Not So Easy

Yesterday, Gavin had another visit to Amplatz for his Kalydeco drug study.

I don’t know if we have been living in a blissful state of ignorance, but when your child has never had a hospitalization, watching him struggle in a hospital setting is heart breaking.

A tough part of the study is that it’s not being conducted at our normal clinic. Instead, its in a completely different area of campus so Gavin doesn’t know anyone on the team (with exception to the research team). And being surrounded by a room full of grown up strangers poking & prodding at you must be so overwhelming.

Yesterday, they failed twice to get an IV started on him. He needed the IV as they had to get several blood draws from him to observe how his body is handling the new medication.

After over 2 1/2 hours of going back and forth, and Gavin (understandingly) completely melting down, Casey and I demanded a regular old blood draw which went off without a hitch.

The research team then suggested we get another person to come upstairs with an ultrasound machine to aid in getting one started. With the help of this machine, they were able to get an IV started before Gavin even knew what was happening. Had I known there was a possibility of the crap Gav had to go through? I would have demanded this from the beginning. I mean, he is there for a RESEARCH study. Yes, a study for a life saving drug, but it’s a STUDY. All we ask is that they at least try to make it as stress free as possible. Why it took several hours and a completely traumatized child to try a different step is a question I’ll probably never get an answer to.

But no worries, I’m sure you’re not surprised that I didn’t keep my opinions to myself! I made it very, very clear that the morning was not tolerated nor was it to ever be repeated. For any kid.

Thankfully, once the IV was started our day went forward as planned. Delayed in time, but moving forward nonetheless.

One of the Child Life Specialists came in and spoke with Gavin several times, including the explanation of the IV using a teddy bear.

They also sent him home with a bag full of real (and safe) medical supplies for him to play with. My biggest concern was creating a fear of doctors in his mind and we’re hoping that having these items at home will help to ease any fears that may have been formed yesterday.

Trying to look your four year old in the eye and explain that yes, what is happening right now does help him tore me apart. Like any parent says – I wish I could have taken all the pain away and placed it on myself. I did manage to sneak off to the gift shop and stocked up on some toys. Well, kind of a lot of toys. Enough for the sales girl to look at me with a curiously raised eyebrow and me responding with a shoulder shrug and a guilty “I’m spoiling my kid and I know it,” glance back.

We spent the rest of the day laying low watching the clock slowly creep towards four o’clock. I don’t think we could have booked it out of there any more quickly. I keep reminding myself that the big picture is what we need to focus on. That the hard days are resulting in a healthier, brighter future. But as much as I try to convince myself of this – the difficult days are just that. Emotionally & physically difficult.

The beginning of a new chapter

On September 17th, 2013, Gavin got his first dose of Kalydeco.

To be honest, I thought I would have this grandiose emotional moment. But like most things, the moment passed in the blink of an eye and it wasn’t until our drive home that it truly hit me.

(in the study, the capsule is opened and the inner tablets are dissolved in applesauce)

Gavin is taking Kalydeco. Our miracle drug is here. In my house. Right now. The drug is in my possession, and there is no more wondering when, if or how. We are beyond lucky that he has started this medication before he has ever once cultured any bad bugs, and that he has never been hospitalized.

People ask, have you noticed any changes? Honestly, not really anything significant. And I hope it stays that way. Why? Because he is SO healthy. So to me, NO changes means he’s staying right where he needs to be. Sure, a slightly more “stable” digestive system is starting to show its face, and any other positive changes will be welcomed with open arms.  But to us, keeping him where he’s at is what we need. Healthy and happy is the goal. Anything extra is just an added benefit.

This visit included another quick check up from the doc, sweat test, and blood draw but wasn’t anything too strenuous.

Still trying to wrap my head around it all.

It may sound trite, but I wonder if there will forever be a “before Kalydeco and after Kalydeco,” state of mind in our home.

It’s happening!

