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dinsdag 31 oktober 2017

The Lancet: [Articles] Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory ...

[Articles] Safety and efficacy of eculizumab in anti-acetylcholine receptor antibody-positive refractory generalised myasthenia gravis (REGAIN): a phase 3, randomised, double-blind, placebo-controlled, multicentre study
The change in the MG-ADL score was not statistically significant between eculizumab and placebo, as measured by the worst-rank analysis. Eculizumab was well tolerated. The use of a worst-rank analytical approach proved to be an important limitation of this study since the secondary and sensitivity analyses results were inconsistent with the primary endpoint result; further research into the role of complement is needed.
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[Comment] Eculizumab: a treatment option for mysthenia gravis?
Eculizumab is an expensive, humanised monoclonal antibody against the terminal complement protein C5. The drug was patented in 2007, and has since been used and approved for two rare diseases, paroxysmal nocturnal haemoglobulinuria and atypical haemolytic ureamic syndrome. Additionally, eculizumab has been used off-label, which constitutes up to a half of its use in some countries,1 for at least 25 different conditions. Refractory and generalised myasthenia gravis with anti-acetylcholine receptor antibodies is a rare disease that involves the complement system and for which eculizumab has a potential effect.
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[Comment] Neurodegeneration in histiocytoses might start in utero
Histiocytic disorders, such as Langerhans cell histiocytosis and Erdheim-Chester disease, are characterised by inflammation and accumulation of cells derived from the monocyte and macrophage lineages, resulting in tissue damage.1 Neurodegenerative lesions in Langerhans cell histiocytosis are a devastating type of CNS involvement—different from tumour infiltration—in which patients present with progressive symmetric cerebellar syndrome, tetrapyramidal syndrome with or without motor deficits, pseudobulbar palsy, or cognitive impairment.
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[Articles] 24-month intervention with a specific multinutrient in people with prodromal Alzheimer's disease (LipiDiDiet): a randomised, double-blind, controlled trial
The intervention had no significant effect on the NTB primary endpoint over 2 years in prodromal Alzheimer's disease. However, cognitive decline in this population was much lower than expected, rendering the primary endpoint inadequately powered. Group differences on secondary endpoints of disease progression measuring cognition and function and hippocampal atrophy were observed. Further study of nutritional approaches with larger sample sizes, longer duration, or a primary endpoint more sensitive in this pre-dementia population, is needed.
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[Comment] Targeting prodromal Alzheimer's disease: too late for prevention?
Prodromal Alzheimer's disease is a novel area of research, with clinical research definitions still under development. The LipiDiDiet trial,1 published in The Lancet Neurology, was one of the first completed trials based on the International Working Group (IWG-1) criteria for prodromal Alzheimer's disease.2 The study was a non-pharmacological intervention (medical food Souvenaid) in prodromal Alzheimer's disease.
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