-
1.
Effect of Delayed-Release and Extended-Release Methylphenidate on Caregiver Strain and Validation of Psychometric Properties of the Caregiver Strain Questionnaire: Results from a Phase 3 Trial in Children with Attention-Deficit/Hyperactivity Disorder.
López, FA, Faraone, SV, Newcorn, JH, Doll, HA, Rhoten, S, Lewis, HB, Khan, TF, DeSousa, NJ, Sallee, FR, Incledon, B
Journal of child and adolescent psychopharmacology. 2021;(3):179-186
-
-
Free full text
-
Abstract
Objectives: Inadequately controlled symptoms and associated impaired functioning have a significant negative impact on caregivers of children with attention-deficit/hyperactivity disorder (ADHD). This study aimed to assess the impact of evening-dosed, delayed-release and extended-release methylphenidate (DR/ER-MPH) treatment on caregiver strain, measured by the Caregiver Strain Questionnaire (CGSQ), and present post hoc psychometric analyses assessing the reliability and validity of the CGSQ, its ability to detect change (responsiveness), and to derive responder definitions. Methods: The CGSQ was an exploratory efficacy endpoint in a phase 3, 3-week, randomized, double-blind, multicenter, placebo-controlled, forced-dose titration trial of DR/ER-MPH in children aged 6-12 years with ADHD (NCT02520388). Psychometric properties of the CGSQ evaluated post hoc included internal consistency using Cronbach's alpha; test/retest reliability using intraclass correlation coefficients (ICCs); construct validity (known groups and convergent/divergent validity); responsiveness to changes in assessments of ADHD severity (ADHD Rating Scale-IV [ADHD-RS-IV], Conners' Global Index-Parent [CGI-P], and Clinical Global Impression-Severity [CGI-S]/CGI-Improvement [CGI-I]); and meaningful change threshold (MCT) using receiver operating characteristic curves, which were used to compare response between DR/ER-MPH and placebo groups. Results: Randomized DR/ER-MPH (54.5) and placebo (54.9) groups had similar mean CGSQ scores at screening. Caregivers of children on DR/ER-MPH reported significant reductions in CGSQ scores after 3 weeks of DR/ER-MPH treatment versus placebo (least-squares mean: 41.2 vs. 49.1; p < 0.001). The CGSQ demonstrated strong internal consistency (Cronbach's alpha = 0.93) and good test/retest reliability (ICC = 0.72). Known groups, convergent/divergent validity, and responsiveness were demonstrated from relationships between the CGSQ and the CGI-S, ADHD-RS-IV, and CGI-P. The mean anchor-based MCT for CGSQ total score was estimated as -9.0 (DR/ER-MPH vs. placebo: 53.2% vs. 29.9% p = 0.003). Conclusions: CGSQ scores significantly decreased after 3 weeks of DR/ER-MPH treatment versus placebo, and the CGSQ was found to be a valid and reliable measure of strain in caregivers of children with ADHD. Clinical trial registration identification number: NCT02520388.
-
2.
Health-related Quality of Life in the Phase III LUME-Colon 1 Study: Comparison and Interpretation of Results From EORTC QLQ-C30 Analyses.
Lenz, HJ, Argiles, G, Yoshino, T, Lonardi, S, Falcone, A, Limón, ML, Sobrero, A, Hastedt, C, Peil, B, Voss, F, et al
Clinical colorectal cancer. 2019;(4):269-279.e5
-
-
Free full text
-
Abstract
INTRODUCTION We used European Organization for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC QLQ-C30) data from the LUME-Colon 1 study to illustrate different methods of statistical analysis for health-related quality of life (HRQoL), and compared the results. PATIENTS AND METHODS Patients were randomized 1:1 to receive nintedanib 200 mg twice daily plus best supportive care (n = 386) or matched placebo plus best supportive care (n = 382). Five methods (mean treatment difference averaged over time, using a mixed-effects growth curve model; mixed-effects models for repeated measurements (MMRM); time-to-deterioration (TTD); status change; and responder analysis) were used to analyze EORTC QLQ-C30 global health status (GHS)/QoL and scores from functional scales. RESULTS Overall, GHS/QoL and physical functioning deteriorated over time. Mean treatment difference slightly favored nintedanib over placebo for physical functioning (adjusted mean, 2.66; 95% confidence interval [CI], 0.97-4.34) and social functioning (adjusted mean, 2.62; 95% CI, 0.66-4.47). GHS/QoL was numerically better with nintedanib versus placebo (adjusted mean, 1.61; 95% CI, -0.004 to 3.27). MMRM analysis had similar results, with better physical functioning in the nintedanib group at all timepoints. There was no significant delay in GHS/QoL deterioration (10%) and physical functioning (16%) with nintedanib versus placebo (TTD analysis). Status change analysis showed a higher proportion of patients with markedly improved GHS/QoL and physical functioning in the nintedanib versus placebo groups. Responder analysis showed a similar, less pronounced pattern. CONCLUSION Analyses of EORTC QLQ-C30 data showed that HRQoL was not impaired by treatment with nintedanib versus placebo. Analysis and interpretation of HRQoL endpoints should consider symptom type and severity and course of disease.
