We had an appt. with Brady’s CF Dr. this morning. It has been 2 whole months since he began
Kalydeco (took first dose Feb. 10th). Before I get to all the juicy details of our
visit, I just want to mention how much different I feel about going to clinic
now, vs. pre-Kalydeco. Most CF parents
can probably relate to the idea that clinic can be a scary place. Besides the risk of cross-infection from
other patients, for me--there was always the fear of what I might learn about
the progression of his disease. Is the
throat culture going to show a new bug causing problems? What new meds will we be adding this
time? Is a hospital visit in our near
future? The list of potential CF
complications is seemingly endless. I’ve
always had a lot of anxiety and fear surrounding clinic visits, but everything
feels different now. Before, I would
spend the week before clinic pacing the floor, not getting much sleep, and
trying to manage my fear. I’m not going
to lie…I’m still not getting any sleep, but it isn’t because of the fear and
worry. It is because I’m so freaking excited. It is because I CAN’T WAIT to see the clinic
team and discuss all the positive changes we have seen in Brady’s health. It is because the game has changed, and I don’t
know all the new rules yet, and my mind has just been racing with possibilities
for the future.
Clinic Visit: Day 62 of Kaydeco for my 4 1/2 yr. old with G551D and DF508
Weight: 48 lbs. His
weight was the same today as it was 1 month ago. We feel totally fine with that because he is
still close to the 90th percentile weigh-for-age. Also, we have been adjusting his diet (not
covering everything he eats in butter, ½ n ½ etc...). I’m sure that his intake of fruits and
veggies has gone up, while intake of pure delicious fat has gone down. We will continue to work with the dietician
to figure out how to manage his caloric needs moving forward.
Pancreatic Function: One of the things we discussed with
both the Dr. and Dietician is the potential enzyme dosage adjustments that
might be needed with the addition of Kalydeco. We very slightly reduced his Zenpep dosage
after he first began Kalydeco, but have been holding steady with 2 pills with
snacks, and 3 with big meals since then.
Of course, those familiar with CF know that the poo is the window to
digestive function. Brady’s bowel
movements have definitely changed since he began Kalydeco—reduced to once per
day, less stinky, more formed, and always sink to bottom of toilet. I think patients and Docs have both been
pleasantly surprised with the increase in digestive function that Kalydeco
seems to provide. No one knows for sure,
but there are a few theories about why Kalydeco is associated with weight gain
in CF patients:
1) For
individuals with significant lung damage, breathing becomes a strenuous activity
that burns a lot of calories. For
patients who see a big increase in lung function with Kalydeco, weight gain
might be linked to the decreased caloric demands of less labored
breathing.
2) Kalydeco
decreases abnormal mucus production in all epithelial cells. Decreased digestive/intestinal mucus might
allow more effective absorption of nutrients and calories (ever change a snotty
CF diaper?—mucus can cause problems in the guts, just as it does in the lungs).
3) Pancreatic
function might be improved with Kalydeco.
The Dr. was really curious about what is going on, if anything, with
Brady’s actual pancreatic function--the ability to release digestive enzymes
into the small intestine where they breakdown nutrients for absorption. He explained that most people view pancreatic
sufficiency as a black or white issue, when it really isn’t that simple. Pancreatic damage likely occurs in a similar
manner to CF lung damage in the respect that it might start out as mucus
plugging and inflammation before it reaches the point of full blown
dysfunctional scar tissue and completely blocked enzyme ducts. The Dr. believes that pancreatic function is
more accurately described on a continuum of function (for example, some
patients might have partial function, but still need enzymes to keep stomache aches away. Others might have enough function to digest
without enzymes and don’t take them at all, but might still potentially benefit from a very
small dose of Zenpep etc...) There isn’t
a clear line between pancreatic sufficiency and pancreatic insufficiency. If this is the case, patients who are somewhere
in the middle of that continuum might be able to clear out some of the mucus
plugging and actually experience an increase in the amount of digestive enzymes
their bodies can naturally release—thereby reducing their need for enzyme
supplements like Zenpep.
