature and an expert panel informed the selection criteria of PPIs. Input from the relevant local clinician population steered the relative weighting of selection criteria. Comparatively scored PPIs, exceeding a defined score threshold, were recommended for selection. Findings Weighted model scores were successfully developed, with 95% CI and 5% margin of error. The model comprised 7 main criteria and 38 subcriteria. Main criteria are indication, dosage frequency, treatment duration, best published evidence, available formulations, drug interactions, and pharmacokinetic and pharmacodynamic properties. Most weight was achieved for the indications selection criteria. Esomeprazole and rabeprazole were suggested as formulary options, followed by lansoprazole for nonformulary use. The estimated effect of the study recommendations was up to a 15.3% reduction in the annual PPI expenditure. Robustness of study conclusions against variabilities in study inputs was confirmed via sensitivity analyses. Implications The implementation of a locally developed PPI-specific comparative MCDA scoring model, which is multiweighted indication and criteria based, into the Qatari formulary selection practices is a successful evidence-based cost-cutting exercise. Esomeprazole and rabeprazole should be the first-line choice from among the PPIs available at the Qatari government hospital health services. [ABSTRACT FROM AUTHOR]

Abstract: There is limited documentation regarding cognitive function in individuals with Friedreich ataxia (FRDA), possibly because FRDA is widely held to predominantly affect the spinal cord, peripheral sensory nerves and cerebellum and not to affect cognition. Traditionally, the cerebellum has been thought to coordinate voluntary movement and motor tone, posture and gait. However, recent studies have implicated the cerebellum in a range of cognitive functions including executive function, visuospatial organisation and memory. We review the available data on cognitive function and neuroimaging in FRDA and the role of the cerebellum in cognitive function. We conclude with recommendations for future research including correlating cognitive function in individuals with FRDA with possible determinants of disease severity, such as age of onset and the causative genetic mutation. [Copyright &y& Elsevier]

Background: Children diagnosed with neurodevelopmental conditions such as cerebral palsy (CP) are at risk of experiencing restrictions in social activities negatively impacting their subsequent social functioning. Research has identified motor and communication ability as being unique determinants of social function capabilities in children with CP, to date, no research has investigated whether communication is a mediator of the relationship between motor ability and social functioning.Aims: To investigate whether early communication ability at 24 months corrected age (ca.) mediates the relationship between early motor ability at 24 months ca. and later social development at 60 months ca. in a cohort of children diagnosed with cerebral palsy (CP).Method: A cohort of 71 children (43 male) diagnosed with CP (GMFCS I=24, 33.8%, II=9, 12.7%, III=12, 16.9%, IV=10, 14.1%, V=16, 22.5%) were assessed at 24 and 60 months ca. Assessments included the Gross Motor Function Measure (GMFM), the Communication and Symbolic Behaviour Scales-Developmental Profile (CSBS-DP) Infant-Toddler Checklist and the Paediatric Evaluation of Disability Inventory (PEDI). A mediation model was examined using bootstrapping.Results: Early communication skills mediated the relationship between early motor abilities and later social functioning, b=0.24 (95% CI=0.08-0.43 and the mediation model was significant, F (2, 68)=32.77, p<0.001, R(2)=0.49.Conclusions and Implication: Early communication ability partially mediates the relationship between early motor ability and later social function in children with CP. This demonstrates the important role of early communication in ongoing social development. Early identification of communication delay and enriched language exposure is crucial in this population. [ABSTRACT FROM AUTHOR]

Summary: Background: Charcot–Marie–Tooth disease type 1A (CMT1A) is the most common inherited nerve disorder. CMT1A is characterised by peripheral nerve demyelination, weakness, and impaired motor function and is caused by the duplication of PMP22, the gene that encodes peripheral myelin protein 22. High-dose ascorbic acid has been shown to have remyelinating potential and to correct the phenotype of a transgenic mouse model of CMT1A by decreasing expression of PMP22. We tested the efficacy and safety of ascorbic acid supplementation in children with CMT1A. Methods: This 12-month, randomised, double-blind, placebo-controlled trial undertaken between June, 2007, and December, 2008, assessed high-dose oral ascorbic acid (about 30 mg/kg/day) in 81 children with CMT1A (2–16 years). Randomisation was done on a 1:1 ratio by a computer-generated algorithm. All investigators and participants were blinded to treatment allocation with the exception of the trial pharmacist. The primary efficacy outcome was median nerve motor conduction velocity (m/s) at 12 months. Secondary outcomes were foot and hand strength, motor function, walking ability, and quality of life. Compliance was measured by plasma ascorbic acid concentration, pill count, and medication diary entries. Analysis was by intention to treat. This trial is registered with the Australian New Zealand Clinical Trials Registry, number 12606000481572. Findings: 81 children were randomly assigned to receive high-dose ascorbic acid (n=42) or placebo (n=39). 80 children completed 12 months of treatment. The ascorbic acid group had a small, non-significant increase in median nerve motor conduction velocity compared with the placebo group (adjusted mean difference 1·7 m/s, 95% CI −0·1 to 3·4; p=0·06). There was no measurable effect of ascorbic acid on neurophysiological, strength, function, or quality of life outcomes. Two children in the ascorbic acid group and four children in the placebo group reported gastrointestinal symptoms. There were no serious adverse events. Interpretation: 12 months of treatment with high-dose ascorbic acid was safe and well tolerated but none of the expected efficacy endpoints were reached. Funding: University of Sydney Research and Development Scheme; National Health and Medical Research Council of Australia; James N Kirby Foundation; New South Wales Podiatrists Registration Board; Australian Podiatry Education and Research Foundation; Charcot–Marie–Tooth Association of Australia. [Copyright &y& Elsevier]

