Primary Prophylaxis for Gastrointestinal Bleeding in Children With Biliary Atresia and Portal Hypertension Candidates for Liver Transplantation: A Single-Center Experience.

BACKGROUND: Cirrhosis for biliary atresia (BA) is associated with risk of gastrointestinal bleeding (GB) from gastroesophageal varices due to portal hypertension. Primary prophylaxis of GB is controversial in children who are candidates for liver transplantation (LT). The aim of the study was to define the management of gastroesophageal varices and to identify the benefit of primary prophylaxis for GB in BA children waiting for LT. METHODS: A retrospective single-center study including all BA children listed for LT in 2008-2016. Clinical, endoscopical, and biochemical data were analyzed. RESULTS: Of 82 children, 50 (61%) did not receive primary prophylaxis and did not present any episode of bleeding, 16 (19.5%) underwent primary prophylaxis, and 16 (19.5%) presented spontaneous GB and received secondary prophylaxis. Children without primary prophylaxis and GB were younger than patients with primary prophylaxis and those with GB (7.7 years [range, 4.1-37.9 years] vs 11.2 years [range, 5.1-43 years]; P = .03 vs 10.7 years [range, 6.9-39.9 years], respectively; P = .004). Seventy-five percent of GB occurred in children older than 8 months. Fifteen (93.8%) children with GB presented esophageal varices (grade III = 10 [62.5%]) and 10 (62.5%) required endoscopic treatments, consisting mainly of sclerotherapy. Median time to LT was similar for children with or without bleeding (2 months [range, 0-17.7 months] vs 2.2 months [0-17.9 months], respectively; P = .89). After 45.5 months (range, 13.7-105.5 months) of follow-up, the overall patient survival was 97.6%. At the intention-to-treat analysis, the survival rate was 100% for patients without bleeding episode and 87.5% for children with GB (P = .16). CONCLUSIONS: Despite the risk of GB being not clinically predictable in children with BA waiting for LT, our experience suggests that primary prophylaxis of GB might be unnecessary in children younger than 6 months, while it should be considered in older children. Thus, the occurrence of GB does not delay the timing of transplantation.

Prevalence of life-threatening complications in pediatric patients affected by intestinal failure.

Intestinal failure (IF) is defined as the reduction of functional gut mass necessary to maintain health and growth in children. Causes of IF include short bowel syndrome (SBS), neuromuscular intestinal disorders (NID), and severe protracted diarrhea (SPD). If patients require long-term parenteral nutrition (PN); they can now be discharged on home PN (HPN), thus improving their quality of life. Children requiring long-term PN are at high risk of developing life-threatening IF complications that hinder HPN, namely, IF associated liver disease (IFALD), catheter-related infections (CRI), and thrombosis. The goal of our study was to retrospectively evaluate the prevalence of life-threatening complications among IF patients according to the HPN indication. From January 1989 to May 2006, 60 IF patients (41 boys and 19 girls) underwent prolonged HPN. Total program duration was 46,391 days (127 total years, mean 2.1 years per patient). Indications for HPN were SBS in 36 cases, SPD in 19 cases, or NID in 5 cases. In our experience patients affected by SBS displayed a significantly higher prevalence of life-threatening complications than patients with other IF causes. Sixteen (27%) among 60 patients developed IFALD. CRI and thrombosis prevalence were 1.4/1000 central venous catheter (CVC) days and 0.2/1000 CVC days respectively. SBS seemed to lead to life-threatening complications more often than other HPN indications. SBS patients on long-term PN therefore require careful management to identify complications early, and they seem to be the candidates for early referral to small bowel transplantation centers.

Association between celiac disease and primary lactase deficiency.

Primary lactase deficiency (PLD) is a common inherited condition caused by a reduced activity of lactase. Two single-nucleotide polymorphisms C/T(-13910) and G/A(-22018) upstream of the lactase gene are associated with lactase nonpersistence. In celiac disease (CD) patients, lactose intolerance could be due to secondary lactase deficiency and to PLD. The aim of this study were to evaluate the association of PLD and CD using genetic test, and to define the prevalence of PLD in celiac subjects compared with a control population. A total of 188 controls and 92 biopsy-proven CD patients were included in the study. More than 70% of all subjects were found homozygous for the polymorphisms. Differences in the prevalence of PLD were not found between CD patients and controls.In conclusions, the hereditary lactase deficiency is frequent in Italian CD children as in control population.

Diagnostic work-up of inflammatory bowel disease in children: the role of calprotectin assay.

BACKGROUND: Some reports highlight the potential application of fecal calprotectin as a direct biomarker of intestinal inflammation and, therefore, as support in choosing candidates for endoscopy. The value of 100 µg/g was recently assumed as the best cutoff for this assay. The purpose of this study was to assess the diagnostic precision of the fecal calprotectin assay, compared to histology, as a stool-screening biomarker for inflammatory bowel disease (IBD) among a group of prospectively identified patients referred for recurrent abdominal pain and altered bowel habits. METHODS: Between 1999 and 2007 we prospectively evaluated the calprotectin assay in a cohort of patients with recurrent abdominal pain and altered bowel habits associated or not with other symptoms suggestive of IBD. All patients suspected of IBD, according to Rome and Porto criteria, provided stool specimens for the calprotectin assay and subsequently underwent endoscopic procedures. RESULTS: Compared to histology, the cutoff of 100 µg/g reached a sensitivity and specificity of 100% and 68%, respectively, and a likelihood ratio (LR) of 3.1. The cutoff value of 160 µg/g, however, in our series produced the best joint estimate of sensitivity and specificity: 100% and 80%, respectively, with an LR of 5. CONCLUSIONS: In pediatric patients with recurrent abdominal pain and changes in stool habits, a positive calprotectin assay is closely associated with IBD; its systematic employment, therefore, seems to improve the process of endoscopy referral. This test, simple and inexpensive, could be included in the first noninvasive phase of an IBD diagnostic work-up.

