Criterion validity of equations as alternatives to reference standards for assessing body composition and energy expenditure in amyotrophic lateral sclerosis - A systematic literature review.

Background and objectives: Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative disease characterized by the relentless loss of upper motor neurones (UMN) and lower motor neurones (LMN), leading to progressive weakness. The cause of ALS is unknown and there are currently no curative treatments. The median survival is 18 months from diagnosis, however, the rate of disease progression and survival vary significantly among individual ALS patients. In the absence of a cure for ALS, discovery of factors that modify disease progression is critical. An understanding of processes that influence the course of ALS could shed light on disease mechanisms and identify therapeutic targets that could be manipulated in order to prolong survival. Systemic energy metabolism is emerging as an important modifying factor in ALS. Of clinical significance, adiposity [in the form of fat mass (FM)] and nutritional status modify survival in ALS. Therefore, the assessment of FM and implementation of nutritional interventions to maintain FM are important components of routine care in ALS patients. Anthropometric measurements, including body mass index (BMI) and body adiposity index (BAI) are commonly used to predict adiposity in order to guide nutritional management. However, the accuracy of these measurements in ALS patients is currently unknown. Hypermetabolism [an increase in measured resting energy expenditure (REE) compared to a predicted REE] has been reported in some ALS patients and is also a possible modifier of disease progression. If this is a causal relationship, then strategies to correct or compensate for increased energy expenditure could possibly modify disease course. However, further studies to clarify the incidence, clinical correlations and consequences of hypermetabolism must be established. The objectives of this thesis were to determine whether commonly used anthropometric measurements are accurate predictors of FM in ALS patients and to study the incidence and clinical correlations of hypermetabolism in ALS patients. Methodology and main findings: Anthropometric measurements of BMI and BAI were compared to percentage FM derived from air displacement plethysmography (ADP) in 44 ALS patients and 35 age- and sex- matched healthy controls. Using Bland-Altman analyses it was found that both anthropometric measurements were less accurate predictors of FM in ALS patients than in controls and that BMI and BAI provided a poor estimate of FM in ALS patients. In a longitudinal assessment of 29 ALS patients, neither BMI nor BAI consistently reflected the change in FM. These results indicate that an isolated measure of BMI and BAI is not an accurate indicator of adiposity in ALS and that longitudinal measurements could be misleading. REE was measured via indirect calorimetry (mREE) and compared to a predicted REE (pREE, derived from a model that accounts for body composition) in 50 ALS patients and 50 age- and sex- matched healthy controls. Hypermetabolism was defined as a metabolic index (mREE/pREE x100) ≥ 120. Individuals with a metabolic index <120 were considered to be normometabolic. Hypermetabolism was found in 16% of controls and 40% of ALS patients. Hypermetabolic ALS patients had a higher LMN disease burden (assessed by clinical examination) and a greater short-term functional decline (assessed by the revised ALS functional rating scale, ALSFRS-R) than normometabolic patients. Conclusions and future directions: This study found that in ALS patients, BMI and BAI are not accurate predictors of FM and that they provide a poor indicator of change in FM over time. It is therefore likely that changes in BMI and BAI in ALS patients occur independent to changes in FM alone and could depend on muscle atrophy and re-distribution of fat. It was also found that when body composition is accounted for, the incidence of hypermetabolism is greater in ALS patients than in healthy matched controls. An association between hypermetabolism and LMN disease burden was observed in ALS patients. As LMN disease burden reflects dysfunction of motor units, it is hypothesized that hypermetabolism in ALS could arise from abnormal motor units which include dysfunctional LMNs, disrupted neuromuscular junctions and denervated muscle. Furthermore, hypermetabolism was associated with a greater functional decline in ALS patients who were studied over time. In the light of these findings it is hypothesized that hypermetabolism could drive progression of ALS and lead to a vicious cycle of denervation, hypermetabolism and further disease progression. Overall, the results of this thesis suggest that BMI and BAI are inadequate markers of nutritional status in ALS and that hypermetabolism is an important metabolic consideration in ALS patients. More accurate ALS-specific predictors of FM are required to guide nutritional therapies and further clinical and physiological studies are needed to understand the cause and prognostic implications of changes in body composition and hypermetabolism.

