Practical Implications of Sum Scores Being Psychometrics' Greatest Accomplishment.

This paper reflects on some practical implications of the excellent treatment of sum scoring and classical test theory (CTT) by Sijtsma et al. (Psychometrika 89(1):84-117, 2024). I have no major disagreements about the content they present and found it to be an informative clarification of the properties and possible extensions of CTT. In this paper, I focus on whether sum scores-despite their mathematical justification-are positioned to improve psychometric practice in empirical studies in psychology, education, and adjacent areas. First, I summarize recent reviews of psychometric practice in empirical studies, subsequent calls for greater psychometric transparency and validity, and how sum scores may or may not be positioned to adhere to such calls. Second, I consider limitations of sum scores for prediction, especially in the presence of common features like ordinal or Likert response scales, multidimensional constructs, and moderated or heterogeneous associations. Third, I review previous research outlining potential limitations of using sum scores as outcomes in subsequent analyses where rank ordering is not always sufficient to successfully characterize group differences or change over time. Fourth, I cover potential challenges for providing validity evidence for whether sum scores represent a single construct, particularly if one wishes to maintain minimal CTT assumptions. I conclude with thoughts about whether sum scores-even if mathematically justified-are positioned to improve psychometric practice in empirical studies.

On the use of the healthy lifestyle index to investigate specific disease outcomes.

The healthy lifestyle index (HLI), defined as the unweighted sum of individual lifestyle components, was used to investigate the combined role of lifestyle factors on health-related outcomes. We introduced weighted outcome-specific versions of the HLI, where individual lifestyle components were weighted according to their associations with disease outcomes. Within the European Prospective Investigation into Cancer and Nutrition (EPIC), we examined the association between the standard and the outcome-specific HLIs and the risk of T2D, CVD, cancer, and all-cause premature mortality. Estimates of the hazard ratios (HRs), the Harrell's C-index and the population attributable fractions (PAFs) were compared. For T2D, the HR for 1-SD increase of the standard and T2D-specific HLI were 0.66 (95% CI: 0.64, 0.67) and 0.43 (0.42, 0.44), respectively, and the C-index were 0.63 (0.62, 0.64) and 0.72 (0.72, 0.73). Similar, yet less pronounced differences in HR and C-index were observed for standard and outcome-specific estimates for cancer, CVD and all-cause mortality. PAF estimates for mortality before age 80 were 57% (55%, 58%) and 33% (32%, 34%) for standard and mortality-specific HLI, respectively. The use of outcome-specific HLI could improve the assessment of the role of lifestyle factors on disease outcomes, thus enhancing the definition of public health recommendations.

Healthy lifestyle change and all-cause and cancer mortality in the European Prospective Investigation into Cancer and Nutrition cohort.

BACKGROUND: Healthy lifestyles are inversely associated with the risk of noncommunicable diseases, which are leading causes of death. However, few studies have used longitudinal data to assess the impact of changing lifestyle behaviours on all-cause and cancer mortality. METHODS: Within the European Prospective Investigation into Cancer and Nutrition (EPIC) cohort, lifestyle profiles of 308,497 cancer-free adults (71% female) aged 35-70 years at recruitment across nine countries were assessed with baseline and follow-up questionnaires administered on average of 7 years apart. A healthy lifestyle index (HLI), assessed at two time points, combined information on smoking status, alcohol intake, body mass index, and physical activity, and ranged from 0 to 16 units. A change score was calculated as the difference between HLI at baseline and follow-up. Associations between HLI change and all-cause and cancer mortality were modelled with Cox regression, and the impact of changing HLI on accelerating mortality rate was estimated by rate advancement periods (RAP, in years). RESULTS: After the follow-up questionnaire, participants were followed for an average of 9.9 years, with 21,696 deaths (8407 cancer deaths) documented. Compared to participants whose HLIs remained stable (within one unit), improving HLI by more than one unit was inversely associated with all-cause and cancer mortality (hazard ratio [HR]: 0.84; 95% confidence interval [CI]: 0.81, 0.88; and HR: 0.87; 95% CI: 0.82, 0.92; respectively), while worsening HLI by more than one unit was associated with an increase in mortality (all-cause mortality HR: 1.26; 95% CI: 1.20, 1.33; cancer mortality HR: 1.19; 95% CI: 1.09, 1.29). Participants who worsened HLI by more than one advanced their risk of death by 1.62 (1.44, 1.96) years, while participants who improved HLI by the same amount delayed their risk of death by 1.19 (0.65, 2.32) years, compared to those with stable HLI. CONCLUSIONS: Making healthier lifestyle changes during adulthood was inversely associated with all-cause and cancer mortality and delayed risk of death. Conversely, making unhealthier lifestyle changes was positively associated with mortality and an accelerated risk of death.