Positive impact of blocking tumor necrosis factor alpha on the nutritional status in pediatric Crohn's disease patients.

BACKGROUND: TNFalpha seems to contribute to inflammation and malnutrition in Crohn's disease (CD) patients. In CD patients, the comparative effects on nutritional status of infliximab and traditional therapy have not yet been determined. The aim of our study was to assess the effects of infliximab as compared with those of standard therapy on nutritional status, disease activity, resting energy expenditure (REE), and food intake in CD children and adolescents. METHODS: From September 1999 to September 2005, all CD patients treated with infliximab (group A) were reviewed and matched with CD patients treated with traditional therapy (mesalazine and azathioprine) (group B). RESULTS: Fourteen CD patients from group A and 14 from group B were included; median interval before follow-up investigation was 10 months. Baseline and final values of weight, height, body mass index (BMI), pediatric CD activity index (pCDAI), REE, and food intake were studied. In treated patients, but not in control group, mean baseline weight (kg) and BMI values, 39.7 +/- 13.1 and 17.9 +/- 3.3, respectively, were significantly lower than their final values 42.6 +/- 13.2 and 18.9 +/- 3.1, and median pCDAI values 23.5 were significantly higher than their final values 10 (P < 0.05). Significant changes in height, REE, and food intake were not found in either group. CONCLUSIONS: In pediatric CD patients, infliximab seems to impact positively on the nutritional status as demonstrated by the improvement in weight and BMI, but not in linear growth; effects on nutritional status seem to be due to amelioration of disease activity, rather than to REE reduction or food intake increase.

Clinical role of calprotectin assay in determining histological relapses in children affected by inflammatory bowel diseases.

BACKGROUND: Inflammatory bowel diseases (IBD) are characterized by periods of remission with recurrent episodes of symptom exacerbation because of acute intestinal inflammation, which is correctly evaluated by endoscopy with biopsy sampling. However, many surrogate markers of intestinal inflammation, including fecal calprotectin (FC), are detected as potential predictors of mucosal inflammation in IBD patients. The aim of our study was to retrospectively assess the clinical efficacy of the calprotectin assay in determining histological relapses of pediatric IBD patients. METHODS: We retrospectively reviewed the histological examinations, clinical records, and FC values of patients who had undergone colonoscopy at our hospital over an 8-year period, from December 31, 1998, to December 31, 2006. Only patients with a first histological examination showing a quiescent IBD who submitted to a second histological examination during the next 3 years were selected. RESULTS: Seventy-three IBD patients, all with a first biopsy showing a quiescent IBD, were studied; at the second histological examination, 32 presented with relapse and 41 presented with remission. Relapsed patients showed significantly increased FC levels compared with nonrelapsed patients. A FC value of 275 mug/g achieved sensitivity and negative predictive value of 97% and specificity and positive predictive value of 85% in predicting histological relapse. CONCLUSIONS: FC seems to be a direct measure of intestinal inflammation and therefore a good marker of the risk of histological relapse in pediatric IBD patients. The application of this test in clinical practice may enable the avoidance of invasive tests as well as targeting treatment.

Clinical efficacy and safety of parenteral nutrition in adolescent girls with anorexia nervosa.

PURPOSE: Anorexia nervosa (AN) is a common chronic disorder characterized by severe malnutrition and psychological disturbances. Parenteral nutrition (PN) is not usually used in nutritional rehabilitation of AN. The aim of our study was to retrospectively evaluate the indications, clinical efficacy, and safety of PN as assessed by short- and long-term outcomes in AN inpatient girls. METHODS: During the last 10 years a total of 198 inpatients were included in our study: 104 (53%) received oral and parenteral refeeding (group A) and 94 (47%) oral refeeding alone (group B). For each nutritional treatment, clinical efficacy was evaluated by short- and long-term outcomes, and safety was assessed by complication rate. RESULTS: Short-term outcome assessment indicated weekly weight gain and maximum caloric intake to be higher in PN-treated patients. Long-term outcome evaluation showed rehospitalization and recovery rate to be similar in the two groups, but failure of first nutritional rehabilitation requiring PN significantly greater in group B (17.5%) than in group A (3%) (p = .01). The number of complications was significantly higher in group A than in group B (p = .004), although all complications resolved. CONCLUSION: Among all nutritional rehabilitation strategies, PN offers an alternative and safe way to successfully treat AN patients. Presence of clinical complications and reduced compliance with individual, group, and family therapy seem to be the main indications for PN, as it promptly improves nutritional status. At pediatric and adolescent ages, psychological disturbances can also contraindicate the use of enteral nutrition, and therefore represent an additional indication for PN.