title: ALSFRS and appel ALS scores: discordance with disease progression.
abstract: Progression of disease and effectiveness of therapy in patients with amyotrophic lateral sclerosis (ALS) are determined by both questionnaire- and examination-based measures. To determine whether both types of measurement tools are equally predictive at all stages of disease, we compared questionnaire-based ALS Functional Rating Scale (ALSFRS) scores to the examination-based Appel ALS (AALS) scores at different stages of disease. Same-day scores were obtained during 174 visits in 62 patients with definite or probable ALS. Using normalized scores, correlation between the scales and predictability were best in mildly affected patients. Predictions of ALSFRS based on AALS scores were less than half as precise in the later stages of disease. Both scales showed significant change with disease progression, but ALSFRS consistently underestimated disease severity defined by AALS (P < 0.001). Questionnaire-based measurements should be compared against objective scales at all stages of disease severity before they are accepted as primary endpoint measures.
authors.affiliation: Department of Neurology and the MDA/ALS Center, Mount Sinai School of Medicine, One Gustave L. Levy Place, Box 1052, New York, New York 10029, USA.
authors.forename: Andrei
authors.initials: A
authors.lastname: Voustianiouk
authors.forename: Gregory
authors.initials: G
authors.lastname: Seidel
authors.forename: Janki
authors.initials: J
authors.lastname: Panchal
authors.forename: Mark
authors.initials: M
authors.lastname: Sivak
authors.forename: Adam
authors.initials: A
authors.lastname: Czaplinski
authors.forename: Albert
authors.initials: A
authors.lastname: Yen
authors.forename: Stanley H
authors.initials: SH
authors.lastname: Appel
authors.forename: Dale J
authors.initials: DJ
authors.lastname: Lange
mesh_terms: D000328:Adult; D000368:Aged; D000369:Aged, 80 and over; D000690:Amyotrophic Lateral Sclerosis / Q000503:physiopathology*; D000704:Analysis of Variance; D018450:Disease Progression; D005260:Female; D006801:Humans; D016014:Linear Models; D008297:Male; D008875:Middle Aged; D012720:Severity of Illness Index; D018434:Sickness Impact Profile; D011795:Surveys and Questionnaires