Abstract Details

Title Health utility declines with advancing Fine'til 9 stage of ALS

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P1 - Poster Session 1 (5:30 PM-6:30 PM)

Poster/Presentation
Number 12-019

Objective Determine health utility of stages of amyotrophic lateral sclerosis (ALS) defined by a novel staging approach called Fine'til 9 (FT9). FT9 assigns stage by the number of ALSFRS-R subscores equal to or less than 9 points (Thakore et al, 2018).

Background To estimate quality-adjusted life years in disease, and thereby cost-effectiveness of any treatment, valuation of stages of disease by a health utility index is necessary.

Design/Methods Multiple self-reported measures including ALSFRS-R and EQ-5D were obtained from ALS patients in waiting rooms using tablet devices and a software system developed in-house at Cleveland Clinic (Knowledge Program). Analyses included descriptive statistics and mixed effects linear models.

Results 1,724 EQ-5D indices (US valuation) and simultaneous FT9 stages were captured in 664 patients between 2010 and 2015. Counts of measures by stage were 148, 465, 497, 353 and 261 for stages 0, 1 2, 3 and 4 respectively. Median EQ-5D indices by stage were 0.83, 0.78, 0.69, 0.60 and 0.46 respectively. Among 219 bulbar onset patients, EQ-5D was higher for each FT9 stage than among non-bulbar onset patients. Adjusting for between-patient variability in EQ-5D intercept and slope, bulbar onset patients scored 0.13 higher on the average than non-bulbar onset patients in the same FT9 stage. EQ-5D declined by about 0.10 for increasing FT9 stage. Visual analog scale (VAS) measures were parallel to EQ-5D, with similar effects of bulbar onset (8.1 points higher than non-bulbar onset), and increasing FT9 stage (7 points lower per stage).

Conclusions Health utility declines in a step-wise fashion with advancing FT9 stage of ALS, in keeping other staging approaches (King's, MITOS). Bulbar impairment registers insufficiently on EQ-5D; a favorable effect of bulbar onset on subjective quality of life needs exploration. Measures reported herein may be useful for health economic studies of ALS in the US.

Authors/Disclosures
Nimish Thakore, MD (Cleveland Clinic) PRESENTER	No disclosure on file
Brittany Lapin	No disclosure on file
Erik P. Pioro, MD, DPhil, FAAN (University of British Columbia)	Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avanir Pharmaceutical, Inc.. Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx Pharmaceuticals. Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Argenx. Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MT Pharma America, Inc.. Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for NeuroTherapia, Inc.. Dr. Pioro has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MT Pharma America, Inc..

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