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  #1  
Old Wed Dec 18, 2013, 09:08 PM
Relentless Against SAA Relentless Against SAA is offline
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Promacta for SAA

Does anyone have info on Promacta being used to treat SAA or Moderate AA? I know they are doing a clinical trial with this med at NIH. Does anyone have any personal experience with this med?
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  #2  
Old Wed Dec 18, 2013, 09:42 PM
Sally C Sally C is offline
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Hi Relentless,
Please read my posts concerning my husband's miracle with this drug. He was the first MDS patient at NIH to use Promacta with unbelievable results. They extended the Promacta trial to include MDS patients after they had such great success with their AA patients.
Please let me know if I can give any further information about Promacta/NIH.
God Bless,
Sally
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  #3  
Old Thu Dec 19, 2013, 05:13 AM
Relentless Against SAA Relentless Against SAA is offline
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Thank you Sally.
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  #4  
Old Thu Dec 19, 2013, 11:05 AM
KMac KMac is offline
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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.
__________________
Kevin, male age 45; dx SAA 02/2012 - Hgb 5.8, platelets 14, ANC 200, 1% cellularity. Received ATG 03/2012. As of 03/2015, significant improvement - Hgb 15, platelets 158, ANC fluctuates around 1000, Lymphocytes 620. Tapering cyclosporine. BMB 20-30% cellularity.

Last edited by KMac : Thu Dec 19, 2013 at 11:23 AM.
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  #5  
Old Thu Dec 19, 2013, 11:24 AM
Relentless Against SAA Relentless Against SAA is offline
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Kevin,
Thank you for your reply. I needed to hear some words of encouragement.
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  #6  
Old Fri Dec 20, 2013, 02:23 PM
Sue H. Sue H. is offline
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Smile Eltrombopag for severe AA

My husband (56 years, diagnosed with severe AA April 2012, one ATG treatment May 2012, only limited response, Ciclosporin since may 2012) has been taking Eltrombopag (Promacta in US, Revolade in Europe where we live) for one year now with encouraging results. Once he was on the full dosage counts began to rise significantly and now he has been platelet transfusion free for one year and transfusion (red blood)free for 3 months with good white blood cells counts too. Hospital here in Switzerland are monitoring closely and informed us at the start of treatment that long term studies for safety of this drug are still not completed. In this game though, as others on this forum have said, you have to "pick your poison" so to speak, so we decided to take the chance and were lucky that our health insurance funded it. I know that NIH in US have just completed one study on use of this drug for AA - findings to be published in Journal of Blood next month apparently.
Wish you lots of luck!
This forum is a wonderful resource and has helped us endlessly. Many thanks and all the very best to all.
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  #7  
Old Fri Dec 20, 2013, 04:34 PM
Relentless Against SAA Relentless Against SAA is offline
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Quote:
Originally Posted by Sue H. View Post
Once he was on the full dosage counts began to rise significantly and now he has been platelet transfusion free for one year and transfusion (red blood)free for 3 months with good white blood cells counts too.
What was your husbands ANC before the Promacta and what are his levels now? How do they monitor for side effects?? Are they doing frequent bone marrow bx?
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  #8  
Old Fri Dec 20, 2013, 09:32 PM
Neil Cuadra Neil Cuadra is offline
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Thanks for your message, Sue H.

Forum member Anja is also from Switzerland and has posted about AA. Anja started the is recovery definitive? and PNH just dignosed forum threads.
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  #9  
Old Sat Dec 21, 2013, 12:59 AM
Sue H. Sue H. is offline
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Thanks Neil.
In answer to previous question - Yes, they do bone marrow biopsies at frequent intervals to monitor for any changes there- last one in August - next one due Jan. At present my husband visits hospital once a month and they take blood and analyse to monitor. Can't recall exact ANC count before Revolade (Promacta) but was out of critical range at that time (ie where infection a big risk) and now one year on is at lower end of normal range. My husband still has big fluctuations in energy levels but is generally feeling so much better than a year ago before Revolade.
As I mentioned before, seems not a lot of long -term experience with this med. so all somewhat experimental but so far we have seen encouraging results. NIH seems to have the most experience using Promacta so probably best to contact them.
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  #10  
Old Sat Dec 21, 2013, 11:34 AM
Relentless Against SAA Relentless Against SAA is offline
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I have actually talked with NIH a couple of times. We are currently gathering medical records for them because they would like to review our case. This is my dilemma: Our current physician is an extremely experienced physician with SAA. He is wanting to start Promacta without a clinical trail. I do not know what to think about this. I am concerned about using Promacta outside of a clinical trial. I am very worried about bone marrow complications and new clone developments. At the same time, we are seeing one of the top docs in the nation, I do trust him and we are fortunate to have him on our side . I wonder if our current expert would promise to do frequent bone marrow bx if we wouldn't be better off staying with him. I am not sure what to think but we need to make a decision soon. Anyone with advice?

Last edited by Relentless Against SAA : Sat Dec 21, 2013 at 12:54 PM.
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  #11  
Old Sat Dec 21, 2013, 12:33 PM
Sue H. Sue H. is offline
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Dear Relentless,

I understand your concerns and it's not an easy decision. Neil Young at NIS (I asked once for his opinion on our situation) had reservations about use of Promacta outisde of a clinical trial as not enough evidence on long - term use.
As we had choice of Revolade (Promacta), another ATG (my husband had many side - effects from first course of horse ATG, only limited response and took a long time to recover) or transplant we decided to opt for Revolade. In terms of quality of life, my husband has had quite a good year this past year and has so far tolerated the drug well.
Ultimately I think you have to go with your gut feeling on which path to take and then trust. Sounds like you have a lot of faith in your physician, which I think is important.
Wishing you all the very best and hoping things become clearer for you.
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  #12  
Old Sat Dec 21, 2013, 12:53 PM
Relentless Against SAA Relentless Against SAA is offline
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Sue,
Thank you for your reply. I find your situation very similar to ours except for the age difference. It would really help me if you would keep me informed of your husband progress. I hope Promacta is a drug that can help people out of this nightmare.
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  #13  
Old Sat Dec 21, 2013, 03:57 PM
Sue H. Sue H. is offline
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Dear Relentless,
I will keep you informed. Wish you much patience and strength in dealing with this huge challenge.
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