Lancet neurology
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Review Comparative Study
Early highly effective versus escalation treatment approaches in relapsing multiple sclerosis.
Treatment decisions in multiple sclerosis are complex given the large number of disease-modifying therapies with diverse safety and efficacy profiles. The importance of early treatment has been recognised but how intensively to treat at onset is not known. Substantial variability exists in treatment selection with weak clinical trial evidence to guide initial treatment choices. ⋯ Whether to use moderately effective and safe medications with escalation as needed, or to use higher efficacy medications from the outset, is a key question in clinical practice. Clinical trials in patients with relapsing multiple sclerosis have focused on pairwise comparisons but the effectiveness of different treatment approaches has not been tested. Future pragmatic randomised clinical trials and observational studies will help to inform more rational selection of initial therapies and improve the quality of life of patients with relapsing multiple sclerosis.
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Multicenter Study Observational Study
Case-mix, care pathways, and outcomes in patients with traumatic brain injury in CENTER-TBI: a European prospective, multicentre, longitudinal, cohort study.
The burden of traumatic brain injury (TBI) poses a large public health and societal problem, but the characteristics of patients and their care pathways in Europe are poorly understood. We aimed to characterise patient case-mix, care pathways, and outcomes of TBI. ⋯ European Union 7th Framework Programme, the Hannelore Kohl Stiftung, OneMind, and Integra LifeSciences Corporation.
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After traumatic brain injury (TBI), plasma concentration of glial fibrillary acidic protein (GFAP) correlates with intracranial injury visible on CT scan. Some patients with suspected TBI with normal CT findings show pathology on MRI. We assessed the discriminative ability of GFAP to identify MRI abnormalities in patients with normal CT findings. ⋯ National Institute of Neurological Disorders and Stroke and US Department of Defense.
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Leukodystrophies comprise a large group of rare genetic disorders primarily affecting CNS white matter. Historically, the diagnostic process was slow and patient prognosis regarded as poor because curative treatment was only available for very few leukodystrophies in early stages of the disease. Whole-exome sequencing has both greatly increased the number of known leukodystrophies and improved diagnosis. ⋯ Improved diagnosis has revealed the phenotypic variability of leukodystrophies, requiring adaptation of prognostication. Technological advance in molecular techniques and improved insight into the pathophysiology of individual leukodystrophies have led to therapeutic developments, including drug design and gene therapy. Despite this progress, therapies are only beneficial early in the disease course, emphasising the need for a speedy diagnosis and for research on regenerative approaches to repair the damage already present.