Amyotrophic lateral sclerosis & frontotemporal degeneration
-
Amyotroph Lateral Scler Frontotemporal Degener · Feb 2020
Respiratory measurements and airway clearance device prescription over one year in amyotrophic lateral sclerosis.
Objective: The rates of decline in respiratory measurements, including Peak Cough Flow (PCF) have not been established in Amyotrophic Lateral Sclerosis (ALS). Additionally, optimal prescription of cough adjuncts which aim to increase cough strength are unknown. The primary aim of this study was to quantify declines in respiratory function in ALS using PCF, Sniff Nasal Inspiratory Pressure (SNIP) and Slow Vital Capacity (SVC). ⋯ Conclusions: This study identified a rapid rate of decline in PCF, a similar decline in SNIP, and slower declines in SVC and ALSFRS-R. Cough adjunct prescription was triggered by declining respiratory measures and recommended PCF thresholds, but also by respiratory symptoms. Chest infections were common in patients regardless of cough adjunct prescription and should be closely monitored.
-
Amyotroph Lateral Scler Frontotemporal Degener · Feb 2020
Epidemiological and clinical features of amyotrophic lateral sclerosis in a Tunisian cohort.
Objective: To describe the phenotypic heterogeneity of amyotrophic lateral sclerosis (ALS) in Tunisian patients, and to define the sociodemographic features, treatments, and survival rate with ALS. Methodology: The study included 210 patients with ALS diagnosed between 2003 and 2019 in The Neurology department, Razi Hospital Tunisia. ALS patients were phenotyped and followed until their death. ⋯ Initial ALSFRS-R scores were higher in our population. Age at onset of Bulbar ALS was younger. Our study highlights the possible presence of genetic and environmental factors that may influence the clinical phenotype of ALS in Tunisia.
-
Amyotroph Lateral Scler Frontotemporal Degener · Nov 2019
ALS/SURV: a modification of the CAFS statistic.
We present a composite endpoint that can be used in amyotrophic lateral sclerosis (ALS) trials, which combines functional status (via the ALS functional rating scale) and survival, denoted ALS/SURV. ALS/SURV modifies and extends the combined assessment of function and survival (CAFS) score and assigns rankings to participants that withdraw or are lost to follow up in a way that does not disproportionately lower and skew ranks for those participants that reach study endpoint (either death or study completion). ALS/SURV has properties of: (1) ordering participants that completed the study from the shortest surviving participant to the last observed death followed by worst function to best function; (2) ordering participants withdrawing at time of withdrawal by their decline in functional status relative to all the participants still in the study; and (3) then maintaining this ordering at time of withdrawal relative to participants still in the study. ⋯ Additionally, ALS/SURV can be summarized as either median functional status or median survival along with interquartile range, thereby adding clinical meaning to the statistic. Finally, by applying normal deviates, confidence intervals can be computed and used to estimate power for future studies. In summary, the above properties support the role for ALS/SURV as a new ALS composite statistic.
-
Amyotroph Lateral Scler Frontotemporal Degener · Nov 2019
Longitudinal analysis of sniff nasal inspiratory pressure assessed using occluded and un-occluded measurement techniques in amyotrophic lateral sclerosis and primary lateral sclerosis.
Objective: Sniff nasal inspiratory pressure (SNIP) is a commonly used clinical measure of respiratory impairment in amyotrophic lateral sclerosis (ALS), which is used to guide the initiation of noninvasive ventilation (NIV). SNIP can be completed with either an occluded or an un-occluded contralateral nostril. The aim of this study was to compare occluded and un-occluded SNIP measurements and to examine the decline in occluded SNIP over time compared to the ALSFRS-R respiratory subscore. ⋯ This showed a linear decline in spinal onset with a floor effect in bulbar-onset ALS. Conclusion: SNIP measured with an occluded and un-occluded contralateral nostril is not interchangeable, which is relevant in interpreting thresholds for initiation of NIV. SNIP declines non-linearly, which is explained in spinal onset ALS by age and diagnostic delay, but an apparent floor effect remains in bulbar onset.
-
Amyotroph Lateral Scler Frontotemporal Degener · Aug 2019
Meta AnalysisPost-hoc analyses of the edaravone clinical trials Study 16 and Study 19: a step toward more efficient clinical trial designs in amyotrophic lateral sclerosis.
Objectives: The edaravone development program established a study design in which a treatment effect slowing functional loss in amyotrophic lateral sclerosis (ALS) could be documented within a 24-week time frame. This report elucidates the strategic enrichment design utilized to create efficiency and precision in the development program. Methods: Post-hoc analyses describe learning, sequential iteration, and evolution in study design. ⋯ The enrichment strategy may have excluded some rapidly progressing patients, potentially supporting the detection of a treatment effect. As previously published, Study MCI186-19 prospectively documented a 33% reduction in rate of progression of ALS (p = 0.0013). Conclusions: Strategic choices in the design of Study MCI186-19 reduced the proportion of minimally progressing patients and supported detection of a treatment effect.