Sirolimus or CellCept

Our 9yr old daughter is currently being treated with IVIG about 1X/month and now it seems to only get us to the low 100s. She has been doing this protocol for almost 3yrs. We are considering trying Sirolimus or Cellcept to improve her quality of life. We did rule out Rituxamab due to her weird medical history. To complicated everything she has partial complex epilepsy and we are told her meds for that can lower platelets. We are not sure how long she has had ITP since she was adopted from China in 2011 and was diagnosed with ITP there. We usually treat when she gets in the teens to low 20s. Our lowest has been 10 and that was 2 weeks ago. In the beginning she responded great to IVIG, but in the last year it is working less and less. Thoughts?

Hmmm. I've never heard of Sirolimus being used as an ITP treatment. Who is suggesting that?

Personally, I am not a fan of using CellCept for children, but that is just my opinion. The strong immunosuppressants carry a cancer risk that could catch up to her later in life. Has her doctor mentioned N-Plate or Promacta?

If IVIG is still lasting a month and she tolerates it well, you could even consider continuing with that. Counts in the 100's are fine and shouldn't be a reason to discontinue treatment. How are symptoms when counts are down?

Her hematologist is recommending. we only get one week in 100 maybe then is heads south. the hem/onc nurse said they are getting good result with sirolimus. It has been tough on our daughter to get IV every month.

The only thing I could find regarding Sirolimus was a clinical trial. I've never heard of it before. It seems to be used mostly for children who have 'cytopenias' due to ALPS.

www.clinicaltrials.gov/ct2/show/NCT00392951

I don't know anything about your daughter's situation or how her symptoms are when counts are down. I do know that many doctors are trending towards the 'watch and wait' method which means no treatment unless symptoms are present. Many children do fine with that if the doctor and parents are on board. There have been several parents here who chose that route and have been successful with it. I'm not advocating that for you, just mentioning it. Many children have eventually obtained remission with no treatment intervention. Sirolimus seems to be a somewhat drastic way to go at this point since I'm not sure that you've done the first and second line treatments yet. It's not necessary to normalize counts when one has ITP. That was the thinking years ago but things have changed.

I know you want to make the best possible decision for your daughter and that would be very hard to do. I have been involved with the PDSA since 1997 and have followed every patient who has come here since then. It all boils down to benefit vs risk. Only you can weigh the pros and cons of treatment risk vs ITP risk.