A propensity score analysis for comparison of T-3b and VATET in myasthenia gravis

OBJECTIVE: We performed propensity score (PS) models to compare the outcome of patients with myasthenia gravis (MG) submitted to 2 different surgical approaches: extended transsternal (T-3b) or thoracoscopic extended thymectomy (VATET). METHODS: Patients' clinical data were retrieved from the MG database of the C. Besta Neurologic Institute Foundation. In the PS analysis, a matching ratio of 1:1 of the main clinical variables was obtained for the 2 groups of patients and treatment effect was estimated by comparing their outcome. RESULTS: A total of 210 patients met the inclusion criteria, by having a complete set of clinical data, and were included in the PS model; a matched dataset of 122 participants (61 per group) showed an adequate balance of all the covariates. Our analysis demonstrated that 68.9% of patients who had thymectomy by the VATET technique reached the pharmacologic remission/remission status at 2 years from thymectomy compared to 34.4% of those operated on by the T-3b technique (p < 0.001), had a lower INCB-MG score (p < 0.001), and had less muscle fatigability (p = 0.004). Similar results were found considering only nonthymomatous patients with MG. Results were also confirmed by paired statistical tests. CONCLUSIONS: Our PS matching analysis showed that VATET is a reliable and effective surgical approach alternative to T-3b in patients with MG who are candidates for thymectomy. CLASSIFICATION OF EVIDENCE: This study provides Class IV evidence that for patients with MG, VATET is more effective than T-3b thymectomy

Validation of the besta neurological institute rating scale for myasthenia gravis

INTRODUCTION: We validated the scale for myasthenia gravis (MG) developed at the Neurological Institute Foundation of Milan (INCB-MG scale). METHODS: A total of 174 patients were evaluated with the INCB-MG and compared with the MG Composite (MGC) as the gold standard. Dimensionality, reliability, and validity of the INCB-MG scale were studied by principal component factor analysis, Cronbach alpha, and Pearson correlation coefficients; interobserver reliability was calculated by the weighted Cohen K coefficient. RESULTS: Generalized and bulbar INCB-MG subscales were unidimensional with excellent consistency; the INCB-MG and MGC scales were strongly correlated. Fatigability assessment was correlated with the INCB-MG generalized, bulbar, and respiratory subscales. CONCLUSIONS: The INCB-MG scale is a reliable tool to assess MG and is strongly correlated with the MGC. The INCB-MG scale is a valid tool for every day practice and should be further investigated for its application in clinical trials

Amifampridine phosphate in the treatment of muscle-specific kinase myasthenia gravis: a phase IIb, randomized, double-blind, placebo-controlled, double crossover study

Objective: The aim of this study is to determine the safety and the efficacy of amifampridine phosphate in muscle-specific kinase antibody-positive myasthenia gravis, in a 1:1 randomized, double-blind, placebo-controlled, switchback, double crossover study. Methods: Eligible patients had muscle-specific kinase myasthenia gravis, >18 years of age, and Myasthenia Gravis Foundation of America class II–IV with a score of ⩾9 on Myasthenia Gravis Composite scale. After the run-in phase, during which amifampridine phosphate was titrated to a tolerable and effective dosage, patients were randomized to receive placebo–amifampridine–placebo sequence or amifampridine–placebo–amifampridine sequence daily for 7 days. Then, patients switched treatment arms twice, for a total of 21 days of double-blind treatment. Safety was determined by serial assessments of adverse events/serious adverse events, physical examinations, and clinical and laboratory tests. The co-primary outcome measures included changes from baseline of Quantitative Myasthenia Gravis score and Myasthenia Gravis–specific Activities of Daily Living Profile score. The secondary outcome measures comprised changes from baseline of Myasthenia Gravis Composite score, Myasthenia Gravis Quality of Life scale—15 questions, Fatigue Severity Scale, and Carlo Besta Neurological Institute–Myasthenia Gravis scale. Statistical analyses were assessed using a switchback model for three-period, two-treatment crossover design. Results: A total of 10 patients were screened, enrolled, and treated. Transient paresthesias (60%) were the only amifampridine phosphate–related adverse events reported. Four patients were randomized to receive placebo–amifampridine–placebo sequence and three patients to receive amifampridine–placebo–amifampridine sequence. The co-primary objectives were statistically met (Quantitative Myasthenia Gravis score: p = 0.0003 and Myasthenia Gravis–specific Activities of Daily Living Profile score: p = 0.0006), as well as all the secondary endpoints (Myasthenia Gravis Composite score: p < 0.0001, Myasthenia Gravis Quality of Life scale—15 questions: p = 0.0025, Fatigue Severity Scale: p = 0.0061, and Carlo Besta Neurological Institute–Myasthenia Gravis scale: p = 0.0014). Conclusion: Despite the low number of patients, MuSK-001 study provided evidence that amifampridine phosphate, in the range of 30–60 mg daily dose, was safe and effective in treating muscle-specific kinase myasthenia gravis, suggesting the need for a large multi-center trial to confirm these results

Spider Functional Trait Diversity is Not Correlated with Plant Functional Trait Diversity

