How can we hope to predict progression accurately

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Unformatted text preview: $50K ALS Predic-on Prize, 7/2012 ­10/2012 •  Goal: Predict rate of disease progression in ALS pa-ents   Dis-nguish the slow progressors from the fast Ques;ons •  What do we mean by disease progression? •  Why is progression predic-on valuable? •  How can we hope to predict progression accurately? Predic-on Progression: What?   ALS Func;onal Ra;ng Scale (ALSFRS) •  Measure of pa-ent func-onality, ranging from 0 ­40 •  Based on 10 ques-ons regarding everyday ac-vity:   Speaking, respira-on, climbing stairs, dressing, wri-ng, …   Ac-vity score of 4 is normal, 0 is complete inability •  Slow progressor loses 0 ­3 points per year •  Fast progressor can lose 20 Speech Respira. Saliv. Visit 0 Month 1 Month 2 Month 3 Swall. Handwr Cuing Dress. Turn. Climb. Walk. Total 3 4 3 3 4 4 3 4 4 4 36 3 4 3 3 4 4 3 4 4 4 36 3 4 2 3 4 4 3 4 4 4 35 3 4 2 3 4 4 3 4 4 3 34 State of Progression Predic-on Clinical Presenta;on:   A 69 year old Caucasian female 19 months aker diagnosis   Bulbar onset (degenera-on in muscles controlling speaking/swallowing)   Weight stable and normal Saliv. Swall. Handwr Cuing Dress. Turn. Climb. Walk. Total 3 4 3 3 4 4 3 4 4 4 36 3 4 3 3 4 4 3 4 4 4 36 3 Visit 0 Speech Respira. 4 2 3 4 4 3 4 4 4 35 3 4 2 3 4 4 3 4 4 3 34 Month 1 Month 2 Month 3 ALSFRS Scores State of Progression Predic-on Clinical...
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