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Objective: Post hoc analyses evaluated the effect of infliximab upon concurrent perianal Crohn disease (CD) in a subpopulation of 31 patients from REACH, a randomized trial of 112 children with moderately to severely active luminal CD., Materials and Methods: The Pediatric Crohn Disease Activity Index perirectal subscore was used to assess perianal symptom activity and therapeutic response. Patients with no symptoms or asymptomatic tags received a score of 0; those with "1-2 indolent fistula, scant drainage, no tenderness" received a score of 5; and those with "active fistula, drainage, tenderness or abscess" received a score of 10. Initial perirectal subscores of 10 or 5 decreasing to 0 were considered complete response. Subscores of 10 decreasing to 5 were considered partial response. All patients were followed for efficacy and safety through week 54., Results: Twenty-two patients with baseline perianal disease were randomized at week 10 following a 3-dose infliximab induction regimen. At week 2, 40.9% (9/22) of patients with signs and symptoms of perianal disease at baseline attained response (4 partial and 5 complete). At week 54, 72.7% (16/22) of patients with signs and symptoms of perianal disease attained response (1 partial and 15 complete). Nine patients developed perianal signs and symptoms during treatment; 7 had complete response and 2 had no response at week 54. The incidence of adverse events for patients with perianal symptoms at baseline and for those in the overall REACH population was similar (95.7% vs 94.6%)., Conclusions: Infliximab rapidly reduced concurrent perianal disease signs and symptoms in this REACH cohort.

Objectives: To assess the efficacy of lansoprazole for the relief of symptoms due to gastroesophageal reflux disease (GERD) in children 1 to 11 years of age. In addition, the efficacy in healing of erosive esophagitis (EE) was determined in those children with EE who were enrolled in the study., Methods: In this phase I/II, open-label, multicenter (11 sites) U.S. study, children with symptomatic GERD, EE by endoscopy, and/or intraesophageal pH < 4 for greater than 4.2% of the time based on 24-hour pH testing were assigned, on the basis of body weight, to lansoprazole 15 mg (< or = 30 kg) or 30 mg (> 30 kg) once daily for 8 to 12 weeks. At the discretion of the investigator, the dosage of lansoprazole was increased up to 60 mg daily in children who continued to be symptomatic after 2 weeks of treatment. Symptom response was assessed by investigator interview and daily diary. Esophagitis healing was evaluated by repeat endoscopy after 8 and, if applicable, 12 weeks of treatment., Results: Sixty-six children were enrolled. At week 8, 78% (21/27) of the children with EE at baseline had healed; the remaining six children were healed by week 12 (100%, 6/6). By investigator interview, 70% of children experienced resolution or improvement in their overall symptoms of GERD by their final visit. Statistically significant reductions from baseline in the severity of each symptom were reported with the exceptions of wheezing, hematemesis, and melena. Based on daily diary data, improvement in overall GERD symptoms was reported in 76% (47/62) of all children. With few exceptions, significant (P < 0.05) reductions from baseline occurred during each of the 2-week treatment intervals of the study period in the percentage of days and the average daily severity of GERD symptoms, the percentage of days antacid was used, and the average number of antacid tablets used per day., Conclusion: In children 1 to 11 years of age, lansoprazole is efficacious in healing EE and in relieving GERD-related symptoms.

Defining and measuring quality of life is a relatively new dimension of health care for many clinicians. The traditional method of evaluating clinical status and response to treatment has been to look at disease-specific symptoms, global assessments (e.g., impressions of the good, better, or worse condition of a patient), days missed at school, and a variety of disease activity scores. These impressions and techniques may or may not reflect how patients are functioning day to day and how they feel about the illness. To address these issues, health-related quality of life questionnaires have been developed to measure the functional effect of an illness and its treatment on a patient, as perceived by the patient. There are four broad domains that are considered part of a health-related quality-of-life questionnaire: 1) physical and occupational function, 2) psychologic state, 3) social interaction, and 4) somatic sensation. In the case of children, the perception of parents or caretakers may be added to complete the picture. Significant social and psychiatric problems have been described in children with IBD, including absenteeism from school, depression, suicide, and major disruption of family patterns. To understand fully the impact of inflammatory bowel disease and its treatment on patient and family function requires one or more quality-of-life instruments that are sensitive to the full range of symptoms, growth and development, and response (including side effects) to many new therapies.

