Intrathecal Trial Update – Highs and Lows

Posted Saturday June 09, 2012 by Melissa

Bunny suit

I’ve often said that a clinical trial is not for the faint of heart and this past month should testify to that.

We were at UNC from May 14-25. We came back for June 7-11. We return again June 17-20. During that time we have missed or will miss Case’s last several days of school, his brothers’ field day and last day of school, Brock’s kindergarten graduation, our annual church concert, Tyson’s first day of camp, and most of Father’s Day, among other things. Par for the course. But now my older boys have been asking more and more questions about why we have to go, why can’t we stay home, why can’t they go with us. Things that are hard to explain to kids that are only six and eight years old.

Meanwhile, we’ve had a few other ups and downs.

Case running

Neither rain, nor sleet, nor 36 hours of blood draws can keep Case from his grocery rounds

Testing his patience. In mid-May, we were here for annual testing, including physical and cognitive testing. Of all times Case decided to be incredibly uncooperative, it was during cognitive testing. Case has learned what he likes and doesn’t like and right now, he doesn’t like being told what to do, especially with medical stuff (sounds pretty normal to me, eh?). We did the best we could, but he was not convinced that he shouldn’t just be playing in a park at that very moment instead of sitting in an office identifying pictures, building blocks, and drawing circles.

Suffice it to say, his score showed his lack of cooperation.

The frustrating part is that this is just a slice of time, but this slice of time is what is used to gauge whether the drug is “working.” Clearly, Case has made many advances since his last testing, advances which include having opinions about what he will and won’t do.

However, all of the other testing went incredibly well and we even survived keeping Case’s IV line in for 36 hours of blood draws. We are so excited about the promise of this drug eventually for all our Hunter boys.

That is if we can figure out a reliable way to keep getting it into the brain.

Ports-a-plenty. So after annual testing was supposed to be Case’s regular dose of IT drug. Well, the port wouldn’t work again. We were just in this situation only 3 months or so ago, so this was incredibly frustrating. The catheter appeared to be fine, so we guessed it was the port device again. Luckily, Case was still able to get his dose via lumbar puncture and we scheduled port surgery after due consideration of whether to just pull the entire device or try one more time to avoid monthly lumbar punctures and thus, monthly general anesthesia.

Bunny suit

Case is a little unsure of my "bunny suit" before surgery

With that being said, with annual testing, failed dose, LP, port surgery, and dosing with stitch removal, Case will have received general anesthesia 5 times in about 5 weeks.

That is sobering. Especially with a disease that, because of airway effects, trains you to consider all general anesthesia a potentially life threatening event.

We are hoping that very soon, news should come about the new port-a-cath that will be used for our boys and for the Phase III clinical trial. Now that everyone is quite familiar with the high activity level of our boys, hopefully this port will be much sturdier.

Case will continue to recover from this port surgery and when we return for the next dose, his stitches will be removed. As with the last one, we hope and pray this port will last until that new one is ready.

Swimming

The "I just fell out of a crayon box" look is big on the swimming runway this year

Life. But life continues. Case continues to do amazingly well and for this we are thankful. For the first time in recent memory, we went to someone’s house (my brother and sister-in-law’s to see their new gorgeous baby) and no one had to be on “Case duty” – watching him so he didn’t run away, hurt someone, hurt himself, break something, or otherwise go crazy. We didn’t even realize it until we’d been there over an hour and I was just sitting on the couch visiting and Case was playing and watching a DVD. Now that is incredible. And when we went swimming, both at the hotel here in NC and over the holiday weekend, Case just played in the pool with his floaties on with his brothers and I actually … wait for it … was able to sit in a pool chair and read. I kept telling my husband how amazing that was.

We look forward to the MPS Family Conference this summer in Boston where I am excited to take all 3 boys by myself on connecting flights, taxicabs, and maybe even a duckboat tour, all in an effort to see our wonderful friends in the MPS community. Another mom and I will even be facilitating an IEP workshop where we hope that families can learn from one another for the benefit of our kids.

So friends, I want you to know that we appreciate you following our journey. We appreciate your support. On days like today (port surgery), it is only the arms of our Lord and the prayers of our friends that keep me from collapsing in a miserable mess. Thank you.

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5 Responses to “Intrathecal Trial Update – Highs and Lows”

  1. Melissa,
    I was about to email/message you this morning. I know what an incredibly tough past fews days (especially) you’ve had, but I thought I’d check here before emailing. Sure enough, a blog. I am with you every step. I could not do this without you.
    Much love,
    Deb

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  2. sarah mitchell // June 9, 2012 at 7:17 pm

    you guys are AWESOME!!! go go new port, and hurry hurry improved port 🙂 i continue to be amazed at case’s progress and dream about days with a break from “kid duty” and reading while he’s “playing in the pool independently”… what???? if i didn’t know you, i wouldn’t believe it for a second! simply amazing. you are a blessing to so many people melissa, i just always want you to know that.

    hugs,
    sarah

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  3. And here I was whinging about missing the Mother’s morning tea and Talia’s class assembly this week and 4 generals in a year! You just put it all in perspective for me!

    Glad you are getting to the Boston Conference, but I wish it was the International Symposium with us instead 😉

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  4. Good to see that IT Elaprase has a lot potential to make a difference.
    Thank you for sharing and updating.

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  5. […] of the problems Easton had with the device [the original port-a-cath had a number of problems – see past post], that I would like him to just be a normal boy, no more surgeries, hospitals, no doctors. I […]

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