Amyotrophic lateral sclerosis & frontotemporal degeneration
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Amyotroph Lateral Scler Frontotemporal Degener · Sep 2013
Randomized Controlled Trial Multicenter StudyRandomized double-blind placebo-controlled trial of acetyl-L-carnitine for ALS.
Our objective was to assess the effects of acetyl-L-carnitine (ALC) with riluzole on disability and mortality of amyotrophic lateral sclerosis (ALS). Definite/probable ALS patients, 40-70 years of age, duration 6-24 months, self-sufficient (i.e. able to swallow, cut food/handle utensils, and walk), and with forced vital capacity (FVC) > 80% entered a pilot double-blind, placebo-controlled, parallel group trial and were followed for 48 weeks. ALC or placebo 3 g/day was added to riluzole 100 mg/day. ⋯ MRC, QoL and adverse events were similar. In conclusion, ALC may be effective, well-tolerated and safe in ALS. A pivotal phase III trial is needed.
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Amyotroph Lateral Scler Frontotemporal Degener · Sep 2013
Prognostic value of efficiently correcting nocturnal desaturations after one month of non-invasive ventilation in amyotrophic lateral sclerosis: a retrospective monocentre observational cohort study.
Abstract NIV adherence ('quantity' of ventilation) has a prognostic impact in amyotrophic lateral sclerosis (ALS). We hypothesized that NIV effectiveness ('quality') could also have a similar impact. NIV effectiveness was evaluated in 82 patients within the first month (M1) and every three months (symptoms, arterial blood bases, and nocturnal pulsed oxygen saturation - SpO2). ⋯ In this subgroup, one-year mortality was not different from that in Group 1. Multivariate analysis identified independent mortality risk factors expectedly including bulbar involvement (HR = 4.31 (1.73 - 10.76), p = 0.002), 'rapid respiratory decline' (HR = 3.55 (1.29 - 9.75), p = 0.014) and vital capacity (HR = 0.97 (0.95 - 0.99), p = 0.010), but also inadequate ventilation in the first month (HR = 2.32 (1.09 - 4.94), p = 0.029). In conclusion, in ALS patients NIV effectiveness to correct nocturnal desaturations is an independent prognostic factor.
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Amyotroph Lateral Scler Frontotemporal Degener · Sep 2013
Prevalence and characteristics of pain in early and late stages of ALS.
The purpose of this study was to compare pain frequency in early and late stages of ALS and to describe the relationship between pain intensity and functional status. Sixty-four patients in different stages of ALS were asked to complete the Neuropathic Pain Scale and to draw the localization of their pain on a body cartoon. The Amyotrophic Lateral Sclerosis Functional Rating Scale-Revised (ALSFRS-R) and Forced Vital Capacity (FVC) values were obtained from the medical record. ⋯ There was no statistically significant difference in the presence of pain among patients in the different stages of ALS. In conclusion, our study showed that pain is common in ALS patients. Although pain intensity did correlate negatively with functional status, as expected, we were surprised to find that pain was also present in the early stages of the disease.
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Amyotroph Lateral Scler Frontotemporal Degener · Sep 2013
The impact of clinical factors, riluzole and therapeutic interventions on ALS survival: a population based study in Modena, Italy.
The prognostic role of riluzole, enteral nutrition (EN), non-invasive ventilation (NIV) and interdisciplinary care in ALS is still debated. A population based study has been performed focusing on ALS survival, with particular attention to prognostic factors and therapeutic intervention. All patients diagnosed with ALS between 2000 and 2009 and residing in Modena, Italy, have been registered. ⋯ In the Cox multivariable model, the factors independently related to a longer survival were age (p < 0.01), site of onset (p = 0.02) and riluzole treatment (p < 0.01), with a median gain in survival of 29 months (patients aged < 55 years compared with patients ≥ 55 years), 20 months (spinal versus bulbar onset), and 12 months (riluzole, yes vs. no), respectively. In conclusion, the study has confirmed the prognostic role of clinical features, but has surprisingly demonstrated that riluzole prolonged life significantly longer than NIV and EN. This observational study described the effects of ALS management in a setting that may approximate routine clinical practice more closely than randomized controlled trial (RCT); effects of uncontrolled potential confounders, however, cannot be excluded.