Abstract Details

Title Validation of an Amyotrophic Lateral Sclerosis Staging System Using Clinical Trial Data

Topic Anterior Horn

Presentation(s) S36 - (-)

Poster/Presentation
Number 005

Objective

Background Clinical staging is important both for guiding patient care, and for the design of clinical trials, because the proper measure of disease progression allows patient care to be planned and health economics analyses of investigational medicines. We have previously proposed a staging system for amyotrophic lateral sclerosis (ALS) in which disease states change in a fixed and logical order (Roche et al. 2012), consisting of Stage 1, onset (first region involved); Stage 2, second region involved; Stage 3, third region involved; Stage 4, need for intervention such as gastrostomy or non-invasive ventilation; Stage 5, death. The stages lend themselves easily to a standardized time scale that accounts for variability in underlying disease progression.

Design/Methods Data from the LiCALS clinical trial was analyzed. ALS-FRS, EQ-5D ordinal questions, EQ-5D visual analogue scale, HADS anxiety and HADS depression scores were correlated with disease progression measured traditionally by time elapsed, and using standardized timings through the ALS clinical stage.

Results Stages occurred in the same order as in our original paper and at very similar standardized times. ALS-FRS score changed by 9 points using time elapsed, but by 20 points using clinical stage. EQ-5D and HADS scores changed by up to six times more using staging than time elapsed.

Conclusions We have validated an ALS clinical staging system and shown that compared with time elapsed, this improved correlation between measurements of disease progression and every functional score studied.

Authors/Disclosures
Ashley Jones PRESENTER	No disclosure on file
	No disclosure on file
	No disclosure on file
Martin R. Turner, MBBS, PhD, MA, FRCP (University of Oxford)	No disclosure on file
Jurgen Koehler	No disclosure on file
Nigel P. Leigh	No disclosure on file
	No disclosure on file

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