Well, we’re back from our appointment with Dr. Schultz and we have still made no decisions. This is not a bad thing, we actually kind of expected it. I just feel like my head is floating, spinning in circles, full of fog…
Dr. Schultz told us today that he is more uncertain about doing the transplant than he was the last time I met with him. He has spoken with all the major players in the metabolic disease transplant game as well as the major MPS doctors (Joe Muenzer, Lorne Clarke, Charlie Peters, Paul Orchard, Paul Szabolcs, Joanne Kurtzberg), and it is VERY controversial to say the least. Many centers will not even consider transplanting a child with Hunter Syndrome, and many feel very strongly about it. Apparently we have raised a few conversations around the transplant tables at meetings and conventions. ALL WE’RE TRYING TO DO IS FIGURE OUT WHAT’S BEST FOR OUR CHILD!!
So here’s what we know: Avery is a perfect match, that cute little bug. And that’s about all we know. We do know that Dr. Schultz prefers related BMT over CBT, but he will consider a CBT if we find a 6 of 6 match. He argues that because there is no hard evidence that Marrow is better than Cord Blood, and because the chance of not grafting is less with a related BM donor and because the chance of GvHD is smaller as well, he wants to do a BMT.
Also, something I did not know until today, if Dr. Schultz did do a BMT at BCCH, he would use only a partial dose of chemo (apparently Busulfan, one of the chemo’s used, is being questioned about causing Central Nervous System problems). This cannot be done with CBT, and although it slightly increases the chance of the transplant not engrafting, the lower dose of chemo is much better for Trey. Because this is the first I have heard of this, I cannot remember scientific terms or the exact pros and cons. I do know that Duke does not do the 1/2 chemo thing, they do the full blown chemo.
A positive is that Dr. Schultz thinks the mortality rate for Trey with either CBT or BMT is 5-10%. However, because Trey has CMV (a virus in his body, like the cold sore virus, that usually causes colds in a person who has a strong immune system, but can cause death in a person with no immune system), this ups the mortlity rate 1-2%.
My biggest question or concern remains over CBT or BMT. I know there is no evidence that Cord Blood is superior to Marrow, or not enough to be decisive. This is why it is controversial. What I want to know is: in 10, 20, 50 years, will there be evidence that Cord Blood is better than Marrow? If anybody out there has an educated argument for why Cord Blood is better than Bone Marrow or why they are the same, I WANT TO HEAR IT.
I understand that Dr. Schultz wants to work with evidence and he does not want to transplant based on a belief or hope or idea. However, because this is my baby, even if there is a possibility that Cord Blood is better than Bone Marrow, I want to do a CBT. Dr. Kurtzberg said: “Cord blood cells have more potential to give rise to non-blood cells in the laboratory.”
Yeah… So what’s next? In February, we go to Duke for a follow-up developmental assessment with Dr. Escolar. Dr. Schultz is very interested to see how Trey does in that.
Also in February, Dr. Schultz is attending a meeting which will be reviewing the article that Dr. Kurtzberg and the Duke team of doctors are publishing about the five children with Hunter Syndrome who have had Cord Blood Transplants. Although Dr. Schultz has read the abstract for this article, he does not believe it is convincing enough for him to do a transplant for Trey. It speaks very briefly about a stabilization or improvement in neurocognitive abilities. Dr. Schultz wants to read the article and hear the presentation before making any definite decision. This is fine with us because we need to get Trey’s developmental test with Dr. Escolar done before we make any decisions anyways.
So now it’s all happening in February. And then we’ll meet with Dr. Schultz on February 16 to make final decisions (for real this time…). So now I just have to read all the articles ever published on transplant in people with Hunter Syndrome, and hopefully get in touch with Dr. Peters, who has much historical information on this. And talk with Duke about all this…and make appointments with Duke for the assessment.
At least now we know what our next steps are. A bit of a relief, although it will be an even bigger relief to make the final decision (although I am now much more comfortable with waiting…playing with Trey is so much fun!!).
In other news, Trey is starting to speak like crazy. It’s really awesome and great and wonderful. And he’s also become way less aggressive than he was a couple of months ago (maybe it was a phase?). These recent happenings allow me to entertain the possibility that maybe Trey does not need a transplant after all.