Color poster with text, images, photographs, and graphs.Ecological communities are subsets of larger-scale species pools. Community assembly may be due to ecological drift, resulting in communities with a random set of species and functional traits. However, ecological selection processes can act as filters and reduce functional trait diversity to a narrow subset, especially where environmental stress is high (aka trait clustering). Alternatively, ecological selection processes such as competition can require resource partitioning and thereby cause communities to have greater than expected functional trait diversity (aka trait overdispersion), especially where environmental stress is low. Trait-based community assembly may also be affected by scale. Environmental filtering should be most evident when comparing local communities to the largest spatial extent of the species pool and assembly should become increasingly random or overdispersed as the species pool extent is reduced in scale. We chose spiders because they are readily found in every terrestrial habitat and because they exhibit a high degree of functional diversity in body size, body shape, eyes, and mouthparts.University of Wisconsin--Eau Claire Office of Research and Sponsored Program

Plant Functional Trait Diversity is Influenced by Spatial Scaling and Moisture

Color poster with text and graphs.Ecological communities are subsets of larger-scale species pools. Community assembly may be due to ecological drift, resulting in communities with a random set of species and functional traits. However, ecological selection processes can act as filters and reduce functional trait diversity to a narrow subset, especially where environmental stress is high (aka trait clustering). Alternatively, ecological selection processes such as competition can require resource partitioning and thereby cause communities to have greater than expected functional trait diversity (aka trait overdispersion), especially where environmental stress is. Trait-based community assembly may also be affected by scale. Competition between plants occurs over short distances, and so trait overdispersion should be most evident in small plots (i.e., small grain sizes). Environmental filtering should be most evident when comparing local communities to the largest spatial extent of the species pool and assembly should become increasingly random or over dispersed as the species pool extent is reduced in scale.University of Wisconsin--Eau Claire Office of Research and Sponsored Program

Psychosocial difficulties of individuals with multiple sclerosis: the PARADISE-24 questionnaire

The aim of this study is to identify the most relevant psychosocial difficulties (PSDs) experienced by persons with multiple sclerosis (PwMS); to evaluate the relation between an overall level of PSDs measured with the PARADISE-24 and other variables; to assess which factors may predict PARADISE-24 overall score. Adults PwMS were consecutively enrolled and completed a battery of eight self-reported instruments (i.e. SCQ, EUROHIS-QOL 8, BRS, BFI10, JSPE, OSS-3, WHODAS-12, PARADISE-24). A clinical evaluation was performed for each patient and the Expanded Disability Status Scale (EDSS) score was determined by neurologists. A total of 80 PwMS, mean age 41.0, 65% women, 62% married, and 76.3% working, 86.3% relapsing-remitting MS, with an EDSS median score of 1.5, took part in the study. The mean disease duration was 7.7 years. The most frequently reported PSDs involved motor and emotional functioning. Free of charge access to medicines; health treatments and family, friends and health professionals' assistance represented the most important facilitators. WHODAS-12 and EUROHIS-QOL 8 were excluded from regression because of their strong correlation with PARADISE-24. EDSS, BRS, OSS-3, and neuroticism were the main predictors of the PARADISE-24 score. Despite MS heterogeneity, a common pattern of PSDs can be observed among PwMS. The level of physical impairment and personal resilience were the main predictors of the overall level of PSDs in PwMS, with a lower but significant additional role played by social support and personality traits. Attention to PSDs and their predictors, using PARADISE-24, can help clinicians to plan tailored and personalized rehabilitation programs. Copyright (C) 2016 Wolters Kluwer Health, Inc. All rights reserved

A comparative study on assessment procedures and metric properties of two scoring systems of the Coma Recovery Scale-Revised items: standard and modified scores

Objective: The study compared the metric characteristics (discriminant capacity and factorial structure) of two different methods for scoring the items of the Coma Recovery Scale-Revised and it analysed scale scores collected using the standard assessment procedure and a new proposed method.Design: Cross sectional design/methodological study.Setting: Inpatient, neurological unit.Participants: A total of 153 patients with disorders of consciousness were consecutively enrolled between 2011 and 2013.Intervention: All patients were assessed with the Coma Recovery Scale-Revised using standard (rater 1) and inverted (rater 2) procedures.Main outcome measures: Coma Recovery Scale-Revised score, number of cognitive and reflex behaviours and diagnosis.Results: Regarding patient assessment, rater 1 using standard and rater 2 using inverted procedures obtained the same best scores for each subscale of the Coma Recovery Scale-Revised for all patients, so no clinical (and statistical) difference was found between the two procedures. In 11 patients (7.7%), rater 2 noted that some Coma Recovery Scale-Revised codified behavioural responses were not found during assessment, although higher response categories were present. A total of 51 (36%) patients presented the same Coma Recovery Scale-Revised scores of 7 or 8 using a standard score, whereas no overlap was found using the modified score. Unidimensionality was confirmed for both score systems.Conclusion: The Coma Recovery Scale Modified Score showed a higher discriminant capacity than the standard score and a monofactorial structure was also supported. The inverted assessment procedure could be a useful evaluation method for the assessment of patients with disorder of consciousness diagnosis

Cognitive function alone is a poor predictor of health-related quality of life in employed patients with MS: results from a cross-sectional study

Depression, anxiety, disease severity, and cognitive functions impact on the quality of life of people with MS. However, the majority of studies were not based on multivariate models and did not target employed patients. The aim of this study was to investigate predictors of HRQoL in persons with MS in the workforce considering cognitive, psychological, disease severity, and disability-related variables