New normal

For the trial schedule, some appointments are harder than others, only a few sweat tests left, some have pre- and post- drug bloodwork, some don't have an LCI which makes them shorter, hopefully. Here are the dates:

Screening- November 8

Day 1- November 15

Day 15- November 29

Week 4- December 12

Week 8- January 9

Week 16- March 6

Week 24- last visit May 1

Week 25- Safety follow-up- May 12

There is a potential for a follow-on trial, up to 96 weeks or until FDA approval. It is sounding like, when our trial ends, so does the supply of the meds until we can get it through insurance after FDA approval, so that probably means, we're in for the follow-up trial so we have continued access to the drug which means more appointments, but the nurse expects them to be quarterly.

I have a packing list that I wrote up when Fawkes was a newborn and I traveled with him a lot. It has evolved over time. It has all his medications, equipment, and miscellaneous things I can't forget. Today, I added the new drug. It's great, and yet another thing to do.

Oh, a funny requirement of the trial is I have to keep the empty packets and return at each appointment. A year of the drug retails for $259,000. That means each one of these babies is worth $356 each.

So, the requirements for Fawkes every day, just the basics, now consist of the following:

Breakfast:  Nebulizer, Vest, Zantac, Creon, Vitamins, New med

Lunch:  Creon

Snack:  Creon

Dinner: Nebulizer, Vest, Zantac, Creon, Vitamins, New med

On top of it all, I scheduled a gastro doctor appointment since we've been having such trouble with his digestion. The first new patient appointment was two months from when I called so I took it. Of course, it fell in line with Week 4 for the trial, so I had to move it. In the midst of it all, we still have to go to our regular quarterly CF appointments at JHU too. We'll have a lot of medical mileage to claim on the FSA. Yay, another thing to file.

Last thing, Grapefruit, seville oranges, tangeloes, orange marmalade, or other grapefruit cousins interfere with the medicine's effectivity. I think we'll just tell people he's allergic; probably easier.

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Day 15

We had a great thanksgiving and did all the Christmas decorations. Last year, I didn't get it all done, so I was determined this year. It was really fun too because Fawkes was helping. We have an abundant amount of ornaments about 15 inches off the ground which is adorable. Fawkes picked out a few favorites too. The tug boat. It's from one of those golden books.

Here we go again to Philly. It's a "quicker" appointment this time. But, it's still a trip. That's the thing about these visits; life doesn't stop for us. There's no break. Just constant CF responsibility. Enzymes, zantac, vitamins, Orkambi, secure the packet, treatments, eat high calorie meals, prescriptions, medical bills, FSA filing, pack the car, get McDonalds to get on the road, save the receipt to put in the expense folder, on and on. It doesn't let up. Oh, and get food, do laundry, go to work, more treatments, more medicine. I would love a break. Sigh. That's not in the cards for us.

We got in late Monday night. We work during the day, do afternoon treatment, pack the car and head out which puts us eating dinner in the car and at the hotel by 8:30. We basically shower and go to bed. Get up next morning, unpack vest, do vest, repack vest, repack everything, eat breakfast and head to the hospital.

This appointment was vitals, EKG, stool sample (oh so fun to collect and transport), spirometry (the skateboard test) pre- and post- blood draws. I was already anxious about this appointment for the two blood draws and had 2 gifts ready. It was terrible. For the pre-dose blood draw she had to poke him 3 times to get enough blood. Poor Fawkes was screaming. It took 3 of us to hold him down and get it done. After they took out the needle, I had an overwhelming need to cry a bit, so I grabbed Fawkes and both of us went to the hallway to get some air. He didn't like that I cried, but it hurt me that he hurt. So bad.

Then we took the drug and ate an early lunch (since he has to take the drug with a high fat meal).  Then, it was waiting. We did our mask treatment that we skipped so we could do the breathing test, played puzzles, colored, all the fun you can possibly have in a hospital room. They came to take the post- blood draw and this time it took 2 pokes. Ugh.