• In this study we determined the optimal behavioral tests for long-term functional assessment in a rodent model of neonatal brain injury • We demonstrate that Z-scores are a useful tool for standardizing behavioral outcomes and can be used to create a functional composite score. • Rats exposed to a hypoxic ischemic brain injury showed deficits in the negative geotaxis test, cylinder test and novel object recognition test. • This study can inform future studies on behavioral tests most appropriate for long-term assessment in rodent models of neonatal brain injury. Hypoxic ischemic (HI) brain injury is a significant cause of childhood neurological deficits. Preclinical rodent models are often used to study these deficits; however, no preclinical study has determined which behavioral tests are most appropriate for long-term follow up after neonatal HI. HI brain injury was induced in postnatal day (PND) 10 rat pups using the Rice-Vannucci method of unilateral carotid artery ligation. Rats underwent long-term behavioral testing to assess motor and cognitive outcomes between PND11-50. Behavioral scores were transformed into Z-scores and combined to create composite behavioral scores. HI rats showed a significant deficit in three out of eight behavioral tests: negative geotaxis analysis, the cylinder test and the novel object recognition test. These individual test outcomes were transformed into Z-scores and combined to create a composite Z-score. This composite z-score showed that HI rats had a significantly increased behavioral burden over the course of the experiment. In this study we have identified tests that highlight specific cognitive and motor deficits in a rat model of neonatal HI. Due to the high variability in this model of neonatal HI brain injury, significant impairment is not always observed in individual behavioral tests, but by combining outcomes from these individual tests, long-term behavioral burden can be measured. [ABSTRACT FROM AUTHOR]

Abstract: Rotavirus infection is a significant cause of childhood morbidity and mortality worldwide. Although infection primarily causes gastroenteritis and dehydration, systemic signs and neurologic manifestations in rotavirus infection are widely recognized. The pathophysiologic origins of neurologic signs in rotavirus infection remain incompletely understood. We present a 4-year-old girl with clinical features of severe cerebellitis in association with abnormalities detected on magnetic resonance imaging. Rotavirus nucleic acid was demonstrated in both serum and cerebrospinal fluid. Severe neurologic sequelae remain after 2 years of follow-up. This report adds further evidence supporting a direct role for rotavirus in neurologic illness. [Copyright &y& Elsevier]

We report on a case of Cobb syndrome, a rare metameric disorder, characterized by a vascular abnormality of the spinal cord, with an associated vascular skin lesion of the same metamere, in an 8-year-old girl presenting with leg weakness. Magnetic resonance imaging revealed a spinal arteriovenous malformation at the level of T10-L5. This finding, together with a large, pigmented nevus at this level, is diagnostic for Cobb syndrome. A systematic review of the literature on Cobb syndrome, with a focus on treatment and outcomes, is presented. Current treatment options include combinations of embolization, neurosurgical intervention, corticosteroid therapy, and radiotherapy. [Copyright &y& Elsevier]

• Repeated doses of UCB improved neuropathology to a greater extent than a single dose. • Repeated doses of UCB reduced long-term functional deficits to a greater extent than a single dose. • Behavioural burden and neuropathology were examined following perinatal brain injury. • A HI insult resulted in cell death. Hypoxic ischemic (HI) insults during pregnancy and birth can result in neurodevelopmental disorders, such as cerebral palsy. We have previously shown that a single dose of umbilical cord blood (UCB) cells is effective at reducing short-term neuroinflammation and improves short and long-term behavioural outcomes in rat pups. A single dose of UCB was not able to modulate long-term neuroinflammation or brain tissue loss. In this study we examined whether multiple doses of UCB can modulate neuroinflammation, decrease cerebral tissue damage and improve behavioural outcomes when followed up long-term. HI injury was induced in postnatal day 10 (PND10) rat pups using the Rice-Vannucci method of carotid artery ligation. Pups received either 1 dose (PND11), or 3 doses (PND11, 13, 20) of UCB cells. Rats were followed with behavioural testing, to assess both motor and cognitive outcomes. On PND50, brains were collected for analysis. HI brain injury in rat pups caused significant behavioural deficits. These deficits were significantly improved by multiple doses of UCB. HI injury resulted in a significant decrease in brain weight and left hemisphere tissue, which was improved by multiple doses of UCB. HI resulted in increased cerebral apoptosis, loss of neurons and upregulation of activated microglia. Multiple doses of UCB modulated these neuropathologies. A single dose of UCB at PND11 did not improve behavioural or neuropathological outcomes. Treatment with repeated doses of UCB is more effective than a single dose for reducing tissue damage, improving brain pathology and restoring behavioural deficits following perinatal brain injury. [ABSTRACT FROM AUTHOR]