scholarly journals Outcome measures in coeliac disease trials: the Tampere recommendations

Gut ◽  
2018 ◽  
Vol 67 (8) ◽  
pp. 1410-1424 ◽  
Author(s):  
Jonas F Ludvigsson ◽  
Carolina Ciacci ◽  
Peter HR Green ◽  
Katri Kaukinen ◽  
Ilma R Korponay-Szabo ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Coeliac Disease ◽  
Outcome Measures ◽  
Patient Reported Outcomes ◽  
Treatment Strategies ◽  
Therapeutic Trials ◽  
Immunogenic Peptides ◽  
Patient Reported ◽  
Patient Representatives

ObjectiveA 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.DesignBased 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.ResultsWe 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.ConclusionCareful evaluation and reporting of outcome measures will increase transparency and comparability of coeliac disease therapeutic trials, and will benefit patients, healthcare and the pharmaceutical industry.

scholarly journals Systematic Review of Outcome Measures Used in Observational Studies of Adults with Eosinophilic Esophagitis

2021 ◽  
pp. 1-25
Author(s):  
Alain M. Schoepfer ◽  
Camilla Schürmann ◽  
Sven Trelle ◽  
Marcel Zwahlen ◽  
Christopher Ma ◽  
...  
Keyword(s):  
Systematic Review ◽  
Outcome Measures ◽  
Eosinophilic Esophagitis ◽  
Observational Studies ◽  
Patient Reported Outcomes ◽  
Case Series ◽  
Open Label ◽  
Topical Corticosteroids ◽  
Therapeutic Trials ◽  
Patient Reported

<b><i>Background:</i></b> Over the last 20 years, diverse outcome measures have been used to evaluate the effectiveness of therapies for eosinophilic esophagitis (EoE). This systematic review aims to identify the readouts used in observational studies of topical corticosteroids, diet, and dilation in adult EoE patients. <b><i>Methods:</i></b> We searched MEDLINE and Embase for prospective and retrospective studies (cohorts/case series, randomized open-label, and case-control) evaluating the use of diets, dilation, and topical corticosteroids in adults with EoE. Two authors independently assessed the articles and extracted information about histologic, endoscopic, and patient-reported outcomes and tools used to assess treatment effects. <b><i>Results:</i></b> We included 69 studies that met inclusion criteria. EoE-associated endoscopic findings (assessed either as absence/presence or using Endoscopic Reference Score) were evaluated in 24/35, 11/17, and 9/17 studies of topical corticosteroids, diet, and dilation, respectively. Esophageal eosinophil density was recorded in 32/35, 17/17, and 11/17 studies of topical corticosteroids, diet, and dilation, respectively. Patient-reported outcomes were not uniformly used (only in 14, 8, and 3 studies of topical corticosteroids, diet, and dilation, respectively), and most tools were not validated for use in adults with EoE. <b><i>Conclusions:</i></b> Despite the lack of an agreed set of core outcomes that should be recorded and reported in studies in adult EoE patients, endoscopic EoE-associated findings and esophageal eosinophil density are commonly used to assess disease activity in observational studies. Standardization of outcomes and data supporting the use of outcomes are needed to facilitate interpretation of evidence, its synthesis, and comparisons of interventions in meta-analyses of therapeutic trials in adults with EoE.

scholarly journals Characteristics and Patient-reported Outcomes Associated to Dropout in Severely Affected Oncological Patients

2020 ◽  
Author(s):  
Pimrapat Gebert ◽  
Daniel Schindel ◽  
Johann Frick ◽  
Liane Schenk ◽  
Ulrike Grittner
Keyword(s):  
Quality Of Life ◽  
Social Support ◽  
Cancer Patients ◽  
Outcome Measures ◽  
Patient Reported Outcomes ◽  
Patient Reported Outcome Measures ◽  
Patient Reported Outcome ◽  
Drop Out ◽  
Patient Reported

Abstract BackgroundPatient-reported outcome measures are commonly used and are surrogates for clinical outcomes in cancer research. In the research setting of very severe diseases such as cancer, it is difficult to avoid the problem of incomplete questionnaires from drop-out or missing data due to patients who deceased during observation period. We aimed to explore patient characteristics and patient-reported outcomes associated with the time-to-dropout. MethodsIn the Oncological Social Care Project (OSCAR) study the condition of participants was assessed four times within 12 months (t0: baseline, t1: 3 months, t2: 6 months, t3:12 months) by validated patient-reported outcome measures. We performed competing-risks regression based on Fine and Gray’s proportional sub-distribution hazards model for exploring factors associated with time-to-dropout. Death was considered a competing risk. ResultsThree hundred sixty-two participants were analyzed in the study. 193 (53.3%) completed follow-up at 12 months, 67 (18.5%) patients dropped out, and 102 patients (28.2%) died during the study period. Poor subjective social support was related to higher risk for drop-out (SHR=2.10; 95%CI: 1.01 – 4.35). Lower values in quality of life (EORTC QLQ-C30) were related to drop-out and death. The subscales global health status/QoL, role functioning, physical functioning, and fatigue symptom were key characteristics associated with drop-out.ConclusionSeverely affected cancer patients with poor social support and poor quality of life seem more likely to drop out of studies compared to patients with higher levels of social support and quality of life. This should be considered when planning studies assessing cancer patients. Methods to monitor drop-outs timely and handle missing outcomes might be used. Results of such studies have to be interpreted with caution in light of the particular drop-out mechanisms.

scholarly journals Use of the patient-reported outcomes measurement information system (PROMIS®) to assess late-onset Pompe disease severity

