Amyotroph Lateral Sc
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Amyotroph Lateral Sc · Oct 2007
Randomized Controlled Trial Clinical TrialOutcome measures for early phase clinical trials.
As the number of potential neuroprotective agents for ALS increases, the need for early phase trials that screen drugs before proceeding to efficacy trials also grows. However, it is not known which outcome measures perform best and also provide the most meaningful information in brief small trials. We assessed the performance of different outcome measures for use in early phase clinical trials in ALS, and determined what degree of change in the ALSFRS-R that patients could perceive. ⋯ One-unit change in patient-perceived clinical function paralleled a 9-point decrease in the ALSFRS-R (p = 0.025; 95% CI 8, 10). This trial assessed just 30 patients over six months, but the standard outcome measures each performed dependably; all could be used in short-duration, early phase trials. The ALSFRS-R most strongly predicted survival and provided the most complete data, but large changes may be necessary before patients perceive treatment effects.
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Amyotroph Lateral Sc · Oct 2007
Distinctions between the dementia in amyotrophic lateral sclerosis with frontotemporal dementia and the dementia of Alzheimer's disease.
The clinical entity of Amyotrophic Lateral Sclerosis with Frontotemporal Dementia (ALS-FTD) has only recently been recognized as an important neurodegenerative disease. As in isolated FTD, the behavioral and personality changes in ALS-FTD might be more characteristic than its cognitive changes. We aimed to characterize the behavioral and cognitive deficits in ALS-FTD, and contrast this profile with that of the most common form of dementia, AD, to assist ALS clinicians in recognizing the syndrome early in its course. ⋯ The FBI-mod was completed by caregivers. We found that the FBI-mod, age-corrected Z scores for the Mini-Mental State Examination (MMSE), a test of delayed recall, and a word fluency measure together discriminated between ALS-FTD and AD. ALS-FTD was characterized by more abnormal FBI scores and poor word fluency, in the presence of relatively normal overall cognitive status (MMSE) and/or delayed recall.
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Amyotroph Lateral Sc · Aug 2007
A role for functional classification in the early identification of prognostic factors in ALS.
The aim of this study was to determine if the interval between onset of symptoms to initial electrodiagnostic studies indicates disease progression in amyotrophic lateral sclerosis (ALS). Fifty consecutive patients referred to our neurophysiology laboratory with clinical evidence of ALS were divided into two groups by outcome scores on the ALS Functional Rating Scale (ALSFRS) using 26 as a cut-off. Our results, which showed a median of four months (range 2-24 months) duration to initial electrodiagnostics for Group I (ALSFRS scores below 26) versus 10 months (range 1-24 months) for Group II (p = 0.02), suggest this measure is a marker of disease progression in ALS.
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Amyotroph Lateral Sc · Apr 2007
Validity of the Dutch version of the Amyotrophic Lateral Sclerosis Assessment Questionnaire, ALSAQ-40, ALSAQ-5.
The objective was to validate the Dutch translation of the 40-item and 5-item Amyotrophic Lateral Sclerosis Assessment Questionnaire (ALSAQ-40, ALSAQ-5). Eighty-one ALS patients participated in this cross-sectional study. The measures used were the ALSAQ-40, the ALSAQ-5, MOS Short-Form-36 (SF-36) and Revised ALS Functional Rating Scale (ALSFRS-R). ⋯ The ALSAQ-5 and ALSAQ-40 showed comparable correlations with the subscales of the SF-36 and the ALSFRS-R, except for Activities Daily Living and Independence (ADL). All other results of the Dutch ALSAQ-40 and ALSAQ-5 were comparable to those of the original UK questionnaires. It is concluded that the psychometric properties of the Dutch version of the ALSAQ-40 and the ALSAQ-5 are good and similar to those of the original English version.
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Amyotroph Lateral Sc · Feb 2007
Comparative StudyDetecting neurobehavioral changes in amyotrophic lateral sclerosis.
Cognitive impairment in patients with amyotrophic lateral sclerosis (ALS) is well documented, but behavioral abnormalities are not well defined. The Frontal Systems Behavior Scale (FrSBe) was used to assess changes in apathy, disinhibition, and executive dysfunction in 45 patients with ALS. ⋯ Apathy and verbal fluency were correlated, lending further support to the hypothesis that an underlying continuum exists between ALS and frontotemporal dementia (FTD), which is characterized predominantly by behavioral disturbance. The FrsBe is useful for detecting behavioral change and abnormalities in patients with ALS.