Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf ·...

23
Renovating the Domain Structure of the Inflammatory Bowel Disease Questionnaire Dahlia Awais, MD 1 Timothy Gathany MEd 2 Peter D.R. Higgins, MD, PhD, MSc 1 1 University of Michigan Medical School 2 Centocor, Inc.

Transcript of Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf ·...

Page 1: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Renovating the Domain Structure gof the Inflammatory Bowel

Disease Questionnaire

Dahlia Awais, MD1

Timothy Gathany MEd2

Peter D.R. Higgins, MD, PhD, MSc1gg

1University of Michigan Medical School2Centocor, Inc.

Page 2: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Disclosures:

I do not have any relevant financial l ti hi ith i lrelationships with any commercial

interests

Page 3: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

IBDQ Background

Th I fl t B l Di• The Inflammatory Bowel Disease Questionnaire (IBDQ) is a widely used measure of disease-specific health-related quality of life in both Crohn’s and UC

• Improvement in HrQoL is regarded as an important therapeutic goal in the treatment of IBD

• IBDQ is used in clinical trials to support• IBDQ is used in clinical trials to support claims of efficacy

Guyatt, et al Gastroenterology 1989

Page 4: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

IBDQ DomainsTh IBDQ i d f 32 it h• The IBDQ is composed of 32 items each measured on a 7 point Likert scale– Higher scores reflect better QoL (32-224)

• Designed to be divided into 4 domainsE h i t f IBD ti t’ lif– Each measuring an aspect of IBD patient’s life

IBDQ

Bowel Emotional Social Systemicy10

items12

items5

items5

items

Page 5: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

IBDQ Background

• The general validity and reliability of the complete IBDQ has been repeatedly confirmed

• However the 4 domain structure of the• However, the 4 domain structure of the IBDQ has not been validated

Page 6: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

IBDQ Domain StructureR t ti d i t t• Reasons to question domain structure:– Problem questions – mix domains

• IBDQ23 How much of the time during the last 2 weeks have you felt embarrassed as a result of your bowel problem?

• IBDQ26 How much of the time during the last 2 weeks have you been troubled by accidental soiling?y y g

– FDA has rejected it as a source for patient reported outcomes

– Although complete IBDQ has been used in many countries, the proposed 4 domain structure has not been successfully reproduced in multiple adaptations including in the UK Norway Sweden Greece andincluding in the UK, Norway, Sweden, Greece, and Spain

FDA Guidance for Industry, 2006

Page 7: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

IBDQ Domain StructureD it ti di th lidit f th• Despite questions regarding the validity of the domain structure, domain subscores are reported in the literature

• Example: Effect of certolizumab on HRQoLIBDQ t t l i d– IBDQ total score improved

– Marked improvement specifically in emotional and systemic domains of the IBDQ

• “alleviates the psychological symptoms associated with Crohn’s disease and improves a patient’s general well-being”

C l i i bl ti d d th– Conclusion is problematic, depends on the unproven validity of the IBDQ domain structure

Rutgeerts, et al Int J Colorectal Dis 2008

Page 8: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Aims

T d t i h th th 4 d i• To determine whether the 4 domain structure of the IBDQ is supported using large IBDQ datasets from randomized clinical trials

• To compare the domain structure of the IBDQ for ulcerative colitis and Crohn’s disease

• If the domain structure is not supported to• If the domain structure is not supported, to propose an alternate domain structure

Page 9: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

MethodsB li i di id l ti t IBDQ d t f th• Baseline individual patient IBDQ data from the ACT and ACCENT trials (725 patients with UC and 876 patients with Crohn’s) were used to analyze the domain structure

• Factor analysis with SAS PROC FACTOR was• Factor analysis with SAS PROC FACTOR was performed to identify # of factors (domains) in the IBDQ– Statistical method that looks for items that group

together to identify a smaller number of umbrella g yvariables called “factors”

Page 10: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

How Many Factors?

Factor 1 Factor 2Emotional Bowel

frustrationsadness

worry

loose stoolsincontinence

frequency

Page 11: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Methods

• Our second step was to depict items that cluster together with clustering analysis and dendrograms

Page 12: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Number of Domains

Factors are assigned values called eigenvalues. By convention, factors with eigenvalues >1 are retained. 7 principal factors were identified in Crohn’s and 6 principal factors in ulcerative colitis.

Page 13: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Crohn’s – 7 DomainsBowel

EmotionalSocial

Systemic Emotional

Social Emotional

Emotional

Bowel

Bowel

BowelSystemic

Page 14: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

UC – 6 DomainsSystemic

Emotional

Systemic Bowel Social Emotional

Bowel Emotional Systemic

Social

Emotional

Bowel

Bowel Emotional Systemic

Page 15: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Brief IBDQ – Crohn’s

Systemic

Abdominal Discomfort

Stool Quality

Emotional

Social

Rectal Emotional

Bowel Incontinence

Emotional

Page 16: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Brief IBDQ – UCSystemic

Rectal Emotional

Abdominal Discomfort

Social

Emotional

Bowel Incontinence

Page 17: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease
Page 18: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Conclusions

• The intended 4 domain structure of the IBDQ is not valid

• The 4 domain subscores of the IBDQ should not be usedshould not be used

• The IBDQ in Crohn’s is better described with a 7 domain structure and the IBDQ in UC is better described with a 6 domainUC is better described with a 6 domain structure

Page 19: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Conclusions

B th B i f IBDQ f C h ’ d• Both our Brief IBDQ for Crohn’s and our Brief IBDQ for UC are highly correlated with the complete IBDQ and DAIs and reflect a clean domain structure

• A modified index with revised domains may be the best approach to the measurement of health-related quality of life in ulcerative colitis and Crohn’slife in ulcerative colitis and Crohn s disease.

Page 20: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Acknowledgments

• Timothy Gathany, MEd (Centocor)• Peter Higgins, MD, PhD, MSc (University

of Michigan)

Page 21: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease
Page 22: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease
Page 23: Renovating the Domain Structure of the Inflammatory Bowel …vid.imedex.com/pdf/5477/awais.pdf · 2009-05-22 · Renovating the Domain Structure of the Inflammatory Bowel Disease

Revised Domain Structure