The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure

Anika Varma; Jennifer Weinstein; Jamison Seabury; Spencer Rosero; Ellen Wagner; Christine Zizzi; Aaron Kaat; Elizabeth Luebbe; Nuran Dilek; John Heatwole; Lawrence Saubermann; Larissa Temple; Scott Rogoff; Chad Heatwole

doi:10.1097/MCG.0000000000001976

The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure

Anika Varma, Jennifer Weinstein, Jamison Seabury, Spencer Rosero, Ellen Wagner, Christine Zizzi, Aaron Kaat, Elizabeth Luebbe, Nuran Dilek, John Heatwole, Lawrence Saubermann, Larissa Temple, Scott Rogoff, Chad Heatwole^*

^*Corresponding author for this work

Medical Social Sciences

Research output: Contribution to journal › Article › peer-review

1 Scopus citations

Abstract

Objective: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. Background: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. Patients and Methods: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. Results: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-Three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. Conclusions: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.

Original language	English (US)
Pages (from-to)	1043-1051
Number of pages	9
Journal	Journal of Clinical Gastroenterology
Volume	58
Issue number	10
DOIs	https://doi.org/10.1097/MCG.0000000000001976
State	Published - Jan 25 2024

Funding

C.H. receives royalties for the use of multiple disease specific instruments. He has provided consultation to Biogen Idec, Ionis Pharmaceuticals, aTyr Pharma, AMO Pharma, Acceleron Pharma, Cytokinetics, Expansion Therapeutics, Harmony Biosciences, Regeneron Pharmaceuticals, Astellas Pharmaceuticals, AveXis, Recursion Pharmaceuticals, IRIS Medicine, Inc., Takeda Pharmaceutical Company, Scholar Rock, Avidity Biosciences, Novartis Pharmaceuticals Corporation, SwanBio Therapeutics, Neurocrine Biosciences, Sanofi, Lupin Pharmaceuticals, and the Marigold Foundation. He receives grant support from the Department of Defense, Duchenne UK, Parent Project Muscular Dystrophy, Recursion Pharmaceuticals, SwanBio Therapeutics, Sanofi, Lupin Pharmaceuticals, the National Institute of Neurological Disorders and Stroke, the Muscular Dystrophy Association, the Friedreich\u2019s Ataxia Research Alliance, Cure Spinal Muscular Atrophy, the Amyotrophic Lateral Sclerosis Association, the University of Miami, Novartis Pharmaceuticals Corporation, and the Michael J. Foxx Foundation. He is the director of the University of Rochester\u2019s Center for Health and Technology. C.Z. has provided consultation to Recursion Pharmaceuticals. The remaining authors declare that they have nothing to disclose.

Keywords

Crohn's disease
disease burden
patient-reported outcome measure
symptom assessment
therapeutic trial

ASJC Scopus subject areas

Gastroenterology

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10.1097/MCG.0000000000001976

Cite this

Varma, A., Weinstein, J., Seabury, J., Rosero, S., Wagner, E., Zizzi, C., Kaat, A., Luebbe, E., Dilek, N., Heatwole, J., Saubermann, L., Temple, L., Rogoff, S., & Heatwole, C. (2024). The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure. Journal of Clinical Gastroenterology, 58(10), 1043-1051. https://doi.org/10.1097/MCG.0000000000001976

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title = "The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure",

abstract = "Objective: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. Background: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. Patients and Methods: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. Results: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-Three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. Conclusions: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.",

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author = "Anika Varma and Jennifer Weinstein and Jamison Seabury and Spencer Rosero and Ellen Wagner and Christine Zizzi and Aaron Kaat and Elizabeth Luebbe and Nuran Dilek and John Heatwole and Lawrence Saubermann and Larissa Temple and Scott Rogoff and Chad Heatwole",

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doi = "10.1097/MCG.0000000000001976",

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Varma, A, Weinstein, J, Seabury, J, Rosero, S, Wagner, E, Zizzi, C, Kaat, A, Luebbe, E, Dilek, N, Heatwole, J, Saubermann, L, Temple, L, Rogoff, S & Heatwole, C 2024, 'The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure', Journal of Clinical Gastroenterology, vol. 58, no. 10, pp. 1043-1051. https://doi.org/10.1097/MCG.0000000000001976

TY - JOUR

T1 - The Crohn's Disease-Health Index

T2 - Development and Evaluation of a Novel Outcome Measure

AU - Varma, Anika

AU - Weinstein, Jennifer

AU - Seabury, Jamison

AU - Rosero, Spencer

AU - Wagner, Ellen

AU - Zizzi, Christine

AU - Kaat, Aaron

AU - Luebbe, Elizabeth

AU - Dilek, Nuran

AU - Heatwole, John

AU - Saubermann, Lawrence

AU - Temple, Larissa

AU - Rogoff, Scott

AU - Heatwole, Chad

PY - 2024/1/25

Y1 - 2024/1/25

N2 - Objective: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. Background: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. Patients and Methods: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. Results: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-Three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. Conclusions: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.

AB - Objective: We sought to develop and validate the Crohn's Disease-Health Index (CD-HI), a disease-specific, patient-reported outcome measure that serially measures Crohn's disease (CD) symptomatic burden in adults with CD. Background: As therapeutic interventions are tested among patients with CD, responsive outcome measures are needed to track disease progression and therapeutic gain during clinical trials. Patients and Methods: We conducted a national cross-sectional study of individuals with CD to identify the most prevalent and impactful symptoms of CD. The most relevant symptoms were included in the CD-HI. We used factor analysis, qualitative patient interviews, test-retest reliability evaluation, and known group validity testing to evaluate and optimize the CD-HI. Results: The CD-HI contains 12 subscales that comprehensively measure CD burden using the patient's perspective. Fifteen adults with CD beta tested the CD-HI and found the instrument to be clear, easy to use, and relevant to them. Twenty-Three adults with CD participated in an assessment of test-retest reliability, which indicated high reliability of individual questions, subscales, and the full instrument (intraclass correlation coefficient = 0.84 for the full instrument). The CD-HI and its subscales demonstrated a high internal consistency (Cronbach α = 0.98 for the full instrument). The CD-HI distinguished between groups of individuals with CD known to differ in disease severity. Conclusions: This research supports the use of the CD-HI as a valid, sensitive, reliable, and relevant patient-reported outcome to determine the multifactorial disease burden of those with CD, assess the relevance and merit of future CD therapies, and support drug labeling claims.

KW - Crohn's disease

KW - disease burden

KW - patient-reported outcome measure

KW - symptom assessment

KW - therapeutic trial

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The Crohn's Disease-Health Index: Development and Evaluation of a Novel Outcome Measure

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