Hi Relentless,
I attended a Webcast this past summer on non-transplant AA treatment options given by Dr. Townsley at NIH. She discussed the Promacta trials, saying she couldn't go into details as the trial wasn't complete, but that to date the results have been excellent, and that she's hopeful Promacta will revolutionize non-transplant AA treatment.
The current trials are ATG+CSA+Promacta for new SAA patients, and she said another trial is underway to supply the same for relapsed SAA patients. There was a slide on possible benefits of Promacta, and I believe one of those was even reduced rates of progression to MDS/AML.
This is all sounds very promising to folks battling AA. But when I mentioned all the above to my doctor, he basically said to try not to get my hopes up, that medical history is filled with pronouncements of 'breakthrough' new treatments, and they often do not pan out. He suggested I reserve my enthusiasm until the trial results come out (I think that will be in 2014 or 2015).
That being said, ATG+CSA was a breakthrough, one that from all indications has saved my life, so certainly sometimes new treatments DO pan out! I'm doing pretty well, and hoping I can finish tapering my CSA, and stop taking any drugs, as opposed to having to take more of them. But if I ever did relapse, I would lean strongly towards getting into the ATG+CSA+Promacta trial at NIH, if they would take me.
Below is the recruitment link for the original trial. I had not found one for the new study for relapsed SAA patients, but perhaps if you contacted Olga Rios (contacts details on bottom of page), she could provide you information on that:
http://clinicalstudies.info.nih.gov/...12-H-0150.html
...I've read in your other threads all the difficult treatment decisions your son is facing. It seems there are often no clear decisions what is the best route, and as you have said, different doctors have different opinions. This is just an anecdote, but one AA patient in my support group did not respond to ATG+CSA, but she is doing very well after her 9/10 MUD transplant. She is about 6 months out, and has some mild GvH, but says she feels much better than she did pre-transplant.
Also in favor of an MUD transplant, is that there is solid data indicating outcomes have been significantly improving over time. Somewhere here on Marrowforums earlier this year was a thread on that, showing very promising outcome improvements in the latest data.