Dodging Bullets

In the interest of full-disclosure, I feel I need to give an update.  Something interesting happened since my last blog post—Brady got sick.  Let me preface this entry by saying that Brady has only had a few minor colds in his lifetime, and a case of Roseola as an infant, but has never had a serious respiratory exacerbation before.   Then, about a month ago, he got hit with a bad virus.  First, vomiting and diarrhea for a full 6 days.  Stomach bugs are no fun, regardless of the circumstances, but I was expecting the illness to run its course pretty quickly.  It didn’t.  This led me to an important question:

The Crisis

What the hell do you do when a patient is unable (for whatever reason) to take their Kalydeco orally?

During the course of the 6 day stomach bug, Brady missed several doses of Kalydeco.  Other doses were thrown-up after just a few minutes, and none of the doses were taken with an appropriate amount of fat (so very little drug was probably absorbed).  Brady was monitored closely by his pediatrician for dehydration and given Zofran to help with nausea.  As the days passed and the missed doses piled up, I began to see some of the symptoms of cystic fibrosis reappear in him.  When he began taking Kalydeco, the sinus inflammation/polyps shrunk within just a couple of days, and he was finally able to breathe through his nose.  After several missed doses of Kalydeco, I could hear the inflammation returning to his sinuses just as quickly as it had left.  Of course, I had to lick the poor kid’s forehead a few times to test for the characteristic “CF saltiness,” and to my horror, it also seemed to be returning.  All I could think about was getting him back on track with his Kalydeco dosing. 

Anecdotally, I have heard several older patients/adults describe their experience in “coming off” Kalydeco as extremely unpleasant.  Some say they can feel their chest tightening and feel that old “heaviness” with just a single missed dose.  The data shows that sweat chloride levels also suffer from a rebound effect when the Kalydeco is removed—shooting to all-time highs before returning to the normal baseline level. 

As Brady’s nausea began to fade, the cough started.  It appeared this virus wasn’t done with him yet.  The cough took ahold quickly and was the worst sound I have ever heard come out of him.  It was an uncontrollable, tight, barky sounding, unproductive cough.  Bronchospasms would wake him in the night and send him into coughing fits that sometimes ended with vomiting.  During Vest treatments, he would cough until he cried and begged to turn the machine off.  It scared me so bad.  After 3 days of CONSTANT coughing, he began a course of Augmentin and a 6 day burst of Prednisone to reduce the inflammation in his lungs.  The steroids helped tremendously with the bronchospasms, but he was still coughing a lot, and the sound had turned deep and phlegmy.   We decided to add Hypertonic Saline back into the treatment schedule, 2X a day, to help clear out the gunk.  The next few weeks brought gradual improvement, but were slow going.  He is finally almost back to 100% and we have dropped Hypertonic Saline again. 

By now you might be asking: “What’s the big deal”?  I mean, ALL KIDS GET SICK.  What is so extraordinary that I had to write a whole entry about this?  For me, this was a frightening experience on a number of levels.  First of all, watching Brady’s CF symptoms reappear was awful.  It illustrated for me what an extremely powerful drug Kalydeco really is.  Kalydeco has a short half-life and is metabolized quickly by the body, which is why timely dosing (every 12 hrs.) with appropriate fat (we shoot for 20 grams) is important to maintain levels in the bloodstream.    When levels in the blood drop and CFTR function turns off again, it doesn’t take long for ol’ shitty CF to come knocking.  It really throws the body for a loop.  I asked our CF Clinic Pharmacist if there was any way to administer Kalydeco via injection or IV for cases where a patient is unable to take the med orally and was told, “Not at this time.”  I don’t even know if that is possible, since Kalydeco is a fat soluble compound.

Also, this was the first bad respiratory bug that Brady has ever had.  I know many of my readers can probably relate when I say that listening to your little CFer cough causes both emotional and physical pain.  I mean, I feel an actual stab of pain in MY chest when HE coughs.  When your kid has CF, it becomes impossible not to analyze every cough, throat clearing, snort, or sneeze that comes out of them.  Unfortunately, a cough is rarely just a cough with CF, and can signify infection with some funky organism or worse.  I began to fear that Brady might end up in the hospital with this virus—something I didn’t think I would have to face once Kalydeco was onboard.  The reality is that Kalydeco may be able to lower Brady’s sweat chloride scores to a normal range, but he still has cystic fibrosis and is just a few missed doses away from his old problems.  Very humbling.  Missing those several doses probably put a big exclamation point on the respiratory symptoms caused by the virus, but the point is…HE GOT BETTER.  He didn’t end up hospitalized and I don’t believe he has an underlying infection.  The pediatrician told me he had been seeing a lot of this bad bug in his office, with the majority of kids presenting with symptoms similar to Brady’s. 

The Lesson

I think Brady just had a bad virus, and his symptoms were punctuated by missing Kalydeco for several days.  Because Brady is a complicated case (has CF, but also has Kalydeco normalizing his CFTR function), his Pediatrician consulted with his CF specialist several times.  Everyone (Brock and I, CF Doc, and Pediatrician) agreed that it was best to err on the side of caution and treat Brady a little more aggressively.  For me, this was a scary little glimpse into pulmonary exacerbations.  Of course, there is no way to know this for sure, but I feel like this virus could have led Brady to his first admission if he hadn’t been able to re-start Kalydeco just as the cough initiated.  It feels like we dodged a bullet.  It also broke my heart to get a little better taste of what many of my CF friends/families regularly endure (and I realize it was a mild, tiny taste).  The strength of this community is unparalleled. 

The Motivation

Brady recovered from the virus just in time for our recent trip to Boise, where I had been invited to speak at a CF education event.  As we packed our bags, I recalled all the lives that CF was currently interrupting.  I wondered how many trips had been cancelled, how many school functions had been missed, and how many sleepless nights CF families had spent because of CF.  I renewed my vow to work even harder and try to do more, because we have the luxury being healthy enough to do it.  I hope I chose the right words at the education event and made an impact on some of the families there.  The clinic team in Boise is really impressive.  One of the providers there, Dr. Perry Brown, is not only administering quality care to his patients, but working on a personal fundraiser to raise money for the CFF.  We all know how busy these Docs are, so I think it says a lot that he is committed to the community on this level.  We are lucky to have Docs like him and I encourage you to throw him a few bucks to help him reach his goal:   http://www.crowdrise.com/500milesforcysticfibrosis/fundraiser/perrybrown. 

I am currently in D.C. for a CFF strategic planning committee meeting on adherence and I fly home in the early a.m.  It has been rewarding work, but I am missing my boys.  I will be avoiding all moving sidewalks in the airport tomorrow…as a precaution.  Cheers to Vertex on their recent announcement of VX-661 combo data!  http://investors.vrtx.com/releasedetail.cfm?ReleaseID=757597