Faculty Opinions recommendation of What does the ALSFRS-R really measure? A longitudinal and survival analysis of functional dimension subscores in amyotrophic lateral sclerosis.

2017 ◽  
Author(s):  
Ammar Al-Chalabi
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Survival Analysis ◽  
Functional Dimension ◽  
Lateral Sclerosis

scholarly journals Prediction of survival in amyotrophic lateral sclerosis: a nationwide, Danish cohort study

BMC Neurology ◽  
2021 ◽  
Vol 21 (1) ◽  
Author(s):  
Anne-Lene Kjældgaard ◽  
Katrine Pilely ◽  
Karsten Skovgaard Olsen ◽  
Anders Hedegaard Jessen ◽  
Anne Øberg Lauritsen ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Survival Analysis ◽  
Prognostic Marker ◽  
Palliative Treatment ◽  
Rating Scale ◽  
Prognostic Markers ◽  
Progression Rate ◽  
Duration Of Symptoms ◽  
Sporadic Als ◽  
Lateral Sclerosis

Abstract Introduction Amyotrophic lateral sclerosis (ALS) is a progressive motor neuron disease with great heterogeneity. Biological prognostic markers are needed for the patients to plan future supportive treatment, palliative treatment, and end-of-life decisions. In addition, prognostic markers are greatly needed for the randomization in clinical trials. Objective This study aimed to test the ALS Functional Rating Scale-Revised (ALSFRS-R) progression rate (ΔFS) as a prognostic marker of survival in a Danish ALS cohort. Methods The ALSFRS-R score at test date in association with duration of symptoms, from the onset of symptoms until test date, (defined as ΔFS’) was calculated for 90 Danish patients diagnosed with either probable or definite sporadic ALS. Median survival time was then estimated from the onset of symptoms until primary endpoint (either death or tracheostomy). ΔFS’ was subjected to survival analysis using Cox proportional hazards modelling, log-rank test, and Kaplan-Meier survival analysis. Results and conclusions Both ΔFS’ and age was found to be strong predictors of survival of the Danish ALS cohort. Both variables are easily obtained at the time of diagnosis and could be used by clinicians and ALS patients to plan future supportive and palliative treatment. Furthermore, ΔFS’, is a simple, prognostic marker that predicts survival in the early phase of disease as well as at later stages of the disease.

Amyotrophic Lateral Sclerosis survival analysis: Swallowing and non-oral feeding

2014 ◽  
Vol 35 (3) ◽  
pp. 535-542 ◽  
Author(s):  
K.F. Luchesi ◽  
S. Kitamua ◽  
L.F. Mourão
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Survival Analysis ◽  
Oral Feeding ◽  
Lateral Sclerosis

scholarly journals Long-term survival analysis of masitinib in amyotrophic lateral sclerosis

2021 ◽  
Vol 14 ◽  
pp. 175628642110303
Author(s):  
Jesus S. Mora ◽  
Walter G. Bradley ◽  
Delia Chaverri ◽  
María Hernández-Barral ◽  
Javier Mascias ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Survival Analysis ◽  
Phase Iii ◽  
Progression Rate ◽  
Term Survival ◽  
Risk Of Death ◽  
Severe Impairment ◽  
Phase Iii Study ◽  
Lateral Sclerosis

