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What are the Most Effective Drug Treatments?

Published on April 2, 2010

Sean Pittock, MD:

Currently, we need much better drug studies, even if you just take, for example, the currently used drugs.

Sean Pittock, MD:

I’ll just give you very briefly, we have azathioprine, which kind of was the mainstay of treatment for NMO. And the use of that drug is based on the published seven patients.

Sean Pittock, MD:

We have a recent paper on mycophenolate mofetil, or CellCept, which is a paper that we did at Mayo, which is a retrospective study of 24 patients. Before that, there was only one case report of CellCept. And we found that that drug also appears to significantly reduce relapse rate.

Sean Pittock, MD:

And then the third drug is rituximab. There was an open label study of rituximab published by Bruce Cree, which only included I think around 10 patients. And there has been a retrospective study of rituximab. I think the numbers were around the thirties.

Sean Pittock, MD:

So, the data we have is really, really limited. We have very, very small kind of case series studies of these drugs in NMO. So there’s a lot of work to be done in terms of, what are the best treatments?

Sean Pittock, MD:

For example, it’s not really clear whether or not you should just use Imuran or CellCept alone, or should you use them with prednisone. In our experience, we found that prednisone is often required for a prolonged period of time in association or in addition to azathioprine and CellCept.

Sean Pittock, MD:

But no big studies have been done, so all of this data and all the reasoning for our use of these drugs is really based on small numbers of patients.

Sean Pittock, MD:

But I think the animal models will be extremely important in systematically looking at different drugs and potentially moving forward in terms of trying to identify drugs that will really stop attacks, rather than just reduce the chances of having a heart attack.


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