Interferon beta-1a in primary progressive MS: an exploratory, randomized, controlled trial.
BACKGROUND
Patients with primary progressive MS have atypical clinical and MRI characteristics and have been excluded from most therapeutic trials. The authors report a randomized, controlled trial restricted to primary progressive MS.


METHODS
Fifty subjects were randomized to weekly IM interferon beta-1a 30 microg, 60 microg, or placebo for 2 years. The primary endpoint was time to sustained progression in disability. Secondary outcomes included the timed 10-meter walk, nine-hole peg test, and on MRI, T2 and T1 brain lesion loads and brain and spinal cord atrophy.


RESULTS
The 30- microg dose of interferon beta-1a was well tolerated, but the 60- microg dose caused severe flulike reactions and raised liver enzymes. No treatment effect was seen on the primary endpoint. Subjects on interferon beta-1a 30 microg had a lower rate of accumulation of T2 lesion load than controls (p = 0.025); subjects on 60 microg had a greater rate of ventricular enlargement than controls (p = 0.025).


CONCLUSIONS
This study has demonstrated that interferon beta-1a 30 microg was well tolerated, identified useful outcome measures, but showed no efficacy on the primary outcome measure or on most of the secondary outcome measures.
Leary
S M
SM
NMR Research Unit, Institute of Neurology, University College London, UK.
Miller
D H
DH
Stevenson
V L
VL
Brex
P A
PA
Chard
D T
DT
Thompson
A J
AJ
D000328:Adult; D001921:Brain / Q000187:drug effects / Q000473:pathology; D004185:Disability Evaluation; D018450:Disease Progression; D004305:Dose-Response Relationship, Drug; D004334:Drug Administration Schedule; D005260:Female; D006801:Humans; D007273:Injections, Intramuscular; D000068556:Interferon beta-1a; D016899:Interferon-beta / Q000009:adverse effects / Q000627:therapeutic use*; D008099:Liver / Q000187:drug effects / Q000201:enzymology; D008279:Magnetic Resonance Imaging; D008297:Male; D008875:Middle Aged; D020528:Multiple Sclerosis, Chronic Progressive / Q000175:diagnosis / Q000188:drug therapy* / Q000276:immunology; D010349:Patient Compliance; D010865:Pilot Projects; D018401:Sample Size; D013116:Spinal Cord / Q000187:drug effects / Q000473:pathology; D016019:Survival Analysis; D016896:Treatment Outcome
D016899:Interferon-beta; D000068556:Interferon beta-1a