Amyotrophic lateral sclerosis & frontotemporal degeneration
-
Amyotroph Lateral Scler Frontotemporal Degener · May 2019
Randomized Controlled Trial Observational StudyPsychological distress and coping styles of caregivers of patients with amyotrophic lateral sclerosis: a longitudinal study.
Objectives: To assess psychological distress levels over time in caregivers of patients in the early stage of amyotrophic lateral sclerosis (ALS) and to investigate the association between caregivers' coping styles and psychological distress over time. Methods: Fifty-four caregivers were followed in a RCT of patients in the early stage of ALS. Questionnaires were administered at baseline, 4, 7, and 10 months. ⋯ Conclusion: Feelings of distress increase in ALS caregivers during the course of the disease of the patient. Emotion-oriented coping is related to psychological distress; however, emotional coping and distress might represent overlapping constructs. Focusing on one coping style in the care for ALS caregivers may be too simplistic for the complex situations they face, as it ignores the fact that people may require different coping strategies in different situations.
-
Amyotroph Lateral Scler Frontotemporal Degener · Nov 2018
Randomized Controlled TrialRelationships between slow vital capacity and measures of respiratory function on the ALSFRS-R.
As declining respiratory muscle function commonly leads to disability and death in amyotrophic lateral sclerosis (ALS), respiratory measurements such as slow vital capacity (SVC) may predict disease progression. This study evaluated the relationship between SVC and symptoms measured by the revised ALS Functional Rating Scale (ALSFRS-R). ⋯ The higher correlation between %predicted SVC and specific ALSFRS-R symptom scores in patients with rapidly versus more slowly progressing disease reinforces the importance of continually monitoring respiratory function throughout the disease course.
-
Amyotroph Lateral Scler Frontotemporal Degener · Jan 2015
Randomized Controlled Trial Comparative StudyA preliminary randomized trial of the mechanical insufflator-exsufflator versus breath-stacking technique in patients with amyotrophic lateral sclerosis.
A major problem faced by patients with amyotrophic lateral sclerosis (ALS) in respiratory failure is the inability to cough effectively. Forty eligible ALS patients were randomized to the breath-stacking technique using a lung volume recruitment bag (n = 21) or mechanical insufflator-exsufflator MI-E (n = 19) and followed up at three-monthly intervals for at least 12 months or until death. Results showed that there were 13 episodes of chest infection in the breath-stacking group and 19 episodes in the MI-E group (p = 0.92), requiring 90 and 95 days of antibiotics, respectively (p = 0.34). ⋯ In conclusion, lack of statistically significant differences due to sub-optimal power and confounders precludes a definitive conclusion with respect to the relative efficacy of one cough augmentation technique over the other. This study however, provides useful lessons and informative data, needed to strengthen the power calculation, inclusion criteria and randomization factors for a large scale definitive trial. Until such a definitive trial can be undertaken, we recommend the breath-stacking technique as a low-cost, first-line intervention for volume recruitment and cough augmentation in patients with ALS who meet the criteria for intervention with non-invasive ventilation.
-
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.
-
Amyotroph Lateral Scler Frontotemporal Degener · Jan 2013
Randomized Controlled TrialDexpramipexole effects on functional decline and survival in subjects with amyotrophic lateral sclerosis in a Phase II study: subgroup analysis of demographic and clinical characteristics.
Our objective was to explore treatment effects in patient subgroups using post hoc analyses of data from part 2 of the dexpramipexole Phase II study. Subjects with amyotrophic lateral sclerosis (ALS) received dexpramipexole 300 mg/day or 50 mg/day for 24 weeks. Treatment effects on the slope of the revised ALS Functional Rating Score (ALSFRS-R) and Combined Assessment of Function and Survival (CAFS) were evaluated in dichotomized subgroups: riluzole use, gender, site of symptom onset. ⋯ Generally, ALSFRS-R decline was reduced and CAFS rankings were higher in the 300-mg vs. 50-mg arm across subgroups. CAFS rankings were significantly higher in the 300-mg vs. 50-mg arm among subjects with ALSFRS-R scores ≤35, symptom duration <18.7 months, or progression rate ≥ 0.7 points/month (p < 0.03). In conclusion, the observed benefit of 300- vs. 50-mg dexpramipexole on functional decline and survival was generally consistent among subjects regardless of baseline characteristics.