Comparison of the EQ-5D-5L and the EQ-5D-3L using individual patient data from the REFORM trial

Background: This study compares the 5-level version of the EQ-5D (5L) with the 3-level version EQ-5D (3L) in older adults using individual patient data from the REFORM (REducing Falls with Orthoses and a Multifaceted podiatry intervention) trial. Methods: EQ-5D-5L and EQ-5D-3L were administered to men and women (n=151) over the age of 65 years alongside the REFORM trial. The two versions of the EQ-5D were assessed in terms of feasibility, level of consistency, ceiling effect and discriminatory power. We also undertook a comparison of the performance of different EQ-5D-3L and EQ-5D-5L value sets. Results: The proportion of participants that returned a complete questionnaire was higher for the 5L (96.7%) than for the 3L (92.7%). Missing values among dimensions were on average 1.59% (5L) and 1.45% (3L). The ceiling effect was reduced from 18.2% (3L) to 6% (5L). On average the proportion of inconsistent responses between both descriptive systems was 3.25%. Redistribution from 3L to 5L showed valid results for the majority of consistent level combinations, with slight inconsistency in the case of Anxiety/Depression. For the 5L, 67 unique health states were observed for the 5L compared to 27 for the 3L. The absolute informatively improved with the new classification system (5.48 for 5L versus 3.91 for 3L) and relative discriminatory power improved slightly on average (0.90 for 5L versus 0.84 for 3L). The mean difference between the EQ-5D-5L and EQ-5D-3L values was 0.091 (range -0.345 to 0.505); whilst the mean difference between the EQ-5D-5L and the crosswalk values was 0.082 (range -0.035 to 0.293). Conclusion: In the REFORM clinical trial involving an elderly population, our study supported the feasibility and convergent validity of both EQ-5D-3L and EQ-5D-5L. Results suggest that the 5L improves the ceiling effect and discriminatory power. The EQ-5D-5L scores were significantly higher than both EQ-5D-3L and crosswalk.

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Estimates of the mean difference in orthopaedic randomized trials: obligatory yet obscure

BMC Medical Research Methodology ◽

10.1186/s12874-021-01249-2 ◽

2021 ◽

Vol 21 (1) ◽

Author(s):

Lauri Raittio ◽

Antti Launonen ◽

Ville M. Mattila ◽

Aleksi Reito

Keyword(s):

Primary Outcome ◽

Outcome Measure ◽

Patient Reported Outcome ◽

Ceiling Effect ◽

Mean Difference ◽

Discussion Section ◽

Power Calculations ◽

Randomized Controlled ◽

Patient Reported ◽

The Mean

Abstract Background Randomized controlled trials in orthopaedics are powered to mainly find large effect sizes. A possible discrepancy between the estimated and the real mean difference is a challenge for statistical inference based on p-values. We explored the justifications of the mean difference estimates used in power calculations. The assessment of distribution of observations in the primary outcome and the possibility of ceiling effects were also assessed. Methods Systematic review of the randomized controlled trials with power calculations in eight clinical orthopaedic journals published between 2016 and 2019. Trials with one continuous primary outcome and 1:1 allocation were eligible. Rationales and references for the mean difference estimate were recorded from the Methods sections. The possibility of ceiling effect was addressed by the assessment of the weighted mean and standard deviation of the primary outcome and its elaboration in the Discussion section of each RCT where available. Results 264 trials were included in this study. Of these, 108 (41 %) trials provided some rationale or reference for the mean difference estimate. The most common rationales or references for the estimate of mean difference were minimal clinical important difference (16 %), observational studies on the same subject (8 %) and the ‘clinical relevance’ of the authors (6 %). In a third of the trials, the weighted mean plus 1 standard deviation of the primary outcome reached over the best value in the patient-reported outcome measure scale, indicating the possibility of ceiling effect in the outcome. Conclusions The chosen mean difference estimates in power calculations are rarely properly justified in orthopaedic trials. In general, trials with a patient-reported outcome measure as the primary outcome do not assess or report the possibility of the ceiling effect in the primary outcome or elaborate further in the Discussion section.

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Convergent validation of a questionnaire to assess the mode and frequency of commuting to and from school

Scandinavian Journal of Public Health ◽

10.1177/1403494817718905 ◽

2017 ◽

Vol 45 (6) ◽

pp. 612-620 ◽

Cited By ~ 27

Author(s):

Palma Chillón ◽

Manuel Herrador-Colmenero ◽

Jairo H. Migueles ◽

Verónica Cabanas-Sánchez ◽

Jorge R. Fernández-Santos ◽

...