Evaluating the Potential of a Novel Metabolic Syndrome Severity Score to Inform Exercise Interventions for People with Complex Chronic Conditions.

Aims: Exercise interventions positively affect numerous cardiometabolic risk factors. To better evaluate the health effects of exercise training, it may be more appropriate to evaluate risk factors together. The Metabolic Syndrome Severity Score (MetSSS) is a composite score representing cardiometabolic risk. Purpose: To evaluate the relationships between physical activity, neuromuscular fitness, exercise capacity, and the MetSSS in a heterogenous sample of people with complex chronic disease. Material and Methods: Fifty-three people with kidney or liver disease and at least one feature of the metabolic syndrome (MetS) were included. Pearson correlations were conducted between physical activity, neuromuscular fitness, exercise capacity, and the MetSSS. Linear regressions were performed for multi-level categorical variables. Independent variables with an association with MetSSS (P ≤ 0.2) were included in a multiple regression analysis. Results: The 6-minute walk test (6MWT) distance was inversely and independently associated with MetSSS [standardized beta coefficient (ß) = -0.31, P = 0.04]. No relationship was found between MetSSS and physical activity or neuromuscular fitness. Mean 6MWT in the highest tertile was 550 m (range: 505-620 m) and 346 m (range: 233-408 m) in the lowest. The analysis showed a medium-large between-group effect for the difference in MetSSS for the lowest and highest tertile of 6MWT [Eta squared (η2) = 0.16, P = 0.01]. Conclusions: Exercise capacity was inversely and independently associated with MetSSS in people with complex chronic disease. Clinical trials with exercise interventions are needed to further investigate if improvements in exercise capacity result in clinically significant changes in the MetSSS.

The relationship between resting metabolic rate and quality of life is moderated by age and body composition in women: a cross-sectional study.

BACKGROUND: Health-related quality of life (HRQOL) is related to body composition, which is also related to resting metabolic rate (RMR). RMR can be increased by exercise and diet interventions that are not dependent on changes in body composition, so a link between RMR and HRQOL may provide interventions that directly improve HRQOL in women. METHODS: One hundred twenty women (median age 63.5 [IQR: 53.0-71.0] years) completed one-time measurement of body composition (multi-frequency bioelectrical impedance), RMR (handheld calorimetry), and HRQOL (RAND-36). Physical (PCS) and mental (MCS) composite scores were calculated for the RAND-36. Pearson correlations were used to identify relationships between RMR, body composition, and HRQOL. Variables at the p < .01 level were entered into multiple regression models. RESULTS: Median body mass index was 26.1 [IQR: 23.2-30.9] kg/m2 and median lean mass index was 16.1 [IQR: 14.6-17.3] kg/m2. Body composition consisted of fat mass (median 27.2 [IQR: 20.3-34.7] kg) and lean mass (median 42.7 [IQR: 38.2-46.9] kg). Median RMR was 1165.0 [IQR: 1022.5-1380.0] kcal/day. Median HRQOL scores were PCS (84.0 [IQR: 74.0-93.0]) and MCS (85.0 [IQR: 74.3-90.0]). RMR was not directly related to PCS, but was directly and negatively related to MCS (p = .002). RMR was significantly and positively related to body composition (lean mass: p < .001; fat mass: p < .001), body mass index (p = .005), and lean mass index (p < .001); but only fat mass (PCS: p < .001; MCS: p < .001) and body mass index (PCS: p < .001; MCS: p < .001) were related to HRQOL, although the relationship was negative. In addition, age was found to be significantly negatively related to RMR (p < .001) and PCS (p = .003). Regression models confirmed the moderating influence of age and body composition on the relationship between RMR and HRQOL. RMR, age, fat mass, and body mass index explained 24% (p < .001) of variance in PCS; and RMR, fat mass, and body mass index explained 15% (p < .001) of variance in MCS. CONCLUSION: In women, the relationship between RMR and HRQOL is moderated by age and body composition. Understanding these pathways will allow clinicians and researchers to direct interventions more effectively.