Again, these are all theories and
individual patients will respond to Kalydeco in their own way anyhow. Patients in the end stages of disease might
see more of a weight bump from the increase in their lung function, while very
young patients might see a weight increase linked to improved pancreatic
function. I know that I’ve read several
accounts of individuals stopping enzymes all together after beginning
Kalydeco. I don’t see that happening for
Brady. His malabsorption issues began
when he was 2 weeks old (even breast milk was running right through him), and
he certainly has some permanent pancreatic damage at this point. Still, there have been a couple times that I’ve
become concerned that we might be OVERDOSING him a bit with enzymes now because
he seems a little constipated and has complained a few times that his tummy
hurts. With the help of his dietician,
our plan to figure out the correct enzyme dosage for Brady is to gradually and
systematically reduce his dosage, while monitoring his stools and weight. We are going to start by decreasing Zenpep
from his normal dose of 3pills/ meal, down to 2.5 and from 2/snack down to 1.5. We will do this dose for 7-10 days, monitor
the poo, and then do another small decrease if he is still absorbing well.
Chest CT Scan
At our March 8th
clinic visit, we discussed the need for a more detailed “roadmap” to guide us in
moving forward with Brady’s care, so we decided to perform a low-dose chest CT
scan. CT scan is the most sensitive and
detailed technology we have right now to measure structural lung disease. Previously, Brady had undergone annual chest
X-rays that had all presented “normal.”
He did a great job the day of the scan and even got to push all the
buttons on this huge cool machine! Brady’s
nurse called us earlier this week with the results. The Dr. and their favorite pediatric
radiologist had both reviewed the results and agreed that there was absolutely
NO VISIBLE DAMAGE. His lungs look
awesome! Imagine me saying that last
part with mascara streaked tears streaming down my ugly “cry face.” Writing about it just doesn’t do justice to
what an emotional basket case I’ve been for the last 2 months. I’ve
been dreaming about this possibility since I first heard of VX-770…and now it
has happened. My dream has come
true. His lungs are currently healthy
and he has Kalydeco working like magic in his body to keep them that way. Discussing
the awesomeness of the CT scan with the Dr. led us right into our next topic—what
do we do now? How do we move forward
with treatments and airway clearance for my little CFer, who has picture perfect lungs and suddenly functional CFTR? Will he eventually be able to "control" CF by simply popping a magic blue pill twice a day?
Breathing Treatments and VEST
This is the conversation that
gets my heart beating so loud that I can barely think over the noise. My hands are sweating like mad just typing
about it! To recap, we requested to
start Brady on Pulmozyme at age 6 mo. in the absence of symptoms in an attempt to
prevent lung issues. We requested and
began HyperSal at 20 mo. for the same reason--still with no lung symptoms at
all. In the 4 ½ years that we have been
doing these treatments, we have NEVER missed a single dose of ANY
treatment. I realize that I am nuts
about it. I don’t care what the
circumstances are, treatments are non-negotiable. One time, I thought I had forgotten to pack
the Pulmozyme in the cooler for a weekend at my parents place…I think I had a
small panic attack and was getting ready to drive 3 ½ hours back home to get it
before Brock found it. I mention this
because the idea of reducing or removing any of his breathing treatments or
VEST scares the crap out of me. I hope
no one assumes we are in any kind of hurry to see treatments go away, because
in reality, I have an irrationally bonkers attitude about it!
We just want to do the right
thing, so we hashed out all our questions and concerns with Brady's Doctor. One of the things I love most about his CF
specialist is that when he doesn’t know the answer, he will say so. EVERYONE is clueless as to the most
appropriate way to proceed with a case like Brady. Before we even got involved with our discussion,
the Dr. stepped out of the room to email some colleagues quickly, to
get their opinion about what to do with this new brand of CF patient like Brady.
In his words, “Brady is a pioneer.
He is the ideal candidate for this.”
This is all new territory. Dr. M
has a very collaborative attitude…I like it.
Another thing I love about Brady’s Doc is that he always asks for our
input and we decide TOGETHER how to best care for Brady. We concluded that our next small step should
be to further reduce his HyperSal treatments.
This sounds strange, but it seems to me like the Hypersal treatment has
become more irritating for Brady now than it was before he started
Kalydeco. Pre-Kalydeco, he would
sometimes cough once or twice during Hypersal, but often not at all. He has always breezed right through it like
no big deal. He still isn’t coughing,
but he is complaining of a sore throat afterward occasionally now. He is just acting like it is bugging him a
little, which he never did before. HyperSal
is used to temporarily restore some surface chloride, which in turn, increases
the “airway surface liquid” (ASL) on the lining of the lungs. This ASL allows those little cilia to beat--to
move and clear particles. I don’t think there is any doubt that this
treatment is effective, but can be a little rough to take. If Kalydeco is working like it is supposed to, his ASL is likely in very close to "normal" condition with Kalydeco alone. We decided that if a treatment was to be eventually be eliminated all together--HyperSal would go before Pulmozyme because it works with a totally different mechanism of action in the lungs. We think Brady might still receive more potential benefit from sticking with this drug for a longer time. Pulmozyme works by chopping up the long strands of extracellular DNA that bind mucus together in CF lungs. http://www.pulmozyme.com/how-pz-works.jsp
Baseline Feb. 10th: Brady used to do HyperSal (7%) twice a day.