Hudson had heard great things about the Franklin Institute and a new dinosaur exhibit just opened. We made our way there only to find the exhibit was at max capacity (which was just as well because it would have been $100 for all of us, and both Fawkes and I desperately needed a nap). We checked out the gift shop and found a battery-operated train. Fawkes had been wanting one around the Christmas tree. I know I'm spoiling him, but it's so hard not to when he has to go through so much that other kids don't even have to think about.

We went home and both Fawkes and I napped in the car while Hudson drove through the gloomy rain. Got home, unpacked, did treatment, ate some food, and got ready for the next day. Back to work. Next time we are staying on Tuesday night too.

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Day 1

All week we were on pins and needles. Would his results come  back within limits, could we keep him well. Could we get him in this life-altering, life-lengthening trial?

Turns out, yes.

As I explained, we never knew what his sweat test results were because our initial collection did not yield enough sweat to send in. The sweat test is the gold standard for CF. Anything over 60 is CF, His was 111; about as high as it gets.  Ouch, still hurts.

Oh, in the midst of it all, I'm supposed to put scar cream on his dishwasher rack cut 3 times a day. I'm lucky if I remember to do it twice.

Some preparations we have to do are urine and stool samples 24 hours before the appointment. Not fun. Also, you have to think about the logistics and transport of said samples which were tripled bagged, frozen, and kept in a separate cooler in the car.

On our way, Fawkes kept asking about the bell. He remembers it was cracked. And he doesn’t like the red and black things (referencing the terminals that induce sweat), and the pinch (bloodwork), but the blue thing (sweat collector) is ok.

It's all worth it. Tomorrow we get our first dose. I might cry when it's in my hands, but probably not until later.

 

Hudson and I both worked today, and so I got in the 2nd treatment, then we packed the car, hit McDonalds drive through, and got on the road around 6. After eating, Fawkes watched Cars the whole way, and I typed all these posts. We got to the hotel, unloaded, took showers, and went straight to bed.

I got up at 5:45 to get myself ready; Fawkes woke up at about 6. We did just his vest. The nurse asked us not to do his mask until after his Spirometry and LCI tests because the study wanted to get his breathing values before albuterol. I refuse to miss a treatment, so we packed up the compressor and masks and did it after his breathing tests at the hospital. We went down for breakfast and got in the car at 7:45 to head to the hospital. We knew where we were going this time and what to expect so it was a little easier.

Started off with vitals, height weight, temp, pulse ox. Easy stuff. Did the EKG, surprisingly Fawkes did not want to be hooked up to the wires even though you don't feel anything. I had to put on Lidocaine since we were about an hour out of the bloodwork. Then we went and did the breathing stuff, LCI and spirometry. It is incredibly difficult to make a toddler stay still, breathe normally, and not fidget. We had to do 7, 5-minute tries to get 3 acceptable runs. You have to wait 3 minutes between each try since he's breathing pure O2. Then came the skateboarder test. Another very difficult activity for a 3 year old. Most of the time, CF patients don't start until 6. He gets really silly and doesn't always blow into the tube for long enough or at all. I think we did 10 tries and only got 2.

Then we had to head back and do the dreaded sweat test. He started kicking and screaming. It took 3 of us to hold him down to connect the leads. He cried the whole time it was on, about 5 minutes. We opened a magic marker frozen book while the blue collector did its job.

Around that time, Fawkes said he was hungry. We started lunch, and a half hour later we gave him the first dose. It has to be taken with enzymes and a high fat meal; so it requires coordination. It's a bunch of white powder we put in ~5mL of applesauce. 

The doctor came in and listened to his lungs, looked in ears, nose, and throat, and signed the EKG papers. Soon after we did bloodwork. This was of course not fun either, but I think has taken 2nd place to worst activity after sweat test. He opened a new game, Kerplunk which I thought he would love to hear all the noisy marbles fall. Turns out, this game was well worth the $9 I paid for it. He literally played with it on and off all day and night. We did his mask while he played Kerplunk, and at this point, all the hard things for the day were done. We were in a 4 hour observation period now. Hudson and Fawkes went on a walk for 20 minutes and I laid down and closed my eyes. The nurse came back and we did another skateboard test. I think he did it 5 times and maybe got 1 good one.