No effect of the Trp64Arg [beta ] 3-adrenoceptor gene variant on weight loss, body composition, or energy expenditure in obese, caucasian postmenopausal women

Objective It is unknown whether the relative contribution to energy imbalance in amyotrophic lateral sclerosis (ALS) is due to decreased energy intake, or increased energy expenditure from hyper-metabolism and/or physical activity, or both. Methods We studied 10 free-living sporadic ALS subjects with mild to moderate disease and 10 matched healthy controls to address this question. We estimated energy intake by 24-h recall in ALS subjects and three-day food diary in all. We estimated body composition by dual energy X-ray absorptiometry and resting metabolic rate by indirect calorimetry; and measured total daily energy expenditure (TEE) and physical activity-energy expenditure using doubly labeled water. Results Daily energy intake was no different between ALS subjects and controls. Despite lower fat-free mass, unadjusted TEE was higher in ALS subjects than controls (2844 ± 319 vs. 2505 ± 261 kcal/d, p = 0.005 by paired t-test). Compared to controls, hyper-metabolism occurred in 80% of ALS subjects. Physical activity-energy expenditure was higher in ALS subjects than controls (718 ± 262 kcal/d vs. 487 ± 196 kcal/d, p = 0.04). In controls, energy intake matched TEE; in ALS subjects TEE was higher than energy intake. Conclusions We found higher TEE in ALS subjects than controls, with larger contribution to difference from physical activity-energy expenditure than hyper-metabolism. Although daily energy intake in ALS subjects was similar to that in controls, they were unable to compensate for increased energy needs. To accurately determine energy balance and optimize nutrition in ALS, future studies should consider measuring energy intake, energy expenditure, and physical activity.

Measuring body composition and energy expenditure in children with severe neurologic impairment and intellectual disability

Estimating daily energy expenditure in individuals with amyotrophic lateral sclerosis

This study aimed to investigate the influence of body fatness on the associations of body composition and energy expenditure (EE) with energy intake (EI). Data from 93 women (BMI = 25.5 [SD 4.2] kg/m2 ) recruited for two studies (Study 1, n = 48, BMI = 25.0-34.9 kg/m2 ; Study 2, n = 45, BMI = 18.5-24.9 kg/m2 ) were examined. Body composition, resting metabolic rate (RMR), and test meal EI were assessed during a laboratory probe day. Physical activity, total daily EE (TDEE), and self-reported free-living 24-hour EI were collected during 7 days. In the whole sample, fat-free mass (r = 0.45; P < 0.001), RMR (r = 0.41; P < 0.001), and TDEE (r = 0.39; P < 0.001), but not fat mass (r = 0.17; P = 0.11), were positively associated with free-living 24-hour EI. Body fat percentage moderated the associations of RMR (β = -1.88; P = 0.02) and TDEE (β = -1.91; P = 0.03) with mean free-living 24-hour EI. Fat mass was negatively associated with test meal EI only in the leaner group (r = -0.43; P = 0.004), and a weak nonlinear association was observed in the whole sample (r2 = 0.092; P = 0.04). Body fat percentage appears to moderate the associations between EE and daily EI. Furthermore, the negative association between fat mass and test meal EI observed in leaner individuals was absent in those with higher body fatness. Therefore, higher levels of body fatness may weaken the coupling between EE and EI.

The Positive and Negative Syndrome Scale (PANSS-30) is the standard instrument for assessing symptoms of schizophrenia and related psychotic disorders. However, its long administration time and structural issues have prompted the development of shorter versions. The PANSS-6, derived through Item Response Theory and Rasch analysis of the PANSS-8, emerged as a potential alternative. Comprising three positive and three negative symptom items, the PANSS-6 offers a more feasible assessment tool. However, its measurement properties have never been systematically reviewed. We applied the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline for systematic reviews and meta-analytical procedures to assess the psychometric properties of the PANSS-6. COSMIN comprises several steps: literature search, risk-of-bias assessments, assessing the updated criteria for good measurement properties, grading the quality of the evidence and feasibility aspects. We included 13 publications. The PANSS-6 showed sufficient content validity, structural validity, measurement invariance, reliability, criterion validity, construct validity and responsiveness according to COSMIN. On some of them only a small body of evidence is currently available. For internal consistency, cross-cultural validity and measurement error there was not enough evidence for a definite rating. Its short administration time of only 15-20 mins renders feasibility good. The PANSS-6 does not cover all schizophrenia symptoms but focuses on the core symptoms in favor of a feasible administration time. The evidence available on its measurement properties yields sufficient results for its purpose - the assessment of symptom severity and its change. According to COSMIN it can potentially be recommended for use.

