Tuesday, February 24, 2009

Geneticist Appointment

Well, we met with four adults in a tiny little exam room today for about two and a half hours. We started with a 101 course on GAMT Deficiency explaining why there's a problem and where supplementing creatine and ornithine and restricting arginine come into play.
We also talked about her diagnosis and that it's basically 100% firm, but they would still like to do a DNA test just to have the information. If any other family members suspect they are at risk or just want to know if they are carriers they could have a much more affordable test ($200-300) because the search of where to look and what to look for would be greatly narrowed down after Samantha's lengthier test ($1500). An example of who may want to know one day is Ellie when she is ready to have children.
The geneticist was an awesome Italian with a super thick accent in his 50's? and he had great things to say about Samantha. He mentioned that when we come back in four months to follow up with him, she should be talking more and doing much better. He referred to her as a "mild" case and was pretty impressed with how well she has developed inspite of the condition.
The nutritionist joined the group towards the end and felt like we were being bombarded with too much information and wouldn't be able to tackle the diet right away. She gave me some basic information (while Trey walked the halls with Samantha for ten minutes) and is going to meet with us next week to get us officially started. Samantha can have 20 grams of protein per day (that number will go up over the years as her weight goes up). I think that if she cuts out all meat and dairy that we can do this without too much difficulty (not that cutting out yogurt, milk, cheese, nuggets, etc isn't difficult!). For example: a glass of cow's milk has 8 grams of protein, but a glass of rice milk has 1 gram. So, having the right foods on hand will be key. The food group with the lowest amount of protein is fruit and luckily she loves fruit. The foods she can have with no limit are "pure fats and sugars" (yum yum!), straight sugar candy, kool aid, popsicles, etc. I'm not sure what "pure fats" are, but I think we'll stick with sugars.
We will have to petition the insurance company to help pay for her creatine and ornithine, but I went ahead and placed a mail order call on our way home to get a kilo of both headed our way. She will be on both of these for the rest of her life and she will be watching her protein intake for the rest of her life. The severity of the diet in the long run will depend on her body and how she responds to the current guidelines. She may regulate to where she can have a higher amount of protein, but will probably never be able to eat totally "carnivore" again.
I hunted down a "scientific grade" scale for measuring out her meds each day and am excited to get started in 2-3 days when the supplements arrive. We will be tracking her food for three days and shooting for about 30 grams of protein per day this week.
Ironically I received an acceptance email today for Samantha to attend a local charter school for Kindergarten in the fall. I'm not sure that she'll be up for that, but it is cool to know it's an option.
I feel like I'm in a little dream right now. I am overwhelmed and ecstatic all at once. I definitely feel like I need to pinch myself every five minutes.

Sunday, February 22, 2009

Results

I got a message from the neurologist's office on Thursday that they wanted to talk to me about the lab work we had done. When I called back, the physician's assistant who had called me was unavailable, so they checked Samantha's chart and said that no results were in. I was of course a wreck and so I called the geneticist. I began explaining that I was looking for lab results and had been rescheduled and without anymore information they knew who I was and transferred me to a geneticist. She answered, "Are you calling about Samantha?" This all made it pretty obvious that we were the buzz around their office. She said she had results, but that we could talk about them Tuesday because it's easier to explain things in person, but I couldn't wait and pushed for more information. It turns out they didn't actually do DNA testing yet; they tested Samantha's blood for creatine in the plasma and her urine for an enzyme that would normally not be there because it is broken down into creatine. Well, Samantha had low levels of creatine in her plasma and high levels of the amino acid in her urine. All of this means that she most likely has "GAMT defficiency". They will do DNA testing now (the insurance wouldn't pay for it until the geneticist had basically proven ahead of time that Sam has what they think she has). Evidently this geneticist that we will be meeting with is amazing and the geneticist I spoke to said he is never wrong and if the results we had so far weren't as definite as they are he would probably pursue other causes of her creatine defficiency.
So, now we have a diagnosis. I won't go into all the facts I've read about this, because I am prepared for all of that to change on Tuesday. Every case study I have read about this disorder is so different that there is no definite prognosis. I will say though that Samantha is MUCH better off than a lot of the kids I've read about, so I still have high hopes. We will begin supplementing creatine and ornithine on Tuesday and we will meet with a nutritionist and begin this diet. The hospital has one other child with this disorder. He is a three year old and has been doing very well. I am hoping to talk to his mom soon.
I can't help but think that all the cooky things we've tried with Samantha (including extreme diets) have helped prepare us to feel a lot less overwhelmed at the work we have ahead of us. And I feel like working with NACD has prepared us to know how to plow forward once we get Samantha's brain healthy and ready. Most of all I think we've learned a lesson in grattitude and patience- lessons I know I sorely needed. :)

Wednesday, February 18, 2009

One more week...

The geneticist appointment has been postponed until next Tuesday. They somehow get test results in one week (not four weeks like the lab and neurologist had told me???), but didn't have them yet. So, we'll see if we are re-scheduled three times before the results are in? I'll post once the visit actually happens.

Monday, February 9, 2009

PLEASE 'go' in the cup!

