Amyotrophic lateral sclerosis & frontotemporal degeneration
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Amyotroph Lateral Scler Frontotemporal Degener · Jan 2015
Non-self-sufficiency as a primary outcome measure in ALS trials.
Our objective was to assess non-self-sufficiency (NSS) in ALS as an outcome measure in therapeutic trials. Using data from the control arm of two randomized trials and an observational study, associations between NSS (score ≤2 in the ALSFRS-R items for swallowing, cutting food and handling utensils, or walking) and the total ALSFRS-R score, forced vital capacity (FVC), and survival at selected time-points until death or 48 weeks, were assessed. These measures were used as surrogates of relevant functional impairment. ⋯ Compared to SS patients (median survival, 27.9 months), individuals becoming NSS at four weeks were at increased risk to die (median survival, 23.6 months, p = 0.02). NSS status at four weeks predicted survival even after adjustment for ALSFRS-R total score, age, gender, site of onset, disease duration, BMI, and FVC. 'Walking' was the only predictor of survival when adjusting for all covariates. In conclusion, NSS status is a possible endpoint to investigate short-term efficacy of treatments of ALS.
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Amyotroph Lateral Scler Frontotemporal Degener · Jan 2015
Compliance with recommendations made in a multidisciplinary ALS clinic.
Multidisciplinary ALS clinics provide recommendations at each visit, but these do little good unless recalled and followed. This study was conducted to determine recall of, and compliance with, these recommendations, and to study factors affecting compliance. Patients were contacted by telephone six weeks after their ALS clinic visit and asked about recommendations made by the multidisciplinary team. ⋯ Monitoring of patients between clinic visits appeared to enhance compliance. In conclusion, for ALS clinic teams seeking to maximize the impact of recommendations, discussions to facilitate understanding, instruction in problem-solving skills, and closer follow-up between clinic visits should facilitate better recall and compliance, and thus improve care. The potential benefits of greater emphasis on mental health and caregiver well-being should be explored.
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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.
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Amyotroph Lateral Scler Frontotemporal Degener · Jan 2015
Prospective study of cost of care at multidisciplinary ALS centers adhering to American Academy of Neurology (AAN) ALS practice parameters.
Multidisciplinary care in ALS is associated with longer survival, improved quality of life, and reduced hospital admissions, but there are no published data on institutional costs associated with multidisciplinary ALS care at U. S. centers. We prospectively examined institutional costs, adherence to AAN Practice Parameters and patient satisfaction in multidisciplinary ALS clinics at 18 U. ⋯ This prospective collaborative study demonstrates the direct financial burden of evidence-based multidisciplinary ALS care in the U. S.; more refined non-salary and overhead cost data are needed to evaluate the full cost impact of care. These data may be useful in supporting evidence-based models of patient centered care for ALS.