The purpose of this study was to compare the relationship of several muscle strength and cardiorespiratory fitness indices with body composition and energy expenditure in obese postmenopausal women. This was a cross-sectional study involving 72 obese postmenopausal women (age: 60.0±4.8 years; body mass index: 34.1±3.5 kg/m²). Muscle strength was determined by hand dynamometer and cardiorespiratory fitness was measured by indirect calorimetry. Muscle strength and cardiorespiratory fitness were expressed in absolute (kg and L/min, respectively) and in relative values (kg/body weight (BW) and kg/lean body mass (LBM) for muscle strength and ml/min/kg BW and ml/min kg LBM for cardiorespiratory fitness). Body composition was measured using dual energy x-ray absorptiometry. Anthropometric (waist and thigh circumference), physical activity energy expenditure and daily number of steps (SenseWear armband) as well as blood pressure were also assessed. Correlations of muscle strength and cardiorespiratory fitness indices with body composition and energy expenditure showed several similarities, however, several variations were also observed. Furthermore, our results showed that age and waist circumference were the primary independent predictors for the muscle strength indices, explaining 22-37% of the variance and % body fat and age were the primary predictors for the cardiorespiratory fitness indices, explaining 18-40% of the variance. In conclusion, the present study indicates that the different methods of expressing muscle strength and cardiorespiratory fitness may display several variations and similarities with body composition and energy expenditure associations. Therefore, interpretations of relationships between muscle strength and cardiorespiratory indices with body composition and energy expenditure factors should take in account the method used to express them.

PurposeWe aimed to list all tests used to assess cognitive change in patients with amyotrophic lateral sclerosis (ALS) and to provide a descriptive synthesis of the psychometric properties of tests that were evaluated in a population of ALS patients.Materials and MethodsThe protocol is registered in PROSPERO (ID: CRD42017055603). We systematically search for literature in 11 databases. Full-text articles, in any language, with original research were included. All included articles were scrutinised by two independent authors. Disagreement was resolved by consensus. The framework of Lezak informed conceptualises of the tests identified. To evaluate methodological quality, we used the Consensus-based Standards for the Selection of Health Measurement Instruments (COSMIN). Data were synthesised using criteria proposed by the Cochrane Back Review Group.ResultsOf 319 included articles, 46 articles reported information on the psychometric properties of cognitive tests used in patients with ALS. We found that the highest level of evidence was supported for the Reading the Mind in the Eye Test (RME), Addenbrooke’s Cognitive Evaluation (ACE) and Frontal Assessment Battery (FAB). Moderate level of evidence was found for the screening tests; Edinburgh Cognitive and Behavioural ALS Screen (ECAS) and the Montreal Cognitive Assessment (MoCA).ConclusionThe screening test, ECAS and the social cognition test, RME, may have some advantages over other tests that have been used for assessing cognitive change in ALS patients. Recommendations of ALS-specific tests with sound psychometric properties are urgently needed.

