PRION-1 scales analysis supports use of functional outcome measures in prion disease.

ABSTRACT:

Objectives

Human prion diseases are heterogeneous but invariably fatal neurodegenerative disorders with no known effective therapy. PRION-1, the largest clinical trial in prion disease to date, showed no effect of the potential therapeutic quinacrine on survival. Although there are several limitations to the usefulness of survival as an outcome measure, there have been no comprehensive studies of alternatives.

Methods

To address this we did comparative analyses of neurocognitive, psychiatric, global, clinician-rated, and functional scales, focusing on validity, variability, and impact on statistical power over 77 person-years follow-up in 101 symptomatic patients in PRION-1.

Results

Quinacrine had no demonstrable benefit on any of the 8 scales (p > 0.4). All scales ha

SUBMITTER: Mead S 

PROVIDER: S-EPMC3208955 | biostudies-literature | 2011 Nov

REPOSITORIES: biostudies-literature