Sunday, February 12, 2012

Baseline

Thursday afternoon, Brady’s Kalydeco was delivered!  We were on the road making our way home as quickly as possible from Arizona, so my parents came to our house to receive the delivery for us.  They were waiting with balloons, champagne, and Kalydeco when we finally arrived home!  The car trip home was fairly torturous.  We just couldn’t drive fast enough!  I could FEEL that the medicine was on its way to our house.  Thursday night, all I did was hold the bottle in my hand and stare at it.  Lots of tears mixed with sheer disbelief.  I’ve been watching VX-770 with an obsession since Brady’s infancy.  The chemist in me has always been impressed with the pharmacological possibilities of VX-770…the mother in me has always been desperate to help Brady escape the inevitable consequences of having cystic fibrosis.  For several hours, I think I really was in a state of medical shock.  My whole body felt numb, heart-rate was racing, clammy hands, and light-headed.  I didn’t get much sleep that night.  I was afraid to put the bottle down.
Meeting my new best friend Kalydeco
Friday morning, we went to Spokane Sacred Heart Children’s Hospital for a sweat test.  This test is typically used to diagnose patients with cystic fibrosis.  Brady was given a sweat test when he was 2 weeks old after a positive newborn screen.  They weren’t able to collect enough sweat off his skinny little leg, and the test came back inconclusive (so we had no previous sweat chloride data).  We moved on to the genetic panel, which is how he was actually diagnosed at 3 weeks old.  Brady also had a blood draw on Friday to establish baseline liver function.  We came home for lunch and then returned to the hospital later that afternoon to see Brady’s CF doc.  The Dr. typically only sees CF patients on Thursday, but he made an exception to see Brady so he could get started on his new medicine before the weekend.   I’ve never been happier to see Brady’s Dr. and nurse.  I shared a couple of the best hugs ever with them!  Then he examined Brady, which is what I really wanted to highlight in this entry. 
Brady's CF Doc
Brady’s Baseline
Brady is probably not your typical 4 ½ year old CFer.  He still has clean cultures, a completely normal chest X-ray, and has NEVER had a cough.  I mean NEVER!  He has only had a few minor colds in his life that never settled in his chest.  He has been on Pulmozyme since 6 mo. of age, and Hypersal since 20 mo. of age, completely as preventative treatment.  We can usually get him to cough a few times at the end of his Hypersal treatments…and then no coughing for the rest of the day.  Brady’s growth has also been exceptional for a child with cystic fibrosis.  This visit was his best ever, 75th percentile for weight (for age), and 90-95th percentile for height.  Brady’s biggest issue with CF at this point in his life has been the involvement with his sinuses.  He has aggressively growing nasal polyps that required surgery last October.  Within 2 months of his sinus clean-out, the polyps had begun growing back.  He has been through several Prednisone bursts since his surgery to slow the growth and help him breathe through his nose.  When his polyps are at their worst, he can’t breathe AT ALL through his nose.  He can’t smell, and loses his appetite.  He also snores like a drunken frat-boy and often snorts himself awake at night.  He recently started a new therapy for his sinuses; a nasal atomizer called the “Nasatouch.” (http://www.sinusdynamics.com/video-atomized-sinus-therapy)  We have been irrigating his sinuses with a steroid solution (Betamethasone), 3X daily to keep the polyp growth to a minimum.  We think this is helping, but it hasn’t completely solved the problem.  We are going to schedule an appointment with Brady’s Ear Nose Throat specialist next week to touch base with him since returning from our vacation.  It is very important to us to collect as much clinical data as we can on how Kalydeco is working in him.  Since he is already on the drug, he obviously won’t be participating in the clinical trial for 2-6 yr. olds starting this summer.  Still, Brady is in the National CF Registry, so we are trying to design the first few months of his treatment similar to the trial so we can add some meaningful data to the registry. Also, the insurance required a “pre-authorization” for the Rx.  When the authorization period has expired (they authorize for a specific length of time.  We are calling Monday to find out how long Brady’s Kalydeco is authorized), the insurance company has the option to re-evaluate the medical necessity of the claim.  Collecting lots of clinical data on how it is affecting his body will help us ensure that his claim will be approved again in the future.    The prescribing info on Kalydeco recommends repeating blood work every 3 months for the first year of treatment.  We are going to check his liver enzymes again in 1 month, just to be completely safe.  Also, we are going to repeat the sweat test in 2-3 weeks.  For this to be meaningful data, all other variables must be held constant—so his CF treatment will stay exactly the same…for now(except the addition of Kalydeco). 
Meal where he took his 1st dose
Baseline Stats: Brady's Genetic Mutations are G551D and DF508