-
3.
Rome III vs Rome IV criteria for irritable bowel syndrome: A comparison of clinical characteristics in a large cohort study.
Vork, L, Weerts, ZZRM, Mujagic, Z, Kruimel, JW, Hesselink, MAM, Muris, JWM, Keszthelyi, D, Jonkers, DMAE, Masclee, AAM
Neurogastroenterology and motility. 2018;(2)
Abstract
BACKGROUND The Rome criteria for irritable bowel syndrome (IBS) have been revised and are expected to apply only to the subset of Rome III IBS subjects with abdominal pain as predominant symptom, occurring at least once a week. The aim of this study was to determine the percentage of Rome III IBS subjects that fulfills Rome IV criteria and to evaluate differences between Rome IV-positive and Rome IV-negative subjects. METHODS Four hundred and four Rome III IBS subjects completed a 14-day end-of-day symptom diary, the Gastrointestinal Symptom Rating Scale (GSRS), Hospital Anxiety and Depression Scale, and RAND 36-item Short-Form Health Survey (SF-36). Diary-based surrogate Rome IV criteria were defined as occurrence of abdominal pain at least 1 day each week with a severity of ≥2 (mild; definition 1) or ≥3 (considerable; definition 2). KEY RESULTS Using surrogate Rome IV criteria, 353 (87.4%, definition 1) and 249 (61.6%, definition 2) subjects were defined as Rome IV positive. These patients were more often female, younger, and recruited from secondary/tertiary care compared with Rome IV-negative subjects. They also presented with higher abdominal pain scores and gastrointestinal (GI) symptom severity on both end-of-day diary and GSRS, higher psychological symptom scores, and lower quality of life compared with Rome IV-negative subjects. CONCLUSIONS AND INFERENCES The Rome IV IBS population likely reflects a subgroup of Rome III IBS patients with more severe GI symptomatology, psychological comorbidities, and lower quality of life. This implies that results from Rome III IBS studies may not be directly comparable to those from Rome IV IBS populations.
-
4.
Using classical test theory, item response theory, and Rasch measurement theory to evaluate patient-reported outcome measures: a comparison of worked examples.
Petrillo, J, Cano, SJ, McLeod, LD, Coon, CD
Value in health : the journal of the International Society for Pharmacoeconomics and Outcomes Research. 2015;(1):25-34
Abstract
OBJECTIVE To provide comparisons and a worked example of item- and scale-level evaluations based on three psychometric methods used in patient-reported outcome development-classical test theory (CTT), item response theory (IRT), and Rasch measurement theory (RMT)-in an analysis of the National Eye Institute Visual Functioning Questionnaire (VFQ-25). METHODS Baseline VFQ-25 data from 240 participants with diabetic macular edema from a randomized, double-masked, multicenter clinical trial were used to evaluate the VFQ at the total score level. CTT, RMT, and IRT evaluations were conducted, and results were assessed in a head-to-head comparison. RESULTS Results were similar across the three methods, with IRT and RMT providing more detailed diagnostic information on how to improve the scale. CTT led to the identification of two problematic items that threaten the validity of the overall scale score, sets of redundant items, and skewed response categories. IRT and RMT additionally identified poor fit for one item, many locally dependent items, poor targeting, and disordering of over half the response categories. CONCLUSIONS Selection of a psychometric approach depends on many factors. Researchers should justify their evaluation method and consider the intended audience. If the instrument is being developed for descriptive purposes and on a restricted budget, a cursory examination of the CTT-based psychometric properties may be all that is possible. In a high-stakes situation, such as the development of a patient-reported outcome instrument for consideration in pharmaceutical labeling, however, a thorough psychometric evaluation including IRT or RMT should be considered, with final item-level decisions made on the basis of both quantitative and qualitative results.
-
5.
Rankin scale as a potential measure of global disability in early Parkinson's disease.
