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Severe bile salt export pump (BSEP) deficiency is a hereditary cholestatic condition that starts in infancy and leads to end-stage liver disease. Three children who underwent orthotopic liver transplantation for severe BSEP deficiency had post-transplantation episodes of cholestatic dysfunction that mimicked the original disease. Remission of all episodes was achieved by intensifying the immunosuppressive regimen. The phenotypic recurrence of the disease correlated with the presence of circulating high-titer antibodies against BSEP that inhibit transport by BSEP in vitro. When administered to rats, these antibodies targeted the bile canaliculi and impaired bile acid secretion., (2009 Massachusetts Medical Society)

Background: The role of early endoscopic retrograde cholangiopancreatography (ERCP) and papillotomy in the treatment of patients who have acute biliary pancreatitis without obstructive jaundice is uncertain., Methods: We conducted a prospective, multicenter study in which 126 patients were randomly assigned to early ERCP (within 72 hours after the onset of symptoms) and endoscopic papillotomy for the removal of stones in the common bile duct, when appropriate, and 112 patients were assigned to conservative treatment. In the conservative-treatment group, ERCP was performed within three weeks if signs of biliary obstruction or sepsis developed. Overall mortality, mortality due to pancreatitis, and complications were compared in the two groups., Results: Early ERCP was successful in 121 of the 126 patients in the invasive-treatment group. Endoscopic papillotomy was performed to remove bile-duct stones in 58 patients; stones were successfully extracted in 57. ERCP was performed in 22 of the 112 patients in the conservative-treatment group; papillotomy for stone removal was successful in 13 patients. Fourteen patients in the invasive-treatment group and 7 in the conservative-treatment group died within three months (P=0.10); 10 patients in the invasive-treatment group and 4 in the conservative-treatment group died from acute biliary pancreatis (P=0.16). The overall rate of complications was similar in the two groups, but patients in the invasive-treatment group had more severe complications. Respiratory failure was more frequent in the invasive-treatment group, and jaundice was more frequent in the conservative-treatment group., Conclusions: In patients with acute biliary pancreatis but without obstructive jaundice, early ERCP and sphincterotomy were not beneficial.

Background: About 90 percent of the deaths from malaria are in African children, but criteria to guide the recognition and management of severe malaria have not been validated in them., Methods: We conducted a prospective study of all children admitted to the pediatric ward of a Kenyan district hospital with a primary diagnosis of malaria. We calculated the frequency and mortality rate for each of the clinical and laboratory criteria in the current World Health Organization (WHO) definition of severe malaria, and then used logistic-regression analysis to identify the variables with the greatest prognostic value., Results: We studied 1844 children (mean age, 26.4 months) with a primary diagnosis of malaria. Not included were 18 children who died on arrival and 4 who died of other causes. The mortality rate was 3.5 percent (95 percent confidence interval, 2.7 to 4.3 percent), and 84 percent of the deaths occurred within 24 hours of admission. Logistic-regression analysis identified four key prognostic indicators: impaired consciousness (relative risk, 3.3; 95 percent confidence interval, 1.6 to 7.0), respiratory distress (relative risk, 3.9; 95 percent confidence interval, 2.0 to 7.7), hypoglycemia (relative risk, 3.3; 95 percent confidence interval, 1.6 to 6.7), and jaundice (relative risk, 2.6; 95 percent confidence interval, 1.1 to 6.3). Of the 64 children who died, 54 were among those with impaired consciousness (n = 336; case fatality rate, 11.9 percent) or respiratory distress (n = 251; case fatality rate, 13.9 percent), or both. Hence, this simple bedside index identified 84.4 percent of the fatal cases, as compared with the 79.7 percent identified by the current WHO criteria., Conclusions: In African children with malaria, the presence of impaired consciousness or respiratory distress can identify those at high risk for death.