By doing the LCI, we were helping the technician get her official qualification to run those tests, so she gave us a button that said, "I am the key" and $25; that will be going in his piggy bank.

Then, more waiting, we did puzzles, action plates, more Kerplunk, a Finding Dori coloring packet, more Kerplunk, and finally did the last spirometry. He was delirious at this point. I think it was 3:30 and he hadn't napped. We did about 5 tries with zero good ones. We packed up and left. Fawkes fell asleep in the car on the way to the hotel which was only about 10 minutes away. Hudson went to get us dinner while I did a little work. We ate and watched a little TV then Fawkes woke up. He was hungry; he wanted popcorn, an Icee, and fruit snacks. This is a snack pack that Hudson had got him at the movies a little while ago. So, we got dressed, walked 2 blocks to the movie theater and got him what he wanted since it was such a hard day. We put lots of butter on the popcorn and gave him yogurt and his second dose of Orkambi. Not the best dinner, but there's only so much you can do. Then we had to do the 2nd treatment of the day, showers, and get ready for bed.

While Fawkes was sleeping I caught up on filing for our medical FSA. I pay our medical bills and turn around to file for tax deduction through our FSA. We max out the $2550 per year, so it's worth my time to keep up with all of it as we incur the costs. There'd be no way for me to keep all the receipts and do it at the end of the year. So, now I'm caught up from July through October with 2 CF appointments, an ER visit, a pediatrician visit, labs countless prescriptions (at least 3 per month). Phew.

Our expenses, hotel, mileage, tolls, and food are covered by the drug company. It's awesome, but it's yet another thing to organize, collect receipts, and turn in. Additionally, Fawkes gets paid for each of the tests he does. So, I guess this is his first job (spoiler alert: he had to pay $86 in Federal taxes).  They apply all the money to a debit card. We plan on putting the money he's earning into a stock account for Fawkes.

Next morning, woke up at 6:30, treatment, breakfast, and got on the road by 9. Hudson and I both have meetings at 1:30. Quick trip this time; returning in two weeks; back to reality?

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Screening

We packed up and on the way he said, mommy why are we going to Philly. I told him we had to go to the dr. He said are they going to give me a shot. No shot buddy, What about a pinch?  I don't know. We'll see. Why do I have to go to the dr mommy? That was it, tears were streaming down my face. It is so unfair, he has to go to the dr and do all these treatments, and the stress of trying to put all this together and the unknown and the trail becoming a reality. It was only 2 min and I was better.

We stopped and saw the liberty bell, which was very influential for Fawkes. He now knows, we're going to Philly and he says, to see the bell? It's cracked. Everything was being prepped for Hillary and a huge show the next night for the day before the election.

We went to dinner at a fancy college bar, but no bar like GT or UF. UPenn is Ivy League. I did not realize. Then we looked up tuition, wow, so expensive.

We woke up the next morning, did treatment, ate breakfast at the hotel, then packed up for a 6 hour dr appt. We wanted to go to Phil museum of art and do the Rocky thing. Turns out the museum is closed on Mondays, but we really didn’t have time anyway. We ran up the stairs, threw our hands up, listened to eye of the tiger, then went down and posed next to Rocky, himself.

Our nurse went through the consent form page by page to ensure we understood what we were getting into.

I signed it.

I had to.

For my baby.

Started off with vitals, easy, blood pressure, temperature, height weight. Reviewed medications, so annoying I need to just make business cards.

Did an EKG, also ok. Fawkes liked the stickers, He wouldn't let me take them off. A doctor came in, look in ears nose throat, listened to lungs, also pretty easy. I think we  ate lunch. I brought him a lunchables, it's amazing, it has a drink and a Reese's peanut butter cup all included.