Keyword(s):

Physical Activity ◽

Convergent Validity ◽

Vigorous Physical Activity ◽

Mean Difference ◽

Activity Levels ◽

Limits Of Agreement ◽

Google Maps ◽

Journey Time ◽

The Mean ◽

Convergent Validation

Aims: The purposes of this research were to study the convergent validity of the Mode and Frequency of Commuting To and From School Questionnaire using objectively assessed steps and time spent in different physical activity intensities and to compare the self-reported versus objective journey time in Spanish youths. Methods: Three hundred and eighty-nine Spanish youth aged 7–19 years were asked to complete the questionnaire and wore an accelerometer for five days. The objective commuting distance and time from home to school were estimated using Google MapsTM. Results: There were significant differences between passive and walking participants on step numbers, sedentary time and physical activity levels (except for vigorous physical activity in children). For children, a mean difference of −4.03 minutes between objective measured and self-reported journey time was found (95% limits of agreement were 13.55 and −21.60 minutes). For adolescents, the mean difference was −1.39 minutes (95% limits of agreement were 15.23 and −18.02 minutes). Conclusions: The findings indicated that the Mode of Commuting To and From School Questionnaire showed a convergent validity to assess this behaviour in Spanish youths. Self-reported journey time for walking is comparable to Google MapsTM in adolescents but not in children.

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A Head-to-head Comparison of the EQ-5D-3L Index Scores Derived from the Two EQ-5D-3L Value Sets for China

10.21203/rs.3.rs-1250025/v1 ◽

2022 ◽

Author(s):

Ruo-Yu Zhang ◽

Wei Wang ◽

Hui-Jun Zhou ◽

Jianwei Xuan ◽

Nan Luo ◽

...

Keyword(s):

Index Score ◽

Altman Plot ◽

Health States ◽

Minimum Important Difference ◽

Absolute Level ◽

Bland Altman Plot ◽

Health Gains ◽

The Mean ◽

Two Indices ◽

Value Sets

Abstract Background: Two EQ-5D-3L (3L) value sets (developed in 2014 and 2018) co-exist in China. The study examined the level of agreement between index scores for all the 243 health states derived from them at both absolute and relative levels and compared the responsiveness of the two indices. Methods: Intraclass correlations coefficient (ICC) and Bland-Altman plot were adopted to assess the degree of agreement between the two indices at the absolute level. Health gains for 29,403 possible transitions between pairs of 3L health states were calculated to assess the agreement at the relative level. Their responsiveness for the transitions was assessed using Cohen effect size.Results：The mean (standard deviation, SD) value was 0.427 (0.206) and 0.649 (0.189) for the 3L2014 and 3L2018 index scores, respectively. Although the ICC value showed good agreement (i.e., 0.896), 88.9% (216/243) of the points were beyond the minimum important difference limit according to the Bland-Altman plot. The mean health gains for the 29,403 health transitions was 0.234 (3L2014 index score) and 0.216 (3L2018 index score). The two indices predicted consistent transitions in 23,720 (80.7%) of 29,403 pairs. For the consistent pairs, Cohen effective size value was 1.05 (3L2014 index score) or 1.06 (3L2018 index score); and the 3L2014 index score only yielded 0.007 more utility gains. However, the results based on the two measures varied substantially according to the direction and magnitude of health change. Conclusion：The 3L2014 and 3L2018 index scores are not interchangeable. The choice between them is likely to influence QALYs estimations.

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T45. THE EFFICACY AND HETEROGENEITY OF ANTIPSYCHOTIC RESPONSE IN SCHIZOPHRENIA: A META-ANALYSIS

Schizophrenia Bulletin ◽

10.1093/schbul/sbaa029.605 ◽

2020 ◽

Vol 46 (Supplement_1) ◽

pp. S248-S249

Author(s):

Rob McCutcheon ◽

Toby Pillinger ◽

Yuya Mizuno ◽

Adam Montgomery ◽

Haridha Pandian ◽

...