2020 ◽  
Vol 4 (1) ◽  
Author(s):  
Melodi Harfouche ◽  
Priya S. Kishnani ◽  
Eva Krusinska ◽  
Jamie Gault ◽  
Sheela Sitaraman ◽  
...  
Keyword(s):  
Clinical Outcome ◽  
Outcome Measures ◽  
Pompe Disease ◽  
Patient Reported Outcomes ◽  
Vital Capacity ◽  
Late Onset ◽  
Walk Distance ◽  
Patient Reported ◽  
Minute Walk Distance ◽  
Minute Walk

Abstract Background Patient-Reported Outcomes provide an opportunity for patients to establish dialogue with pharmaceutical or biotechnology companies about their health conditions without interpretation by a clinician or anyone else. However, Patient-Reported Outcomes that can be widely applicable for use in patient-focused drug development or clinical trial designs are not yet validated for all diseases. The aim of this study report was to provide supportive evidence of the construct and content validity of selected Patient-Reported Outcomes Measurement Information System (PROMIS®) questionnaires compared with other disease-relevant clinical outcome measures, including the 6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test, in late-onset Pompe disease and to provide supportive evidence that the selected PROMIS measures are relevant and important to these patients. Methods Thirty patients with late-onset Pompe disease completed five PROMIS questionnaires that were chosen based on patient and provider feedback, along with discussion with key opinion leaders who are experts in Pompe disease. The Amicus Pompe Patient Advisory Board also provided patient experience feedback using the PROMIS questionnaires. Clinical outcome measures (6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test) were collected at the Duke University Pompe Disease Clinical Research Program during a single visit. Results The Patient Advisory Board rated the questionnaires as representative of an unmet need. Correlation data demonstrated moderate to strong correlations of PROMIS questionnaires with the specified clinical outcome measures (6-Minute Walk Distance, forced vital capacity, and Manual Muscle Test). These data supported the construct and content validity of the PROMIS questionnaires because they confirmed the motor signs and symptoms of functional disability observed in patients with Pompe disease. Conclusions The correlations indicate that the clinical outcome measures assess important concepts related to patient-reported experiences. The Patient Advisory Board findings suggest that the selected PROMIS questionnaires are meaningful and address important concepts to patients with Pompe disease. The data were collected from a small number of patients at a single time point; further studies are needed with additional PROMIS questionnaires, which should include measures of motor function and health-related quality of life, in a larger number of patients followed up longitudinally.

P048 QUALITY OF LIFE AND OTHER PATIENT REPORTED OUTCOMES IN PATIENTS WITH POUCHITIS

2020 ◽  
Vol 158 (3) ◽  
pp. S107
Author(s):  
Edward Barnes ◽  
Millie Long ◽  
Laura Raffals ◽  
Xian Zhang ◽  
Anuj Vyas ◽  
...  
Keyword(s):  
Quality Of Life ◽  
Patient Reported Outcomes ◽  
Patient Reported

Understanding quality of life and treatment history of patients with Bertolotti syndrome compared with lumbosacral radiculopathy

2019 ◽  
Vol 31 (2) ◽  
pp. 222-228 ◽  
Author(s):  
Joshua L. Golubovsky ◽  
Arbaz Momin ◽  
Nicolas R. Thompson ◽  
Michael P. Steinmetz
Keyword(s):  
Quality Of Life ◽  
Mental Health ◽  
Propensity Score ◽  
Disc Herniation ◽  
Symptom Onset ◽  
Patient Reported Outcomes ◽  
Lumbosacral Radiculopathy ◽  
Patient Reported ◽  
Age And Sex

OBJECTIVEBertolotti syndrome is a rare spinal condition that causes low-back pain due to a lumbosacral transitional vertebra (LSTV), which is a pseudoarticulation between the fifth lumbar transverse process and the sacral ala. Bertolotti syndrome patients are rarely studied, particularly with regard to their quality of life. This study aimed to examine the quality of life and prior treatments in patients with Bertolotti syndrome at first presentation to the authors’ center in comparison with those with lumbosacral radiculopathy.METHODSThis study was a retrospective cohort analysis of patients with Bertolotti syndrome and lumbosacral radiculopathy due to disc herniation seen at the authors’ institution’s spine center from 2005 through 2018. Diagnoses were confirmed with provider notes and imaging. Variables collected included demographics, diagnostic history, prior treatment, patient-reported quality of life metrics, and whether or not they underwent surgery at the authors’ institution. Propensity score matching by age and sex was used to match lumbosacral radiculopathy patients to Bertolotti syndrome patients. Group comparisons were made using t-tests, Fisher’s exact test, Mann-Whitney U-tests, Cox proportional hazards models, and linear regression models where variables found to be different at the univariate level were included as covariates.RESULTSThe final cohort included 22 patients with Bertolotti syndrome who had patient-reported outcomes data available and 46 propensity score–matched patients who had confirmed radiculopathy due to disc herniation. The authors found that Bertolotti syndrome patients had significantly more prior epidural steroid injections (ESIs) and a longer time from symptom onset to their first visit. Univariate analysis showed that Bertolotti syndrome patients had significantly worse Patient-Reported Outcomes Measurement Information System (PROMIS) mental health T-scores. Adjustment for prior ESIs and time from symptom onset revealed that Bertolotti syndrome patients also had significantly worse PROMIS physical health T-scores. Time to surgery and other quality of life metrics did not differ between groups.CONCLUSIONSPatients with Bertolotti syndrome undergo significantly longer workup and more ESIs and have worse physical and mental health scores than age- and sex-matched patients with lumbosacral radiculopathy. However, both groups of patients had mild depression and clinically meaningful reduction in their quality of life according to all instruments. This study shows that Bertolotti syndrome patients have a condition that affects them potentially more significantly than those with lumbosacral radiculopathy, and increased attention should be paid to these patients to improve their workup, diagnosis, and treatment.

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