Background: A randomized, placebo-controlled phase III study (AB10015) previously demonstrated that orally administered masitinib (4.5 mg/kg/day) slowed rate of functional decline, with acceptable safety, in amyotrophic lateral sclerosis (ALS) patients having an ALS Functional Rating Scale-revised (ALSFRS-R) progression rate from disease onset to baseline of <1.1 points/month. Here we assess long-term overall survival (OS) data of all participants from study AB10015 and test whether a signal in OS is evident in an enriched patient population similar to that prospectively defined for confirmatory study AB19001. Methods: Survival status of all patients originally randomized in AB10015 was collected from participating investigational sites. Survival analysis (using the multivariate log-rank test and Cox proportional hazards model, with stratification factors as covariates) was performed on the intention-to-treat population and enriched subgroups, which were defined according to initial randomization, baseline ALSFRS-R progression rate and baseline disease severity. Results: A significant survival benefit of 25 months ( p = 0.037) and 47% reduced risk of death ( p = 0.025) was observed for patients receiving 4.5 mg/kg/day masitinib ( n = 45) versus placebo ( n = 62) in an enriched cohort with ⩾2 on each baseline ALSFRS-R individual component score (i.e. prior to any complete loss or severe impairment of functionality) and post-onset ALSFRS-R progression rate <1.1 (i.e. exclusion of very fast progressors) [median OS of 69 versus 44 months, respectively; hazard ratio, 0.53 [95% CI (0.31–0.92)]]. This corresponds to the population enrolled in confirmatory phase III study, AB19001. Conclusions: Analysis of long-term OS (75 months average follow-up from diagnosis) indicates that oral masitinib (4.5 mg/kg/day) could prolong survival by over 2 years as compared with placebo, provided that treatment starts prior to severe impairment of functionality. This trial was registered at www.ClinicalTrials.gov under identifier NCT02588677 (28 October 2015).

scholarly journals Survival Analysis of Irish Amyotrophic Lateral Sclerosis Patients Diagnosed from 1995–2010

PLoS ONE ◽  
2013 ◽  
Vol 8 (9) ◽  
pp. e74733 ◽  
Author(s):  
James Rooney ◽  
Susan Byrne ◽  
Mark Heverin ◽  
Bernie Corr ◽  
Marwa Elamin ◽  
...  
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Survival Analysis ◽  
Lateral Sclerosis

Automated Acoustic Analysis of Oral Diadochokinesis to Assess Bulbar Motor Involvement in Amyotrophic Lateral Sclerosis

2020 ◽  
Vol 63 (1) ◽  
pp. 59-73 ◽  
Author(s):  
Panying Rong
Keyword(s):  
Amyotrophic Lateral Sclerosis ◽  
Acoustic Analysis ◽  
Speaking Rate ◽  
Disease Stage ◽  
Healthy Controls ◽  
Tongue Movement ◽  
Major Barrier ◽  
Syllable Repetition ◽  
Oral Diadochokinesis ◽  
Lateral Sclerosis

Purpose The purpose of this article was to validate a novel acoustic analysis of oral diadochokinesis (DDK) in assessing bulbar motor involvement in amyotrophic lateral sclerosis (ALS). Method An automated acoustic DDK analysis was developed, which filtered out the voice features and extracted the envelope of the acoustic waveform reflecting the temporal pattern of syllable repetitions during an oral DDK task (i.e., repetitions of /tɑ/ at the maximum rate on 1 breath). Cycle-to-cycle temporal variability (cTV) of envelope fluctuations and syllable repetition rate (sylRate) were derived from the envelope and validated against 2 kinematic measures, which are tongue movement jitter (movJitter) and alternating tongue movement rate (AMR) during the DDK task, in 16 individuals with bulbar ALS and 18 healthy controls. After the validation, cTV, sylRate, movJitter, and AMR, along with an established clinical speech measure, that is, speaking rate (SR), were compared in their ability to (a) differentiate individuals with ALS from healthy controls and (b) detect early-stage bulbar declines in ALS. Results cTV and sylRate were significantly correlated with movJitter and AMR, respectively, across individuals with ALS and healthy controls, confirming the validity of the acoustic DDK analysis in extracting the temporal DDK pattern. Among all the acoustic and kinematic DDK measures, cTV showed the highest diagnostic accuracy (i.e., 0.87) with 80% sensitivity and 94% specificity in differentiating individuals with ALS from healthy controls, which outperformed the SR measure. Moreover, cTV showed a large increase during the early disease stage, which preceded the decline of SR. Conclusions This study provided preliminary validation of a novel automated acoustic DDK analysis in extracting a useful measure, namely, cTV, for early detection of bulbar ALS. This analysis overcame a major barrier in the existing acoustic DDK analysis, which is continuous voicing between syllables that interferes with syllable structures. This approach has potential clinical applications as a novel bulbar assessment.

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