Presented at the 29th International Symposium on ALS/MND in Glasgow, Scotland on December 8, 2018.
Background: We have previously reported on the development of models for predicting ALSFRS-R, vital capacity and survival. Here we report on the development of regression models for the ALSFRS-R subscores (including bulbar function, fine and gross motor function & respiratory function) and percent expected vital capacity. In addition, we also report on the development of time-to-event models, that predict loss of speech, wheelchair use, use of feeding tube and time to 50% expected vital capacity. These time-to-event models can potentially be used to predict the ALS King’s Stage.
Objectives: To validate all twelve models using the VITALITY-ALS placebo arm data set.
Conclusions: Validation of a dozen models is discussed along with the useful ranges for each model. Regression models for ALSFRS-R, sub-scores, including bulbar and gross motor, and % expected vital capacity and all listed time-to-event models have been validated with external, contemporary datasets for 6 month and one-year timepoints.
Authors: Danielle Beaulieu, Jonavelle Cuerdo, Albert A. Taylor, Andrew Conklin, Amy Bian, Lisa Meng, Andrew A. Wolff, David L. Ennist