This subproject is one of many research subprojects utilizing the resources provided by a Center grant funded by NIH/NCRR. The subproject and investigator (PI) may have received primary funding from another NIH source, and thus could be represented in other CRISP entries. The institution listed is for the Center, which is not necessarily the institution for the investigator. Crohn's disease is the most common type of inflammatory bowel disease (IBD). In a recent meta-analysis, the number of Crohn's patients in North America is estimated to be between 400,000 and 600,000. While the disease can affect any age group, it is most commonly diagnosed in young adults between the ages of 15 and 35, with average age at onset of 22 to 23 years. Overall, approximately 25% of new cases in the population occur in individuals below 20 years of age. Most pediatric cases are diagnosed between the ages of 10 and 19 years; Crohn's disease in infants and young children occur less frequently. Crohn's disease is a serious, life-long illness. It is characterized by a transmural, usually granulomatous, inflammation that leads to ulceration of the mucosa, intestinal stricture, and fistula formation. In many cases of pediatric Crohn's disease, the onset is insidious with nonspecific gastrointestinal (GI) symptoms or extraintestinal manifestations leading to delayed or incorrect diagnosis. The most common presenting symptoms of the disease include diarrhea, abdominal pain, fever, and weight loss. Symptoms associated with small bowel involvement or upper GI involvement include postprandial cramping, early satiety, nausea, anorexia, and diarrhea. As with adults, there is considerable variability in the clinical course of Crohn's disease in children. The general principles of treatment of pediatric Crohn's disease mirror those that are applicable to adults. However, the management of pediatric Crohn's disease presents greater and unique challenges because of the impact of the disease on their growth, development, quality of life, long-term disease-related complications, and potential sequelae of therapy in later life. The primary objective of this study is to characterize the safety and clinical effects of sargramostim treatment with and without concomitant corticosteroid induction therapy, after the first cycle and after repeated cycles if protocol requirements are met, in pediatric patients with active Crohn's disease. Secondary objectives are to: (1) Characterize the pharmacokinetic and pharmacodynamic properties of sargramostim treatment with and without concomitant corticosteroids in pediatric patients with Crohn's disease; and (2)Evaluate the efficacy of sargramostim treatment with and without concomitant corticosteroids, as measured by the Pediatric Crohn's Disease Activity Index (PCDAI; response defined as decrease greater than or equal to 12.5 points), Physician's Global Assessment (PGA), and IMPACT-III questionnaire (patients 10 years of age and older).
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