Journal of neurology, neurosurgery, and psychiatry
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J. Neurol. Neurosurg. Psychiatr. · Sep 2010
Effect sizes can be misleading: is it time to change the way we measure change?
Previous comparisons of the ability to detect change in the Barthel Index (BI) and Functional Independence Measure motor scale (FIMm) have implied these two scales are equally responsive when examined using traditional effect size statistics. Clinically, this is counterintuitive as the FIMm has greater potential to detect change than the BI and raises concerns about the validity of effect size statistics as indicators of rating scale responsiveness. To examine these concerns, in this study a sophisticated psychometric analysis was applied, Rasch measurement to BI and FIMm data. ⋯ These findings demonstrate that effect size calculations are limited and potentially misleading indicators of rating scale responsiveness at the group comparison level. Rasch analysis at the individual person level showed the superior responsiveness of the FIMm, supporting clinical expectation, and its added value as a method for examining and comparing rating scale responsiveness.
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J. Neurol. Neurosurg. Psychiatr. · Aug 2010
Case ReportsThe enigma of 7q36 linked autosomal dominant limb girdle muscular dystrophy.
Two families with autosomal dominant limb girdle muscular dystrophy (LGMD) have previously been linked to a locus on chromosome 7q36 10 years ago. The locus has been termed both LGMD1D and 1E, but because of lack of additional families to narrow down the linked region of interest, this disease has remained elusive. ⋯ Advancement towards the causative gene defect in the 7q36 linked disease needs new additional families to narrow the region of interest. The phenotype in the previously linked families has not been reported in full detail, which may be one reason for the shortage of additional families. A comprehensive clinical and morphological phenotype of chromosome 7q36 linked autosomal dominant LGMD with a restricted and updated 6.4 Mb sized haplotype is reported here.
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J. Neurol. Neurosurg. Psychiatr. · Aug 2010
Delayed onset of a second paraneoplastic neurological syndrome in eight patients.
The occurrence of a second delayed paraneoplastic neurological syndrome (PNS), different from the first, in the same patient, is not well known. However, recognition of this possibility is important in the management of the patient with PNS. ⋯ A second PNS can reveal a cancer relapse but can also arise in its absence. The long survival of patients with a second PNS suggests that the immune response might be more effective in controlling the cancer.
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J. Neurol. Neurosurg. Psychiatr. · Aug 2010
Randomized Controlled TrialAnalysis of clinical outcomes according to original treatment groups 16 years after the pivotal IFNB-1b trial.
Evidence for efficacy of disease-modifying drugs in multiple sclerosis (MS) comes from trials of short duration. We report results from a 16 y, retrospective follow-up of the pivotal interferon beta-1b (IFNB-1b) study. ⋯ The original treatment assignment could not be shown to influence standard assessments of long-term efficacy. On-study behaviour of patients was influenced by factors that could not be controlled with the sacrifice of randomisation and blinding. Mortality was higher in patients originally assigned to placebo than those who had received IFNB-1b 50 microg or 250 microg. The dataset provides important resources to explore early predictors of long-term outcome.