Anyone using Promacta?

[jhassen]

I have failed ATGAM and rabbit serum. My doc wants to avoid a BMT if possible as I only have a 90% match. He has started me on Promacta. Anyone else have this experience?

[Sally C]

Hi Jhassen,
My husband has had great success using Promacta after being extremely transfusion dependent for a few years. Even after coming off of it due to his platelets getting high enough for concern of clots, his red cells as well as his platelets have continued to stay up.
I have gone into great detail about his Promacta journey under the "Clinical Trials" forum.
Please feel free to ask any questions you may have - either through the forums or my personal e-mail - shcalvert3@aol.com.
I wish you the best.
God Bless,
Sally

[Janire]

My doctor told me that promacta could cause a clonal evolution in aplastic anemia turnig into MDS or leukemia. Does anybody know anything about it? How much time does it to be taken? It has to be taken forever or can it be a good and long treatment to put into remission aplastic anemia?

[Sally C]

Hi Janire,
One of the concerns in the clinical trial for Promacta at NIH was an increase in blasts with MDS. My husband took it for about 4 months before coming off. It normalized his red cells and put his platelets over 100,000 so he had to stop taking it. Even after being off since 7/12, his platelets are in the 80,000's and his RBC is near normal. There has been no increase in blasts in his case.
Take care and God Bless,
Sally

[tytd]

Hello Janire, If you do not mind wading through a technical medical journal you may want to read the published report of an NIH trial of Promacta in AA patients in the July 5, 2012 issue of the New England Journal of Medicine. I think about 40+% of refractory AA patients responded to it in that article and the ones that did were continued on it. The NIH probably have more up to date data now and perhaps an on-going trial that you could possibly access if you have not already started on it. If you search through the forums by putting in New England Journal of Medicine + Promacta into the marrowforums search box you can find a link to the article that Sally C. kindly provided in July 2012. Good Luck and I hope it works for you.
Sally C. , am so glad to hear that things are going well for your husband. Just goes to show you how clinical trials sometimes can be a lifesaver.

[Janire]

Thank you for the information. I will search for it carefully to know what is happenig with that.
I am from Spain and here promacta is only given to people affected with lupus and when I asked for promacta to my doctor, she told me what I said above.
Maybe there are other reasons or in Spain this treatment is not approved and my doctor told me this like a excuse....
I dont know what to think....

[Sally C]

Tytd,
Thank you for your kind words. And you are so right about clinical trials. Don is a living example. It took 6 months until finally going to NIH to get a diagnosis for Don. He entered the clinical trial for Campath and that eliminated his Trisomy 8 and I believe saved his life. But he was still very transfusion dependent for RBC's and platelets. Then he entered the Promacta trial and has been transfusion independent for almost 2 years.

Janire,
The way the US gets FDA approval for these drugs is through clinical trials like my husband has participated. Promacta is not yet an approved drug for MDS nor AA (I don't think). So it would have to be obtained through a clinical trial here. Do not know about in Spain.
Maybe you could access the article that Tytd referred to regarding AA/Promacta and show it to your doctor.
Best wishes and God Bless to you both,
Sally