Clinical trial adaptation by matching evidence in complementary patient sub-groups of auxiliary blinding questionnaire responses

Clinical trial adaptation refers to any adjustment of the trial protocol after the onset of the trial. Such adjustment may take on various forms, including the change in the dose of administered medicines, the frequency of administering an intervention, the number of trial participants, or the duration of the trial, to name just some possibilities. The main goal is to make the process of introducing new medical interventions to patients more efficient, either by reducing the cost or the time associated with evaluating their safety and efficacy. The principal challenge, which is an outstanding research problem, is to be found in the question of how adaptation should be performed so as to minimize the chance of distorting the outcome of the trial. In this paper we propose a novel method for achieving this. Unlike most of the previously published work, our approach focuses on trial adaptation by sample size adjustment i.e. by reducing the number of trial participants in a statistically informed manner. We adopt a stratification framework recently proposed for the analysis of trial outcomes in the presence of imperfect blinding and based on the administration of a generic auxiliary questionnaire that allows the participants to express their belief concerning the assigned intervention (treatment or control). We show that this data, together with the primary measured variables, can be used to make the probabilistically optimal choice of the particular sub-group a participant should be removed from if trial size reduction is desired. Extensive experiments on a series of simulated trials are used to illustrate the effectiveness of our method.

Reproducibility of the online food4me food-frequency questionnaire for estimating dietary intakes across Europe

BACKGROUND: Accurate dietary assessment is key to understanding nutrition-related outcomes and is essential for estimating dietary change in nutrition-based interventions. OBJECTIVE: The objective of this study was to assess the pan-European reproducibility of the Food4Me food-frequency questionnaire (FFQ) in assessing the habitual diet of adults. METHODS: Participants from the Food4Me study, a 6-mo, Internet-based, randomized controlled trial of personalized nutrition conducted in the United Kingdom, Ireland, Spain, Netherlands, Germany, Greece, and Poland, were included. Screening and baseline data (both collected before commencement of the intervention) were used in the present analyses, and participants were included only if they completed FFQs at screening and at baseline within a 1-mo timeframe before the commencement of the intervention. Sociodemographic (e.g., sex and country) and lifestyle [e.g., body mass index (BMI, in kg/m(2)) and physical activity] characteristics were collected. Linear regression, correlation coefficients, concordance (percentage) in quartile classification, and Bland-Altman plots for daily intakes were used to assess reproducibility. RESULTS: In total, 567 participants (59% female), with a mean ± SD age of 38.7 ± 13.4 y and BMI of 25.4 ± 4.8, completed both FFQs within 1 mo (mean ± SD: 19.2 ± 6.2 d). Exact plus adjacent classification of total energy intake in participants was highest in Ireland (94%) and lowest in Poland (81%). Spearman correlation coefficients (ρ) in total energy intake between FFQs ranged from 0.50 for obese participants to 0.68 and 0.60 in normal-weight and overweight participants, respectively. Bland-Altman plots showed a mean difference between FFQs of 210 kcal/d, with the agreement deteriorating as energy intakes increased. There was little variation in reproducibility of total energy intakes between sex and age groups. CONCLUSIONS: The online Food4Me FFQ was shown to be reproducible across 7 European countries when administered within a 1-mo period to a large number of participants. The results support the utility of the online Food4Me FFQ as a reproducible tool across multiple European populations. This trial was registered at clinicaltrials.gov as NCT01530139.

The association between anxiety symptoms and sleep in school-aged children: a combined insight from the Children's Sleep Habits Questionnaire and actigraphy

The current study assessed the association between anxiety symptoms and sleep in 90 school-aged children, aged 6-12 years (Mage = 108 months, 52.2% male). The Children's Sleep Habits Questionnaire (CSHQ) and 14 nights of actigraphy were used to assess sleep. Anxiety was assessed using the Spence Children's Anxiety Scale (SCAS). A significant association was found between parent-reported anxiety symptoms and current sleep problems (i.e., CSHQ total scores ≥ 41). An examination of SCAS subscales identified a specific association between generalized anxiety disorder (GAD) symptoms and increased parental sleep concerns, including sleep onset delay, sleep duration, and daytime sleepiness. Regarding actigraphy, whilst anxiety was not associated with average sleep variables, a relationship was identified between anxiety and the night-to-night variability of actigraphy-derived sleep schedules.

