Mycophenolate vs. Interferon-beta: an underpowered study

Mycophenolate is an anti-rejection agent widely used in transplantation. This was a randomised, 6-monthly, study to compare the effectiveness of mycophenolate to interferon beta in 35 untreated patients with RRMS. Not surprisingly there was no difference between mycophenolate and interferon beta therapy treatment groups. The mycophenolate group showed a trend toward a lower accumulation of lesions on MRI, but this was not significant. This study was simply too small to draw any conclusions (underpowered). It should have been appropriately powered to give a definitive answer; the worrying consequence of this study is will anybody be brave enough now to do a definitive trial? Has this study killed a potentially promising oral agent?

Frohman et al. Ther Adv Neurol Disord. 2010 Jan;3(1):15-28.

Is it ethical to do under-powered studies? NO!

Should people with MS who volunteer to participate in treatment trials expect the studies to be appropriately powered? YES!

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