Abstract Details

Title Post-onset ALSFRS-R Slope (POS) as a Predictor of Disease Progression in Patients with Amyotrophic Lateral Sclerosis (ALS)

Topic Neuromuscular and Clinical Neurophysiology (EMG)

Presentation(s) P12 - Poster Session 12 (11:45 AM-12:45 PM)

Poster/Presentation
Number 11-001

Objective To validate whether POS predicts disease progression.

Background The ALSFRS-R is a validated questionnaire-based functional rating scale for determining ALS disease progression. Change in ALSFRS-R slope has also been used as a qualifying parameter for trial entry and as an efficacy endpoint. Recently, change in ALSFRS-R between symptom onset and diagnosis (post-onset slope, POS) has been discussed as a predictor of progression and survival.

Design/Methods A retrospective analysis of real-world data from the ALS/MND Natural History Consortium was performed on patients with an initial ALSFRS-R score documented at diagnosis (or within 14 months of disease onset) and the 12-month follow-up interval using a 3-month threshold. POS was defined as [(ALSFRS-R at diagnosis - 48) divided by (duration from onset to diagnosis)]. Interval slope was defined as [(ALSFRS-R at 12 months - ALSFRS-R at diagnosis) divided by (duration from diagnosis to interval visit)].

Results Analysis of 210 subjects who had available 12-month follow-up data showed an average POS of -0.972 +/- 0.818 and interval slope of -0.890 +/- 0.649 points/month. A linear regression model was then performed, demonstrating that the POS is a statistically significant predictor of 12-month slope when controlling for sex, site of onset, and baseline ALSFRS-R and FVC scores (t(176) = 2.542, p = 0.0119). For every point/month decrease in POS, the interval slope at 12-month follow-up decreases by 0.206.

Conclusions

The ALSFRS-R is an accessible and reproducible scale reflecting function in ALS. The positive relationship between POS and 12-month interval slope indicates that individuals who experience a faster decline in ALSFRS-R between onset and diagnosis are likely to show a greater decline in their score at 12-months follow up. Therefore, POS can be used to predict disease progression, guide prognostic discussions and planning, and act as a lead-in period for therapeutic interventions.

Authors/Disclosures
Larisa Ibric, Medical student PRESENTER	Ms. Ibric has nothing to disclose.
John Furey (Temple University Hospital)	Mr. Furey has nothing to disclose.
Alexander Sherman (Massachusetts General Hospital)	The institution of Mr. Sherman has received research support from The ALS Association. The institution of Mr. Sherman has received research support from NIH. The institution of Mr. Sherman has received research support from FDA. The institution of Mr. Sherman has received research support from Biogen. The institution of Mr. Sherman has received research support from Amylyx Pharmaceuticals. The institution of Mr. Sherman has received research support from Mitsubishi-Tanabe Pharma America. Mr. Sherman has a non-compensated relationship as a Member, Board of Directors with ALD Connect that is relevant to AAN interests or activities.
Terry D. Heiman-Patterson, MD (Temple University Lewis Katz School of Medicine)	Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for MTPA. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Amylyx. Dr. Heiman-Patterson has received personal compensation in the range of $0-$499 for serving as a Consultant for novartis. Dr. Heiman-Patterson has received personal compensation in the range of $500-$4,999 for serving as a Consultant for biogen. The institution of Dr. Heiman-Patterson has received research support from MTPA. The institution of Dr. Heiman-Patterson has received research support from State of Pennsylvania . The institution of Dr. Heiman-Patterson has received research support from ALS Association. The institution of Dr. Heiman-Patterson has received research support from ALS United. The institution of Dr. Heiman-Patterson has received research support from ALS Hope Foundation.

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