Kalydeco. It’s happening for Gavin.

When Gavin was first diagnosed shortly after birth through the newborn screening test, we heard about Kalydeco (then known as VX-770) at one of his first appointments. We really didn’t  understand much about it at that time other than he had the “right mutation” (G551d) and that he might be able to get on the  medication by high school.

A few months later we were told – he might be able to get on the medication by middle school.

Which then became elementary school. And then WHAM. The results were far better than anyone could have ever imagined and after the studies were completed it was crazy fast tracked by the FDA and it looked like he’d be on it by preschool.

On January 31st, 2012 the FDA approved Kalydeco (you may see it called ivacaftor) for all CF patients with the G551d mutation aged six and older. At this time, Gavin was just a couple weeks away from turning three.

We knew that it would be awhile for the medication to get started in pediatric trials (ages 2-5) but had no idea it would be until August 2013! Yet, we are ever so thankful that it is available and Gavin is one of the rare few that are able to use the drug. Thankfully, because the medication is already approved by the FDA, the study would not have any placebos – but patients are still unaware of which dosage they are taking.

One of the delays was that the lab rats started to develop cataracts (still uncertain if this will also happen in humans). This became apparent after the medication was approved for those over six, but weighed heavily on the start dates of the pediatric trials. And then came the issue of, “who pays for the cataract issues if they do develop?” Of course no one wanted the responsibility. That is what kept our clinic out of the Part A section of the study – needless to say, I wasn’t thrilled that those “politics” were keeping my child off this potentially life saving medication and made my point clear to the clinic/research team.

After several potential start dates that kept falling through, we were told in July of this year that they’d start enrolling in August. Only one spot in our HUGE clinic was available and GAVIN GOT IN!

Yesterday morning, Gavin and I got up around 5:30 am and headed up to Amplatz Children's Hospital to begin his evaluation for the study – just to make sure he was “healthy” enough to participate.

I could not believe how well he behaved. Not only was he amazing at last week’s regular quarterly check up with his pulmonologist – yesterday he was happy, carefree and more than willing to do whatever the research team asked!

He got his vitals checked, followed by an EKG to make sure that ticker is working well.

He has had a slight murmur since birth, something both his pulm. and pediatrician said isn’t anything to worry about – but of course showed up on the EKG. It doesn’t exclude him from the study, but they want him to meet with a cardiologist at his next appointment in September and possibly needs an ECHO, will update on that when I know more. Nothing needed to “fix,” they just are covering all their bases – as many of you know, every t has to be crossed when participating in a research study, especially one this big!

A lot of the day was waiting. Waiting for this doctor. Waiting for this test etc. And the research coordinators kept complimenting him on his amazing behavior!

Following the EKG, we went to get his blood drawn. He whimpered – but only for a moment. THANK GOODNESS for that numbing cream! It sure is a life saver! He completed a urine sample and then had a sweat test done. Gavin last had a sweat test completed when he was an infant (just a few weeks old) – it is a diagnostic tool for CF.

To make a long story short, a chemical is placed on a small patch of skin which “forces” the skin to sweat. After a half hour that sweat is measured – and the results indicate whether or not that person has the disease. A high number (over 50/60) indicates CF, but the number does not indicate the severity of the disease. Many adults on Kalydeco have seen a HUGE drop in their sweat test numbers, some even into the normal range! Basically, it shows that the body is able to function more normally, producing more normal results.

In the midst of all of this, a pulmonologist from our clinic that is running the study stopped by to do a quick evaluation. I’m sure the poor man thinks I’m a crazy mom – I had never met him in person before, he only “knows” me as the incredibly angry mom that want on several email rants when our clinic didn’t get into Part A of the study. (Hey, when you’re at one of the best clinics in the world you expect only the best care – and when that’s not there mama bear comes out to play!). But nonetheless he was extremely friendly and great with Gavin – so even if he thinks I’m nuts at least he’s great with my child! That’s all I ask for!

