Presented at the 27th International Symposium on Amyotrophic Lateral Sclerosis and Motor Neurone Disease, Dublin Ireland, December 2016.
- To develop a meaningful operational definition of “ALS resistance” which captures patients with unexpectedly long plateaus or large and sustained reversals
- To determine whether ALS resistance is regional (occurring across multiple ALSFRS-R questions related to the same body part) or random (and thus more likely to be due to noise).
- To look for differences in demographics, medications and laboratory values between resistant patients and those with more typical progression.
Conclusions: We compared resistant and more typically progressive patients in 40 demographic categories, 333 medications, 114 adverse events and 111 labs using t-tests. After adjusting for multiple comparisons, we found no significant differences. There may be a genetic component, as seen in HIV elite controllers. Our definition of ALS resistance (2 ALSFRS-R points at least 6 months apart that are 2 standard deviations better than predicted) appears clinically meaningful and should allow prospective identification of a reasonably large cohort of resistant patients for further studies.
Authors: Richard S. Bedlack, David Ennist, Albert Taylor, Mike KeymerFinal-P197-Duke-Origent-Resistance-2016-ALS-MND-Symp