Immunogenicity of Diphtheria Booster Vaccination in Adolescents With Inflammatory Bowel Disease.

The aim of this prospective study was to assess the immunogenicity and safety of booster vaccine against diphtheria in children with inflammatory bowel disease on and without immunosuppression treatment. Immunoprotection was achieved in 93% of the children. No significant differences depending on the treatment used and no serious adverse events or flares of inflammatory bowel disease were observed.

Immunogenicity of Pertussis Booster Vaccination in Children and Adolescents with Inflammatory Bowel Disease: A Controlled Study.

BACKGROUND: There are limited data on antibody response to vaccination in patients with inflammatory bowel disease (IBD). In this study, we aimed to assess the immunogenicity of a booster dose of pertussis vaccine in pediatric patients with IBD and to compare their response with healthy controls. METHODS: We performed a multicenter, prospective, and controlled trial. Eligible for inclusion were children and adolescents (11-18 year olds), with no history of pertussis booster immunization after the age of 6 years or history of pertussis. Study population was divided into 4 groups: patients with IBD receiving no immunosuppressive therapy (group 1), those on thiopurines only (group 2), those on thiopurines and TNF-α agents (group 3), and healthy controls (group 4). Patients and controls received 1 dose of pertussis vaccine intramuscularly and were asked to record adverse effects for 3 days after vaccination. The primary outcome measure was adequate vaccine response, defined as the concentration of anti-Bordetella pertussis antibodies >11 µg/mL, measured between 4 and 8 weeks after the vaccination. RESULTS: In total, 138 subjects (111 patients and 27 controls) were enrolled in the study. Rates of adequate vaccine response did not differ among the 4 study groups (P = 0.11). Moreover, those patients with IBD who were on immunosuppressive therapy did not differ from those who were not (90.6% versus 88.2%, P = 0.37). No serious adverse effects in relation to the administration of vaccine were noted. CONCLUSIONS: Booster dose of pertussis vaccine was immunogenic and safe in pediatric patients with IBD.

Genetic architecture differences between pediatric and adult-onset inflammatory bowel diseases in the Polish population.

Most inflammatory bowel diseases (IBDs) are classic complex disorders represented by common alleles. Here we aimed to define the genetic architecture of pediatric and adult-onset IBDs for the Polish population. A total of 1495 patients were recruited, including 761 patients with Crohn's disease (CD; 424 pediatric), 734 patients with ulcerative colitis (UC; 390 pediatric), and 934 healthy controls. Allelotyping employed a pooled-DNA genome-wide association study (GWAS) and was validated by individual genotyping. Whole exome sequencing (WES) was performed on 44 IBD patients diagnosed before 6 years of age, 45 patients diagnosed after 40 years of age, and 18 healthy controls. Altogether, out of 88 selected SNPs, 31 SNPs were replicated for association with IBD. A novel BRD2 (rs1049526) association reached significance of P = 5.2 × 10-11 and odds ratio (OR) = 2.43. Twenty SNPs were shared between pediatric and adult patients; 1 and 7 were unique to adult-onset and pediatric-onset IBD, respectively. WES identified numerous rare and potentially deleterious variants in IBD-associated or innate immunity-associated genes. Deleterious alleles in both groups were over-represented among rare variants in affected children. Our GWAS revealed differences in the polygenic architecture of pediatric- and adult-onset IBD. A significant accumulation of rare and deleterious variants in affected children suggests a contribution by yet unexplained genetic components.

Assessment of induction therapy with infliximab in children with moderate to severe ulcerative colitis: a multi-center study.

