Primary hyperoxalurias are rare inborn errors of metabolism with deficiency of hepatic enzymes that lead to excessive urinary oxalate excretion and overproduction of oxalate which is deposited in various organs. Hyperoxaluria results in serious morbidity, end stage kidney disease (ESKD), and mortality if left untreated. Combined liver | 5 of 6 KOTB eT al.

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The off-label use of medications is a “right” for pediatricians, owing to lack of enough safety and effectiveness drug trials in pediatric age group. Pediatricians have to rely on their personal judicial use of medications in children. We studied off-label use of ursodeoxycholic acid (UDCA) retrospectively during 2005 to 2015 among those who attended the Pediatic Hepatology Unit, Cairo University. We analyzed data of 779 neonates and infants with cholestasis. 15% dropped out. Males comprised 374 (56.5%). Cholestasis was due to surgical causes in 129 (19.5%), neonatal hepatitis in 445 (67.2%), and paucity of intrahepatic bile ducts in 88 (13.3%). Three hundred sixty (54.4%) received UDCA (15–30 mg/kg/d), and 302 (45.6%) did not. Both groups were matched as regards causes and severity of cholestasis. Those who received UDCA had worse outcome (P < .001), and more complications (P < .001). A total of 73.1% (221) achieved cure without UDCA compared to only 45.8% (165) of those on UDCA (P < .001). UDCA is not effective and not safe in Egyptian neonates and infants with cholestasis. UDCA use compromises chance of cure, and is associated with serious morbidity, progression of disease, and death. UDCA off-label use mortality was absolutely preventable. Off- label use of UDCA in neonates and children should be utterly prohibited. Information of use of off-label medications, effectiveness, and safety, should be recorded, analyzed, and made available within context of Off-label Use Registry Studies with informed consent of parents.

Background: With an incidence of twelve tuberculosis (TB) cases for every 100,000 persons in 2019, Egypt is considered a medium-burden country. Nevertheless, guidelines and studies for admission of pediatric TB cases are lacking in developing countries. Aim of the Work:To describe the clinical presentations of hospitalized pediatric TB cases. Materials and Methods: A retrospective analytical study of data collected from the inpatient medical files of children with confirmed TB, from the archives of Giza Chest Hospital (Omraneya), Egypt was implemented during January 2009 to the end of 2018. Results: Of the 76 children hospitalized with TB, 42 were males with a male to female ratio of 1.2:1, 61 (80.3%) patients were from rural areas, and 65 (85.5%) were above six years of age. Extra-pulmonary TB was more common (45 patients; 59.2%) than pulmonary TB (31 patients; 40.8%). Tuberculous LN affection was (N=20) 26.2% of total patients, followed by pleural affection (N=17), then affection of bones (N=4). CNS involvement was among least common presentations (N=4; 5.3%), but was the only type of TB to occur more in preschool children. Moreover, there was an annual rate of change of -2.714 in the recorded numbers of pediatric TB inpatients with no cases in the last two years. Conclusion: Extra-pulmonary TB was more common than pulmonary TB among the hospitalized children. Lymph nodes were the most common extra-pulmonary site of affection. School-aged children were a majority as well as rural residents. These findings warrant evoking awareness among physicians about the various presentations of pediatric TB.

PurposeWe aimed to define the clinical presentations, course and outcome of cholestasis in infants with Down syndrome (trisomy 21) who presented to the Pediatric Hepatology Clinic, New Children Hospital, Cairo University, Egypt.MethodsRetrospective analysis of data of cohort of infants with Down syndrome and cholestasis who followed up during 2005–2015.ResultsAmong 779 infants with cholestasis who presented during 2005–2015, 61 (7.8%) had Down syndrome. Six dropped out. Among the 55 who followed-up for a mean duration +SD = 12.1 ± 16.7 months, none had extrahepatic biliary atresia (EHBA), 37 (63.3%) had neonatal hepatitis and 18 (32.7%) had non-syndromic paucity of intrahepatic biliary radicals. Fourteen (25.4%) had associated congenital heart disease. Only 35 (63.3%) cleared the jaundice. Twenty-nine (52.7%) received ursodeoxycholic acid (UDCA); of them, 13 cleared the jaundice, one improved, 14 progressed and one died, compared to 22 who cleared the jaundice of the 26 who did not receive UDCA. Only three of those who did not receive UDCA progressed and none died. UDCA carried a 3.4-fold risk of poor prognosis (p= 0.001). UDCA use was associated with more complications (p= 0.016) in those with Down syndrome and cholestasis.ConclusionWe did not come across EHBA among neonates and infants with Down syndrome in 10 years. Non-syndromic paucity is associated with favorable outcome in infants with Down syndrome. UDCA use in cholestasis with Down syndrome is associated with poor outcome.