Need input on a drug

Hi,
My name is Melissa and ive been diagnosed with ITP in 2006. Recently I was told that i need a bone marrow transplant in the next couples of months but until i do, doctors are now starting me on a new drug that i never tried before named Rapamude or Sirolimis. It is an immunesuppresant and they expect it to bring my counts up and maintain or decrease my symptoms like bleeding from the nose and the gums. Has anyone taken this medication before, if yes how high did the counts go up? Any major side effects that i should be worried about?

As far as I know, Rapamude is not a common drug used for ITP. Your situation is different because of the fibrosis, so I can't really offer much advice. I haven't seen anyone here using that drug, sorry.

How are your counts doing?

They havent been above 15k for atleast 2 months. Theyve been giving me 80mg of prednisone and im on platlet transfusions every 3 days with no rise in my platlet counts...

Here is a very recent article I had missed:

www.bloodjournal.org/content/127/1/17
Sirolimus is effective in relapsed/refractory autoimmune cytopenias: results of a prospective multi-institutional trial
Key Points

• Sirolimus monotherapy is a safe and effective steroid-sparing agent, improving autoimmune cytopenias in highly refractory patients.
• Sirolimus is particularly active in ALPS and should be an early therapy option for patients who require chronic therapy.

Publisher's Note: There is an Inside Blood Commentary on this article in this issue:
www.bloodjournal.org/content/127/1/5

Maybe you could ask to try eltrombopag. I was found to have under productive bone marrow and am too old for a transplant to be offered but 75mg daily eltrombopag seems to be supporting my marrow and increasing my red and white cell counts to near normal. My platelets remain very low but just above bleeding level, about 10 but I can live normally with these counts. A number of studies have highlighted the beneficial effects of eltrombopag on marrow.

Margaret - From Melissa's earlier thread, she has bone marrow fibrosis which is believed to have been caused by Nplate. I doubt that her doctor would try eltrombopag, since it is similar to Nplate.

Melissa - Sirolimus is used to prevent graft vs. host disease (GVHD), to keep the foreign transplant from attacking the body of the host (you), so it might be used following your transplant. Your doctor's choice to use sirolimus prior to the transplant is interesting. Perhaps there is the added benefit of it being a "trial run" to make sure your body tolerates it well (better to find out now rather than later).

www.ncbi.nlm.nih.gov/pubmed/15273708
Sirolimus for GVHD prophylaxis in allogeneic stem cell transplantation.

How long were you on NPlate before this happened? I have been on it for about 6 months

MaryV: i was on nplate the highest dose for about 9 months and lower doses for another 2 to 3 months.

Thanks rob and magaret for the helpfull information. My specialist told me i would not be a good candidate for the eltrombag because it is similar to nplate and is scared that the fibrosis will worsen.

Rob16- my doctor is using sirolimus as a treatment to try and bring up my platelets since my counts havent been above 15K for at least 2 months and most recently having problems with bleeding. My platlet transfusion are not working and neither is prednisone. If that does not work, i am going to have to take treatments of chemo therapy as part of and immunesuppresant therapy. Its very complicated with my case.

Mel:

Because of the fibrosis, you have more going on than just ITP. Your doctors choices of treatment are not typical for ITP (other than Prednisone), so I wouldn't even try to give advice. I'm sorry, I wish I could.