Then, it got harder. The sweat test. I have bad memories of sweat test. We tried to do one of his earlier CF appointments when he was a month old or so. They had done bloodwork and took way too many vials from a newborn (and then didn't even use 2 of them), and then put these little electrodes on an already upset newborn and wrapped his arm with cellophane. Afterward they put on the sweat collector and he was screaming the whole time. Finally the time was up and they looked and not enough sweat had collected, it needed 2 spins, so they didn’t even turn it in. All that for nothing. Anyway, the sweat test consists of hooking up red and black leads to his arm to stimulate sweat. It's a definite current, and Fawkes immediately did not like it. It was 5 minutes; might as well have been an eternity. Finally she disconnected them and put on a blue bracelet with a plastic coil. It stayed on his arm for 20 minutes to collect sweat. We got enough to complete the test. It was so awful, I gave him his little gift for the day. It was a game of go fish. I tried to teach him to play, but instead he sorted all the cards into like piles. Such an organizer; just like mommy.

Breathing LCI- He gets hooked up to a mask that blows ambient air and then pure oxygen. Here's the explanation, "This test determines how well your child’s lungs are functioning. For this test, your child will be required to wear either a mouth piece or a mask while resting comfortably and breathing at rest. During this test your child will breathe in room air for a short period followed by pure oxygen. Your child will not be able to detect the difference between the room air and pure oxygen." They collect his output ratio. We had to do it 3 times. At first Fawkes was holding his breath and not breathing regularly, he's a toddler, not CF-related, so we had to do it again. We had Thomas the train on so he went in zombie mode and we got a great test in. the 3rd test was not as good, then the 4th test was ok.

Had to walk back to get dreaded Bloodwork. We call it a pinch. An hour before I put lidocaine on his arm and a dressing which is supposed to numb him, but it never works. I hold him down while he screams for what seems like forever. While the blood is collected, I show him the big present and open it to try and distract him. They were action plates, like fashion plates but with super heroes. It turned out this was a great idea because next we went to get an eye appt.

Something happened  and we were not scheduled an appointment and no kidding, there were probably 20 people in line. Luckily, when you're in a trial that brings in a bunch of money for your hospital, the coordinator has some people she can call.  We still had to wait for 45 min, but we made a lot of superheroes. He went back and was so amazing. At first the lady gave him a laminated sheet with a T, H, O, and V. She asked him to point to the letter he saw on the screen of various sizes. After the first letter he called out, T, then O, then H, she threw the sheet aside and just did the exam so fast. We only paused when V came up, it wasn't because he couldn't see the letter, it was because I said it was a hard one, but he got it! A few other follow the sticker tests, and then dilation drops.

While the drops needed to take effect, 30 min, we went and did PFTs. Now, these are hard. Usually, CF patients don't start these until 5 or 6 because it's a very specific test. They put a clip on his nose and have him practice with a birthday noise maker. Then, you have a screen with a skateboarder that has to get across a gap. At this point, it's late in the afternoon, Fawkes hasn't had a nap which he normally takes by 1230 and he's doing something really difficult. He was being silly and blowing through his nose, or not putting his mouth on the thing. And all the adults are tired too, and we were about to quit. I had already taken him out of the room to give him a pep talk, and he did it. We needed 2 more. He did them in a row. We all cheered, and he was so happy.

Then we went back to the eye place and they checked his pupils. That was it, then we could get all our stuff and go back to the hotel. The little guy was exhausted. So were we. She sent us home with a toilet hat so we could collect urine sample and turn it in the next day. Fawkes did not like to pee in the bowl which is funny because he just quit doing that 2 months ago.

The hotel has a free happy hour and appetizers. I went down and had glasses of wine. I went back up to the room and just bawled for about 10 minutes. It was so much bottled up from the day and weeks prior. All the anxieties, the unknown, the stress.

Hudson went and picked us up some food. We did treatment, took a shower, and went to bed.