Amyotrophic lateral sclerosis (ALS) is the most common motor neuron disease in adults. There are many patient-reported outcome measures (PROMs) for measuring quality of life (QoL) and health-related QoL (HRQoL) within this population; however, there is limited consensus regardingwhich are most valid, reliable, responsive, and interpretable. This systematic review assesses the psychometric properties and interpretability of QoL and HRQoL PROMs for individuals with ALS. This review was conducted following the Consensus-based Standards for the selection of health Measurement Instruments(COSMIN) methodology for systematic reviews of PROMs. MEDLINE, EMBASE, and CINAHL databases were searched. Studies were included if their aim was to evaluate one or more psychometric properties or the interpretability of QoL or HRQoL PROMs in individuals with ALS. We screened 2713 abstracts, reviewed 60 full-text articles, and included 37 articles. Fifteen PROMs were evaluated including generic HRQoL (e.g., SF-36), ALS-specific HRQoL (e.g., ALSAQ-40), and individualized QoL (e.g., SEIQoL) measures. Evidence for internal consistency and test-retest reliability were acceptable. For convergent validity, 84% of hypotheses were met. For known-groups validity, outcomes were able to distinguish between healthy cohorts and other conditions. Responsiveness results ranged from low to high correlations with other measures over 3-24months. Evidence for content validity, structural validity, measurement error, and divergent validity was limited. This review identified evidence in support of the ALSAQ-40 or ALSAQ-5 for individuals with ALS. These findings canguide healthcare practitioners when selecting evidence-based QoL and HRQoL PROMs for patients and provide researchers withinsight into gaps in the literature.

Bulbar impairment represents a hallmark feature of Amyotrophic Lateral Sclerosis (ALS) that significantly impacts survival and quality of life. Speech and swallowing dysfunction are key contributors to the clinical heterogeneity of ALS and require well-timed and carefully coordinated interventions. The accurate clinical, radiological and electrophysiological assessment of bulbar dysfunction in ALS is one of the most multidisciplinary aspects of ALS care, requiring expert input from speech-language pathologists (SLPs), neurologists, otolaryngologists, augmentative alternative communication (AAC) specialists, dieticians, and electrophysiologists—each with their own evaluation strategies and assessment tools. The need to systematically evaluate the comparative advantages and drawbacks of various bulbar assessment instruments and to develop integrated assessment protocols is increasingly recognized. In this review, we provide a comprehensive appraisal of the most commonly utilized clinical tools for assessing and monitoring bulbar dysfunction in ALS based on the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) evaluation framework. Despite a plethora of assessment tools, considerable geographical differences exist in bulbar assessment practices and individual instruments exhibit considerable limitations. The gaps identified in the literature offer unique opportunities for the optimization of existing and development of new tools both for clinical and research applications. The multicenter validation and standardization of these instruments will be essential for guideline development and best practice recommendations.

For individuals who face barriers to care assessment, there is a need for remote administration or self-administration of physical performance measures that assess mobility to determine current functional status and to monitor and predict future changes in functional status. The primary purpose of this review is to evaluate the available measurement properties of scores for remotely or self-administered lower extremity mobility performance measures in adults. This review also outlines the test procedures and population suitability of these measures. Data sources were Ovid MEDLINE, Ovid Embase, EBSCOhost CINAHL, Ovid AMED, and Cochrane CENTRAL-which were searched from inception to January 26, 2021-and the reference lists of relevant studies. Two individuals independently screened studies that assessed at least 1 prespecified measurement property of scores for a remote and/or self-administered lower extremity physical performance measure assessing mobility in an adult population. Two individuals independently extracted data on study characteristics, measurement properties, feasibility, and interpretability using piloted extraction forms. The COSMIN (COnsensus-based Standards for the selection of health Measurement INstruments) Risk of Bias tool was used to assess methodological quality. Data were qualitatively summarized, and results were compared against COSMIN's criteria for good measurement properties. Level of evidence was determined using COSMIN's modified GRADE approach. Fourteen studies detailing 19 outcome measures were included. Many studies displayed "sufficient" measurement properties based on COSMIN's criteria; however, risk of bias for most of the included studies was rated adequate or doubtful. Clinicians and researchers can consider the measurement properties of scores and feasibility of different approaches presented in this review when determining how to assess or monitor mobility in adult populations. Assessing mobility via remote or self-administered physical performance measures in adult populations appears to be feasible using a variety of methods including simple tools (chair, stopwatch), videoconferencing, and smartphone applications. This strategy may be particularly valuable for self-management of chronic conditions and decreasing barriers to accessing care.