Simuni, T, Luo, ST, Chou, KL, Fernandez, H, He, B, Parashos, S
Journal of clinical neuroscience : official journal of the Neurosurgical Society of Australasia. 2013;(9):1200-3
-
-
Free full text
-
Abstract
We conducted an exploratory analysis of the utility of the modified Rankin Scale (mRS) as a global measure of disability in early Parkinson's diesase (PD) using the baseline data from a large cohort of PD patients enrolled in a longitudinal study of creatine. The mRS is scored 0-6 with lower scores reflecting less disability. For the analysis the mRS score was dichotomized at <2 versus ≥2. We explored the association of the mRS with multiple measures of PD-related impairments, including the Unified Parkinson Disease Rating Scale (UPDRS); cognitive function characterized by the Symbol Digit Modalities--verbal, and Scales for Outcomes in Parkinson's disease--cognition (SCOPA-COG); quality of life (Parkinson's disease questionnaire [PDQ-39]) and EuroQOL; Beck Depression Inventory II (BDI); and Total Functional Capacity (TFC). We also investigated the interaction between variables. One thousand seven hundred forty-one patients were included in the analysis of which 374 had a mRS score of 2 or above. In the univariate model, all interested measures except SCOPA-COG (p=0.23) had significant association with mRS (p<0.001) after controlling for confounders. In the multivariate model, UPDRS Part II and III (activities of daily living and motor), BDI, TFC and PDQ-39 were significant (p<0.05). The mRS has a significant association with the wide spectrum of measures of impairment and quality of life in early PD and shows good potential to be a global measure of disability in early PD. The sensitivity of the mRS to change and performance of the scale in more advanced PD will have to be established longitudinally.
-
6.
Effectiveness and cost-effectiveness of early screening and treatment of malnourished patients.
Kruizenga, HM, Van Tulder, MW, Seidell, JC, Thijs, A, Ader, HJ, Van Bokhorst-de van der Schueren, MA
The American journal of clinical nutrition. 2005;(5):1082-9
-
-
Free full text
-
Abstract
BACKGROUND About 25-40% of hospital patients are malnourished. With current clinical practices, only 50% of malnourished patients are identified by the medical and nursing staff. OBJECTIVE The objective of this study was to report the cost and effectiveness of early recognition and treatment of malnourished hospital patients with the use of the Short Nutritional Assessment Questionnaire (SNAQ). DESIGN The intervention group consisted of 297 patients who were admitted to 2 mixed medical and surgical wards and who received both malnutrition screening at admission and standardized nutritional care. The control group consisted of a comparable group of 291 patients who received the usual hospital clinical care. Outcome measures were weight change, use of supplemental drinks, use of tube feeding, use of parenteral nutrition and in-between meals, number of consultations by the hospital dietitian, and length of hospital stay. RESULTS The recognition of malnutrition improved from 50% to 80% with the use of the SNAQ malnutrition screening tool during admission to the hospital. The standardized nutritional care protocol added approximately 600 kcal and 12 g protein to the daily intake of malnourished patients. Early screening and treatment of malnourished patients reduced the length of hospital stay in malnourished patients with low handgrip strength (ie, frail patients). To shorten the mean length of hospital stay by 1 d for all malnourished patients, a mean investment of 76 euros (91 US dollars) in nutritional screening and treatment was needed. The incremental costs were comparably low in the whole group and in the subgroup of malnourished patients with low handgrip strength. CONCLUSIONS Screening with the SNAQ and early standardized nutritional care improves the recognition of malnourished patients and provides the opportunity to start treatment at an early stage of hospitalization. The additional costs of early nutritional care are low, especially in frail malnourished patients.
-
7.
Agreement between a brief mailed screener and an in-depth telephone survey: observations from the Fresh Start study.
Snyder, DC, Sloane, R, Lobach, D, Lipkus, I, Clipp, E, Kraus, WE, Demark-Wahnefried, W, ,
Journal of the American Dietetic Association. 2004;(10):1593-6
Abstract
Brief screening instruments can roughly characterize individual behavior and target those most in need of change. However, the level of agreement between abbreviated and full-scale instruments is often unknown. We determined agreement between a brief screener and an in-depth survey for assessing eligibility into a randomized controlled trial to improve lifestyle behaviors among cancer survivors who consumed diets with >or=30% total energy from fat or fewer than five servings of fruits and vegetables per day, and/or who exercised <150 min/wk. Responses of 203 subjects to mailed screeners, which included scales from the National Cancer Institute (NCI)Percent Energy from Fat Screener and 5-A-Day trials, and one item on exercise, were compared with data from telephone interviews using the Diet History Questionnaire and the 7-Day Physical Activity Recall. Moderate correlations and fair agreement existed between screener and survey for intakes of fat [ r =0.54 ( P <.0001)/kappa statistic (ks)=0.35] and fruits and vegetables [ r =0.50 ( P <.0001)/ks=0.32], whereas agreement was low for exercise (ks=0.15). NCI and 5-A-Day screening instruments perform relatively well in targeting cancer survivors most in need of dietary change. Decisions to use brief screening instruments should be based on available resources and tolerance for misclassification.
