Journal of the neurological sciences
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Randomized Controlled Trial
Planning future clinical trials in Machado Joseph disease: Lessons from a phase 2 trial.
In a recent phase 2 clinical trial in spinocerebellar ataxia type 3/Machado Joseph disease (SCA3/MJD), a neurogenetic disorder without specific therapy, benefits of lithium carbonate were found only on secondary efficacy outcomes, all related to ataxic features. In order to help designing future studies, we further analyzed the trial data searching for treatment response modifiers and metric properties of spinocerebellar ataxia (SCA) scales. ⋯ Lithium efficacy on cerebellar NESSCA, and on SCAFI and CCFS in the primary analysis, together with the lack of effect on non-ataxia features suggests that lithium should be tested in phase 3 trials in SCA3/MJD and that ataxia scales should be preferred to multisystem neurological instruments as the primary outcome. The inclusion of early stage patients is advisable in future clinical trials in SCA3/MJD.
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Amyotrophic lateral sclerosis (ALS) patients can suffer episodes of lower respiratory tract infections (LRTI) leading to an acute respiratory failure (ARF) requiring noninvasive ventilation (NIV). ⋯ NBS can predict noninvasive management failure during LRTI in ALS.
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Randomized Controlled Trial
Cognitive outcome in acute simvastatin treatment for aneurysmal subarachnoid hemorrhage: A propensity matched analysis.
Experimental evidence has indicated the benefit of simvastatin in the treatment of subarachnoid hemorrhage (SAH). Recently, acute simvastatin treatment was not shown to be beneficial in neurological outcome using modified Rankin Scale. Cognitive function is another important dimension of outcome assessment and yet had not been investigated in statin studies for aneurysmal subarachnoid hemorrhage. We therefore explored whether acute simvastatin treatment would improve cognitive outcomes. ⋯ The current study does not support that acute simvastatin treatment improves cognitive outcome after aneurysmal subarachnoid hemorrhage.
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The Global Health Delivery (GHD) framework (Farmer, Kim, and Porter, Lancet 2013;382:1060-69) allows for the analysis of health care delivery systems along four axes: a care delivery value chain that incorporates prevention, diagnosis, and treatment of a medical condition; shared delivery infrastructure that integrates care within existing healthcare delivery systems; alignment of care delivery with local context; and generation of economic growth and social development through the health care delivery system. Here, we apply the GHD framework to epilepsy care in rural regions of low- and middle-income countries (LMIC) where there are few or no neurologists.
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Randomized Controlled Trial
Canalith repositioning in apogeotropic horizontal canal benign paroxysmal positional vertigo: Do we need faster maneuvering?
A correct diagnosis and a proper treatment may yield a rapid and simple cure for benign paroxysmal positional vertigo (BPPV). Although the Gufoni maneuver is widely used to treat apogeotropic horizontal-canal BPPV (HC-BPPV), few studies have clarified the relationship between the speed and intensity of maneuver execution and successful canalith reposition. To evaluate the effect of accelerated execution of the Gufoni maneuver, a prospective randomized controlled study was conducted with HC-BPPV patients in a single dizziness clinic. ⋯ The overall resolution rate was 48% (24 of 50; p=1.00), regardless of acceleration. Our results suggest that a faster, more intense execution of the Gufoni maneuver provides little benefit in treating apogeotropic HC-BPPV. Detachment of the otolith from the cupula or the gravitational force-when the otolith is in the anterior arm of the HC-may be more important contributors to treatment efficacy.