Ursodeoxycholic acid use is associated with significant risk of morbidity and mortality in infants with cholestasis: A strobe compliant study.

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.

Natural history and response to treatment of HCV infection among Egyptian survivors of childhood malignancy.

BACKGROUND: Children with cancer are at a high risk for hepatitis C virus infection due to immunosuppression secondry to chemotherapy and multiple transfusions of blood products. We aim to evaluate the presence of HCV infection in children with malignant diseases, risk factors, clinical course, laboratory, histopathological findings, and response to HCV treatment. METHOD: We described 31 patients referred to the pediatric hepatology clinic at Cairo University pediatric hospital and presenting with postmalignant virus C infection. Data collected included that of medical history, physical examination, and periodic evaluation clinically, laboratory, and histopathologically during their follow up. RESULTS: The mean age at diagnosis of HCV infection was 8 ± 3.3 years, the period of follow up of the patients in the hepatology clinic ranged from 0.3 to 15 years with a mean of 2.6 ± 2.3 years. Risk factors for HCV acquisition were chemotherapy in 93.5%, blood transfusions in 83.9%, and operations in 64.5%. Out of the 31 cases, 51.6% had leukemia. At first presentation, serum ALT level was elevated in 83.9% and AST level was elevated in 80.6%. Liver biopsy was performed in 26 cases; 96.1% had mild to moderate activity, 32% had no fibrosis, and 68% had mild to moderate fibrosis. Eighteen cases received HCV treatment. The response to HCV treatment was 27.7%. Although hepatitis C infection acquired by childhood cancer survivors was presented initially with high rate of elevated liver enzymes and PCR positivity, it seems to have a relatively benign clinical course with mild to moderate chronic hepatitis.