Amyotrophic lateral sclerosis & frontotemporal degeneration
-
Amyotroph Lateral Scler Frontotemporal Degener · Mar 2014
Non-invasive ventilation effectiveness and the effect of ventilatory mode on survival in ALS patients.
Non-invasive ventilation (NIV) prolongs survival in amyotrophic lateral sclerosis (ALS), but there are no data with which to compare the effectiveness of the different ventilator modes - volume (Vol-NIV) or pressure-cycled (Pres-NIV) ventilation - in ALS. We aimed to determine whether the ventilatory mode has an effect on ventilation effectiveness and survival of ALS patients using NIV. We used a retrospective study that included all ALS patients for whom NIV was indicated in two referral units: one using Vol-NIV and the other using Pres-NIV. ⋯ Ventilator mode (OR 12.066 (4.251-32.270), p < 0.001) and severity of bulbar dysfunction (OR 1.07 (1.011-1.133), p = 0.02) were the variables correlated with effective NIV. In conclusion, although Vol-NIV provides more effective ventilation, Vol-NIV and Pres-NIV present similar survival in ALS. Effectiveness of NIV is related to the severity of bulbar dysfunction.
-
Amyotroph Lateral Scler Frontotemporal Degener · Mar 2014
ReviewPredicting success: optimizing phase II ALS trials for the transition to phase III.
Amyotrophic lateral sclerosis (ALS) research is advancing quickly, but the transition from phase II to phase III trials remains particularly challenging. In part, this is because of the paradox of phase II ALS trials - they are expected to inform researchers about safety, tolerability, dosage selection, and efficacy using a small number of patients, and relying on essentially the same outcome measures used in phase III trials. We examined pharmacokinetics in the cerebrospinal fluid and pharmacodynamic markers to demonstrate target engagement. ⋯ Primary endpoints should be pre-specified. Inclusion criteria should be used to reduce heterogeneity and target a relevant subpopulation of people with ALS when possible. Multiple phase II trials might be required before moving to a large phase III trial.
-
Amyotroph Lateral Scler Frontotemporal Degener · Mar 2014
Emotion processing deficits distinguish pure amyotrophic lateral sclerosis from frontotemporal dementia.
Amyotrophic lateral sclerosis (ALS) is a multisystem disease that overlaps with frontotemporal dementia (FTD). Although FTD patients exhibit prominent deficits in emotion perception and social cognition, these domains have received relatively little attention in ALS. Moreover, direct comparisons between ALS and FTD on emotion processing tasks remain lacking. ⋯ We conclude that patients with FTD-ALS and FTD show similar, significant impairments in emotional processing. By contrast, ALS patients without dementia exhibit preserved emotion processing. Performance on emotion processing tasks may provide a useful clinical tool in identifying those with early FTD-ALS.