title: Sample size estimations for MRI-monitored trials of MS comparing new vs standard treatments.
abstract: 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.
authors.affiliation: Neuroimaging Research Unit, Department of Neuroscience, Scientific Institute and University Ospedale San Raffaele, Milan, Italy.
authors.forename: M P
authors.initials: MP
authors.lastname: Sormani
authors.forename: M
authors.initials: M
authors.lastname: Rovaris
authors.forename: F
authors.initials: F
authors.lastname: Bagnato
authors.forename: P
authors.initials: P
authors.lastname: Molyneux
authors.forename: P
authors.initials: P
authors.lastname: Bruzzi
authors.forename: C
authors.initials: C
authors.lastname: Pozzilli
authors.forename: D H
authors.initials: DH
authors.lastname: Miller
authors.forename: G
authors.initials: G
authors.lastname: Comi
authors.forename: M
authors.initials: M
authors.lastname: Filippi
mesh_terms: 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
chemical_list: D000276:Adjuvants, Immunologic; D010455:Peptides; D000068576:Interferon beta-1b; D000068717:Glatiramer Acetate; D016899:Interferon-beta; D000068556:Interferon beta-1a