Lancet neurology
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Many of the available disability outcome measures used in clinical trials of multiple sclerosis are insensitive to change over time, inadequately validated, or insensitive to patient-perceived health status or quality of life. Increasing focus on therapies that slow or reverse disability progression makes it essential to refine existing measures or to develop new tools. Major changes to the expanded disability status scale should be avoided to prevent the loss of acceptance by regulators as a measure for primary outcomes in trials that provide substantial evidence of effectiveness. ⋯ Conversely, although substantial data support the multiple sclerosis functional composite as an alternative measure, changes to its component tests and scoring method are needed. Novel approaches, including the use of composite endpoints, patient-reported outcomes, and measurement of biomarkers, show promise as adjuncts to the current disability measures, but are insufficiently validated to serve as substitutes. A collaborative approach that involves academic experts, regulators, industry representitives, and funding agencies is needed to most effectively develop disability outcome measures.
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Diagnostic criteria for multiple system atrophy are focused on motor manifestations of the disease, in particular ataxia and parkinsonism, but these criteria often cannot detect the early stages. Non-motor symptoms and signs of multiple system atrophy often precede the onset of classic motor manifestations, and this prodromal phase is estimated to last from several months to years. Autonomic failure, sleep problems, and respiratory disturbances are well known symptoms of established multiple system atrophy and, when presenting early and preceding ataxia or parkinsonism, should be regarded as evidence of premotor multiple system atrophy. An early and accurate diagnosis is becoming increasingly important as new neuroprotective agents are developed.
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Randomized Controlled Trial Comparative Study
Prognostic factors for time to treatment failure and time to 12 months of remission for patients with focal epilepsy: post-hoc, subgroup analyses of data from the SANAD trial.
Epilepsy is a heterogeneous disorder, with outcomes ranging from immediate remission after taking a first antiepileptic drug to frequent unremitting seizures with multiple treatment failures. Few prognostic models enable prediction of outcome; we therefore aimed to use data from the SANAD study to predict outcome overall and for patients receiving specific treatments. ⋯ National Institute for Health Research (UK).