Systematic review: patient-reported outcome measures in coeliac disease for regulatory submissions (original) (raw)
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Outcome measures in coeliac disease trials: the Tampere recommendations
Gut, 2018
A gluten-free diet is the only treatment option of coeliac disease, but recently an increasing number of trials have begun to explore alternative treatment strategies. We aimed to review the literature on coeliac disease therapeutic trials and issue recommendations for outcome measures. Based on a literature review of 10 062 references, we (17 researchers and 2 patient representatives from 10 countries) reviewed the use and suitability of both clinical and non-clinical outcome measures. We then made expert-based recommendations for use of these outcomes in coeliac disease trials and identified areas where research is needed. We comment on the use of histology, serology, clinical outcome assessment (including patient-reported outcomes), quality of life and immunological tools including gluten immunogenic peptides for trials in coeliac disease. Careful evaluation and reporting of outcome measures will increase transparency and comparability of coeliac disease therapeutic trials, and w...
Quality-of-Life Evaluation in Coeliac Patients on a Gluten-Free Diet
Nutrients
The treatment for coeliac disease (CD) has a considerable psychological impact on patients, which may vary depending on subjects and clinical characteristics. The aim of this study was to describe the quality of life (QoL) in CD patients during follow-up, evaluating which factors can influence it. Patients with CD who consecutively visited the outpatient clinic of CD Unit of the University Hospital of Padua from January to September 2019 were enrolled. Demographics and clinical information were collected, and all patients were asked to answer the CD-QoL and Biagi’s validated questionnaires. Student’s t-test and chi-square test were used to compare the continuous and categorical variables, respectively. One hundred patients were enrolled (86 females, mean age at test ± SD: 39.73 ± 13.51; mean age at diagnosis ± SD: 33.09 ± 12.92), with 61% of them having been diagnosed with CD within the previous 5 years. At the time of diagnosis, 43 CD patients reported classical CD presentation, 32...
The accuracy of diagnostic indicators for coeliac disease: A systematic review and meta-analysis
PLOS ONE, 2021
Background The prevalence of coeliac disease (CD) is around 1%, but diagnosis is challenged by varied presentation and non-specific symptoms and signs. This study aimed to identify diagnostic indicators that may help identify patients at a higher risk of CD in whom further testing is warranted. Methods International guidance for systematic review methods were followed and the review was registered at PROSPERO (CRD42020170766). Six databases were searched until April 2021. Studies investigating diagnostic indicators, such as symptoms or risk conditions, in people with and without CD were eligible for inclusion. Risk of bias was assessed using the QUADAS-2 tool. Summary sensitivity, specificity, and positive predictive values were estimated for each diagnostic indicator by fitting bivariate random effects meta-analyses. Findings 191 studies reporting on 26 diagnostic indicators were included in the meta-analyses. We found large variation in diagnostic accuracy estimates between studie...
Nutrients
Gluten proteins are known as immunological triggers for inflammation resulting in mucosal lesions in patients with coeliac disease (CD). Adherence to a strict gluten-free diet (GFD) is currently known as the only effective treatment for CD. In this study, we performed a systematic review and dose-response meta-analysis on data from previous studies to investigate the association between different gluten doses administered and the risk of CD relapse. Electronic databases were systematically searched to retrieve studies that investigated the response of CD patients to different amounts of gluten intake and evaluated the clinical, serologic, and/or histologic evidence to recognize disease relapse. Study-specific relative risks (RRs) were combined using a random effects model. A total of 440 identified published papers were screened, of which 7 records were selected following full-text reviewing and eligibility assessment for dose-response meta-analysis. According to our analysis, the r...
Alimentary Pharmacology & Therapeutics, 2007
Background Increasing numbers of individuals are now being diagnosed with coeliac disease. The only accepted treatment for coeliac disease is lifelong adherence to a strict gluten-free diet (GFD). Individuals' ability to adhere to the GFD varies, but systematic studies guiding the assessment of adherence are currently lacking. Aim We sought to compare the predictive value of self-report and four serologic tests compared to expert nutritionist evaluation. Methods In all, 154 individual adults with biopsy-proven coeliac disease rated their adherence to the GFD on a Likert scale. Serum antibody titres of IgA anti-tissue transglutaminase, and IgA and IgG anti-deamidated gliadin peptides were determined. Using ANOVA and ROC analyses, results were compared to a standardized evaluation by an expert nutritionist blinded to the participants' self-rated adherence and serology results. Results All serologic measures as well as participant reported adherence were significantly associated with GFD adherence as assessed by expert nutritionist evaluation. However, on ROC analysis no measure performed satisfactorily. The performance of serologic testing, but not self-report, improved with increased time on the GFD. Conclusion Although current serologic tests have very high sensitivities and specificities for the diagnosis of coeliac disease, they cannot replace trained nutritionist evaluation in the assessment of GFD adherence.
British Journal of Nutrition, 2012
A dietary interview performed by expert personnel is the best method to check whether patients with coeliac disease follow a strict glutenfree diet (GFD). We previously developed a score based on four fast and simple questions that can be administered even by non-expert personnel. The aim of the present study is to verify the reliability of our questionnaire in a new cohort of patients. The questionnaire has a five-level score. From March 2008 to January 2011, the questionnaire was administered to 141 coeliac patients on a GFD, who were undergoing re-evaluation. The score obtained was compared with persistence of both villous atrophy and endomysial antibodies (EMA). The rate of lower scores was higher among the patients with persistence of either villous atrophy (Fisher's exact, P,0·001; test for trend, P,0·001) or positive EMA (Fisher's exact, P¼ 0·001; test for trend, P¼0·018). Given that the coeliac patients have been well instructed on what a GFD means and on how to follow it, our questionnaire is a reliable and simple method to verify compliance to a GFD.
Quality of Life and Psychological Disorders in Coeliac Disease: A Prospective Multicentre Study
Nutrients
Coeliac disease (CeD) has been associated with psychological disorders and reduced quality of life. Our prospective study evaluated the changes in the quality of life, anxiety and depression in CeD patients up to two years after diagnosis. We recruited adult patients residing in the Veneto region with a new diagnosis of CeD. Several validated questionnaires were administered to measure quality of life, psychological symptoms and adherence to a gluten-free diet (GFD) at the time of diagnosis and after 1 and 2 years. Ninety-three patients reached the 1-year follow-up (81.7% were females with a median age at diagnosis of 35 years), and 55 patients reached the 2-year follow-up. We observed a significant improvement in quality of life, anxiety and depression scores at 1 year after diagnosis, particularly in patients who complied with a GFD. The improvements among classical CeD patients were similar to those observed in nonclassical patients except for anxiety, which improved only in pati...