Liver transplantation for fulminant Wilson's disease in children.

BACKGROUND: Fulminant Wilson's disease (FWD) is rare and fatal condition in children unless liver transplantation is performed, however introduction of new technologies could change this poor prognosis. The aim of our study was retrospective analysis of clinical course, treatment and outcome of children with FWD treated in our institution. MATERIAL/METHODS: Between 1999-2007 we've treated in our hospital 13 patients with mean age of 15.5 yrs with FWD. We performed retrospective analysis of clinical course, biochemical parameters, MELD/PELD score, Wilson score and Kings'-College criteria for LTx in acute liver failure in all these patients. Type of treatment and final outcome were analyzed, as well as qualification for transplantation was reevaluated in each case in accordance to pathological examination of explanted during transplantation livers. RESULTS: The initial symptoms of FWD were typically weakness, abdominal pain and developing later after 5-60 days (mean 20 days), jaundice. Eleven patients developed neurological symptoms with coma lasting for 2-11 days before transplantation or death. Maximal serum bilirubin concentration ranged between 4.5-71.6 mg% (mean 42.24 mg%), INR 2.9-10.0 (mean 5.4). MELD/PELD score was between 21-58 (mean 38), 10 patients fulfilled general King's-College criteria for transplantation in acute liver failure. Wilson's index ranged between 11 and 17 points (mean 13 points). In 11 children urgent liver transplantation (LTx) was performed, 1 child recovered on albumin dialysis and chelating treatment, 1 child died shortly after very late referral to our center. Actual follow-up of living patients is 0.36-7.43 years (mean 2.57 yrs), all are doing well with good liver function. CONCLUSIONS: FWD lead to death in almost all pediatric patients if LTx can not be performed, however early introduction of albumin dialysis (MARS) and chelating therapy allowed for survival without transplantation in single patient. It seems also that MARS therapy allows for at least prolongation of waiting time for LTx. Wilson's was slightly better predictor of need for LTx in our patients than classical King's-College criteria.

Short-term effects of parenteral nutrition of cholestatic infants with lipid emulsions based on medium-chain and long-chain triacylglycerols.

OBJECTIVE: Infants with chronic cholestasis may require parenteral nutrition with lipid emulsions to provide energy and essential fatty acids but the optimal strategy is controversial. METHODS: We studied the effects of parenteral lipid emulsions with long-chain triacylglycerols (LCTs) or a mixture of LCTs and medium-chain triacylglycerols (MCTs/LCTs) on serum bilirubin and lipid metabolism in cholestatic infants who received these 20% emulsions in alternating order for 3 d each, together with a glucose and amino acid infusion. RESULTS: Of 11 recruited infants, two dropped out because enteral feeding could be established. In nine infants (2-8 mo of age, mean age 4.2 mo) who completed the study, serum bilirubin decreased from baseline to 6 h after the end of LCT infusion (from 8.5 +/- 2.0 to 7.8 +/- 1.8 mg/dL, mean +/- SEM, P < 0.05) and MCT/LCT infusion (7.9 +/- 6.5 to 7.1 +/- 6.5 mg/dL, P < 0.05). Cholesterol, triacylglycerol, and phospholipid concentrations in plasma and in chylomicrons, very low-density lipoprotein, low-density lipoprotein, and high-density lipoprotein were not changed by either emulsion. Total polyunsaturated fatty acid contents in high-density lipoprotein phospholipids increased during LCT infusion (from 29.8 +/- 0.9 to 35.9 +/- 1.4% wt/wt, P < 0.05) and MCT/LCT infusion (from 30.4 +/- 1.0 to 33.0 +/- 0.7%, P < 0.05). The long-chain polyunsaturated fatty acid docosahexaenoic acid increased only with the LCT infusion. Because docosahexaenoic acid availability during infancy is important for early visual and cognitive development, the use of soybean oil-based lipid emulsions may be preferable for infants with severe progressive cholestasis. CONCLUSION: The MCT/LCT and LCT emulsions showed a good metabolic tolerance in infants with chronic cholestasis but had a differential effect on high-density lipoprotein phospholipid contents of arachidonic and docosahexaenoic acids.

Long-chain PUFA supplementation improves PUFA profile in infants with cholestasis.

Long-chain PUFA (LCP) deficiency is a frequent complication in cholestatic infants. We investigated the effects of LCP-supplemented formula on EFA status in infants with cholestasis. Twenty-three infants with cholestasis (biliary atresia after surgery, 8; intrahepatic cholestasis, 15) aged 1.9 to 4.9 mon (median 3.1 mon) were randomized to receive commercial infant formulas either without LCP or with LCP from egg phospholipids for 1 mon. Liver tests, nutrient intakes, and plasma phospholipid FA (%w/w) were determined at baseline and after intervention. At baseline, patients had high serum direct bilirubin levels (5.9 +/- 3.0 mg/dL; mean +/- SD), they were malnourished (body fat mass: 40 +/- 13% of normal) and presented with PUFA deficiency [plasma phospholipid PUFA: 28.43%w/w (26.56-30.53) in patients vs. 37.02%w/w (34.53-39.58) in controls; median (1st-3rd quartile)] with elevated Mead acid and palmitoleic acid. LCP-supplemented (n = 11) and -nonsupplemented groups (n = 12) did not differ in age, indicators of liver function, and EFA status at baseline. After the intervention, LCP-supplemented infants had higher levels of arachidonic acid [7.2 (5.9-8.8) vs. 4.2 (3.0-5.3) %w/w; P < 0.001] and DHA [2.8 (2.2-3.2) vs. 1.6 (1.0-2.1) %w/w; P < 0.05], accompanied by increased TBARS concentration: 1.9 (1.4-2.2) vs. 1.3 (1.1-1.6) nmol/mL; P < 0.05]. We concluded that LCP-supplemented formulae improve LCP status of infants with severe cholestasis but may enhance lipid peroxidation.