Finally, we headed across the street from the hospital to the ophthalmologist. Because those darn lab rats began to develop cataracts, part of the study now includes extensive eye exams. This doc had to go through special training just for this one study, so we are eternally grateful for her willingness to help! She was FANTASTIC. (If anyone in MN is in need of an ophthalmologist for their child, I am more than happy to pass on her name!) He went through an array of tests – and his only complaint of the entire day was the eye drops causing his eyes to dilate.

I think that Gavin will talk about this part of the study for a long time. They were a fantastic crew and they actually made the eye exam fun for him.

And I couldn’t be more thankful than the two study coordinators that trooped through this nearly six hour day. They were so sweet with my son, and so unbelievably friendly. Just when I think I couldn’t love our clinic more, I meet even more of the team and become more and more impressed!

At the end of it all, Gavin and I stopped at Perkins for pancakes to celebrate a job well done. I asked what the worst part of the day was. Two things (1) the eye drops (2) his own pulmonologist wasn’t there. I mean seriously?!? What kid gets bummed about not seeing their doctor, we are so blessed to have her!

I am not allowed to publicly reveal any results of the study – but I am able to let close friends/family know. Basically – they don’t want anyone going on social media blaring the results before they are analyzed and published. But if you really want to know, you can message me personally and I can let you know how things progress.

All in all, it was a long but successful day! Just a couple more weeks until his first dose!

Those Moments

Before becoming  a parent, you see images/clips/tv shows/movies etc. of parents gushing over those “proud parenting moments.” Before kids, we can all live to imagine what that must feel like, how it must feel to see a part of you succeeding.

But nothing can prepare you for those moments. Whether it’s a first step or a gold medal, watching your heart and soul accomplishing milestones is hard to describe.

One of my favorite characteristics of Gavin is that whatever he tries, he gives it his full attention and dedication. Nothing is ever done halfway with this little guy – like his mama, it’s all or nothing.

Since getting his bike tire fixed, getting Gavin off his bike has become a challenge. Every free moment is filled with, “can I go ride mama? please?” We luckily live on a dead end street and in the last 48 hours I’ve lost track of how many laps he has pedaled. As he crosses the street to enter our driveway (after cautiously looking both ways) I immediately hear, “AGAIN!” And either Casey or I have to say, “Ready….set…go!!!!” And he’s off, pedaling as quickly as those little legs will take him.

If this keeps up, I’d be willing to bet he’ll toss those training wheels aside by the end of the summer. It’s more than fun to watch him enjoy this new sense of freedom, and nothing is better than spending the last few minutes of dusk (in a season before mosquitoes) watching him ride and listening to his little voice singing happily as he circles the block again and again.

Gavin is also finishing up his first year of preschool. After swallowing my fears (and pride) of sending our kiddo with some extra needs into a place I can’t control – my fears were quickly diminished. In this last year school year, Gavin has thrived. He loves making new friends, learning letters/numbers/simple math, and getting some time away from the chaos of our home. He was blessed to have two of the best teachers I could have asked for.

When you see someone everyday, it can be difficult to see the physical changes happening right before your eyes. While I knew he had grown since his first day, seeing the images side by side really show the difference the last few months have brought.

 

The baby face of three has disappeared into the “bigger” kid looks of four. We couldn’t be more proud of the kid he has become.

Catching Up

Finally. Spring.

After a snowstorm at the beginning of May left us with 15.5 inches of snow – we’re finally past one of the worst winters I can remember.

Mother nature really messed with our heads this year

Tuesday, April 30th

Thursday, May 2nd

Sunday, May 5th

Yep, shoveling snow in shorts. That pretty much sums up the last month or so!

In the midst of this we had our FIFTH Great Strides Walk! Once all the checks are deposited, we will have raised nearly $7,000! Thank you so very much for your generosity! We feel very blessed to have so many people fighting alongside us to keep our kiddo healthy and strong!

