We met with Dr. Stockler today (Trey also got x-rays to check a ridge on his head, that could be craniosynostosis…which Dr. Stockler didn’t seem concerned about and neither are we after looking into it), who gave us more information about transplant that made everything even more confusing.
Until now, we’ve been seriously leaning towards CBT. It’s what has been working and it’s what is being done. Bone Marrow Transplantation scares the crap out of me because they stopped doing it 10 years ago because it didn’t work. And what’s frustrating about it all is that Duke and BCCH don’t agree.
Duke is here and BCCH is there. Here’s where they stand: Duke wants to do a CBT. BCCH wants to consider a related BMT first (it comes down to them wanting to find the best possible match for Trey, whether bone marrow or cord blood). Duke thinks cord blood is superior to bone marrow. BCCH thinks there is no difference between cord blood and bone marrow. Duke thinks that CBT’s are working for Hunter Syndrome because cord blood is superior to bone marrow. BCCH thinks that CBT’s are working for Hunter Syndrome because transplant procedures have improved (I found out what some of those procedures are: 1. radiation is no longer used, which can cause brain damage and 2. instead of transplanting all the bone marrow like they used to, transplanters now use only the stem cells from the bone marrow, which is what the cord blood is made up of, which is what we know gets into the brain).
So I think that’s it. It is just such a frustrating place to be because we do not know enough about transplant 10 years ago versus transplant today and bone marrow versus cord blood to make an educated decision for Trey. Even the doctors who specialize in this cannot agree, at least when it comes to Hunter Syndrome. There are no answers and we’re having to make a somewhat blind decision regarding our child’s life…and people say that buying a house is the biggest decision they’ll ever make.
I will hopefully be talking with Dr. Kurtzberg (the head of transplant at Duke) and Dr. Szabolcs at the end of this week to hear more of their thoughts on this, and then we’ll meet with Dr. Schultz at BCCH again on December 15th to hopefully make some decisions.
On another front, if we choose not to go with a transplant and choose ERT instead, it’s ready to go. All we have to do is give the go ahead and then it’s a matter of sending the drug up to Canada. Which is so freaking awesome. It was a bit of a fight, but nothing like some of our friends have gone through and nothing like we were prepared for.
I don’t often hear positive things about our government, especially when it comes to rare drugs for rare diseases, but in this instance they have come through for us and we are so thankful. It would be so nice to know that Trey’s brain wasn’t affected and then we could jump on the ERT gravy train…just dreams, I know.