The Short Questionnaire to Assess Health-Enhancing (SQUASH) physical activity in adolescents: a validation using doubly labeled water

Accurate assessment of physical activity energy expenditure (PAEE) among adolescents is important for surveillance, evaluating interventions, and understanding the relation between energy balance and normal physiological and behavioral growth and development. The purpose of this study was to examine the validity of the Short Questionnaire to Assess Health-Enhancing Physical Activity (SQUASH)13 for measuring PAEE among adolescents. Methods: The participants were seventeen adolescents (9 females; Mean age = 17.53; SD = 0.62). Energy expenditure was measured during a 9-day period with doubly labeled water (DLW). The SQUASH was self-administered on the morning of the 10th day and assessed commuting activities, leisure time activities, household activities, and activities at work and school over the previous 9 days. Results: A Bland-Altman plot indicated that the SQUASH underestimated PAEE compared with DLW by a mean difference of 126 kcal·d-1 (95% limits of agreement:-1,207 to 1,459 kcal·d-1), representative of a 10% underestimation. The Spearman rank order correlation coefficient showed there was a significant association between the SQUASH and DLW (r =.50, P =.04), for estimating PAEE. Conclusion: When using a sample of highly active adolescents, the SQUASH is a valid self-report tool for measuring PAEE at the group and individual rank order level.

Text analytics visualisation of Course Experience Questionnaire student comment data in science and technology

A version of the Course Experience Questionnaire (CEQ) has been included in the Graduate Careers Council of Australia national survey of university graduates from 1993 onward. In addition to the quantitative response items noted above, the CEQ also includes an invitation to respondents to write open-ended comments on the best aspects (BA) of their university course experience and those aspects most needing improvement (NI).

Development and preliminary evaluation of the OsteoArthritis Questionnaire (OA-Quest): a psychometric study

OBJECTIVE: This study reports the development of the OsteoArthritis Questionnaire (OA-Quest) - a new measure designed to comprehensively capture the potentially modifiable burden of osteoarthritis.

DESIGN: Item development was guided by the a priori conceptual framework of the Personal Burden of Osteoarthritis (PBO) which captures 8 dimensions of osteoarthritis burden (Physical distress, Fatigue, Physical limitations, Psychosocial distress, Physical de-conditioning, Financial hardship, Sleep disturbances, Lost productivity). One hundred and twenty three candidate items were pretested in a clinical sample of 18 osteoarthritis patients. The measurement properties of the OA-Quest were assessed with exploratory factor analysis (EFA), Rasch modelling, and confirmatory factor analysis (CFA) in a community-based sample (n = 792).

RESULTS: EFA replicated 7 of the 8 PBO domains. An exception was PBO Fatigue domain, with items merging into the Physical distress subscale in the OA-Quest. Following item analysis, a 42-item 7-subscale questionnaire was constructed, measuring Physical distress (seven items, Cronbach's α = 0.93), Physical limitations (11 items, α = 0.95), Psychosocial distress (seven items, α = 0.93), Physical de-conditioning (four items, α = 0.87), Financial hardship (four items, α = 0.93), Sleep disturbances (five items, α = 0.96), and Lost productivity (four items α = 0.90). A highly restricted 7-factor CFA model had excellent fit with the data (χ(2)(113) = 316.36, P < 0.001; chi-square/degrees of freedom = 2.8; comparative fit index [CFI] = 0.97; root mean square error of approximation [RMSEA] = 0.07), supporting construct validity of the new measure.