The influence of preoperative Veterans RAND-12 physical composite score in patients undergoing anterior lumbar interbody fusion [Retrospective Review].

No study has evaluated the preoperative impact of Veterans RAND-12 Physical Composite Score (VR-12 PCS) on anterior lumbar interbody fusion (ALIF) patients. This study examines its influence on physical function, mental health, pain, and disability outcomes. Two cohorts of ALIF patients with preoperative VR-12 PCS scores were formed using a single-surgeon registry: VR-12 PCS < 30 and VR-12 PCS ≥ 30. Demographics, perioperative characteristics, and patient-reported outcome measures (PROMs) were collected. PROMs of VR-12 PCS/Mental Composite Score (MCS), Short Form-12 (SF-12) PCS/MCS, Patient-Reported Outcomes Measurement Information System-Physical Function (PROMIS-PF), Patient Health Questionnaire-9 (PHQ-9), Visual Analog Scale-Back/Leg Pain (VAS-BP/LP), and Oswestry Disability Index (ODI) were collected pre/postoperatively up to 2-years. Demographics, perioperative characteristics, and preoperative PROMs were compared. Intercohort postoperative 6-week/final PROMs and improvements were compared. Of 80 patients, there were 41 in the VR-12 PCS < 30 cohort. Besides VR-12 PCS, VR-12 PCS < 30 patients reported inferior preoperative VR-12 MCS/SF-12 PCS/PROMIS-PF/PHQ-9/ODI scores (p ≤ 0.003, all). At 6-weeks postoperatively, VR-12 PCS < 30 reported inferior VR-12 PCS/SF-12 PCS/PROMIS-PF/PHQ-9 (p ≤ 0.030, all). There was greater improvement up to 6-weeks postoperatively in VR-12 PCS < 30 for VR-12 PCS/MCS and SF-12 PCS (p ≤ 0.020, all). VR-12 PCS < 30 reported superior improvement by final follow-up in VR-12 PCS/SF-12 PCS/PHQ-9 (p ≤ 0.006, all). MCID achievement rates were higher in VR-12 PCS < 30 for PHQ-9 and ODI (p ≤ 0.013, both). VR-12 PCS < 30 patients reported inferior postoperative physical function, mental health, and disability, yet superior magnitude of improvement in physical function and mental health. Rates of clinically meaningful improvement for VR-12 PCS < 30 were greater in mental health and disability.

Associations between metabolic profiles and incident CKD in the Chinese population aged 45-85 years.

PURPOSE: The roles of metabolic indices in predicting chronic kidney disease (CKD) were lacking. This study aimed to examine the concomitant impact of metabolic and novel anthropometric indices on incident CKD in the Chinese populations. METHODS: This prospective cohort study included 1825 males and 2218 females aged between 45 and 85 years, derived from the ongoing prospectively cohort of China Health and Retirement Longitudinal Study (CHARLS), from 2011 to 2015. The outcome was defined as an estimated glomerular filtration rate (eGFR) < 60 mL/min/1.73 m2. RESULTS: During the 5-years follow-up period, 3.0% (55/1825) of males and 4.1% (90/2218) of the females developed CKD. After multivariable adjustment, elevated triglyceride (TG), low high-density lipoprotein cholesterol (HDL-C), serum uric acid (sUA), elevated visceral fat index (VFI), elevated body shape index (BSI) and elevated body roundness index (BRI) in males, and sUA, and BRI in females were the independent predictors for CKD. Composite scores, composed of sUA, history of cardiovascular disease (CVD), waist circumstance (WC), HDL-C, and BRI in males and sUA, hypertension, and BRI in females were constructed that could accurately predict CKD. CONCLUSION: Our study found that elevated levels of TG, sUA, BSI, BRI, and diminished HDL in males and elevated levels of sUA, and BRI in females, are indicative of the incident CKD. The composite score, integrating a history of disease, metabolic indices, and noval anthropometric indices, could accurately differentiate individuals with and without incident CKD, proving useful for CKD care and management.

Effect of Complex Decongestive Therapy on Quality of Life and Physical Parameters in Head and Neck Lymphedema.

