Jennifer L. Dotson, MD, MPH Assistant Professor of Pediatrics

1. Feasibility and Validity of the Pediatric Ulcerative Colitis Activity Index (PUCAI) in Routine Clinical Practice Jennifer L. Dotson, MD, MPH, Wallace V. Crandall, MD, PeixinZhang, PhD, ChristopherB Forrest, MD, PhD, L. Charles Bailey, MD, PhD, Richard B. Colletti, MD, and Michael D. Kappelman, MD, MPH Jennifer L. Dotson, MD, MPH Assistant Professor of Pediatrics Division of Gastroenterology, Hepatology and Nutrition The Ohio State University College of Medicine Principal Investigator, Center for Innovation in Pediatric Practice The Research Institute at Nationwide Children's Hospital December 13, 2013

2. I have no financial disclosures or conflicts of interest

3. Background: PUCAI • Standardized assessment tool of UC disease activity • Rigorous development process1 • Outstanding clinimetricproperties • Widely adopted by clinical researchers as a non-invasive measure of disease activity2-7 • Recommended in the clinical management of patients and incorporated into recent clinical guidelines2,8 1.Turner D, et al. Gastroenterology. Aug 2007 2.Turner D, et al. Am J Gastroenterol. Apr 2011 3.Gray FL, et al. Journal of pediatric surgery. Jul 2013 4.Teitelbaum JE, et al. J PediatrGastroenterolNutr. Jun 2013 5.Turner D, et al. ClinGastroenterolHepatol. May 2013 6.Watson S, et al. Inflamm Bowel Dis. Jan 2011 7.Turner D, et al. Inflamm Bowel Dis. Jan 2011 8.Turner D, et al. J PediatrGastroenterolNutr. Sep 2012

4. Background: PUCAI • Although the use of PUCAI has been evaluated in single-center and small multi-center research studies,1-6little is known about its feasibility and performance when used in routine clinical practice 1.Turner D, et al.. Gastroenterology. Aug 2007 2.Turner D, et al. ClinGastroenterolHepatol. May 2013. 3.Turner D, et al. Inflamm Bowel Dis. Jan 2012;18(1):55-62. 4.Turner D, et al.. Journal of clinical epidemiology. Apr 2009 5.Turner D, et al. InflammBowel Dis. Apr 2010 6.Lee JJ, et al. J PediatrGastroenterolNutr. Jun 2011

5. Turner D, et al. Gastroenterology. Aug 2007

6. Turner D, et al. Gastroenterology. Aug 2007

7. Objective • Evaluate the feasibility, validity, and responsiveness to clinical change of PUCAI in a large, diverse collection of pediatric GI practices

8. Methods: Study Design • ImproveCareNow (ICN): Network of pediatric GI practices established in 2007 to improve the health of children with IBD • Demographic, disease and treatment data collected prospectively and longitudinally during all routine outpatient encounters • Patients diagnosed and managed according to the usual practice of the primary GI provider

9. Methods: Study Design • Extracted data from the 2 most recent encounters for all patients with UC (September 2006-December 2012) • Demographics, disease duration, disease extent (Paris classification), Physician Global Assessment (PGA), and PUCAI components

10. Methods: Feasibility Analysis • Percentage of patients for whom all PUCAI components were recorded at their most recent visit

11. Methods: Validity Analysis • We examined the correlation between PUCAI and PGA: • Distribution of PUCAI scores across PGA categories using boxplots and compared differences using Kruskal-Wallis test • Pearson’s correlation coefficient

12. Methods: Responsiveness Analysis • Responsiveness of an instrument is • Its ability to detect minimal clinically important differences • Directly related to the magnitude of change • Extent to which PUCAI changes in relation to a corresponding change in PGA over time

13. Methods: Responsiveness Analysis • PGA was unchanged between visits: • Assessed the test-retest reliability of the PUCAI with intra-class correlation coefficient using ANOVA • PGA changed between visits: • Evaluated the distribution of change in PUCAI according to change in PGA using boxplots with the Kruskal-Wallis test  

14. Methods: Responsiveness Analysis • Change in PUCAI defined by: • Subtracting the follow-up PUCAI score from the previous visit PUCAI score • Change in PGA between the 2 most recent visits defined by: • Small change = change in 1 category (e.g. severe to moderate) • Moderate change = change in 2 categories (e.g. moderate to remission) • Large change = change in 3 categories (e.g. severe to remission)

15. Results: Demographics (most recent visit)

16. Results: Demographics (most recent visit)

17. Results: Feasibility(n=2503)

18. Results: Feasibility(n=2503)

19. Results: Validity Kruskal-Wallis p<0.001 • Good correlation with PGA by Pearson’s correlation [r=0.76 (p<0.001)]

20. Results: Responsiveness • 1236 patients whose PGA was unchanged • 1040 (84%) remission • 145 (12%) mild • 44 (4%) moderate • 7 (<1%) severe • Test-retest reliability of PUCAI (p<0.001)

21. Results: Responsiveness Kruskal-Wallis p<0.001

22. Key Limitations • Small sample size at the periphery of the distribution of the change in PGA categories • Data derived from an outpatient database, so few UC patients had severe disease activity

23. Conclusions • First large-scale, multicenter evaluation of PUCAI (approximately 2000 patients from 35 sites) supports the broad generalizability and ease of use in routine outpatient care • Demonstrated strong feasibility and validity between PUCAI and PGA • Responsiveness of change in PUCAI by change in PGA over time was good

24. Summary • PUCAI is highly feasible, valid and responsive to change • Findings support the use of PUCAI as a clinical and research tool, including serving as a basis for inpatient and outpatient care algorithms

25. Mentorship and Funding • Wallace V. Crandall, MD • Michael D. Kappelman, MD, MPH • Kelly Kelleher, MD, MPH • This project was supported by a grant from the Agency for Healthcare Research and Quality (R01 HS020024) • MDK was supported by a grant from the NIH/NIDDK (K08 DK088957) • JLD was supported by the NASPGHAN Foundation/CCFA Young Investigator Development Award