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Old Fri Aug 22, 2014, 10:53 PM
Hopeful Hopeful is offline
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Join Date: Jan 2009
Location: California, USA
Posts: 766
More random thoughts...

Hi Alcof,

Currently, I see that Eltrombopag is only used in NIH clinical trials without IST and as a first line treatment for cases of moderate AA. Unfortunately, there are no results yet as to its effectiveness without IST. Ethically this makes sense to me because ATG/CyA is the gold standard for AA and has a 60-80% success rate. Moderate AA typically gets a watch-and-wait approach in the US. So, it seems like a less risky approach to try this experimental drug without IST on patients who aren't in crisis mode yet.

I still believe that if there is an immune component to a person's AA, that has to be addressed as well. However, there are probably some people who's AA was caused by something else like a toxin or virus that has since left their body but that has left their marrow too fragile to recover. So perhaps, Eltrombopag alone will help these people. It will be interesting to see the results from this trial! Who knows..perhaps Eltrombopag alone can alter the course of a person's immune response. It works for ITP, another auto immune disease.

If you do get a hold of Eltrombopag, you should write/call NIH on their suggested dosage/duration.

On a side note...
That link that I included previously shows that Danazol has a 47% success rate as a first line of treatment for AA.
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55 yo female, dx 9/08, AA/hypo-MDS, subclinical PNH, ATG/CsA 12/08, partial response. small trisomy 6 clone, low-dose cyclosporine dependent
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