OBJECTIVE: To assess the effect of complex decongestive therapy treatment (CDT) on quality of life, neck disability, cervical range of motion, and facial and neck lymphedema size using specific anatomical landmark points. METHODS: This prospective study was conducted in a tertiary cancer center in Turkey. Thirty patients included in the study were treated with CDT for 21 days. The patients were evaluated before and after CDT with MD Anderson Cancer Center Head and Neck Lymphedema (MDACC HNC) staging system, The Neck Disability Index, European Organization for Research and Treatment of Cancer Quality of Life 30 (EORTC-QLOC30), Facial Composite score and Neck Circumferences, cervical range of motion. RESULTS: Median follow-up was 7.2 months. After treatment of 30 patients significant cognitive function, emotional function, and social function (p < 0.001). After CDT treatment, the quality of life sub-parameters of EORTC QLQ-C30 showed significant improvement (p < 0.001). The facial composite score and neck circumferences indices showed significant improvement (p < 0.001). A decrease of 2% or more in the facial composite score and neck circumferences was observed in all patients participating in the study. CONCLUSION: There have been few studies on the effectiveness of CDT on the EORTC QLQ-C30, facial composite score, and neck circumferences in head and neck lymphedema. In patients with head and neck lymphedema following head and neck cancer, our study demonstrated the positive effects of complex decongestive therapy (CDT) on neck disability, range of motion, quality of life, and facial and neck lymphedema tissue size. LEVEL OF EVIDENCE: 4 Laryngoscope, 134:3152-3157, 2024.

ADSS: A Composite Score to Detect Disease Progression in Alzheimer's Disease.

Background: Composite scores have been increasingly used in trials for Alzheimer's disease (AD) to detect disease progression, such as the AD Composite Score (ADCOMS) in the lecanemab trial. Objective: To develop a new composite score to improve the prediction of outcome change. Methods: We proposed to develop a new composite score based on the statistical model in the ADCOMS, by removing duplicated sub-scales and adding the model selection in the partial least squares (PLS) regression. Results: The new AD composite Score with variable Selection (ADSS) includes 7 cognitive sub-scales. ADSS can increase the sensitivity to detect disease progression as compared to the existing total scores, which leads to smaller sample sizes using the ADSS in trial designs. Conclusions: ADSS can be utilized in AD trials to improve the success rate of drug development with a high sensitivity to detect disease progression in early stages.

Assessment of Humoral Immune Responses to Repeated Influenza Vaccination in a Multiyear Cohort: A 5-Year Follow-up.

The long-term effects of host factors on vaccine-elicited immune responses have not been well studied, and the interactions of host factors with annual influenza vaccinations are yet to be explored. We analyzed data from a cohort of 386 individuals who received the standard-dose influenza vaccine and enrolled in ≥2 seasons from 2016 to 2020. Our analyses indicated disparate vaccine-elicited immune responses between males and females in adults when they were repeatedly vaccinated for at least 2 seasons. Notably, we found interactive effects between age and body mass index (BMI) on overall immune responses, and between sex at birth and BMI in adults.

Cognitive training for children with ADHD: composite cognitive score outcome in a randomized controlled trial.

PURPOSE OF THE ARTICLE: Cognitive training for Attention Deficit/Hyperactivity Disorder (ADHD) has shown promising, although mixed results. In post-hoc analyses, we evaluate effects of cognitive training using a novel composite cognition score as the outcome for children attending at least 16 sessions of training, dose-response of training and associations between symptoms and cognitive functioning. MATERIALS AND METHODS: Children (age 6-13) with ADHD were randomized to intervention (n = 26) or control (n = 34). For the current analysis, we restricted the intervention group to children, who completed at least 16 sessions of cognitive training (n = 26) and examined a dose response within that group. RESULTS: Cognition improved significantly in the intervention, but not control group. Amount of the completed training sessions correlated significantly with the amount of cognitive improvement. CONCLUSION: Variations in dose and frequency of training may be an important source of the variance in previous studies.

Clinical Profile and Quality of Life in Myasthenia Gravis Using MGQOL15 R(Hindi): An Indian Perspective.

