Blog · March 9, 2016

Personal Decisions


For many years now I often had people that care about Nicky telling me about or asking me why he hasn’t had a Bone Marrow Transplant. While I wrote in the book extensively about Nicky’s decision (because, make no mistake, he is in charge of his body and I’ve told him that since he was old enough to understand), I wanted to delve a little more into it. Nicky has given me a free pass to talk about EB as it pertains to his life long ago, so please do not think I am doing this behind his back. I wouldn’t do that. Never.

As soon as I heard that Minnesota started doing the transplants back in… oh… 2007 I think, I was hungry for any detail that came my way. I followed blogs from parents, gathered info any way I could. When I then heard that they started doing transplants from a donor match that wasn’t a sibling (since Nicky does not have a sibling match), I started getting the ball rolling in getting a counsel. Nobody would give me a referral, it took me a year to go through the red tape and finally in October 2010 we flew to Minnesota to talk to all the Doctors and get all the information we needed to make an informed decision. I felt that to go through that kind of effort and expense required not only as much information as possible, but deep personal thought.

We have to remember that everyone has their reasons for doing this kind of extensive procedure or not, there are many different situations, including especially the financial aspect of it, which would allow or disallow a 6-8 month stay in MN. This is a long time and hard for a family to be away for so long without any guarantees, especially since this was not a cure and at the time it proved to be extremely risky. My husband was still not independent from having had his strokes at that time and I could not leave him behind for this long of a time as well. Not sure I could do this NOW either. This is by far a very personal decision.

I took the picture above just before they put Nicky “under” for testing. They wanted to take photos of his body, do blood tests, do biopsies etc.

After it was all said and done MN told me that Nicky had many unrelated donor matches available (unlike some of their other patients) and that all they needed was our “go” to get the ball rolling. Nicky was skeptical at best. As of then, of the 12 RDEB transplants done, only 4 were done with unrelated donors. The first child died, albeit it was a chemotherapy issue. Second child was okay but did not see much of an improvement. I heard later on she developed other issues because of the transplant. Third child didn’t work at all and the fourth was having major complications at that time and he was one of Nicky’s friends. He later died. Needless to say, Nicky never gave his “go”. That did not mean we would “never” do it. Never say never.
Nicky felt the transplant was just “too much”, his life was already so painful. It’s his body. His life. He decided to wait for other treatments to become available. Something less risky. I can’t fault him. Can you?

Of course, this was 5.5 years ago, and as I hear, transplants are a lot less invasive and a lot less risky as they were back then. They’ve also been more successful, which is all well and good, but Nicky is going to be 20 years old this year and he’s a lot weaker than he was nearly 6 years ago. I feel it would be impossible to convince him to do it now. It would be, of course, a completely different story if he was much, much younger. I remember them telling me the older the patient the lesser the chances of a good outcome, which is depressing. In my view, I would still “find a way” if there were some guarantees in place that it could significantly improve his life and it could be done locally, either at CHLA, USC or Stanford. While I still don’t have the luxury to leave my husband for 6-8 months, I would find a way, somehow. Nicky always comes first.

I am not against the transplants at all. I am very happy it has helped many EB children. Heck, if there was ONE condition that needed any help it could get, it is this one. As a matter of fact, I am SURE that if this procedure had been available when Nicky was little, we would have done it! I have no doubts in my mind. But it was a different time and place and we were not meant to have that option.

Since trying the genetically modified skin-grafts at Stanford, we learned that Nicky also has anti-bodies that attack the collagen as well. This is why in the end they didn’t work. This means that most likely the transplant wouldn’t have worked either. The antibodies will attack the collagen no matter how it’s introduced. Does this mean he’ll be disqualified from all the upcoming trials, namely the Protein Replacement, the Fibrocell or the Gentamicin Therapies or perhaps a future BMT (more on these in my Caringbridge Blog)?

Good question. Yes, he could be disqualified for that. But that does not mean I will not enroll him to make sure. I will never give up. If there is a way, we will find it.

Love & Light,