-
8.
The Yale Physical Activity Survey for older adults: predictions in the energy expenditure due to physical activity.
Kruskall, LJ, Campbell, WW, Evans, WJ
Journal of the American Dietetic Association. 2004;(8):1251-7
Abstract
OBJECTIVE To evaluate the accuracy of the Yale Physical Activity Survey (YPAS) for older adults. DESIGN Fourteen-week strictly controlled diet study. SUBJECTS/SETTING Eleven men and 17 women, age range 55 to 78 years, spent 10 weeks in an outpatient setting and 4 weeks in an inpatient setting at the General Clinical Research Center, Noll Physiological Research Center, The Pennsylvania State University, University Park. INTERVENTION Subjects were provided dietary energy to maintain body weight within +/-0.5 kg of baseline weight. The daily menus contained 0.8 g protein per kilogram body weight and nonprotein energy as 60% carbohydrate and 40% fat. MAIN OUTCOME MEASURES Metabolizable energy intake (MEI) was measured at week 14 from the gross energy contents of food, urine, and feces, with corrections for any body composition changes during the last 6 weeks of weight maintenance. Resting energy expenditure (REE) was determined using indirect calorimetry. The thermic effect of feeding (TEF) was estimated to be 10% of the MEI. The energy expenditure due to physical activity (EEPA) was derived by the formula: EEPA=MEI-REE-TEF. This value was compared with the EEPA estimated from the YPAS. Statistical analyses performed Two-factor analysis of variance with repeated measures and paired t tests. RESULTS At week 14, after a minimum of 6 weeks of sustained weight stability, the derived EEPA was not different from that estimated using the YPAS for the men and the men and women combined, while the YPAS estimate was more than the measured mean value for the women (P<.05). There was wide variability in the accuracy of the EEPA prediction for individual subjects (range=-637 to 794 kcal). APPLICATIONS/CONCLUSIONS The YPAS may be used, with caution, to estimate the EEPA for groups of older individuals, and may provide inaccurate estimates of the EEPA in older individuals.
-
9.
Measuring adherence in a hypertension clinical trial.
Hamilton, GA
European journal of cardiovascular nursing. 2003;(3):219-28
Abstract
BACKGROUND Non-adherence in hypertension is a global problem and promoting adherence is necessary to decrease cardiovascular mortality. AIMS The purpose of this paper is to examine the measurement of adherence to medication taking in hypertensive patients. Adherence was evaluated primarily by means of MEMS (Medication Event Monitoring System, Aprex Corporation, Fremont, California) an electronic system that records the date and time of opening of the study medication container. Additional measurements such as change in urinary potassium level, capsule count, client self report and physician estimate of adherence were recorded. METHODS A randomised clinical trial was used to assign patients to receive the study medication (potassium) or placebo. Descriptive statistics were used to answer the research questions. Frequency and percentage of responses to different measures of adherence were carried out as well as correlation between the measures. RESULTS One hundred and seven subjects between the ages of 26 and 80 participated in the clinical trial. The results showed that adherence measures varied with lowest adherence from two items of self-report related to forgetfulness (46 and 55%) and stringent electronic monitoring with the MEMS (58%) to percentages in the 80-90 range for other self-report items and the general adherence scale. Electronic monitoring correlated best with capsule count at visit 5. Implications for health care providers are discussed.
-
10.
Comparability of epidemiological information between self- and interviewer-administered questionnaires.
Okamoto, K, Ohsuka, K, Shiraishi, T, Hukazawa, E, Wakasugi, S, Furuta, K
Journal of clinical epidemiology. 2002;(5):505-11
Abstract
The aim of the present study was to determine the extent to which respondents provided the same answers to a health-related lifestyle questionnaire in self- and interviewer-administered forms. A total of 234 subjects completed a 110-item questionnaire in both interviewer and self-administered forms. Modes of administration were separated by a 2-week interval. The order was determined by random allocation. The presence and the extent of the tendency to give socially acceptable responses were evaluated using percentage of bias calculated as the ratio of the difference in proportion of positive responses or the mean between interviews and questionnaires and those in questionnaires. All percentages of bias were in the positive direction, ranging from 1.4% (physical exercise) to 26.1% (general life stress). The average percentage of bias was higher in women than in men and were stronger for younger respondents. The age differences between interviewer and respondent were inversely and most strongly related to percentage of bias. Self- and interviewer-administered questionnaires yield very similar results in discriminating between subjects, but the interviewer-administered questionnaire showed systematically more desirable responses to questions related to lifestyle factors. The differences in characteristics between interviewer and respondent may be important determinants of the socially desirability bias in the interview.