Background: Myasthenia Gravis (MG) is a chronic fluctuating illness, due to the dysfunction of neuromuscular junction which is autoimmune in nature. The disease severely affects the Quality Of Life (QOL). Objective: The primary objective of our study was to assess the QOL in patients with MG using Short Form 36 (SF 36) and MGQOL 15 R (Hindi translated). The secondary objective was to assess the correlation of age, sex, illness duration, clinical characteristics, severity, and treatment with the QOL in MG patients. Methodology: A cross sectional study of 55 MG patients was done to analyse and evaluate the clinical status using Hybrid Myasthenia Gravis Foundation of America (HMGFA), Myasthenia gravis composite score (MGCS) and The Myasthenia Gravis Activities of Daily Living (MG - ADL). QOL was assessed by SF 36 and Hindi version of Myasthenia Gravis Quality of Life 15 - Revised (MG-QOL15R) score. Results: 78.2% patients had generalized MG. The mean MGC and MG-ADL scores were 5.27 and 3.29 (95% CI: 2.24 -4.34) respectively. The mean MGQOL15R score was 6.52 ± 7.7 and the score correlated with the symptoms. The SF 36 scores were the best and the worst in the bodily pain (93.72 ± 13.52) and general health subset (61.81 ± 39.64) respectively. Except for steroid dose, there was no significant correlation between SF36 and other factors. Conclusion: QOL in MG was found to be affected due to the disease. The MGQOL 15 R scores correlated with the clinical features, remission or active status, steroid use and thymectomy. No Significant association was observed between MG QOL scores and various lab parameters and repetitive nerve stimulation (RNS) test results. Higher dose of steroid was associated with poor QOL, while thymectomy was associated with better QOL scores. MGQOL15R (Hindi) is a quick and simple tool to assess the QOL in MG patients.

Cost-effectiveness of the Perioperative Pain Management Bundle a registry-based study.

Introduction: The Perioperative Pain Management Bundle was introduced in 10 Serbian PAIN OUT network hospitals to improve the quality of postoperative pain management. The Bundle consists of 4 elements: informing patients about postoperative pain treatment options; administering a full daily dose of 1-2 non-opioid analgesics; administering regional blocks and/or surgical wound infiltration; and assessing pain after surgery. In this study, we aimed to assess the cost-effectiveness of the Bundle during the initial 24 h after surgery. Materials and methods: The assessment of cost-effectiveness was carried out by comparing patients before and after Bundle implementation and by comparing patients who received all Bundle elements to those with no Bundle element. Costs of postoperative pain management included costs of the analgesic medications, costs of labor for administering these medications, and related disposable materials. A multidimensional Pain Composite Score (PCS), the effectiveness measurement, was obtained by averaging variables from the International Pain Outcomes questionnaire evaluating pain intensity, interference of pain with activities and emotions, and side effects of analgesic medications. The incremental cost-effectiveness ratio (ICER) was calculated as the incremental change in costs divided by the incremental change in PCS and plotted on the cost-effectiveness plane along with the economic preference analysis. Results: The ICER value calculated when comparing patients before and after Bundle implementation was 181.89 RSD (1.55 EUR) with plotted ICERs located in the northeast and southeast quadrants of the cost-effectiveness plane. However, when comparing patients with no Bundle elements and those with all four Bundle elements, the calculated ICER was -800.63 RSD (-6.82 EUR) with plotted ICERs located in the southeast quadrant of the cost-effectiveness plane. ICER values differ across surgical disciplines. Conclusion: The proposed perioperative pain management Bundle is cost-effective. The cost-effectiveness varies depending on the number of implemented Bundle elements and fluctuates across surgical disciplines.

Should we Oppose or Combine Waveforms for Spinal Cord Stimulation in PSPS-T2 Patients? A Prospective Randomized Crossover Trial (MULTIWAVE Study).

Refractory persistent spinal pain syndrome after surgery (PSPS-T2) can be successfully addressed by spinal cord stimulation (SCS). While conventional stimulation generates paresthesia, recent systems enable the delivery of paresthesia-free stimulation. Studies have claimed non-inferiority/superiority of selected paresthesia-free stimulation compared with paresthesia-based stimulation, but the comparative efficacy between different waveforms still needs to be determined in a given patient. We designed a randomized controlled 3-month crossover trial to compare pain relief of paresthesia-based stimulation versus high frequency versus burst in 28 PSPS-T2 patients implanted with multiwave SCS systems. Our secondary objectives were to determine the efficacy of these 3 waveforms on pain surface, quality of life, functional capacity, psychological distress, and validated composite multidimensional clinical response index to provide holistic comparisons at 3-, 6-, 9-, and 15-month post-randomization. The preferred stimulation modality was documented during the follow-up periods. No difference between the waveforms was observed in this study (P = .08). SCS led to significant pain relief, quality of life improvement, improvement of multidimensional clinical response index, and of all other clinical outcomes at all follow-up visits. Forty-four percent of the patients chose to keep the paresthesia-based stimulation modality after the 15-month follow-up period. By giving the possibility to switch and/or to combine several waveforms, the overall rate of SCS responders further increased with 25%. In this study, high frequency or burst do not appear superior to paresthesia-based stimulation, wherefore paresthesia-based stimulation should still be considered as a valid option. However, combining paresthesia-based stimulation with paresthesia-free stimulation, through personalized multiwave therapy, might significantly improve SCS responses. PERSPECTIVE: This article assesses clinical SCS efficacy on pain relief, by comparing paresthesia-based stimulation and paresthesia-free stimulation (including high frequency and burst) modalities in patient presenting with PSPS-T2. Switching and/or combining waveforms contribute to increasing the global SCS responders rate.

