So we’ve made a decision. ERT. And there’s a whole lot of emotions which go along with that decision. It is possible that one day Ryan and I may have to make more difficult decisions in our lives than this one, but I am not sure about this. This is easily the most difficult decision I have ever made and I don’t see many decisions being more difficult. And because of this, we are exhausted. Relieved to have made a decision, but exhausted.
Since we learned about transplant as a potential option for Trey, I have really not slept a lot or done much else other than trying to have fun with the kids as often as possible. I have spoken with most of the experts in the field of transplant and MPS, I have talked to parents who have gone through with transplant and ERT, I have read their journals, I have read articles on historical transplants, we have travelled to North Carolina to see one of the leading experts in MPS II to talk about transplant and ERT, we have had transplant assessments done in North Carolina and here…
Basically, we learned that Duke is transplanting Cord Blood in children with Hunter Syndrome. We knew that BMT’s in Hunter Syndrome were stopped 10 years ago because they did not work (meaning that they did not help the brain), although after reading the research, there were not a huge number of transplants done (and the ones that were done were done on all ages and severities of disease). However, there is enough evidence for all the transplanters and MPS II specialists in the world, except Duke, to stop transplanting for Hunter Syndrome.
So we asked the question: why is Duke transplanting? Duke believes that Cord Blood is superior to Bone Marrow and they believe that this will change the outcomes of transplant in Hunter Syndrome. I want to believe this, with every part of my soul and being, and I was even willing to transplant based on a hope or belief, but… Theoretically it makes sense that Cord Blood can do more than Bone Marrow, and apparently this has been shown at a cellular level. Unfortunately, other than Duke, no one I spoke with has seen any difference in outcomes in humans or animals from Bone Marrow to Cord Blood.
This is the entire reason why we were considering transplant. Cord Blood will be different than Bone Marrow. When this idea was shot down, we began to wonder. We began talking to everyone we could. Then we heard that newer transplant procedures could make the difference. And again, most (all but two) doctors said that this will not change the outcome.
One of the doctors we spoke with, who has a way with his words…in a sick to your stomach kind of way…really hit it home for us. Basically, Duke is the only team in the world transplanting children with Hunter Syndrome. We knew this and we were okay with this, for a while. The problem is, no one else we have spoken to believes or has seen evidence that Cord Blood or newer transplant procedures will make a difference for Trey.
And although Duke is hopeful of what their transplants will show, we will not know the outcomes of the Hunter children transplanted at Duke for a few years, as they are still young and were transplanted recently. So we cannot transplant based on how they are doing. It is simply to early to tell.
And if we wait to do another assessment in February, not only are we not giving Trey the ERT that he needs right now, but if his brain is involved, we are waiting to see when his development drops as demonstrated by developmental tests, and by that time it will be too late. His brain will already be involved.
And if we did decide to transplant and it did not work, not only are we cutting at least a year out of Trey’s life to do the procedure, but we are also possibly giving Trey another disease (GvHD) on top of Hunter Syndrome, we are excluding him from any trials that may happen down the road that pertain to Hunter Syndrome, and we may also possibly kill him in the process.
We have made our decision and we are comfortable with the decision we have made, but we have very mixed feelings about it. Transplant was our only hope for Trey if his brain becomes involved. That is gone. We spent yesterday sobbing because of that.
I asked some mamas I know from the MPS forum if this ever gets any easier because although this has not been nearly as hard as adjusting to the news of diagnosis, it is definitely not easy. And it seems as though we are faced with decisions or news like this every few months.
I am so thankful for all the support we receive from the MPS families we know out there. If it wasn’t for you, I would feel very alone during these times. But on the other hand, we do not yet know if Trey’s brain is involved. And until the time that we do, I am going to believe that it’s not.
And we have been granted ERT. And we are lucky and grateful for that because we know families who are still struggling to get it. I know that life is about the lens through which you view it.
So, I will take the time I need to be sad, but I will come up from that and realize that I have amazing family and friends who have been so incredible through all of this. And I have an awesome and thoughtful and wonderful and talented and loving son who has taught me more than I could ever learn on my own and whom I love with all of my body and soul. Thank you to all of you who have helped us get this far. We love you.