Cough: None
Chest X-ray: Normal
Sweat Chloride: 105
Liver Enzymes: We don’t have results yet, but in his last blood draw (3 mo. ago), enzymes were normal.  I will report the actual numbers of this draw when we get them.
Weight: 47 lbs
Height: 42 ½ inches
Blood Oxygen Saturation: 99%
Sinuses: Aggressive polyp growth
Pancreatic Function: Takes 3 Zenpep (10,000 lipase per) with meals, 1-2 with a small snack. Brady eats super slowly also, so I occasionally open a pill and give him a few extra beads (4-5) in the middle of a meal if he is eating a lot of fat and taking FOREVER!
Breathing Treatments and Airway Clearance: Morning--Albuterol and Hypersal via nebulizer followed by 20 minutes on his VEST. Evening--Albuterol, Pulmozyme, and Hypersal via neb.  followed by 20 minutes in the VEST.

Meds: Source CF vitamins, Prevacid, Singulair, Ursodiol, Periactin, Zenpep, and Kalydeco! Brady does an occasional round of Prednisone and Augmentin for his sinuses, but is currently not taking either one of those.

Forehead Lick: Salty.  I know, real scientific right?!  I just want to see if I can notice his saltiness going away.

I’ve seen a lot of questions about what will happen to the regular treatment regimen when patients begin Kalydeco.  My opinion is that each patient will be different and have to make those decisions with their Dr., based primarily on how much permanent lung damage is present.  Kalydeco can’t fix scar tissue, which many CF patients have a significant amount of in their lungs.  My hope for Brady is that he will be able to stop his breathing treatments and Vest one day in the future because he still has clinically normal lungs.  We are going to move very slowly and didn’t even get into too much detail with his Dr. about this yet.  We are determined to stay the course for now, re-test his sweat chloride and blood work soon, and then go from there.  My guess is that one day we will begin to taper him down off of treatments.  For example, he does Hypersal 2x a day right now.  Maybe we will drop down to once a day for a while before we stop it altogether.  Same with Pulmozyme and Vest.  I honestly have a hard time imagining the day when we don’t wake up and do his treatments.  I’m in no hurry to stop his regular treatments…but I also don’t want to have him doing hours of unnecessary breathing treatments if he has “normal” functioning lungs and functioning CFTR.  Brady’s Dr. told us that we probably won’t see much change in Brady’s lung health because his lungs are so good to begin with.  That is just music to my ears.  I have always had the dream of getting this drug to Brady in time to truly offer him a “normal” life expectancy.  I believe that dream is coming true right now and I couldn’t be happier.  I COULDN’T BE HAPPIER!  So far, Brady has taken 4 doses.  The only changes I've noticed are that he seems very energetic and is eating less.  He says he feels "very good."  This morning he had a huge bowel movement.  All sinkers, which indicates he is not passing a lot of fat into his stool and digesting pretty well. 
I want to conclude this post by promising to report the details of the changes we see in him physically and in his labwork as we go along.  Someone asked me for the link to our Great Strides fundraising video from last year so I watched it again this morning.  I cried my eyes out thinking about how we felt the day of his diagnosis compared to how we feel right now.  I didn't think I could ever feel this good again.  Take a look at how much has changed for us since last year... Keep working and hoping.  Anything is possible.

5 comments:

  1. I have a son who is almost three and has the same CF mutations that your son has. I would love it if you could contact me so I can ask you some questions. How do I do that?
    Stacy

    ReplyDelete
    Replies
    1. my email is rebecca_schrdr@yahoo.com

      Delete
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