Keyword(s):

Individual Patient Data ◽

Meta Analysis ◽

Symptomatic Improvement ◽

Patient Data ◽

Mean Difference ◽

Sensitivity Analyses ◽

Response To Treatment ◽

Antipsychotic Treatment ◽

Symptom Change ◽

Lower Variability

Abstract Background Antipsychotics are more effective than placebo in reducing symptoms in schizophrenia. However, response to treatment appears to vary, and as such it has been proposed that different subtypes of schizophrenia exist, defined by treatment-response. This has not been formally examined using meta-analysis. Methods Randomised controlled trials comparing placebo and antipsychotics for the acute treatment of schizophrenia published between January 1 1950 and November 30, 2018 were examined. Mean change and variance of change in symptoms were extracted from each study, alongside publication year, participant age and gender, baseline symptom severity, antipsychotic dose, and use of placebo lead-in. Relative variability of symptomatic improvement in antipsychotic-treated individuals compared to placebo-treated individuals was quantified using coefficient of variation ratio (CVR). Mean difference in symptom change was quantified using Hedges’ g. The significance of potential moderating factors was assessed using meta-regression and sensitivity analyses. In addition, individual patient data from two clinical trials (N=522) was examined in terms of both the distribution of total symptom change, and the variability of individual symptoms and symptom factors. Results 11,006 articles were identified. 66 met inclusion criteria, reporting on 17,202 participants. Compared with placebo, antipsychotic-treated patients demonstrated both greater symptomatic improvement (g=0.47, p<0.001) and reduced variability in symptomatic improvement (CVR=0.86, p<0.001). Lower variability in antipsychotic-response was associated with studies including younger patients (z=3.07, p=0.002), those published earlier (z=3.98, p<0.001), with higher dose treatments (z=-2.62, p=0.009), and greater mean-difference in symptom-change (z=-5.70, p<0.001). In the individual patient data antipsychotic treated patients did not show significantly increased variability for any individual symptom, and there was no evidence of a bimodal distribution of response. Discussion Compared to placebo, in addition to a greater mean change, antipsychotic treatment shows lower variability of change in total, positive, and negative symptoms. This is contrary to the hypothesis that there exists a subtype of antipsychotic non-responsive schizophrenia, instead providing evidence for a relatively homogeneous effect of antipsychotic treatment in improving symptoms of schizophrenia.

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Pharyngeal Electrical Stimulation for Treatment of Poststroke Dysphagia: Individual Patient Data Meta-Analysis of Randomised Controlled Trials

Stroke Research and Treatment ◽

10.1155/2015/429053 ◽

2015 ◽

Vol 2015 ◽

pp. 1-8 ◽

Cited By ~ 12

Author(s):

Polly Scutt ◽

Han S. Lee ◽

Shaheen Hamdy ◽

Philip M. Bath

Keyword(s):

Electrical Stimulation ◽

Length Of Stay ◽

Functional Outcome ◽

Randomised Controlled Trials ◽

Rating Scale ◽

Individual Patient Data ◽

Patient Data ◽

Mean Difference ◽

Controlled Trials ◽

Randomised Controlled

Background. Dysphagia after stroke is common, associated independently with poor outcome, and has limited treatment options. Pharyngeal electrical stimulation (PES) is a novel treatment being evaluated for treatment of poststroke dysphagia.Methods. We searched electronically for randomised controlled trials of PES in dysphagic patients within 3 months of stroke. Individual patient data were analysed using regression, adjusted for trial, age, severity, and baseline score. The coprimary outcomes were radiological aspiration (penetration aspiration score, PAS) and clinical dysphagia (dysphagia severity rating scale, DSRS) at 2 weeks; secondary outcomes included functional outcome, death, and length of stay in hospital.Results. Three completed trials were identified: 73 patients, age 72 (12) years, severity (NIHSS) 11 (6), DSRS 6.7 (4.3), mean PAS 4.3 (1.8). Compared with no/sham stimulation, PES was associated with lower PAS, 3.4 (1.7) versus 4.1 (1.7), mean difference −0.9 (p=0.020), and lower DSRS, 3.5 (3.8) versus 4.9 (4.4), mean difference −1.7 (p=0.040). Length of stay in hospital tended to be shorter: 50.2 (25.3) versus 71.2 (60.4) days (p=0.11). Functional outcome and death did not differ between treatment groups.Conclusions. PES was associated with less radiological aspiration and clinical dysphagia and possibly reduced length of stay in hospital across three small trials.

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