THE AIM OF THE STUDY: Assessment of clinical and endoscopic efficacy of induction therapy with infliximab in children with ulcerative colitis. MATERIAL AND METHODS: This is a retrospective analysis of medical records of pediatric patients with moderate to severe UC who had received at least one infusion of infliximab in Polish pediatric academic clinical centers from 2003 to 2013. The primary endpoint was clinical remission rate at week 10, (PUCAI score <10 points) while the secondary endpoints were: clinical response rate (>19-points decrease in PUCAI), mucosal response rate (defined as an improvement of the Baron score), and mucosal healing rate (Baron score 0 or 1). RESULTS: 44 patients, at mean age of 14±3.9 years, were included into the study. 38 (86%) patients completed induction therapy regimen with infliximab and were finally included into the analysis. Clinical response and remission rates at week 10 there were 36% and 25% respectively. There was significant drop of PUCAI (58.31±15.5 vs. 24.23±23.83) and Baron score (2.63±0.49 vs. 1.44±0.99) at this time point. Mucosal response and mucosal healing rate were 57% and 48% respectively. Infliximab failure defined as non-clinical and non-mucosal response at week 10, occurred in 16 patients. Infliximab-associated adverse events occurred in 3 patients, with all severe hypersensitivity reactions to infliximab. CONCLUSIONS: Infliximab induction therapy was safe and effective in Polish moderate to severe UC pediatric patients with 50% rate of mucosal improvement. However, clinical response rate was lower than previously reported.

Overweight and obesity in children with newly diagnosed inflammatory bowel disease.

PURPOSE: Determination of overweight and obesity prevalence in children with inflammatory bowel disease (IBD) at the time of diagnosis. MATERIAL AND METHODS: This was a multicenter retrospective study. The study group consisted of children with new cases of IBD diagnosed in 2005-2013 according to the Porto criteria. Hospital admission records were reviewed for demographic and clinical characteristics. BMI-for-age and gender percentile charts were used to define overweight as ≥85th BMI percentile and obesity as ≥95th BMI percentile. RESULTS: 675 patients were evaluated: 368 with Crohn's disease (CD) and 307 with ulcerative colitis (UC). Of these, 54.8% were boys and 45.2% were girls. There were no statistically significant differences in age, weight, height and disease activity between the CD and UC patients. The UC patients had higher BMI values than the CD patients. The prevalence of overweight and obesity was higher in the UC than the CD patients (4.89% CI95 2.76-7.93 vs. 2.45% CI95 1.12-4.59 and 8.47% CI95 5.61-12.16 vs. 1.9% CI95 0.77-3.88, respectively); the differences were statistically significant (-2.44% CI95 -5.45 to 0.49 and -6.57% CI95 -10 to -3.1, respectively). The risk of overweight/obesity was 3.5 times higher for patients with UC (OR=0.272, CI95 0.14-0.49, p=0.0004). CONCLUSIONS: The prevalence of overweight and obesity in newly diagnosed children with IBD was 8.4% and was higher in patients with UC than in patients with CD. The results of this study have shown that not only malnourished children may suffer from IBD but also children who are overweight or obese at the time of diagnosis.

Juvenile polyposis syndrome.

Juvenile polyposis syndrome (JPS) is an autosomal dominant predisposition to the occurrence of hamartomatous polyps in the gastrointestinal tract. Diagnosis of JPS is based on the occurrence of numerous colon and rectum polyps or any number of polyps with family history and, in the case of juvenile polyps, their occurrence also outside the large intestine. The JPS is caused by mutations in SMAD4 and BMPR1A. Products of the SMAD4 gene are involved in signal transduction in the transforming growth factor ß pathway and BMPR1A protein is a receptor belonging to the family of transmembrane serine/threonine kinases. Both proteins are responsible for processes determining appropriate development of colonic mucosa. The JPS belongs to the group of hamartomatous polyposes. The hamartomatous polyposis syndromes constitute a group of diseases in which manifestations differ slightly and only molecular diagnostics gives the possibility of verifying the clinical diagnosis.

Liver disease in cystic fibrosis.

