An exploratory, randomized, controlled trial
S.M. Leary, MRCP, D.H. Miller, FRCP, V.L. Stevenson, MRCP, P.A. Brex, MRCP, D.T. Chard, MRCP and A.J. Thompson, FRCP
From the NMR Research Unit, Institute of Neurology, University College London, UK.
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.
Fifty subjects were randomized to weekly IM interferon ß-1a 30 µg, 60 µg, 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.
The 30-µg dose of interferon ß-1a was well tolerated, but the 60-µg dose caused severe flulike reactions and raised liver enzymes. No treatment effect was seen on the primary endpoint. Subjects on interferon ß-1a 30 µg had a lower rate of accumulation of T2 lesion load than controls (p = 0.025); subjects on 60 µg had a greater rate of ventricular enlargement than controls (p = 0.025).
This study has demonstrated that interferon ß-1a 30 µg was well tolerated, identified useful outcome measures, but showed no efficacy on the primary outcome measure or on most of the secondary outcome measures.
© 2003 American Academy of Neurology