Myopathic changes are not uncommon in children with GSD III. Myopathic changes tend to occur in older age and are associated with higher CPK level. Cardiac muscle involvement is less common in this age group and may, on occasion, occur alone without skeletal muscle involvement. Despite mild degrees of affection in this age group, it is recommended to perform prospective annual screening using EMG and echocardiography in order to augment dietary therapy regimen to prevent progression to life threatening complications.

Treatment with all-oral DAAs (SOF/LED) for 12 weeks was well tolerated in Egyptian children and adolescents infected with genotype 4 HCV, with 100% SVR12 and negligible side effects.

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Combined treatment with pegylated interferon (PEG-IFN)-α2b and ribavirin (RBV) is the only currently approved treatment for hepatitis C virus (HCV) infection in children. The aim of this study was to assess the safety and efficacy of combined treatment with PEG-IFN-α2b and RBV in Egyptian children and adolescents with genotype 4 (GT4) HCV infection. The study included 66 patients (3-17 years of age), of both sexes, infected with HCV GT4, treated with PEG-IFN-α2b (60 μg/m(2)), subcutaneously once weekly plus RBV (15 mg/kg/day) in 2 divided oral doses. Efficacy was assessed by achievement of sustained virological response (SVR). Safety was assessed by questionnaires directed to the patients at specific intervals, growth assessment and laboratory tests. SVR was achieved in 28 patients (42.4%). Nonresponders had significantly commoner history of treated malignancies (P = 0.03), baseline lower absolute neutrophil count (ANC; P = 0.009), higher gamma glutamyl transpeptidase (GGT; P = 0.003), and higher viral load (P = 0.03). Fever was the most frequently reported side effect occurring in 98.5% of the patients followed by musculoskeletal symptoms. Neutropenia was observed in 36 patients (54.6%) and necessitated treatment discontinuation in 1 patient. Decline in both weight and height percentiles was observed in 70% of children who received the combined therapy for a total of 48 weeks. In conclusion, the currently available treatment for HCV GT4 in pediatric patients has modest SVR with numerous adverse events necessitating meticulous monitoring to optimize care of the patients. Side effects could be managed with dose modifications and specific treatment when necessary.

Background: The widespread use of abdominal ultrasonography in children documented increased detection rate of gallstones. The aim of this study was to assess the possible risk factors of gallstones and to detect the complication rate and outcome of the disease in Egyptian infants and children from a hepatologist perspective. This prospective study included 35 cases, with ultrasound-detected gallstones, who presented to the Pediatric Hepatology Unit from July 2015 to October 2017. All patients were initially evaluated by full history, general and abdominal examination, laboratory assessment, and ultrasound findings to detect possible risk factors of gallstones. Management approach was individualized for each patient according to his/her condition, and the patients were followed up at 3 months interval. Results: The patients' ages ranged from 3.5 months up to 17 years. The median age of gallstone detection was 2 years. Gallstones were symptomatic in 62.9% with abdominal pain in 51.4%. Complications in the form of choledocholithiasis were detected in 7 patients (20%). Laparoscopic cholecystectomy was done for 7 patients while conservative management was applied for 24 patients. Complete resolution occurred in 8.3% and partial resolution in 33.3%. Chronic hemolytic anemia was the most commonly identified risk factor for gallstones. Patients needed surgical intervention had significant higher serum cholesterol and LDL levels (P = 0.006 and P = 0.002 respectively). Conclusion: Risk factors for gallstones could be identified in most of pediatric cases. Symptomatic presentation is common among the pediatric population. Conservative management is recommended for asymptomatic cases of gallstones in children. Laparoscopic cholecystectomy is a safe and recommended procedure for complicated and symptomatic cases of gallstones.

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Infection with hepatitis C virus (HCV) is a major health problem worldwide. A large proportion of perinatal HCV infections are silent and may present later in adulthood with long-term complications. HCV has no effective immune prophylaxis and hence appropriate follow-up of all infants born to HCV-infected mothers is necessary. Universal antenatal screening for HCV is largely debatable. Intrauterine and partum transmission of HCV are both possible and higher rates are associated with a high maternal serum viral load (> 10 6 copies per milliliter), concomitant HIV infection, prolonged or difficult delivery, and invasive fetal monitoring during delivery. Infection during pregnancy and infancy needs to be investigated more in order to design management strategies for perinatal transmission of HCV most effectively. The recently approved new-generation, oral, direct-acting antiviral drugs may open a new era in HCV therapy for pregnant women and infected infants if proved to be safe during conception and infancy.

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