Today I went to the children's hospital expecting to get instructions on collecting a sample for genetic testing. It turned out they also needed blood and that it had to be drawn at roughly the same time as the urine (within an hour), plus they were sending their shipment (to a lab in Texas) very soon. So, my great plan (as approved by the physician's assistant on the phone) to hang out at Faith's for lunch while filling Samantha with juice and comfortably waiting for the sample did not work. Instead I had to hold down Samantha again (I hope she won't remember all this and hate me forever) while they tried to draw blood (at least they only failed once).
After failure to cooperate in the bathroom, a visit to the cafeteria for 32 ounces of orange soda and more failure in the bathroom, Samantha was a crying mess, Ellie wet her pants while watching and started crying and I, sitting on the dirty bathroom floor holding a specimen cup (empty still), started crying too. Will just sat there with a confused look on his face as if to say "what the heck is going on?" I will spare you the details of how we finished this visit, but we did, and now we get to wait approximately four weeks for results.
I got home feeling like this was definitely on the top ten list of "worst days of my life" until the neurologist called to say we had gotten in with the Metabolic Geneticist for next Tuesday and that he is very eager to meet with us!! Even though there won't be any test results yet, she said he may want to run more tests (ugh) and he also may want to get us started on some creatine supplementation and possibly excluding argenine from Samantha's diet.
I am excited that they aren't making us wait for weeks to get the ball rolling. I hate everyday thinking that there is something I can do to help Samantha and I'm not doing it. Though there is no diagnosis, it seems like, regardless of the cause, if her brain is low on creatine, it can only help to get her started on some supplementation.
This is the first attempt to "cure" Samantha that I don't secretly suspect won't work before we've even begun. I am so anxious to fast forward through the next week I can barely stand it.

All of this reminds me of the wise counsel of one of Samantha's preschool teachers which was "there are two things you cannot force a child to do: talk and 'go' in the potty".

Friday, February 6, 2009

Hoping for the worst

Samantha had her MRI on Wednesday afternoon and was not able to wake up until the next morning at seven. (It was a little freaky.) We got a C.D. copy of the MRI on Wednesday and got home and started checking it out. Everything structurally looked fine (according to the technician's notes- I'm not claiming to be an expert). In addition to the standard MRI, the neurologist had ordered a "spectroscopy" which is done with the same machine, but it's a way of checking what levels of chemicals are present in the brain. The note regarding this scan said that she had a decline in creatine which could indicate a creatine deficiency state. He mentioned specifically a condition called Guanido acetate methyl transferase deficiency. I quickly did some reading up on this and here is my summary.
Creatine is a major source of energy in the brain, helping the brain work in general (yes, same stuff as body builders take). The specific deficiency mentioned shows the following symptoms in children: developmental delays from birth (not "she developed normally till 18 months and then regressed"), major speech delays and seizures. Pretty much Samantha to a T.
This is a genetic condition. It is pretty rare and occurs when both parents have the recessive gene and both give the child that gene (like two brown eyed parents having a blue eyed baby). This deficiency was only recently discovered. So why am I hoping for my child to have a rare genetic disorder??? Because the final adjective describing this deficiency is "TREATABLE".
Of course this is just speculation by a technician viewing her MRI. I haven't wanted to get my hopes up too much (or anyone else's). I have been calling the neurologist's office for two days driving them crazy to get the doctor's opinion. They finally called late this afternoon and want us to come in Monday for a urine sample to be sent to a special lab that handles genetic testing and they are setting up an appointment for Samantha to see the genetecist that specializes in metabolic issues as soon as possible.
All of this information was conveyed to me very delicately as though the physician's assistant was scared that she was going to drive another poor mother to tears. Finally I said "we've seen the report. Are these tests to pursue the possibility of the creatine deficiency disorder mentioned?" And she very tentatively replied, "Well, yes... but the good news is that it's treatable." My mind said "woo hoo" and my mouth said something more socially restrained and I hung up.
Treatment is mellow in the sense that it's totally safe. It involves oral supplementation of creatine which has no side effects, but it involves the most difficult diet I have ever heard of. The diet is to eat nothing containing arganine. Arganine is an amino acid found in:
  • Animal sources: dairy products (e.g. cottage cheese, ricotta, milk, yogurt, whey protein drinks), beef, pork (e.g. bacon, ham), poultry (e.g. chicken and turkey light meat), wild game (e.g. pheasant, quail), seafood (e.g. halibut, lobster, salmon, shrimp, snails, tuna in water)
  • Vegetable sources: wheat germ and flour, buckwheat, granola, oatmeal, nuts (coconut, pecans, cashews, walnuts, almonds, Brazil nuts, hazelnuts, pinenuts, peanuts), seeds (pumpkin, sesame, sunflower), chick peas, cooked soybeans
So, basically vegan plus gluten and casein free, plus a few more things. Of course, I will be happy to put her on this diet;)
The concept of "we're going to an MRI and may find a cure today" had never even entered my mind. I was thinking the closest thing to that would be, "there's a huge tumor that needs to be removed" or more likely, "her brain is full of glucose which is causing her inability to focus on the words coming out of her mouth. We think you should put her on ritalin". Really I expected nothing.
I am scared that I have my hopes up for nothing, but this is the most real thing we've learned about Samantha and I am going to keep my hopes up for now. I feel like these seizures had their sudden increase for a reason and were truly a blessing in disguise.