WHAT'S KNOWN ON THIS SUBJECT: Studies on adult patients who have Crohn disease have shown a comparative benefit of antiTNFa versus placebo and thiopurines. These same studies have not been done in children, because of time, cost, and ethical (withholding an efficacious treatment) challenges.
WHAT THIS STUDY ADDS:Anti-TNFa therapy administered in routine practice to children with Crohn disease was more effective than usual care at achieving clinical and corticosteroid-free remission. Using data from the ImproveCareNow learning health system for observational research is feasible and produces valuable evidence. abstract OBJECTIVES: ImproveCareNow (ICN) is the largest pediatric learning health system in the nation and started as a quality improvement collaborative. To test the feasibility and validity of using ICN data for clinical research, we evaluated the effectiveness of anti-tumor necrosis factor-a (anti-TNFa) agents in the management of pediatric Crohn disease (CD).METHODS: Data were collected in 35 pediatric gastroenterology practices (April 2007 to March 2012 and analyzed as a sequence of nonrandomized trials. Patients who had moderate to severe CD were classified as initiators or non-initiators of anti-TNFa therapy. Among 4130 patients who had pediatric CD, 603 were new users and 1211 were receiving anti-TNFa therapy on entry into ICN.RESULTS: During a 26-week follow-up period, rate ratios obtained from Cox proportional hazards models, adjusting for patient and disease characteristics and concurrent medications, were 1.53 (95% confidence interval [CI], 1.20-1.96) for clinical remission and 1.74 (95% CI, 1.33-2.29) for corticosteroid-free remission. The rate ratio for corticosteroid-free remission was comparable to the estimate produced by the adult SONIC study, which was a randomized controlled trial on the efficacy of anti-TNFa therapy. The number needed to treat was 5.2 (95% CI, 3.4-11.1) for clinical remission and 5.0 (95% CI, 3.4-10.0) for corticosteroid-free remission.
CONCLUSIONS:In routine pediatric gastroenterology practice settings, anti-TNFa therapy was effective at achieving clinical and corticosteroidfree remission for patients who had Crohn disease. Using data from the ICN learning health system for the purpose of observational research is feasible and produces valuable new knowledge. Over thelastseveraldecades,therapeutic advances in the treatment of pediatric Crohn disease (CD) have included the widespread use of immunomodulators such as 6-mercaptopurine, azathioprine, and methotrexate. [1][2][3] More recently, the anti-tumor necrosis factor a (anti-TNFa) biological agents (eg, infliximab and adalimumab) have been adopted as a treatment of moderate to severe pediatric CD. 4 Although several studies on adult patients who have CD have shown a comparative benefit of anti-TNFa versus placebo 5 and thiopurines, 6 these same studies have not been done in children because of practical (time and cost) and ethical (withholding an efficacious treatment) challenges.The cost of anti-TNFa is in the range...