Longitudinal assessment of neurocognitive function in people with relapsing multiple sclerosis initiating alemtuzumab in routine clinical practice: LEM-COG study results.

BACKGROUND: Alemtuzumab is effective in reducing relapse rate and disability, but limited data exist on its effect on cognitive function in relapsing multiple sclerosis (RMS). The present study assessed neurocognitive function and safety associated with alemtuzumab treatment in RMS. METHODS: This longitudinal, single-arm, prospective study included people with RMS (aged 25-55 years) who were treated with alemtuzumab in clinical practice in the United States of America and Canada. The first participant was enrolled in December 2016. The primary endpoint was the change from baseline to post-baseline (month [M] 12/24) in MS-COGnitive (MS-COG) composite score. Secondary endpoints included Paced Auditory Serial Addition Test (PASAT), Symbol Digit Modalities Test (SDMT), Brief Visuospatial Memory Test-Revised (BVMT-R), Selective Reminding Test (SRT), Controlled Oral Word Association Test (COWAT), and Automated Neuropsychological Assessment Metrics (ANAM) scores. Depression and fatigue were assessed using Hamilton Rating Scale-Depression (HAM-D) and Fatigue Severity Scale (FSS)/Modified Fatigue Impact Scale (MFIS), respectively. Magnetic resonance imaging (MRI) parameters were assessed when available. Safety was assessed throughout the study. Descriptive statistics were used for the pre-specified statistical analyses. Since the study was terminated early (November 2019) because of operational and resource difficulties, post hoc analyses for statistical inference were performed among participants who had a baseline value and at least one complete post-baseline assessment for cognitive parameters, fatigue, or depression. RESULTS: Of the 112 participants enrolled, 39 were considered as the primary analysis population at M12. At M12, a mean change of 0.25 (95% confidence interval [CI]: 0.04, 0.45; p = 0.0049; effect size [ES]: 0.39) was observed in the MS-COG composite score. Improvements were observed in processing speed (based on PASAT and SDMT; p < 0.0001; ES: 0.62), as well as in individual PASAT, SDMT and COWAT scores. An improvement was also noted in HAM-D (p = 0.0054; ES: -0.44), but not in fatigue scores. Among MRI parameters, decreases in burden of disease volume (BDV; ES: -0.12), new gadolinium-enhancing lesions (ES: -0.41) and newly active lesions (ES: -0.07) were observed at M12. About 92% of participants showed stable or improved cognitive status at M12. There were no new safety signals reported in the study. The most common adverse events (≥10% of participants) were headache, fatigue, nausea, insomnia, urinary tract infection, pain in extremity, chest discomfort, anxiety, dizziness, arthralgia, flushing, and rash. Hypothyroidism (3.7%) was the most frequent adverse event of special interest. CONCLUSION: The findings from this study suggest that alemtuzumab has a positive impact on cognitive function with significant improvements in processing speed and depression in people with RMS over a period of 12 months. The safety profile of alemtuzumab was consistent with previous studies.

Comparative evaluation of weighted mean and composite score for combining assessments.

OBJECTIVES: Approaches for combining scores have been based on weighted mean (WM) without consideration for psychometric characteristics of each individual assessments. This study evaluates consequences of WM and composite score (CS) approach. METHODS: Data from two longitudinal cohorts (n = 219) were utilized for performance in three Operative Dentistry courses as basis to compare the two score-combining methods. Four assessments (two written and two practical exams) from each course were combined using WM and CS approaches. WM scores were calculated by multiplying the score by its weight and summing across assessments. The CS approach follow a modification of the Kane and Case method, by standardizing scores, taking into account the reliability and associations between each assessment score. t-Tests and Pearson's correlation were used to evaluate the consequences of the WM and CS approaches. In addition, changes in each student's rank across WM and CS were determined. RESULTS: Combining scores using CS method produced lower scores and higher percentage failure in all courses compared to WM. Students ranks were changed significantly when CS was used with only 15% of the cohorts retained their ranks. CONCLUSIONS: CS produced a composite that is correlated with WM but still being substantively different providing meaningful and psychometrically rigorous information.

