Mitoxantrone doesn't work for PPMS

[SuzE-Q]

Darn. :(

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J Neuroimmunol.
2014 Nov 20.

Lack of efficacy of mitoxantrone in primary progressive Multiple Sclerosis irrespective of pharmacogenetic factors: A multi-center, retrospective analysis.

Grey Née Cotte S1, Salmen Née Stroet A1, von Ahsen N2, Starck M3, Winkelmann A4, Zettl UK4, Comabella M5, Montalban X5, Zipp F6, Fleischer V6, Kruse N7, Gold R1, Chan A8.

Abstract
BACKGROUND:
Mitoxantrone is used on an off-label basis in primary progressive MS (PPMS). ABC-transporter-genotypes are associated with therapeutic response in relapsing/secondary progressive MS (RP/SPMS).

OBJECTIVE:
To evaluate potential pharmacogenetic response markers for mitoxantrone in PPMS.

METHODS:
41 mitoxantrone-treated PPMS-patients, 155 mitoxantrone-treated RP/SPMS-patients and 43 PPMS-controls were retrospectively assessed for clinical therapy-response and in correlation with four single-nucleotide-polymorphisms in ABCB1- and ABCG2-genes.

RESULTS: 53.7% PPMS-patients were mitoxantrone-responders, in comparison to 78.1% of RP/SPMS-patients (p=0.039). There was no association between genotype and treatment response.

CONCLUSION:
Our data discourages the use of mitoxantrone in PPMS regardless of pharmacogenetic response markers previously described in RP/SPMS.

Copyright © 2014 Elsevier B.V. All rights reserved.

[SuzE-Q]

...why do you think they are saying that PPMSer's shouldn't take it when they consider over 1/2 of them to have been "responders", regardless of genotype?!??

[agate]

...why do you think they are saying that PPMSer's shouldn't take it when they consider over 1/2 of them to have been "responders", regardless of genotype?!??

I can hazard a couple of guesses. The authors seem to be saying that a 53.7% response among PPMS patients isn't good enough to warrant use of a drug like mitoxantrone, which is pretty risky in terms of potential serious side effects. I think they're saying that that 53.7% response isn't nearly as good as the 78.1% response seen in those progressive MS patients who were still having relapses.

They're differentiating sharply between the PPMS people and the RPMS people.

And this periodical isn't on the list of peer-reviewed scientific journals that the AAAS puts out:

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This is one guideline that can be used to sort out the wheat from the chaff when it comes to scientific articles, I think. There are so MANY scientific periodicals that it's tricky to figure out which articles are trustworthy.

Sometimes an abstract will make it clear whether the authors were funded by pharmaceutical companies but often they don't. The periodicals that aren't peer-reviewed tend to be less trustworthy than the ones that take part in the peer-review process.

But there are different kinds of peer-review. Some journals claim they are peer-reviewed but they're not on the AAAS list.

Unless you have far more scientific training than I do, you have to play it by ear. This is a small study coming from a source that's not on the AAAS list, and so maybe it doesn't get taken very seriously in the scientific community.

I'm no scientist though. This is sheer guesswork on my part.

[SuzE-Q]

Thanks, Joan! Great info to look for in figuring out how much weight to give to a study result! All studies are NOT created equal, that's for sure!

[Lazarus]

Novantrone was my favorite chemo. I was strong and energetic. There is risk associated and even though it has been years since I used up the FDA allowed lifetime amount of it, I still have heart tests periodically to make sure it is not causing damage.