The Positive and Negative Syndrome Scale (PANSS) is widely used to assess schizophrenia symptoms. Initially designed with three subscales, Marder et al.´s 5-factor-Model (M5M) first proposed in 1997 has been frequently used in treatment trials, but it has never been systematically reviewed for its measurement properties. We utilized the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guideline for systematic reviews and meta-analytical procedures to assess the psychometric properties of the M5M-PANSS. COSMIN comprises several steps: literature search, risk-of-bias assessments, assessing the updated criteria for good measurement properties, feasibility aspects and grading the quality of the evidence. We further assessed the goodness of fit of other PANSS factor models. We included 95 publications. The M5M-PANSS showed good construct validity, but “insufficient” structural validity. Evidence of other COSMIN domains is largely lacking. Among the multiple (73) factor solutions examined with confirmatory methods, several other 5-factor solutions had better model fit. According to COSMIN rules the M5M should not be recommended for use. Other five-factor models such as the one proposed by Wallwork et al.1 warrant further evaluation. Nevertheless, the factor composition of the M5M and these other models was relatively similar, so previously published results should not be disregarded.

Characteristics of chronic obstructive pulmonary disease (COPD) can include shortness of breath, chronic cough, sputum production and reduced exercise capacity. The sit-to-stand (STS) test variations (e.g., 5-repetition STS, 30-second STS) may be appropriate outcome measures to assess exercise capacity in people with COPD. To date, the measurement properties of the various STS tests in people with COPD have not been synthesized in a systematic review since the publication of the COnsensus-based Standards for the selection of health Measurement Instruments (COSMIN) guidelines. The purpose of this proposed systematic review is to synthesize the literature of the measurement properties of the STS test variations among people with COPD. The review will be conducted with methods consistent with the COSMIN guidelines. Peer-reviewed publications will be included if they assessed the measurement properties (reliability, validity, responsiveness) of a STS test in community-dwelling adults with COPD. We will search six databases from inception. Study selection and data extraction will be conducted independently and in duplicate. We will assess the risk of bias using the COSMIN Risk of Bias tool, assess results against the COSMIN updated criteria for good measurement properties, and summarize certainty of evidence using the modified Grading of Recommendations Assessment, Development and Evaluation (GRADE) tool. Study results will be pooled by calculating weighted means and 95% confidence intervals or summarized narratively, as appropriate. This will be the first systematic review to synthesize the measurement properties of the STS tests in people with COPD using the methods recommended by COSMIN. Consequently, its results will be robust and may help clinicians or researchers identify the best variation of the STS test to use in their practice.

The 41-item Quality of Life Questionnaire of the European Foundation for Osteoporosis (QUALEFFO-41) is a widely used and freely available patient-reported outcome measure (PROM). However, data on its reliability, validity, and responsiveness remain unclear. Therefore, this study aimed to systematically review the measurement properties of the QUALEFFO-41. A systematic search of MEDLINE, EBSCOhost, and Cochrane Library from their inception up to December 2022 was performed. Data were extracted, and the methodological quality of each measurement property was evaluated according to the COnsensus-based Standards for the selection of health Measurement INstruments (COSMIN) guidelines. The evidence of the measurement properties was rated against the updated criteria for good measurement properties, and the quality of evidence was graded using the modified GRADE approach. A total of 99 articles were identified, of which eight studies were included in the review. The QUALEFFO-41 is categorized as B as it demonstrated moderate quality evidence for sufficient content validity, moderate-to-high quality evidence for sufficient hypothesis testing for construct validity (except for the social function domain for convergent validity), and very low-quality evidence for sufficient responsiveness. For structural validity and internal consistency, only the domains of pain and general health perception were sufficient with low-quality evidence. For reliability, only the domain of physical function was sufficient with low-quality evidence. None of the studies reported measurement error, cross-cultural validity, and criterion validity. The QUALEFFO-41 may be a promising, valid, and reliable PROM to assess HRQoL in osteoporosis patients with vertebral fractures. However, future studies must focus on good methodological quality to strengthen the evidence of measurement properties, especially on structural validity, reliability, responsiveness, and cross-cultural validity. The systematic review evaluated the measurement properties of the QUALEFFO-41 questionnaire for assessing Health-Related Quality of Life (HRQoL) in osteoporosis patients. The review found moderate-to-high-quality evidence for construct validity but limited evidence for responsiveness and other properties. Future studies should focus on strengthening the evidence, particularly for structural validity, reliability, responsiveness, and cross-cultural validity. The QUALEFFO-41 shows promise as a valid and reliable PROM for HRQoL assessment in osteoporosis patients.