This paper tells about the prognostics in MSA by listing the...

This paper tells about the prognostics in MSA by listing the clinical features of poor outcomes in 210 MSA patients. Now, as we know, ATH also has another biometric phase 2 study going on. It has no control or placebo group, but only the 15 cases of MSA patients. With this info in the article below it is perhaps possible to find a comparison group (even in this 210 MSA population?? or just a theoretical comparison group) to these 15 MSA patients in this ATH434 treatment group. Prof Claassen was not among the authors of this paper but I would think that he could get the needed extra info from these authors if needed. The ATH434 is a biometric study, but this kind of possible comparison could add value to this study in demonstrating that ATH434 works ( if it really works as we believe today).



"Development and Validation of a Prognostic Model to Predict Overall Survival in Multiple System Atrophy" by Eschlboeck et al

Abstract

Background: Multiple system atrophy (MSA) is a devastating disease characterized by a variable combination of motor and autonomic symptoms. Previous studies identified numerous clinical factors to be associated with shorter survival.Objective: To enable personalized patient counseling, we aimed at developing a risk model of survival based on baseline clinical symptoms.Methods: MSA patients referred to the Movement Disorders Unit in Innsbruck, Austria, between 1999 and 2016 were retrospectively analyzed. Kaplan-Meier curves and multivariate Cox regression analysis with least absolute shrinkage and selection operator penalty for variable selection were performed to identify prognostic factors. A nomogram was developed to estimate the 7 years overall survival probability. The performance of the predictive model was validated and calibrated internally using bootstrap resampling and externally using data from the prospective European MSA Study Group Natural History Study.Results: A total of 210 MSA patients were included in this analysis, of which 124 patients died. The median survival was 7 years. The following clinical variables were found to significantly affect overall survival and were included in the nomogram: age at symptom onset, falls within 3 years of onset, early autonomic failure including orthostatic hypotension and urogenital failure, and lacking levodopa response. The time-dependent area under curve for internal and external validation was >0.7 within the first 7 years of the disease course. The model was well calibrated showing good overlap between predicted and actual survival probability at 7 years.Conclusion: The nomogram is a simple tool to predict survival on an individual basis and may help to improve counseling and treatment of MSA patients.