Sample size estimations for MRI-monitored trials of MS comparing new vs standard treatments.
The authors estimated the sample sizes needed for exploratory trials of MS assessing the efficacy of new treatments in reducing the number of new enhancing lesions vs those of interferon-beta or glatiramer acetate. The sample sizes per arm ranged from 868 (effect: 20%) to 94 (effect: 50%) for patients with relapsing-remitting MS and from 2,484 (effect: 20%) to 361 (effect: 50%) for patients with secondary progressive MS. In MS, exploratory trials of new vs available therapies require large numbers of patients, even when MR end-points are used.
Sormani
M P
MP
Neuroimaging Research Unit, Department of Neuroscience, Scientific Institute and University Ospedale San Raffaele, Milan, Italy.
Rovaris
M
M
Bagnato
F
F
Molyneux
P
P
Bruzzi
P
P
Pozzilli
C
C
Miller
D H
DH
Comi
G
G
Filippi
M
M
D000276:Adjuvants, Immunologic / Q000627:therapeutic use*; D000328:Adult; D001921:Brain / Q000187:drug effects / Q000473:pathology; D017322:Clinical Trials, Phase II as Topic / Q000706:statistics & numerical data; D017326:Clinical Trials, Phase III as Topic / Q000706:statistics & numerical data; D004305:Dose-Response Relationship, Drug; D005260:Female; D000068717:Glatiramer Acetate; D006801:Humans; D000068556:Interferon beta-1a; D000068576:Interferon beta-1b; D016899:Interferon-beta / Q000627:therapeutic use*; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000175:diagnosis / Q000188:drug therapy*; D020529:Multiple Sclerosis, Relapsing-Remitting / Q000175:diagnosis / Q000188:drug therapy*; D009460:Neurologic Examination / Q000187:drug effects; D010455:Peptides / Q000627:therapeutic use*; D016032:Randomized Controlled Trials as Topic / Q000706:statistics & numerical data; D015203:Reproducibility of Results; D013557:Switzerland; D016896:Treatment Outcome
D000276:Adjuvants, Immunologic; D010455:Peptides; D000068576:Interferon beta-1b; D000068717:Glatiramer Acetate; D016899:Interferon-beta; D000068556:Interferon beta-1a