I've always wondered if you will ever or have ever considered BMT? I am sure it has always been in the back of your mind but wondering if you have a specific "condition" on what would make you decide that "now" is the time (i.e. # of hospital visits, # of infections, etc). Also, has your insurance company been beneficial to you or is it a constant nightmare trying to get things covered?
Well, I wish I had a better answer... but the BMT (Bone Marrow Transplant) is a BIG FAT Question Mark for me right now. I'm not sure that in our situation, that we will ever "know the right time" to make that decision. But yes... I know exactly what you mean when you ask that. There is just no easy answer. Here are my feelings on it... The BMT is a huge, huge risk (especially for Tripp, who is not 100% healthy) and some children don't make it through the actual transplant. But aside from that... if it were a chance for a CURE- then I think 100%, we would take the chance. But it's NOT a chance for a cure... as of right now, it's just a chance to live a better life with EB (if you make it through). But I want to remind you- that all of this BMT in EB stuff is BRAND, spankin' new-- we don't know what's going to happen for these kids down the road. Maybe it will take years for the "new cells" to do what they are expected to do, and eventually end up being "a cure"... who knows? BUT... does Tripp have years to wait and see what happens with these other kids who are going through the transplant...?? Maybe... maybe not. But then again, how much damage do you allow this horrible disease to do?
SO... the question is: Do you sit back and wait? Or do you do something about it? The part that I'm torn on is... What if we do take him to do the transplant and he doesn't make it through? Could I selfishly have had 2, 5, 10 more years with him? Do you put him in a situation where you are 99% sure that he will be on life support, dialysis, organ failure... just to "see" if he will make it through and have a chance at "better skin" but also a chance of so many other complications that come along with it? After following the stories of all the kids that have gone through with the transplant... I try and picture "us" in that situation. And it's a really hard decision to "knowingly" put your child through something worse- for a chance at something better. I don't know.. I'm rambling, sorry:)
But when you ask "what would make you decide that 'now' is the time?" I totally get what you're saying. Yes, I think if we were in a situation where we "knew" he wasn't getting better and we knew that we didn't have much time left with him anyway... I think we may opt to do the transplant. BUT he also has to meet certain requirements to be able to go through with it... he needs to be somewhat healthy to even have a chance, I would think.
Oh, and insurance has been excellent!! So far, (if I jinx myself, I will have a nervous breakdown) they have covered all of Tripp's supplies, from wound care to feeding supplies to tracheostomy supplies and machines. Before a few months ago, I had not even appiled for Medicaid... so we were paying co-pays for hospital stays, doctor's appointments (which were sometimes as often as 3x week) and presctiptions (which were outrageous)... but I finally got the chance to apply for the Medicaid as Tripp's secondary insurance, and that has helped out tremedously. They pick up most of the remaining co-pays. We are blessed.
However, if there are any volunteers who would like to be my secretary with no pay, feel free to send me a resume'. Just playing... but really- I feel like I am on the phone with someone all. day. long. And it's been 18 months since we've been getting supplies, right? (well, about 15 since his feeding tube supplies)... but you would think they could get an order right, huh? Nope... something is wrong, different, or missing each month. That'll make-a-momma-crazy. Then doctor's offices... sheesh- if I don't say Tripp's name and birthdate at least 47 times a week, then it's not week. Thank God his doctor's that I have to deal with on a daily/weekly basis give me their personal numbers and e-mails. Because those poor doctor's office secretaries just don't know the stress an EB momma carries around:)
I would like to know more about BMT. How it works and what it's supposed to do and if I can get tested somewhere to see if I am a suitable donor. I would also like to know, if you know that, if they are looking into the cord blood treatments?
A Bone Marrow Transplant or Cord Blood Transplant both replace your unhealthy blood-forming cells with healthy ones. The cells used in these transplants can come from three sources:
-Peripheral (circulating) blood (also called peripheral blood stem cell or PBSC transplants)
-Blood collected from an umbilical cord after a baby is born
Right now at the University of Minnesota, the Bone Marrow Transplants (BMT) and the Cord Blood Transplants are being done. Some children have have both marrow and cord blood infused together. Researchers have been studying the use of stem cells to treat children with severe EB- Recessive Dystrophic and Junctional forms. So far, the results have showed that stem cells from a healthy donor find their way (home) to the skin and secrete the protein that is missing in EB. For the child, this can mean a substantial reduction in blistering and enhanced healing of the skin. However, the BMT journey is brutal to these children and their organs and some don't even make it through the transplant.
The first thing that would have to be done if we decided on doing the transplant would be a complete workup. This consists of complete laboratory and diagnostic tests in order to make sure that your child is physically ready for the transplant. The tests will help doctor's to evaluate the child's lung, liver, kidney, and heart function. The workup evaluation takes approximately one week. We would have to go up to Minnesota and Tripp would spend every day for a week having these tests done... whoo hoo.
The evaluation includes:
An in-depth medical history and physical exam
Blood, urine, and other cultures
Kidney function evaluation
Bone marrow aspiration and biopsy
Heart evaluation, including an electrocardiogram (ECG or EKG) and an echocardiogram
A spinal tap (if needed)
Radiation therapy consultation (if needed)
CT or MRI scans (if needed)
Consultation with other medical specialties as needed
Then you meet with one of the transplant doctors to discuss the workup evaluation results and decide if BMT is the right treatment for your child, and then decide on the best timing for the transplant.
To prepare their body for the transplant, the kids are given chemotherapy, sometimes along with radiation therapy. This is called the conditioning regimen. The standard transplant uses a very strong treatment that destroys their diseased cells, and it also destroys their immune system so it will not attack the cells that have been donated.
After this "conditioning regimen," the blood-forming cells are given to the child or "infused" through a central line that goes into a vein in their chest. (One of the most common central lines is called a Hickman catheter). The actual transplant (infusing the cells through the Hickman) takes only about an hour. These transplanted cells move into the spaces inside their bones where they create new marrow. They grow and make healthy new red blood cells, white blood cells and platelets. Sometimes it can take several weeks before the transplanted cells produce new blood cells. During this time the body is unable to produce white blood cells, which fight infection. This means the child is at great risk of infection.
Red blood cells carry oxygen to all parts of your body.
White blood cells help your body fight infection.
Platelets help control bleeding.
The actual day of the transplant is called Day Zero. When the transplanted cells begin to grow and make new blood cells, this is called engraftment (and is usually expected around day 30). The amount of time spent in the hospital following the transplant depends on each child's circumstances.
To learn more about Bone Marrow Transplants in EB, visit this website.