March 8th: Last month, we dropped it down to HyperSal once
a day.
Today, April 12th: We
decided to move down to Hypersal 3X/week.
This doesn’t sound like much…but what it means to us is that tomorrow
morning, for the first time in over 4 years, we are going to wake up and NOT DO
ANY NEBULIZER TREATMENTS!! He normally
does albuterol to prep for HyperSal, so during the actual appt., I had
it in my head that we would be stopping the HyperSal some days, but still continue doing
albuterol. On the ride home from clinic,
it hit me that the Dr. may have actually meant no nebs at all those
mornings so I called the clinic back and talked to his nurse. She said “no need for the albuterol either”
that it was just to prep him for the HyperSal.
I had her repeat it to me one more time just to be crystal clear and
convince myself I was hearing correctly—no nebs at all in the morning on the 4
days he skips the HyperSal. “You might need to find some new hobbies,” his
Dr. told us. We are going to do this for
a month and see how it goes. If all is
well then…
The next step will be to decrease the VEST from 20 min. twice a
day, down to 15 min. twice a day. Baby
steps. We will re-evaluate again when we
see the Dr. in 2 months. My mind is so
blown right now. I swear I have been
watching this happen in my head for years.
I have been visualizing this exact miracle that is playing out in front
of me—intervene before irreversible damage has been done and give him an honest-to-goodness second chance at a long healthy life. It is like I am watching a super awesome Lifetime
Original movie starring MY FAMILY—a real tearjerker, but with a fantastic
conclusion. I’d like to say that having
Kalydeco has calmed me down a bit, but on the contrary, I remain firmly perched
on the edge of my seat. Every change is
so exciting. Every test result has been
so fantastic. I’m just amazed time and
time again.
Other Oral Meds: Holding steady with current schedule
Sinuses: Still totally clear with no steroid rinses or oral prednisone! The polyp growth seems to be a thing of the past! Amazing!! No snoring or mouth breathing, and he is making lots of comments about
smells.
I feel so unnaturally lucky that
I’ve begun having really irrational fears about other parts of my life. Everything is so perfect, something has to
give. Is Brock going to get into a car
accident?? Do I have cancer?? Where is the cat?? Kitty!! You get the picture. I’ve never denied that I am slightly
off-balance, and it just doesn’t compute how or why this is happening to us. I mean, treatments are one of the ways CF
parents “take care” of their kids. We
wake up every day swinging because every day with CF is a fight…but what do you
do when your enemy stops swinging back??
How long and how hard do we keep swinging when CF has retreated back to
its corner of the ring? The transition
is hard. Knowing the right thing to do
is impossible and is driving me crazy. But,
as Brady’s Dr. said, “this is a great problem to have.” I am trying hard to
live in the present and stay out of the swirling thoughts in my head (not very
successfully, but I’m trying). Kalydeco
may have changed the way CF is affecting Brady’s body, but it hasn’t changed
the fact that CF messes constantly with my mind!! Brady honestly couldn’t care less about
treatments because he gets to watch Puss in Boots and I massage his legs. I have so much fear about backing down from
treatments because it has always represented the path to health. BUT KALYDECO IS A GAMECHANGER, and I also want to take full advantage of
the opportunities that lie before us. Spending
all those hours sucking in meds and getting shaken silly might actually be totaI
overkill for someone with perfectly healthy lungs and functional CFTR. I don’t want to sentence my lovely son, who
has already been through so much, to unnecessary treatment time because of my
fears. I will be calling into the clinic
in a couple weeks to inquire about the responses the Dr. is getting from his
colleagues in regard to Brady’s case.
Lastly, I want to just “radio in”
a message to my CF families still waiting on that rooftop, with the floodwaters
rising around you, wondering if you will be rescued in time. . http://bennettgamel.blogspot.com/2012/01/will-helicopters-return-in-time-for-us.html I often imagine how painful it has to be
watching another child receive a treatment like this while your own precious babies
continue to fight CF. My message to everyone still waiting is this: “WE HEAR YOU! We will never forget where you are and we are working to get back to
you as fast as we can! DO NOT EVEN THINK ABOUT GIVING UP, WE ARE ALMOST THERE!”
Roger.
