Systematic review: patient‐reported outcome measures in coeliac disease for regulatory submissions

Canestaro, William J.; Edwards, Todd C.; Patrick, Donald L.

doi:10.1111/apt.13703

Cited by 33 publications

(35 citation statements)

References 102 publications

(107 reference statements)

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Section: Discussionmentioning

confidence: 99%

“…The poor correlation of histology and serology with symptoms has highlighted the need for suitable tests to monitor relevant clinical responses to gluten in trials of novel therapies intended for coeliac disease patients on gluten-free diet. 4,5,29 Symptoms measured by patient-reported outcome instruments are increasingly used as efficacy endpoints in clinical trials. 30 Here, we show that standardised oral gluten challenge, combined with cytokine assessment and patient-reported outcomes adapted for hourly rating (as described for the first time in this study), is well suited to monitoring the effects of gluten in coeliac disease patients.…”

Section: Discussionmentioning

confidence: 99%

See 1 more Smart Citation

Elevated serum interleukin‐2 after gluten correlates with symptoms and is a potential diagnostic biomarker for coeliac disease

Tye–Din

Daveson

et al. 2019

Aliment Pharmacol Ther

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Summary Background Coeliac disease patients on a gluten‐free diet experience reactions to gluten, but these are not well characterised or understood. Systemic cytokine release was recently linked to reactivation of gluten immunity in coeliac disease. Aim To define the nature and time‐course of symptoms and interleukin‐2 changes specific for coeliac disease patients. Methods 25 coeliac disease patients on a gluten‐free diet and 25 healthy volunteers consumed a standardised 6 gram gluten challenge. Coeliac Disease Patient‐Reported Outcome survey and global digestive symptom assessment were completed hourly up to 6 hours after gluten. Adverse events over 48 hours were recorded. Serum interleukin‐2 was measured at baseline, and 2, 4 and 6 hours. Results Serum interleukin‐2 was always undetectable in healthy controls, whereas it was undetectable at baseline and elevated >0.5 pg/ml at 4 hours in 92% of coeliac disease patients. All patient‐reported outcome severity scores increased significantly after gluten in coeliac disease patients (P < .001 Wilcoxon signed rank test), but not in controls. Symptoms began after 1 hour, and peaked in the third. Nausea and vomiting characterised severe reactions, but mild reactions were limited to headache and tiredness. Peak interleukin‐2 correlated with symptom severity, particularly for nausea and vomiting. Conclusions Serum interleukin‐2 elevations correlate with timing and severity of symptoms after gluten in coeliac disease. Standardised bolus gluten food challenge and interleukin‐2 assessment could provide a valuable clinical test to monitor and diagnose coeliac disease in patients established on a gluten‐free diet.

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Section: Discussionmentioning

confidence: 99%

Section: Discussionmentioning

confidence: 99%

Elevated serum interleukin‐2 after gluten correlates with symptoms and is a potential diagnostic biomarker for coeliac disease

Tye–Din

Daveson

et al. 2019

Aliment Pharmacol Ther

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“…A key finding in this study was that only coeliac disease-specific PROs met all of our stringent evaluative criteria 15. A recently published systematic review reported four appropriate PROs for use in coeliac disease trials 51. It should be noted, however, that only two of these, the CDSD and the CeD-PRO, are coeliac disease-specific PROs that were developed according to the FDA SEALD criteria and can thus be used for labelling purposes.…”

Section: Discussionmentioning

confidence: 99%

Disease activity indices in coeliac disease: systematic review and recommendations for clinical trials

