Document Detail

The Combined Assessment of Function and Survival (CAFS): A new endpoint for ALS clinical trials.
MedLine Citation:
PMID:  23323713     Owner:  NLM     Status:  Publisher    
Our objective was to describe a new endpoint for amyotrophic lateral sclerosis (ALS), the Combined Assessment of Function and Survival (CAFS). CAFS ranks patients' clinical outcomes based on survival time and change in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Each patient's outcome is compared to every other patient's outcome, assigned a score, and the summed scores are ranked. The mean rank score for each treatment group can then be calculated. A higher mean CAFS score indicates a better group outcome. Historically, ALS clinical trials have assessed survival and function as independent endpoints. Combined endpoints have been used in other diseases to decrease the confounding effect of mortality on analysis of functional outcomes. We explored the application of a similar approach in ALS, the CAFS endpoint, which was used as a pre-specified secondary analysis in a phase II study of dexpramipexole. Those results and some hypothetical examples based on modeling exercises are presented here. CAFS is the primary endpoint of a dexpramipexole phase III study in ALS. In conclusion, the CAFS is a robust statistical tool for ALS clinical trials and appropriately accounts for and weights mortality in the analysis of function.
James D Berry; Robert Miller; Dan H Moore; Merit E Cudkowicz; Leonard H Van Den Berg; Douglas A Kerr; Yingwen Dong; Evan W Ingersoll; Donald Archibald
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Publication Detail:
Type:  JOURNAL ARTICLE     Date:  2013-1-17
Journal Detail:
Title:  Amyotrophic lateral sclerosis and frontotemporal degeneration     Volume:  -     ISSN:  2167-9223     ISO Abbreviation:  Amyotroph Lateral Scler Frontotemporal Degener     Publication Date:  2013 Jan 
Date Detail:
Created Date:  2013-1-17     Completed Date:  -     Revised Date:  -    
Medline Journal Info:
Nlm Unique ID:  101587185     Medline TA:  Amyotroph Lateral Scler Frontotemporal Degener     Country:  -    
Other Details:
Languages:  ENG     Pagination:  -     Citation Subset:  -    
Massachusetts General Hospital, Neurological Clinical Research Institute , Boston, Massachusetts , USA.
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