Minimum clinically important difference for drug effectiveness in an area of patient-oriented therapeutic goals in amyotrophic lateral sclerosis

Amyotrophic Lateral Sclerosis and Frontotemporal Degeneration, Journal Year: 2025, Volume and Issue: unknown, P. 1 - 10

Published: March 11, 2025

Objective: In this review, we will examine the more common endpoints incorporated in randomized controlled trials (RCTs) and their strength of evidence, focusing on definition what constitutes a clinically meaningful change. We also reflect perspective patients families regarding design RCTs amyotrophic lateral sclerosis (ALS). Methods: Authors performed scoping review literature around clinical meaningfulness ALS field minimum important difference to deem treatment effective. Results: The use survival as an RCT endpoint, well functional rating scale-revised slope, has been criticized, relevance for remains debated. Biomarkers are promising alternatives surrogate endpoints, but currently, only cerebrospinal fluid plasma neurofilaments have emerged reliable sensitive biomarkers disease progression. Incorporating patients' preferences priorities care when treatments selected is minimize burden limit potential harms overtreatment. Patients' interest acceptance new therapy determined by its impact quality life. Discussion conclusion: While scientifically sound must be conducted, balanced with patient expectations limiting trial burden, duration placebo usage. An approach uniting these diverging needs inclusion people organizations advise execution trials, facilitating focused while retaining high scientific rigor.

Language: Английский

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The spectrum of behavioral disorders in amyotrophic lateral sclerosis: current view

Journal of Neural Transmission, Journal Year: 2024, Volume and Issue: unknown

Published: Oct. 14, 2024

Language: Английский

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