Hindryckx

Levesque

Holvoet³

et al. 2016

Gut

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ObjectiveAlthough several pharmacological agents have emerged as potential adjunctive therapies to a gluten-free diet for coeliac disease, there is currently no widely accepted measure of disease activity used in clinical trials. We conducted a systematic review of coeliac disease activity indices to evaluate their operating properties and potential as outcome measures in registration trials.DesignMEDLINE, EMBASE and the Cochrane central library were searched from 1966 to 2015 for eligible studies in adult and/or paediatric patients with coeliac disease that included coeliac disease activity markers in their outcome measures. The operating characteristics of histological indices, patient-reported outcomes (PROs) and endoscopic indices were evaluated for content and construct validity, reliability, responsiveness and feasibility using guidelines proposed by the US Food and Drug Administration (FDA).ResultsOf 19 123 citations, 286 studies were eligible, including 24 randomised-controlled trials. Three of five PROs identified met most key evaluative criteria but only the Celiac Disease Symptom Diary (CDSD) and the Celiac Disease Patient-Reported Outcome (CeD PRO) have been approved by the FDA. All histological and endoscopic scores identified lacked content validity. Quantitative morphometric histological analysis had better reliability and responsiveness compared with qualitative scales. Endoscopic indices were infrequently used, and only one index demonstrated responsiveness to effective therapy.ConclusionsCurrent best evidence suggests that the CDSD and the CeD PRO are appropriate for use in the definition of primary end points in coeliac disease registration trials. Morphometric histology should be included as a key secondary or co-primary end point. Further work is needed to optimise end point configuration to inform efficient drug development.

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“…Some coeliac‐specific measures have been developed, for example, the Coeliac Disease Questionnaire (CDQ) and the Coeliac Disease Quality of Life Survey (CD‐QOL) . A systematic review identified four candidate coeliac‐specific PROMs for use in clinical trials and concluded that none of these meet the standards of the US Food and Drug Administration . Another systematic review focused on patient‐reported symptom scores and identified two coeliac disease indices that have been approved by the US Food and Drug Administration.…”

Section: Introductionmentioning

confidence: 99%

Quality of life in coeliac disease: item reduction, scale development and psychometric evaluation of the Coeliac Disease Assessment Questionnaire (CDAQ)

Crocker

Jenkinson

Peters

2018

Aliment Pharmacol Ther

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SummaryBackgroundA better understanding of coeliac disease can be achieved by assessing health‐related quality of life alongside clinical factors. Existing patient‐reported outcome measures (PROMs) evaluating quality of life in coeliac disease have not been developed in accordance with the US Food and Drug Administration guidelines.AimTo develop a PROM in accordance with best practice guidelines, capturing all aspects of quality of life important to adults with coeliac disease.MethodsCandidate items for the Coeliac Disease Assessment Questionnaire (CDAQ) were refined through item appraisal, expert review, cognitive interviews, and a translatability assessment. A cross‐sectional survey determined further item reduction and the CDAQ's structure. The final CDAQ was administered alongside the Short Form Health Survey Version 2 (SF?36v2) in a second survey to assess construct validity and test‐retest reliability.ResultsPre‐testing the 64 candidate items revealed a range of issues which guided their refinement and reduction, resulting in the final CDAQ with 32 items representing 5 subscales: stigma (eight items), dietary burden (eight items), symptoms (five items), social isolation (five items), and worries and concerns (six items). Cronbach's alpha ranged between 0.82 and 0.88 for all domains. Further results showed CDAQ scores were more strongly correlated with the SF‐36v2's mental health dimensions, as expected. Intraclass correlation coefficients ranged from 0.79 to 0.89.ConclusionThe CDAQ is a reliable and valid coeliac‐specific measure that captures all aspects of quality of life important to adults with coeliac disease. Further work is underway to assess the CDAQ's responsiveness to change.

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Systematic review: patient‐reported outcome measures in coeliac disease for regulatory submissions

Cited by 33 publications

References 102 publications

Elevated serum interleukin‐2 after gluten correlates with symptoms and is a potential diagnostic biomarker for coeliac disease

Elevated serum interleukin‐2 after gluten correlates with symptoms and is a potential diagnostic biomarker for coeliac disease

Disease activity indices in coeliac disease: systematic review and recommendations for clinical trials

Quality of life in coeliac disease: item reduction, scale development and psychometric evaluation of the Coeliac Disease Assessment Questionnaire (CDAQ)

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