Amyotrophic lateral sclerosis & frontotemporal degeneration
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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.
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Amyotroph Lateral Scler Frontotemporal Degener · May 2019
Meta AnalysisPlasma creatinine and amyotrophic lateral sclerosis prognosis: a systematic review and meta-analysis.
Background: Plasma creatinine has been described as a prognostic biomarker for Amyotrophic Lateral Sclerosis (ALS), but with conflicting results in the literature. We performed a systematic review followed by a meta-analysis to address this question. Methods: We performed a systematic review of Pubmed, Embase and Cochrane databases and retrieved 14 distinct cohorts (19 studies) reporting results regarding the relationship between plasma creatinine and a clinical marker for ALS progression, notably ALSFRS (ALS Functional Rating Scale) and survival. ⋯ The overall quality of the studies was low mainly due to potential attrition bias, and several studies did not report analyzable results raising concern regarding a potential reporting bias. Conclusions: Plasma creatinine seems to be a promising prognostic biomarker for ALS. However, new studies with sound methodology and standardized criteria for the evaluation of ALS progression should be conducted to validate plasma creatinine as a clinical biomarker for ALS prognosis.
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Amyotroph Lateral Scler Frontotemporal Degener · Nov 2017
Review Meta AnalysisNocebo in motor neuron disease: systematic review and meta-analysis of placebo-controlled clinical trials.
Nocebo is very prevalent among neurological disorders, resulting in low adherence and treatment outcome. We sought to examine the AEs following placebo administration in placebo-controlled randomised clinical trials (RCTs) for motor neuron disease (MND). ⋯ Our study indicates significant nocebo in trials for MND treatment, adversely affecting adherence and efficacy of current treatments in clinical practice, with additional implications for trial design.
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Amyotroph Lateral Scler Frontotemporal Degener · May 2013
Review Meta AnalysisStatins and amyotrophic lateral sclerosis: a systematic review and meta-analysis.
To evaluate the association between statins and the incidence and progression of amyotrophic lateral sclerosis (ALS). ⋯ No definite association between statin use and ALS incidence and progression has been found. Existing results are currently inconclusive to make scientifically supported conclusions. Further prospective cohort studies are still needed.