Pediatric Network Initiative

Background

Evidence unfortunately points to a rise in the number of pediatric patients diagnosed with inflammatory bowel diseases (IBD). Of these children, some show mild disease activity and do well with basic medication. However, many others exhibit more a more severe disease trajectory with growth failure, continued active gastrointestinal symptoms, and the eventual development of disease complications requiring surgery. To decide an appropriate course of treatment, it is important to identify at time of diagnosis those children most likely to require aggressive medical therapy. However, good quality, pediatric-specific data is either limited or non-existent so treatment is based on adult data. Much-needed pediatric-specific information can only be obtained by a large-scale multi-center collaborative effort.

Project Description

The first study of the Pediatric Network utilizes a design called risk stratification of an inception cohort. The purpose of the RISK Stratification study is to enroll pediatric patients at the time of diagnosis of Crohn’s disease to participate in long-term studies to understand progression of disease. The largest study of its kind, the RISK study gathers patient biospecimens for identifying genetic, clinical, microbial, and immunologic markers. By integrating and contrasting the genetic and microbial data with disease progression of the patient, investigators expect to be able to predict aggressive versus benign disease as well as response to therapy. This study is currently operating across 29 centers with over 1,709 patients enrolled.

Building on the success of RISK, study centers are also collaborating on the Predicting Response to Standardized Pediatric Colitis Therapy, or PROTECT study. Researchers in the PROTECT study will conduct a clinical trial of standardized medical therapy for 410 children newly diagnosed with ulcerative colitis at 17 pediatric IBD centers in North America. Investigators will follow patients for two years, collecting data that will prove invaluable for the study of IBD. The primary clinical goal of both of these projects is remission for one year without the use of steroid medications or the need for more potent immunosuppressive medications or surgery.

Significance

If successful, the Network will illustrate how risk factors present in children with IBD influence the natural history of the disease. The remarkable success of the RISK Stratification study has allowed for accumulation of biospecimens from an unprecedented number of patients. This information can be translated into clinical studies testing the hypothesis that early, aggressive therapy in selected patients may decrease the likelihood of developing severe complications. The potential is to identify myriad predictors of how patients will respond to various medications. Studies will identify best outcomes and educate physicians as to the best treatment options for their young patients, producing more targeted and effective therapies for individual patients.

For more information about the Pediatric Network Initiative, click here.

You can support CCFA by clicking here or you can speak with Judith Brown at (646) 943-7441 to learn how to support the Pediatric Newtork Intiative.