Effectiveness of anti-TNFα for Crohn disease: research in a pediatric learning health system.

Journal Article (Journal Article)

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-α (anti-TNFα) 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-TNFα therapy. Among 4130 patients who had pediatric CD, 603 were new users and 1211 were receiving anti-TNFα 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-TNFα 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-TNFα therapy was effective at achieving clinical and corticosteroid-free 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.

Full Text

Duke Authors

Cited Authors

  • Forrest, CB; Crandall, WV; Bailey, LC; Zhang, P; Joffe, MM; Colletti, RB; Adler, J; Baron, HI; Berman, J; del Rosario, F; Grossman, AB; Hoffenberg, EJ; Israel, EJ; Kim, SC; Lightdale, JR; Margolis, PA; Marsolo, K; Mehta, DI; Milov, DE; Patel, AS; Tung, J; Kappelman, MD

Published Date

  • July 2014

Published In

Volume / Issue

  • 134 / 1

Start / End Page

  • 37 - 44

PubMed ID

  • 24935993

Pubmed Central ID

  • PMC4531278

Electronic International Standard Serial Number (EISSN)

  • 1098-4275

Digital Object Identifier (DOI)

  • 10.1542/peds.2013-4103


  • eng

Conference Location

  • United States