Cystic fibrosis-associated liver disease (CFLD) affects ca. 30% of patients. The CFLD is now considered the third cause of death, after lung disease and transplantation complications, in CF patients. Diagnostics, clinical assessment and treatment of CFLD have become a real challenge since a striking increase of life expectancy in CF patients has recently been observed. There is no elaborated "gold standard" in the diagnostic process of CFLD; clinical evaluation, laboratory tests, ultrasonography and liver biopsy are used. Clinical forms of CFLD are elevation of serum liver enzymes, hepatic steatosis, focal biliary cirrhosis, multilobular biliary cirrhosis, neonatal cholestasis, cholelithiasis, cholecystitis and micro-gallbladder. In children, CFLD symptoms mostly occur in puberty. Clinical symptoms appear late, when damage of the hepatobiliary system is already advanced. The CFLD is more common in patients with severe mutations of CFTR gene, in whom a complete loss of CFTR protein function is observed. CFLD, together with exocrine pancreatic insufficiency and meconium ileus, is considered a component of the severe CF phenotype. Treatment of CFLD should be complex and conducted by a multispecialist team (gastroenterologist, hepatologist, dietician, radiologist, surgeon). The main aim of the treatment is to prevent liver damage and complications associated with portal hypertension and liver cirrhosis. Ursodeoxycholic acid is used in the treatment of CFLD. There is no treatment of proven long-term efficacy in CFLD. Liver transplantation is a treatment of choice in end-stage liver disease.

Disease specific knowledge about cystic fibrosis, patient education and counselling in Poland.

INTRODUCTION AND OBJECTIVE: The presented study assesses levels of specific knowledge of the disease among cystic fibrosis (CF) patients and their families, and evaluates the effectiveness of a targeted, disease specific education programme. MATERIALS AND METHODS: A cross-sectional survey among 462 families with a CF child evaluated their knowledge of the disease. A one year follow up survey among 200 families assessed the effectiveness of an educational programme developed to correct gaps, errors and misconceptions identified in the previously administered survey. Self-administered, comprehensive, 5-domains, 45-item multiple-choice CF Disease Knowledge Questionnaire (CFDKQ) was anonymously completed by 462 subjects. RESULTS: 228 respondents were male (49%), 234 female (51%). The level of disease-specific knowledge in the age groups 0-6 and 7-10 years, was significantly higher than in 11-14 and 15-18 years of age groups (p<0.005). General medical and Genetics/Reproduction knowledge was low in all patients. Significant predictors of patient and parental knowledge were age and domicile. Patients and parents rely heavily on doctors for information about CF (77%). The follow-up survey (CFDKQ) emphasized that special education programmes significantly improved levels of disease specific knowledge (p<0.0001). CONCLUSIONS: If left uncorrected, the misconceptions, gaps and errors in CF knowledge identified in the presented study could result in inadvertent non-adherence to treatment, and impact on the progression and outcome of the disease. Secondly, the results demonstrate the effectiveness of targeted, disease specific information in improving disease knowledge of CF patients and their families, and highlights the value and need for the development of educational programmes for chronically ill patients and their families.

Prevalence and correlates of vitamin K deficiency in children with inflammatory bowel disease.

Although vitamin K deficiency has been implicated in adult inflammatory bowel disease (IBD), its prevalence in pediatric IBD remains unknown. We carried out a cross-sectional study in 63 children with Crohn's disease (CD) and 48 with ulcerative colitis (UC) to assess the prevalence of vitamin K deficiency and to search for potential correlation between vitamin K status and pediatric IBD activity. Vitamin K status was assessed using protein induced by vitamin K absence-II (PIVKA-II; ELISA). Prevalence of vitamin K deficiency was 54.0% in CD and 43.7% in UC. Vitamin K deficiency was more common in patients with higher CD activity, in CD patients with higher mass Z-scores, and less common among children with CD treated with infliximab. Relation of vitamin K deficiency to pediatric IBD clinical course and treatment demand further research.

Influenza vaccination coverage in children with inflammatory bowel disease.

The aim of this study was to evaluate the influenza vaccination status among paediatric patients with inflammatory bowel disease (IBD) in Poland. This was a questionnaire-based study. 242 patients with IBD and 142 controls were enrolled in the study. Of patients with IBD, 7.8% received an influenza vaccine, compared to 18.3% of controls (P = 0.0013). There were no statistically significant differences in time from IBD diagnosis, disease activity and in drugs, between vaccinated and non-vaccinated IBD children. In conclusion, the data of our study demonstrate an alarmingly poor influenza vaccination status in the majority of children with IBD. Therefore, there is an unmet need to implement better influenza vaccination strategies for this group of patients.