Clinical and biochemical spectrum of metabolic cardiomyopathy in Egyptian children

BackgroundInborn errors of metabolism (IEMs) commonly present with pediatric cardiomyopathy. Identification of the underlying cause is necessary as it may lead to improved outcomes.ObjectivesWe aimed to investigate the diagnostic rate, the clinical, and biochemical spectra of IEMs among Egyptian pediatric patients presenting with cardiomyopathy, and their outcome measures.MethodsWe retrospectively analyzed the clinical, biochemical, and radiological data of 1512 children diagnosed with cardiomyopathy at Cairo University Children''s Hospital over a 5-year duration.ResultsTwo hundred twenty-nine children were clinically suspected as IEMs and underwent metabolic workup. Nineteen different IEMs were confirmed in 57 (24.4%) of the suspected children. Their median age at presentation was 2.6 years and the majority had extra-cardiac manifestations. Hypertrophic cardiomyopathy represented 43/57 (75.4%) of confirmed cases, while dilated cardiomyopathy represented 13/57 (22.8%), and one patient presented with a mixed phenotype. Twenty- six patients (45.6%) survived, while 31 patients (54%) either died or were lost to follow up and assumed deceased.ConclusionsWe developed for the first time a database and a diagnostic scheme for metabolic cardiomyopathies in Egyptian children. With the recent introduction of enzyme replacement therapy, many metabolic disorders became treatable, thus establishing an early and accurate diagnosis is extremely important.     

Synthesis of a New Nitrate-Fertilizer Form with a Controlled Release Behavior via an Incorporation Technique into a Clay Material

The current study shows an advanced synesthetic technique of a nitrate-fertilizer with a controlled release behavior into different soils (normal and acidic agriculture soils solutions) at different climate temperatures. The environmentally friendly and the biocompatible layered double hydroxide (LDH) clay material was used as a host to accommodate the nitrate anion into its interlayer gallery by applying a reconstruction-incorporation technique (the memory effect property of LDH that allows calcined LDH to memorize its original structure). The prepared materials were characterized by different spectroscopic techniques including; X-ray powder spectroscopy, IR, SEM, TEM, and TGA analyses. A remarkable loading ratio of the nitrate anion fertilizer was recorded into the LDH structure. Around 90 wt% of the intercalated nitrate anion was released in a sustained controlled behavior over around 24 days, while the same amount of nitrate was released over 10 days in the acidic soil. Accordingly, the present study offers a new passway for the formulation of controlled release fertilizers by using the hosting anionic clay LDH materials.     

Successes and shortfalls of community Plusoptix photoscreening: results from the iSee study in Southwestern Ontario

ObjectiveThe purpose of this work is to provide the final results from a community-wide photoscreening program in Southwestern Ontario for children aged 18 to 72 months, and to estimate the prevalence of amblyogenic risk factors in this population.Study DesignProspective, multisite photoscreening program.Participants5959 children aged 18–72 months were recruited and screened in Southwestern Ontario at 210 locations over a period of 3 years and 4 months.MethodsOphthalmic screening examinations were performed with the Plusoptix S12 photoscreener. The threshold for the referral criteria used was the manufacturer's criteria on receiver operating characteristics 4.ResultsThe screening was negative in 5386 children (90.4%), positive in 403 (6.8%), and unreadable in 170 (2.9%); 42% of all screened children were ≤36 months old. The estimated amblyogenic risk factor prevalence of anisometropia was 4.0%, astigmatism was 3.1%, hyperopia was 1.1%, myopia was 0.4%, and strabismus was 0.4%. Of the 403 referred children, 99 (24.5%) completed a formal eye examination based on the responses returned to the study site.ConclusionsThis is the first Canadian study that provides data on amblyogenic risk factors based on a volunteer-led photoscreening program. Photoscreening is an effective screening tool, particularly for pre-school-aged children; however, the lack of mandatory follow-up to ensure that children receive proper treatment based on cycloplegic refraction reduces the efficacy of screening.     