This year Gavin really understood that the money we raised was for him – and as much as it breaks my heart that at 4 he already understands his life will always be a fight for health, it makes me proud that he is becoming just as determined as we are to work hard and raise awareness.

It was far from being the warmest weather we’ve walked in…

But we trudged through the slush & snow! Another successful walk in the books!

And thanks to a group on Facebook filled with fellow CF mamas – I became aware that this little guy was “free” for our our family since Gavin uses the Hill Rom vest. Gavin named him Button, and loves that he’s no longer the only one with a vest in our house!

At four, he’s already beginning to learn to read. He’s working hard on sounding out the letters and trying to come up with the word. I love listening to him in the evenings as he lays in bed (after we’ve read him his stories) trying to reread some of his favorites on his own.

He’s fascinated with simple addition & subtraction problems and when watching a sport on tv he can quickly figure out who is in the lead, by how much & what the other team needs to do to catch up/win.

His imaginary friend, “Brainy,” (quite the name I know, he must be smart!) makes an “appearance” fairly often these days. Gavin loves to pretend that he’s playing sports with this buddy and talks about him often. Thankfully we haven’t had to set a plate for Brainy at the table! (yet!)

Getting him back inside when he’s playing outside is always an adventure in itself. This kid loves the outdoors – especially when he’s playing some sort of sport. He still loves to drive his John Deere tractor around the yard, peddle his bike around the cul-de-sac, play in the sand at the park  and swing on our swing set in the backyard.

As much as he doesn’t like doing his vest he rarely complains about it. As long as he’s got the cartoon of his choice on tv, he calmly & bravely sits for each thirty minute session!

And it continues…

We’re over it.

Winter, that is.

Nearly every morning, Gavin asks me if summer will be here by bedtime. Once again, on the 18th of March, we’re in the middle of a winter storm. A bunch melted over the weekend, just to get replenished today.

Yech.

In December, the first sprinkle of snow brought out all the neighbors – almost as if we’re racing to be the first to clear the driveway. By mid-March? Well, it’s 3 in the afternoon and I don’t think anyone on our block has touched a shovel. Looks like we’re not the only ones over it!

I hate to complain about the weather, as we do love living in a state with four distinct seasons, but too much is too much. The grey skies and never ending snow has put all of us in a funk.

But in typical Gavin fashion, he made the best of what we have and we headed out to build a snowman. We completely cheated this year by buying a snowman kit but when we only have one teeny little tree (no sticks for arms!) and wood chips for landscaping anything helps!

picture perfect snowman


that even got a hug…


and a kiss!

Enjoying the snow

 

What a bizarre winter this has been! From rain, to sleet to snow – and don’t forget the ice! It took until March to get a combination of enough snow on the ground and weather warm enough to enjoy being outside for more than 10 minutes. I kid you not, In January the daycare kids had out our summer bikes/toys as we had no snow! Anytime we’d get some on the ground, it would rain – bummer for the littles in my house needing to escape the house!

The last 24-48 hours brought about 10 inches of the fluffy stuff and we’ve been loving every second of it! It took a lot of convincing to get Gavin to come inside yesterday evening, even after being outside for nearly two hours.

Yesterday brought back a lot of childhood memories. Helping shovel the driveway. Digging tunnels in the large snow piles. The excitement of enjoying the outdoors trumping the wet and cold winter clothing. The shrieks of neighborhood children combined with the loud whirring of snow plows clearing driveways and sidewalks.

The news may have “complained” about digging out – but honestly our street was full of laughter and joy. Even amidst the piles of the snow, the warmer weather and fast melting snow was proof enough that spring is coming quickly – the end of cabin fever is near!

As ready as we are to no longer need 4o minutes to get dressed warmly enough to head outside to play, we’re enjoying what is here now and soon enough we’ll be sweating and cursing the heat, wishing for a quick relief of cool weather!