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Jennifer L. Dotson, MD, MPHAssistant 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

Feasibility and Validity of the Pediatric Ulcerative Colitis Activity Index

(PUCAI) in Routine Clinical PracticeJennifer L. Dotson, MD, MPH, Wallace V. Crandall, MD, Peixin

Zhang, PhD, Christopher B Forrest, MD, PhD, L. Charles Bailey, MD, PhD,

Richard B. Colletti, MD, and Michael D. Kappelman, MD, MPH

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I have no financial disclosures or conflicts of interest

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Background: PUCAI• Standardized assessment tool of UC disease activity• Rigorous development process1 • Outstanding clinimetric properties• 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 20113.Gray FL, et al. Journal of pediatric surgery. Jul 20134.Teitelbaum JE, et al. J Pediatr Gastroenterol Nutr. Jun 20135.Turner D, et al. Clin Gastroenterol Hepatol. May 20136.Watson S, et al. Inflamm Bowel Dis. Jan 20117.Turner D, et al. Inflamm Bowel Dis. Jan 20118.Turner D, et al. J Pediatr Gastroenterol Nutr. Sep 2012

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Background: PUCAI

• Although the use of PUCAI has been evaluated in single-center and small multi-center research studies,1-6 little is known about its feasibility and performance when used in routine clinical practice

1.Turner D, et al.. Gastroenterology. Aug 20072.Turner D, et al. Clin Gastroenterol Hepatol. 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 20095.Turner D, et al. Inflamm Bowel Dis. Apr 20106.Lee JJ, et al. J Pediatr Gastroenterol Nutr. Jun 2011

Item Points

1. Abdominal pain No pain Pain can be ignored Pain cannot be ignored

0510

2. Rectal bleeding None Small amount only, in <50% of stools Small amount with most stools Large amount (>50% of stool content)

0102030

3. Stool consistency of most stools Formed Partially formed Completely unformed

0510

4. Number of stools per 24 hours 0-2 3-5 6-8 >8

051015

5. Nocturnal stools (any episode causing wakening) No Yes

010

6. Activity level No limitation of activity Occasional limitation of activity Severe restricted activity

0510

Sum of PUCAI 0-85Turner D, et al. Gastroenterology. Aug 2007

Item Points

1. Abdominal pain No pain Pain can be ignored Pain cannot be ignored

0510

2. Rectal bleeding None Small amount only, in <50% of stools Small amount with most stools Large amount (>50% of stool content)

0102030

3. Stool consistency of most stools Formed Partially formed Completely unformed

0510

4. Number of stools per 24 hours 0-2 3-5 6-8 >8

051015

5. Nocturnal stools (any episode causing wakening) No Yes

010

6. Activity level No limitation of activity Occasional limitation of activity Severe restricted activity

0510

Sum of PUCAI 0-85

Disease Severity

PUCAI Cut-Points

Remission <10

Mild 10-34

Moderate 35-64

Severe 65-85

Turner D, et al. Gastroenterology. Aug 2007

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Objective

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

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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

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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

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Methods: Feasibility Analysis

• Percentage of patients for whom all PUCAI components were recorded at their most recent visit

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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

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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

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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  

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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)

Results: Demographics (most recent visit)Variable n (%)Total number of patients 2503Gender   Male 1237 (49.4)Age 15.2 ± 4.1 yearsRace/Ethnicity   White 1920 (81.9) Black 199 (8.5) Hispanic or Latino 87 (3.7) Asian 42 (1.8) Other 97 (4.1)Disease duration 3.7 ± 3.2 yearsParis Classification (n=1773 (70.8%))   E1: ulcerative proctitis 154 (8.7) E2: left-sided (distal to splenic flexure) 330 (18.6) E3: extensive (hepatic flexure distally) 135 (7.6) E4: pancolitis (proximal to hepatic flexure) 1154 (65.1)PGA   Remission 1703 (70.0) Mild 518 (21.3) Moderate 183 (7.5) Severe 30 (1.2)

Results: Demographics (most recent visit)Variable n (%)Total number of patients 2503Gender   Male 1237 (49.4)Age 15.2 ± 4.1 yearsRace/Ethnicity   White 1920 (81.9) Black 199 (8.5) Hispanic or Latino 87 (3.7) Asian 42 (1.8) Other 97 (4.1)Disease duration 3.7 ± 3.2 yearsParis Classification (n=1773 (70.8%))   E1: ulcerative proctitis 154 (8.7) E2: left-sided (distal to splenic flexure) 330 (18.6) E3: extensive (hepatic flexure distally) 135 (7.6) E4: pancolitis (proximal to hepatic flexure) 1154 (65.1)PGA   Remission 1703 (70.0) Mild 518 (21.3) Moderate 183 (7.5) Severe 30 (1.2)

Results: Feasibility (n=2503)

PUCAI Components # of visits recorded

% of visits recorded

Abdominal pain 2478 99.0%

Rectal bleeding 2451 97.9%

Stool consistency of most stools 2446 97.7%

Total number of stools 2459 98.2%

Nocturnal stools 2438 97.4%

Activity level 2476 98.9%

Patients with all components 2402 96%

Results: Feasibility (n=2503)

PUCAI Components # of visits recorded

% of visits recorded

Abdominal pain 2478 99.0%

Rectal bleeding 2451 97.9%

Stool consistency of most stools 2446 97.7%

Total number of stools 2459 98.2%

Nocturnal stools 2438 97.4%

Activity level 2476 98.9%

Patients with all components 2402 96%

• Good correlation with PGA by Pearson’s correlation [r=0.76 (p<0.001)]

Results: Validity

Kruskal-Wallis p<0.001

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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)

Results: Responsiveness

Kruskal-Wallis p<0.001

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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

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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

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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

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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