Design of paediatric trials with benefit-risk endpoints using a composite score of adverse events of interest (AEI) and win-statistics.

A fundamental problem in the regulatory evaluation of a therapy is assessing whether the benefit outweighs the associated risks. This work proposes designing a trial that assesses a composite endpoint consisting of benefit and risk, hence, making the core of the design of the study, to assess benefit and risk. The proposed benefit risk measure consists of efficacy measure(s) and a risk measure that is based on a composite score obtained from pre-defined adverse events of interest (AEI). This composite score incorporates full aspects of adverse events of interest (i.e. the incidence, severity, and duration of the events). We call this newly proposed score the AEI composite score. After specifying the priorities between the components of the composite endpoint, a win-statistic (i.e. win ratio, win odds, or net benefit) is used to assess the difference between treatments in this composite endpoint. The power and sample size requirements of such a trial design are explored via simulation. Finally, using Dupixent published adult study results, we show how we can design a paediatric trial where the primary outcome is a composite of prioritized outcomes consisting of efficacy endpoints and the AEI composite score endpoint. The resulting trial design can potentially substantially reduce sample size compared to a trial designed to assess the co-primary efficacy endpoints, therefore it may address the challenge of slow enrollment and patient availability for paediatric studies.

Preoperative Veteran RAND-12 Mental Composite Score of >60 Associated With Increased Likelihood of Patient Satisfaction After Total Hip Arthroplasty.

BACKGROUND: The present study aimed to determine the distribution of Veterans RAND 12-Item health survey (VR-12) mental component scores (MCS) of patients undergoing primary total hip arthroplasty (THA) and the thresholds of VR-12 MCS scores that predict higher health care utilizations and 1-year patient-reported outcome measures (PROMs). METHODS: A prospective cohort of 4,194 primary THA patients (January 2016 to December 2019) were included. Multivariable and cubic spline regression models were used to test for associations between preoperative VR-12 MCS and postoperative outcomes, including: 90-day hospital resource utilization (nonhome discharge, prolonged length of stay [LOS](ie, ≥3 days), all-cause readmission), attainment of patient acceptable symptom state (PASS) at 1-year postoperative and substantial clinical benefit (SCB) in the hip disability osteoarthritis outcome score (HOOS)-pain and HOOS-physical short form. RESULTS: Lower VR-12 MCS was associated with older age, obesity, Black race, women, and smokers (all P < .001). Preoperative VR-12 MCS<20 was associated with more than twice the odds of nonhome discharge (odds ratio [OR]:2.31) and prolonged LOS (OR: 3.46). VR-12 MCS >60 was associated with higher odds of achieving PASS (OR: 2.00) and SCB in HOOS-joint related (JR) (OR: 1.16). Starting VR-12 MCS ≤40, there were exponentially higher odds of worse outcomes. CONCLUSION: Low preoperative VR-12 MCS, specifically less than 40, may predict increased health care utilization. Furthermore, preoperative VR-12 MCS>60 predicts greater satisfaction at 1 year and higher odds of achieving SCB in HOOS-JR. Quantifiable thresholds for VR-12 MCS may aid in shared decision-making and patient counseling in setting expectations or may guide specific care pathway interventions to address mental health during THA. LEVEL OF EVIDENCE: II.

Altered Developmental Trajectory in Male and Female Rats in a Prenatal Valproic Acid Exposure Model of Autism Spectrum Disorder.

Early motor and sensory developmental delays precede Autism Spectrum Disorder (ASD) diagnosis and may serve as early indicators of ASD. The literature on sensorimotor development in animal models is sparse, male centered, and has mixed findings. We characterized early development in a prenatal valproic acid (VPA) model of ASD and found sex-specific developmental delays in VPA rats. We created a developmental composite score combining 15 test readouts, yielding a reliable gestalt measure spanning physical, sensory, and motor development, that effectively discriminated between VPA and control groups. Considering the heterogeneity in ASD phenotype, the developmental composite offers a robust metric that can enable comparison across different animal models of ASD and can serve as an outcome measure for early intervention studies.