Next morning, treatment, breakfast, packing, decided to head home and call it a day. We dropped off a urine sample, goodbye Philly. Be back soon.


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Bloodwork #1

Today began blood work 1 of 9 for Fawkes in the next 6 months. Clearly, my subconscious was anxious because last night I had a dream both Fawkes and I had our molars pulled out at the dentist. Losing teeth is a classic dream dictionary troupe about losing control. That's certainly how I'm going to feel starting these next 6 months.

We are desperately hoping my son gets accepted to one of the three spots available for an event I can't fully disclose until it's finished, in Philly. The Baltimore location where we normally do our CF appointments is not participating, so we are making the trip to Philadelphia. We plan to make some fun on the Rocky steps and the Liberty bell, but each appointment will be 6-8 hours long, with blood work, and other assorted poking and prodding of my toddler. There's a requirement that he not have respiratory infections or be on antibiotics 28 days before day 1. If we don't get in, we have to wait until medication is approved by FDA which could be over a year away. My son needs this potentially life-lengthening drug to slow the progression of his terminal disease. The stakes are high; we are not attending any birthday parties for awhile.

A CF mom blogger I follow gave a metaphor that she went into "Roly-Poly" mode recently, where she said she did the equivalent of balling up and ignored anything that wasn't critical to maintain day-to-day. She was overwhelmed by the procedures her CF son was enduring. If she was in roly-poly mode for a few weeks, then this blog is evidence I've been in more of an Arctic Woolly Bear Moth mode. After three years, I'm finally ready to start writing things on a blog, much like that moth that spends around seven years to build up enough resources to finally pupate into the adult moth. Look them up, they're seriously incredible. I'm not quite sure if it's narrative, educational, or cathartic, but I have so many thoughts and things to say about CF to try and explain, so let's begin.

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Prepping

We went to his Quarterly CF appointment without a hitch. Then, Hudson got the email we've been waiting for; we could sign up for a screening appointment. The drug is approved for 6 and up, and we wanted to get Fawkes on it as soon as we could in his lifetime. If we waited for FDA approval, it could be as late as his 5th birthday. We believe that science is the only way to combat his disease. Reading the consent form was a terrifying venture. As a mother, what am I signing him up for. So much testing, so much blood work, so many appointments. Eight visits over 5 months, but front loaded. 4 visits in the first month! Our normal JHU in Baltimore wasn't participating, so we had to go to CHOP in Philly. In the consent form, you see all the risks of the trial. It was like one of those medicine commercials where it could cause this or that.

The scariest thing is risk of developing cataracts. So what am I doing, trading his sight for years of life?

You can correct cataracts right?

Reward far outweighs the risk; we have to do it.  While we were discussing the paperwork, Fawkes decided to take the dishwasher rack and push it like a train. It stopped, his momentum didn’t and well, we landed in the ER

He developed a fever, think it was from flu shot but took him to doctor anyway since he had the suture glue.  I had to do it anyway for his annual bloodwork for CF. I had since decided to make that a separate appointment at our pediatrician because it's too long at CF appointments in the hospital and I can run him up to our doctor and get in and out. They won't do labs without a visit. So I took my JHU orders and did the whole 2 birds things. I knew ahead of time he would not like it so I ordered a companion jewel book to one he and I sing every night before bad. I wrapped it to distract him. It didn’t really help. It's bloodwork. I don't even like it.

For the screening I had to Request paperwork. Which meant I had to sign a release for JHU to send it to CHOP. We saw genotyping results for the first time. We knew the outcome, double delf508, but seeing it on paper still hurts your heart.

In order to get in the trial you have to do a screening and make sure certain things are within acceptable limits. The biggest thing is he cannot be on antibiotics or sick 30 days before day 1. He had just been on antibiotics 2 times in a row; how was this going to be possible. No birthday parties, no indoor playgrounds, limited interaction with kids and people, bubble time. In the midst of all that, it's the holidays; how will this can we keep up with work and life and this trial.


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