CONCLUSIONS: The OA-Quest is a new measure of osteoarthritis burden that is founded on a comprehensive conceptual model. It has strong evidence of construct validity and provides reliable measurement across a broad range of osteoarthritis burden.

Intensive care nurses' knowledge of enteral nutrition: a descriptive questionnaire

BACKGROUND: Nurses have an important role in the delivery and management of enteral nutrition in critically ill patients, to prevent iatrogenic malnutrition. It is not clear how nurses source enteral nutrition information. 

Validity of short food questionnaire items to measure intake in children and adolescents: a systematic review

BACKGROUND: Short food questions are appealing to measure dietary intakes. METHODS: A review of studies published between 2004 and 2016 was undertaken and these were included in the present study if they reported on a question or short item questionnaire (≤50 items, data presented as ≤30 food groups) measuring food intake or food-related habits, in children (aged 6 months to 18 years), and reported question validity or reliability. Thirty studies met the inclusion criteria. RESULTS: Most questions assessed foods or food groups (n = 29), with the most commonly assessed being fruit (n = 22) or vegetable intake (n = 23), dairy foods and discretionary foods (n = 20 studies each). Four studies assessed food habits, with the most common being breakfast and meal frequency (n = 4 studies). Twenty studies assessed reliability, and 25 studies determined accuracy and were most commonly compared against food records. Evaluation of question performance relied on statistical tests such as correlation. CONCLUSIONS: The present study has identified valid and reliable questions for the range of key food groups of interest to public health nutrition. Questions were more likely to be reliable than accurate, and relatively few questions were both reliable and accurate. Gaps in repeatable and valid short food questions have been identified that will provide direction for future tool development.

Cultural adaptation and validation of the Health Literacy Questionnaire (HLQ): robust nine-dimension Danish language confirmatory factor model

Health literacy is an important construct in population health and healthcare requiring rigorous measurement. The Health Literacy Questionnaire (HLQ), with nine scales, measures a broad perception of health literacy. This study aimed to adapt the HLQ to the Danish setting, and to examine the factor structure, homogeneity, reliability and discriminant validity. The HLQ was adapted using forward-backward translation, consensus conference and cognitive interviews (n = 15). Psychometric properties were examined based on data collected by face-to-face interview (n = 481). Tests included difficulty level, composite scale reliability and confirmatory factor analysis (CFA). Cognitive testing revealed that only minor re-wording was required. The easiest scale to respond to positively was 'Social support for health', and the hardest were 'Navigating the healthcare system' and 'Appraisal of health information'. CFA of the individual scales showed acceptably high loadings (range 0.49-0.93). CFA fit statistics after including correlated residuals were good for seven scales, acceptable for one. Composite reliability and Cronbach's α were >0.8 for all but one scale. A nine-factor CFA model was fitted to items with no cross-loadings or correlated residuals allowed. Given this restricted model, the fit was satisfactory. The HLQ appears robust for its intended application of assessing health literacy in a range of settings. Further work is required to demonstrate sensitivity to measure changes.

Meaning in life in earlier and later older-adulthood : confirmatory factor analysis and correlates of the meaning in life questionnaire

OBJECTIVE: To validate the Meaning in Life Questionnaire (MLQ) in earlier and later older-adulthood, and examine its correlates. METHOD: Participants in earlier (n = 341, M age = 68.5) and later older-adulthood (n = 341, M age = 78.6) completed the MLQ and other measures. Confirmatory multigroup analysis, correlations, and regression models were conducted. RESULTS: A two-factor (presence and search), eight-item model of the MLQ had a good fit and was age-invariant. Presence and search for meaning were largely unrelated. Meaning was associated with life satisfaction, well-being across a range of domains, and psychological resources. Searching for meaning correlated negatively with these variables, but to a lesser degree in later older-adulthood. DISCUSSION: The MLQ is valid in older-adulthood. Meaning in life is psychologically adaptive in older-adulthood. Searching for meaning appears less important, especially in later older-adulthood. Findings are discussed in the context of aging and psychosocial development.