Infliximab for the treatment of pediatric ulcerative colitis.

Ulcerative colitis is a chronic, idiopathic, inflammatory disease of the colon and rectum that may be associated with growth failure, nutritional derangements and psychosocial ramifications in affected children. Multiple medical options are available to achieve disease remission; however, some of these medications can have unwanted side effects, especially in younger patients. With increased understanding of the etiology of the disease, newer therapeutic alternatives have arisen in the form of biologic therapies, namely monoclonal antibodies targeted to a specific protein or receptor. Specifically, infliximab, an anti-TNF-α agent, has been shown to be safe and effective for the treatment of moderate-to-severe pediatric ulcerative colitis.

Genetic modifiers of nutritional status in cystic fibrosis.

BACKGROUND: Improved nutrition early in life is associated with better pulmonary function for patients with cystic fibrosis (CF). However, nutritional status is poorly correlated with the CFTR genotype. OBJECTIVE: We investigated the extent to which modifier genes influence nutrition in children with CF. DESIGN: BMI data were longitudinally collected from the CF Twin-Sibling Study and Cystic Fibrosis Foundation Patient Registry for twins and siblings from 2000 to 2010. A nutritional phenotype was derived for 1124 subjects by calculating the average BMI z score from 5-10 y of age (BMI-z(5to10)). The genetic contribution to the variation in BMI-z(5to10) (ie, heritability) was estimated by comparing the similarity of the phenotype in monozygous twins to that in dizygous twins and siblings. Linkage analysis identified potential modifier-gene loci. RESULTS: The median BMI-z(5to10) was -0.07 (range: -3.89 to 2.30), which corresponded to the 47th CDC percentile. BMI-z(5to10) was negatively correlated with pancreatic insufficiency, history of meconium ileus, and female sex but positively correlated with later birth cohorts and lung function. Monozygous twins showed greater concordance for BMI-z(5to10) than did dizygous twins and siblings; heritability estimates from same-sex twin-only analyses ranged from 0.54 to 0.82. For 1010 subjects with pancreatic insufficiency, genome-wide significant linkage was identified on chromosomes 1p36.1 [log of odds (LOD): 5.3] and 5q14 (LOD: 5.1). These loci explained ≥16% and ≥15%, respectively, of the BMI variance. CONCLUSIONS: The analysis of twins and siblings with CF indicates a prominent role for genes other than CFTR to BMI variation. Specifically, regions on chromosomes 1 and 5 appear to harbor genetic modifiers of substantial effect.

Pediatric ulcerative colitis: current treatment approaches including role of infliximab.

Ulcerative colitis is a chronic inflammatory bowel disease that can lead to derangements in the growth, nutritional status, and psychosocial development of affected children. There are several medical options for the induction and maintenance of disease remission, but the benefits of these medications need to be carefully weighed against the risks, especially in the pediatric population. As the etiology of the disease has become increasingly understood, newer therapeutic alternatives have arisen in the form of biologic therapies, which are monoclonal antibodies targeted to a specific protein or receptor. This review will discuss the classical treatments for children with ulcerative colitis, including 5-aminosalicylates, corticosteroids, thiopurine immunomodulators, and calcineurin inhibitors, with a particular focus on the newer class of anti-tumor necrosis factor-α agents.

Nutritional outcomes following gastrostomy in children with cystic fibrosis.

OBJECTIVE: In 2005 the Cystic Fibrosis (CF) Foundation recommended that children with CF maintain a body mass index (BMI) ≥ 50th percentile. Our study evaluated if gastrostomy (GT) placement increases the likelihood of reaching that goal compared to a standardized nutrition protocol. STUDY DESIGN: Retrospective study of 20 children with CF ages 2-20 years with GTs placed from 2005 to 2010. Each case was pair-matched on age, sex, pancreatic status, BMI, and lung function with a nonGT child with CF. Outcome measures included nutritional status and lung function at 6 months and 1 year. RESULTS: At baseline, mean ± SD BMI Z-scores were similar (cases -1.19 ± 0.60, controls -1.10 ± 0.50; P = 0.10). Cases had a significant 6-month increase in mean BMI Z-score to -0.29 ± 0.84 compared to -1.02 ± 0.67 for controls (P < 0.001). By 1 year, the change in mean BMI Z-score was less different (cases -0.41 ± 0.76, controls -0.71 ± 0.51; P = 0.07). Both groups had stable lung function. From exact logistic regression analysis, the odds ratio for cases compared to controls of reaching BMI ≥ 50th percentile was 9.70 (95% CI: 1.05-484.7; P = 0.04) at 6 months and 3.65 (95%CI: 0.69-25.86; P = 0.16) at 1 year. CONCLUSION: Our study suggests that children with CF who receive GTs are more likely to achieve BMI ≥ 50th percentile than matched children without GTs.