The Paediatric-Onset Inflammatory Bowel Disease Cohort and Treatment Study (PICTS)

Inflammatory bowel disease (IBD) is a common problem in children and teenagers, with significant embarrassing symptoms, effects on growth and development, and interference with school progress, family and social life. There have been few successful research trials of therapies in childhood IBD, so we draw conclusions from studies in adults with IBD, where the issues can be different. Throughout Scotland, many children and teenagers (more than 450) have already joined our research study into the causes (in terms of genetic makeup and triggers in the environment) of IBD. This group is called a cohort, and our studies have been very successful in scientific terms to date. Although we have studied all potentially important genes that may have a role in the cause of IBD, none have been shown to have a major role ? the responsible genetic causes have yet to be found!

We are increasing the numbers of Scottish children and young people in the cohort, which will help identify more potential genetic and environmental causes. We also wish to perform trials of therapies which will answer key questions such as what is the best treatment?, what side effects can occur? With a larger group, we can also show the expected course of some of the rarer forms of childhood IBD, such as oral Crohn?s disease, perianal-only Crohn?s disease, and ulcerative colitis of only the rectum, which have been poorly studied in childhood to date.

This project will focus on the consolidation and expansion of a proven, scientifically important cohort of Scottish patients with childhood-onset inflammatory bowel disease (IBD). Scottish families with IBD strongly support this research, with over 450 cases in the cohort to date, all with comprehensive phenotypic description and extensive genetic analysis. Expansion will allow a near-complete ascertainment of cases of IBD diagnosed 16 years of age in Scotland, provide a complete geographical spread throughout Scotland, and expand numbers in the rarer subtypes of IBD. Follow-up will allow characterisation of disease progression, natural history, responsiveness to therapy, and of pathogenetic factors.

SUCCESS OF COHORT TO DATE:
? Enrollment of over 450 cases of childhood-onset IBD in Scotland
? Built on epidemiological study of this population for more than 4 decades and genetic studies for the last 8 years
? Very high acceptance by children and families ( 99% recruitment in SE Scotland)
? Rigorous and extensive phenotypic characterisation of cohort
? Demonstration of extensive disease at childhood diagnosis and rapid progression in extent and disease behaviour
? Demonstration of geographical and socio-economic variance in disease prevalence within Scotland
? High impact publications on paediatric phenotype and genotype-phenotype relationships
? Research group at international forefront of phenotypic and genotypic analysis of childhood-onset IBD
? Close links with international groups with similar childhood-onset IBD cohorts, and also more widely in adult Gastroenterology
? Development of paediatric clinician scientists with skills in translation medicine
? 500,000 SNP genetic profiling of 410 Scottish children with IBD available shortly

EXPECTED RESULTS OF EXPANSION OF THIS COHORT:
? Increase in cohort size to 1000 by 3 year study end
? Completion of full geographical ascertainment will permit further study of epidemiological and environmental risk factors for IBD
? Characterisation of pathogenetic factors by parallel epidemiological, genetic, serological, and proteomic studies
? Fuller characterisation of disease progression and natural history, especially in rarer sub-types of IBD
? Intervention studies ? pilot feasibility RCT and full RCT for both induction of remission and maintenance of remission in childhood-onset IBD
? Characterisation of responsiveness to therapy
? Determination of safety of exposure to new biological therapies in IBD