Identification,isolation, structural characterization,in silico toxicity prediction and in vitro cytotoxicity assay of simeprevir acidic and oxidative degradation products

Simeprevir is a new direct-acting antiviral drug used for the treatment of chronic hepatitis C. In this work, a simple, fast and economical chromatographic method was developed for the determination of simeprevir in the presence of its acidic and oxidative degradation products. The stress studies performed herein showed that simeprevir degraded under acidic and oxidative conditions but was stable under thermal and alkaline conditions. Chromatographic separation was achieved on a reversed-phase Eclipse XDB C₁₈ column (4.6 × 150 mm, 5 μm). The mobile phase consisted of methanol-0.05 M ammonium acetate (pH 4) (90 : 10, v/v) and was used at a flow rate of 1 mL min⁻¹. The column effluent was monitored at 237 nm. The calibration curve was linear over the concentration range of 0.1–20 μg mL⁻¹. The relative standard deviations for the intra-day and inter-day precision were less than 2%, and good percentage recoveries that met the acceptance criteria of the International Conference on Harmonization (ICH) guidelines were obtained. The robustness was assessed using the Plackett–Burman design. The simeprevir degradation products were isolated by flash chromatography and confirmed by ¹H NMR and LC-MS/MS techniques. The fully validated chromatographic method can be applied as a stability-indicating method for simeprevir and for routine analysis during quality control. Additionally, in silico toxicity prediction of the degradation products demonstrated a hepatotoxicity alert for DP 1, DP 2, DP 4 and DP 5 and a carcinogenicity alert for DP 3. In view of safety aspects, an in vitro cytotoxicity assay was carried out for simeprevir degradation products. They were found to be non-toxic in vitro at the tested concentrations.

Simeprevir is a new direct-acting antiviral drug used for the treatment of chronic hepatitis C.     

Neurocognitive Outcome and White Matter Anisotropy in Childhood Acute Lymphoblastic Leukemia Survivors Treated with Different Protocols

Neurocognitive outcome affects the quality of life of ALL survivors. This study is aimed to assess the prevalence of neurocognitive dysfunction by psychometric and imaging tools in survivors of childhood ALL, treated with 3 different protocols and the effect of time elapsed since the end of chemotherapy. Sixty-two ALL survivors aged 6–18 years and treated in the period 1997–2007 and 60 healthy age and sex matched controls were subjected to neurocognitive testing using Wechsler Intelligence Scale for Children, Benton visual retention (BVRT) and Trail Making test (TMT), followed by diffusion weighed and diffusion tensor MRI for calculation of fraction anisotropy (FA). Survivors underwent revision of protocol and type of CNS therapy. Three different protocols were used: modified BFM 83, BFM 90, and CCG. Survivors treated with modified CCG protocol showed a significant decrease in all cognitive tests compared to control (p<.05); BFM 90 group had a significant lower IQ and longer TMT compared to both control and BFM 83 group and no significant difference was found in results of cognitive tests between BFM 83 and control group. Frontal FA was lower in CCG treated group compared to control, BFM 90 and BFM 83 groups (p<.05); meanwhile it was significantly lower in BFM 90 and BFM 83 groups compared to control group. We concluded that patients treated with modified CCG protocol showed the worst neurocognitive outcome among three assessed protocols. Frontal lobe FA might be an early marker for predicting the neurotoxicity in childhood ALL survivors.     

Predictors of cardiac morbidity and related mortality in children with acquired immunodeficiency syndrome

OBJECTIVES: The aim of this study was to determine the prevalence of cardiovascular dysfunction and its predictors in children with acquired immunodeficiency syndrome (AIDS). BACKGROUND: Cardiovascular manifestations are common among children with AIDS but may be clinically occult. METHODS: We reviewed the medical records, echocardiograms, electrocardiograms, and Holter monitor studies of 68 children with AIDS. We tested clinical and demographic characteristics at the time of AIDS diagnosis for their ability to predict serious cardiac events, death, and cardiac death. RESULTS: The median time from AIDS diagnosis to death or end of follow-up was 1.0 year (range, 1 week to 7.9 years). Nineteen patients (28%) experienced serious cardiac events after AIDS diagnosis. Of 43 patients who died, 15 (35%) had cardiac dysfunction. Multivariable analyses revealed that recurrent bacterial infections, wasting, encephalopathy, male gender, and an earlier year of AIDS diagnosis were predictors of serious cardiac events (relative risk [RR] = 9.3, 6.9, 4.7, 4.1, and 0.76, respectively, p < 0.05). Wasting, encephalopathy, a low age-adjusted CD4 count, a low age-adjusted immunoglobulin G (IgG) level, and an earlier year of AIDS diagnosis increased the risk of all-cause mortality (RR = 8.9, 5.1, 2.7, 0.82, and 0.8, respectively, p 相似文献