Dataset Information

Clinical outcome assessment in malignant glioma trials: measuring signs, symptoms, and functional limitations.

ABSTRACT: The shared goal of all parties developing therapeutics against malignant gliomas is to positively impact the lives of people affected by these cancers. Clinical outcome assessment (COA) tools, including measures of patient-reported outcome, performance outcome, clinician-reported outcome, and observer-reported outcome, allow patient-focused assessments to complement traditional efficacy measures such as overall survival and radiographic endpoints. This review examines the properties of various COA measures used in malignant glioma clinical trials to date and cross references their content to the priority signs, symptoms, and functional limitations defined through a community survey conducted by the National Brain Tumor Society. The overarching goal of this initiative is to identify COA measures that are feasible and have appropriate psychometric properties for use in this patient population as well as highlight where further development is needed.

SUBMITTER: Blakeley JO

PROVIDER: S-EPMC4795998 | biostudies-literature | 2016 Mar

REPOSITORIES: biostudies-literature

ACCESS DATA

Publications

Clinical outcome assessment in malignant glioma trials: measuring signs, symptoms, and functional limitations.

Blakeley Jaishri O JO Coons Stephen Joel SJ Corboy John R JR Kline Leidy Nancy N Mendoza Tito R TR Wefel Jeffrey S JS

Neuro-oncology 20160301

The shared goal of all parties developing therapeutics against malignant gliomas is to positively impact the lives of people affected by these cancers. Clinical outcome assessment (COA) tools, including measures of patient-reported outcome, performance outcome, clinician-reported outcome, and observer-reported outcome, allow patient-focused assessments to complement traditional efficacy measures such as overall survival and radiographic endpoints. This review examines the properties of various C ...[more]

PMID: 26989128

Similar Datasets

Project description:ObjectivesDepressive symptoms can be both a cause and a consequence of functional limitations and medical conditions. Our objectives were to determine the association of depressive symptoms with subsequent total healthcare costs in older women after accounting for functional limitations and multimorbidity.DesignProspective cohort study (Study of Osteoporotic Fractures [SOF]).SettingFour US sites.ParticipantsA total of 2508 community-dwelling women (mean age = 79.4 years) participating in the SOF year 10 (Y10) examination linked with their Medicare claims data.MeasurementsAt Y10, depressive symptoms were measured using the 15-item Geriatric Depression Scale (GDS) and functional limitations were assessed by number (range = 0-5) of impairments in performing instrumental activities of daily living. Multimorbidity was ascertained by the Elixhauser method using claims data for the 12 months preceding the Y10 examination. Total direct healthcare costs, outpatient costs, acute hospital stays, and skilled nursing facility during the 12 months following the Y10 examination were ascertained from claims data.ResultsAnnualized mean (SD) total healthcare costs were $4654 ($9075) in those with little or no depressive symptoms (GDS score = 0-1), $7871 ($14 534) in those with mild depressive symptoms (GDS score = 2-5), and $9010 ($15 578) in those with moderate to severe depressive symptoms (GDS score = 6 or more). After adjustment for age, site, self-reported functional limitations, and multimorbidity, the magnitudes of these incremental costs were partially attenuated (cost ratio = 1.34 [95% confidence interval {CI} = 1.14-1.59] for those with mild depressive symptoms, and cost ratio = 1.29 [95% CI = 0.99-1.69] for those with moderate to severe depressive symptoms vs women with little or no depressive symptoms).ConclusionDepressive symptoms were associated with higher subsequent healthcare costs attributable, in part, to greater functional limitations and multimorbidity among those with symptoms. Importantly, even mild depressive symptoms were associated with higher healthcare costs. J Am Geriatr Soc 67:1596-1603, 2019.

Project description:Symptomatic relief is an important treatment goal for patients with COPD. To date, no diary for evaluating respiratory symptoms in clinical trials has been developed and scientifically-validated according to FDA and EMA guidelines. The EXACT - Respiratory Symptoms (E-RS) scale is a patient-reported outcome (PRO) measure designed to address this need. The E-RS utilizes 11 respiratory symptom items from the existing and validated 14-item EXACT, which measures symptoms of exacerbation. The E-RS total score quantifies respiratory symptom severity, and 3 domains assess breathlessness, cough and sputum, and chest symptoms.This study examined the performance of the E-RS in each of 3 controlled trials with common and unique validation variables: one 6-month (N = 235, US) and two 3-month (N = 749; N = 597; international). Subjects completed the E-RS as part of a daily eDiary. Tests of reliability, validity, and responsiveness were conducted in each dataset.In each study, RS-Total score was internally consistent (Cronbach ?) (0.88, 0.92, 0.92) and reproducible (intra-class correlation) in stable patients (2 days apart: 0.91; 7 days apart: 0.71, 0.74). RS-Total scores correlated significantly with the following criterion variables (Spearman's rho; p < 0.01, all comparisons listed here): FEV1% predicted (-0.19, -0.14, -0.15); St. George's Respiratory Questionnaire (SGRQ) (0.65, 0.52, 0.51); Breathlessness, Cough, and Sputum Scale (BCSS) (0.89, 0.89); modified Medical Research Council dyspnoea scale (mMRC) (0.40); rescue medication use (0.43, 0.42); Functional Performance Inventory Short-Form (FPI-SF) (0.43); 6-minute walk distance (6-MWT) (-0.30, -0.14) and incremental shuttle walk (ISWT) (-0.18) tests. Correlations between these variables and RS-Breathlessness, RS-Cough and Sputum, RS-Chest Symptoms scores supported subscale validity. RS-Total, RS-Breathlessness, and RS-Chest Symptoms differentiated mMRC levels of breathlessness severity (p < 0.0001). RS-Total and domain scores differentiated subjects with no rescue medication use and 3 or more puffs (p < 0.0001). Sensitivity to changes in health status (SGRQ), symptoms (BCSS), and exercise capacity (6MWT, ISWT) were also shown and responder definitions using criterion- and distribution-based methods are proposed.Results suggest the E-RS is a reliable, valid, and responsive measure of respiratory symptoms of COPD suitable for use in natural history studies and clinical trials.MPEX: NCT00739648 ; AZ1: NCT00949975 ; AZ 2: NCT01023516.

Dataset Information

Clinical outcome assessment in malignant glioma trials: measuring signs, symptoms, and functional limitations.

Publications

Clinical outcome assessment in malignant glioma trials: measuring signs, symptoms, and functional limitations.

Similar Datasets

OmicsDI is part of the ELIXIR infrastructure

Tweets