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Trina Ward
March 14th, 2004, 04:13 AM
Hello there

My daughter Daria has MPS 1 (hurler). She received a sibling cord blood transplant (CBT) in September 2001. Whilst we have seen some fabulous results from the transplant, and she continues to do well, her orthopaedic problems are quite dramatic, especially in her spine. I have read that ERT assists with the bones.

I have read so many conflicting stories about the availability of ERT to the MPS 1 patients. I know a lot it all depends on the approval of the government/s where you live. However, I have been told that until clinical trials of ERT in transplanted kids takes place, no transplanted child will ever get ERT. Then I read in websites that transplanted patients have received, or are being seriously considered for, ERT as well. I am confused !!!!

Can anyone shed any light on this? Have you heard of a BMT or CBT patient getting ERT as well?

Thanks
Trina

Allimom
March 14th, 2004, 01:20 PM
Trina~
I have heard of kids recieving ERT and a BMT!
Julia: http://www.caringbridge.com/page/hopeforjulia
Christopher is going through transplant now and still receiving ERT:
http://www.christopherjoseph.com
Niki is also going through transplant and started with ERT. I'm not sure if she is continuing with the ERT though.
http://www.caringbridge.org/il/niki
Evline is on ERT but planning to go through transplant and continue with ERT.
http://www.caringbridge.org/ky/evline

I hope this helps!
Jennifer

MelI
March 14th, 2004, 07:04 PM
Hi Trina,

We posed this same question to Jacob's doctor, Dr. Kurtzberg, at Duke when we were there a few weeks ago as we had heard different things about this as well. I am by no means an expert on this and can only pass on what we were told by one doctor! My understanding is that different doctors have different opinions on this issue (probably the reason we are hearing different things about it! :D ) Dr. Kurtzberg thinks that the ERT is fine to begin immediately upon diagnosis, even if you are going to go ahead with CBT or BMT sometime in the future but then to stop the ERT directly prior to the actual transplant. I know that some of the websites Jennifer posted are doing this. The other issue/question is doing ERT AFTER CBT or BMT...Dr. K is more wary of this. She has concerns as to what the Aldurazyme does to bodies that already have a 'healthy' enzyme level. There is a test that can be performed in the lab to see what happens (when the Aldurazyme is put into a body that has been transplanted) but to her knowledge this has not happened yet and/or this has been done and the data has not been released on what happens. I think everyone knows that I have been very happy with Dr. K as Jake's doctor and trust her on this one...that said I am interested to hear if anyone else has heard anything different! Also, how we see it is that Jake is 100% engrafted and has a higher enzyme level than any of us (Mommy, Daddy, & Brother) so I'm not sure how giving him more enzyme can help him UNLESS it can somehow be targeted to his 'trouble spots'! Hope that makes sense!!!

Take care, Melanie Irish
http://www.caringbridge.org/wa/jacobirish