The safety and efficacy of olsalazine sodium was compared to sulfasalazine over 3 months in a multicenter, randomized, double-blind study of 56 children with mild to moderate ulcerative colitis. Twenty-eight children received 30 mg/kg/day of olsalazine (maximum, 2 g/day) and 28 received 60 mg/kg/day of sulfasalazine (maximum, 4 g/day). Side effects were frequent in both groups. Eleven of 28 patients (39%) on olsalazine reported headache, nausea, vomiting, rash, pruritus, increased diarrhea, and/or fever. Thirteen of 28 on sulfasalazine (46%) reported similar side effects and/or neutropenia, and four patients had the drug stopped because of an adverse reaction. After 3 months, 11 of 28 (39%) on olsalazine were asymptomatic or clinically improved, compared to 22 of 28 (79%) on sulfasalazine (p = 0.006). In addition, 10 of 28 patients on olsalazine versus one on sulfasalazine required prednisone because of lack of response or worsening of colitis (p = 0.005). The dose of olsalazine used in this clinical trial was thought to be equivalent to a standard dose of sulfasalazine, but fewer patients on olsalazine improved and a greater number had progression of symptoms when compared to sulfasalazine. Although side effects were slightly less frequent for olsalazine, the number of patients was too small to detect a clinically significant difference.

The Pediatric Crohn's Disease Activity Index (PCDAI) has been proposed as a simple instrument to aid in the classification of patients by disease severity. The PCDAI includes subjective patient reporting of symptoms, physical examination, nutritional parameters, and several common laboratory tests (hematocrit, erythrocyte sedimentation rate, albumin). In this report we examine the relationship of each of the laboratory parameters to the PCDAI, as well as to a modified Harvey-Bradshaw Index score and physician global assessment of disease activity. Data were gathered from the clinical and laboratory observations from 133 children and adolescents at 12 pediatric gastroenterology centers in North America. A statistically significant relationship (p less than 0.05) was noted between each of the laboratory tests and the PCDAI for patients with either disease limited to the small bowel or in those with colonic involvement. For patients with disease limited to the small bowel, a statistically significant (p less than 0.05) relationship was also noted between the three laboratory parameters and the modified Harvey-Bradshaw Index and global assessment. For patients with large-bowel involvement, the erythrocyte sedimentation rate was statistically related to the modified Harvey-Bradshaw Index and global assessment (p less than 0.01), as was hematocrit to global assessment (p less than 0.01). Although the laboratory parameters used in the PCDAI appear to generally reflect disease activity in most patients, no single laboratory test is adequate to reflect disease activity in all patients. Future work will need to identify additional laboratory measures to reflect the inflammatory process and serve as important adjuncts in the assessment of disease activity.

This case describes a patient with cholesteryl ester storage disease who underwent liver transplantation for progressive cirrhosis, portal hypertension, ascites, and uncontrollable gastrointestinal bleeding. Four and one-half years posttransplant, her growth improved, cholesterol levels have returned to normal, and she is clinically well except for mild hypersplenism and an elevated blood urea nitrogen (BUN) and creatinine. Serum triglycerides remain elevated, but there have been no signs of progressive renal, intestinal, vascular, or pulmonary disease.

When conventional treatment with upright positioning and thickened feedings fails to control vomiting associated with gastroesophageal reflux (GER) of infancy, malnutrition and growth failure may result. If this occurs, fundoplication is usually recommended. In this study, 12 infants with growth failure associated with GER were given a trial of short-term (11.1 +/- SE 1.6 days) continuous-drip nasogastric (NG) feedings prior to surgical referral. Five of 10 infants for whom long-term follow-up (3-12 months) was obtained showed immediate weight gain, cessation of vomiting, and long-term resolution of growth failure without the need for surgery. All infants who had a favorable long-term response showed evidence of catch-up growth during the first 7 days of NG feeding. Infants who did not begin catch-up growth during the first 7 days did not benefit from longer periods (up to 21 days) of NG feeding. Poor response to NG feedings was associated with the presence of other medical problems (p = 0.024), including chronic pulmonary disease, malabsorption, cerebral palsy, and laryngomalacia. Four infants who did require fundoplication still showed no improvement in growth 2-6 months after surgery. In infants with GER and growth failure without other complicating disorders, a 7-10-day course of NG feeding may improve nutrition and bring about a permanent resolution of vomiting.