Saturday, October 13, 2012

NACFC Update--Caring For Patients on Kalydeco: Emerging Topics in CF Management.

Brock and I started off today's session by filming an interview for the CF Foundation about the positive impact of Kalydeco on Brady and our entire family.  We also filmed "My Dream for CF" videos.  After that we headed to the third and final Plenary Session of this event entitled: Adherence...Where's the App for That?  I will go into the details of this session later, but will say this--new therapies and medicines are of absolutely zero use if they do not get utilized by the patient.  As a parent of a young CFer, I understand that there are challenges in maintaining the rigorous CF treatment schedule.   This session focused on ways to help patients overcome those barriers to become more compliant with their treatments, which ultimately leads to better health outcomes and fewer hospitalizations.  There are all sorts of cool new apps and web based technologies coming along to make staying adherent EASIER on the patient.  The future of tracking your PFTs may become as easy as simply blowing into your smartphone, which will then automatically send the data to your care center's database.  How awesome is that? 

We attended talks all morning, which I will also summarize at some point, but right now I want to focus on the most interesting information I got today, which came from a "Luncheon Roundtable" session.  There are dozens of highly specialized topics to choose from in these sessions, which are discussed in an informal manner by the 10 or so attendees that fit around your table.  There was no question which discussion I wanted to be a part of--Caring For Patients on Kalydeco: Emerging Topics in CF Management.  Each table has a "moderator" or expert who leads the discussion.  Our moderator was Dr. Frank Accurso M.D. University of Colorado, Denver.  We didn't have a ticket to attend this talk, but eventually got seated and entered the discussion already in progress.  We had a nice variety of guests at our table, consisting of nurses, a Pulmonologist, a GI doc, and a pharmacist.  When we were finally seated, we were asked to introduce ourselves and describe why we were there and what we hoped to learn.  When I announced that we were parents of a 5 year old that had been on Kalydeco for over 8 months who showed a drop from 105 mmol/L to 17 mmol/L on Kalydeco and a CT scan showing "no visible damage," and our goal was to learn how to proceed with his care...well, we suddenly became the focus of interest for everyone else at the table.  I'm not sure what they were discussing before we arrived because as soon as WE got there, we were bombarded with questions.  First of all, every one of them was surprised that Brady had Kalydeco and wanted to know first and foremost how we had obtained it.  I described that we had been discussing Kalydeco with Brady's Pulmonologist since infancy and had prepared a strong case for his medical necessity of the drug.  We didn't do anything special, or secret...we simply obtained a prescription from Brady's Dr., submitted it to CF Services Pharmacy, submitted the required "prior authorization form," and provided a load of Brady's medical information at the request of our insurance company.   It was approved and shipped to our house just like any other medication that Brady takes (except you have to sign for this super expensive one!).

On a side note, I think there is a big misconception out there that no one can get Kalydeco unless they are G551D and over 6 years old.  That simply is not true.  The only thing you need to obtain Kalydeco is a prescription and a way to pay for it (either by being INCREDIBLY independently wealthy or having insurance coverage).  Of course, getting insurance coverage for "off-label" cases has proven to be the tricky part, but many patients/parents don't even try because they have been told that it is "impossible."  Everyone told us that also, but I hope our experience illustrates that the "impossible to get" concept is BULLSHIT.  Vertex pharmaceuticals has ABSOLUTELY NOTHING to do with who is able to take Kalydeco.  They can't provide any financial assistance to patients attempting an off-label Rx, because they don't want to get sued, but drug makers do not provide medicines to patients directly (except on compassionate grounds in the case of Kalydeco), pharmacies do.  I mean, do you think that every time a new prescription is submitted, that the pharmacy calls Vertex to ask if it is OK??  Hell no.  That is not how it works.  All the pharmacy cares about is how you are going to pay for it.  If you have insurance coverage, you are golden.

After describing all the incredible changes we've seen in Brady, everyone turned their focus back to the topic of--How do you move forward with treating CF patients on Kalydeco?  Should the regular schedule of medicines and therapies be maintained, or is it OK to drop some?  Are there some patients that may eventually be able to stop ALL of their regular CF therapies?  I wish I could say that there were clear answers to these questions, but the consensus at the table was that NO ONE KNOWS!  Everyone agreed that the treatment of these new Kalydeco patients must be addressed on a case-by-case basis, with the most important factor being how much permanent lung damage/scar tissue/bronchiectasis each patient had obtained before the initiation of Kalydeco treatment.  The idea is that the Kalydeco has the ability to halt further progression of the disease in certain patients (like those with G551D or other gating mutations).  If a patient starts Kalydeco BEFORE much damage has occurred, there is an opportunity to drop some of the regular CF therapies.  On the other hand, patients in the end stage of disease with considerable scar tissue may need to remain on all of their maintenance meds in addition to Kalydeco.  Think of it like this--if someone smokes for 30 years and then stops, they will certainly experience an improvement in their lung function, but the lungs have suffered some permanent damage and will never reach the same level of function as someone who has never smoked in their life.  It is a similar situation with CFers on Kalydeco.  With that  being said, the discussion then moved back to Brady's case and the question then turned to, Which medication to you remove first and why?  Is it more important to stay on Hypertonic Saline or Pulmozyme?  What about airway clearance?    We described our rationale for choosing to slowly wean Brady off of Hypertonic Saline first (which Brady began at 20 months old, and used to do twice a day).   Hypertonic Saline is designed to temporarily rehydrate the airway surface liquid (ASL) on the lining of the lungs, allowing for better clearance of particles and pathogens.  Kalydeco has also been shown to rehydrate the ASL, but through the mechanism of restoring proper chloride transport out of epithelial cells by activating CFTR function.  We decided that because Pulmozyme works by a different mechanism of action (cuts up long strands of DNA left behind when cells die in the lung.  Those strands can contribute to the formation of biofilms by binding mucus together).   Some people at the table agreed with us, others thought that Pulmozyme should be the first of those two meds to go because of its high cost.

We also discussed whether patients might be able to clear infections from their lungs and no longer require antibiotic therapy.  Most agreed that they would need to see more convincing evidence than a "clean" throat or sputum culture to be really sure that the infection had been cleared (there is lots of evidence that throat cultures do NOT give an accurate picture of what is growing in the lung).  Bugs residing deep in the lower airways are often not reflected in throat or sputum culture, but can be picked up by techniques like broncheoalveolar lavage (BAL).  BAL is an invasive technique where a liquid is squirted inside the lung and then collected and cultured.  You can imagine that BAL is more difficult to perform, but yields a much deeper understanding of the bacterial environment inside the lung, than swabbing the back of the throat.  Again, no one knew any single correct path forward.

This year, new data was presented that Kalydeco changes the pH in both the GI tract and the airways, restoring the body's natural ability to kill bacteria and pathogens.  CF patients typically have a more acidic environment in their airways and gut because of the body's impaired ability to secrete biocarbonate (a base that neutralizes acid).  The more acidic the environment, the less natural "killing power" your body has.  Patients taking Kalydeco were able to restore normal pH in their airways and gut, which leads us to believe that complete eradication of infection is at least possible

At this point, Dr. Accurso announced that he had to leave for a different meeting, and he brought in a new moderator for our discussion.  I desperately asked him to answer some questions about the "trials of 1" initiating in Denver, before he left, but his response was that I could email him with those questions...which I will certainly do.  I'm so sorry I can't provide more details about this. I promise I will try to get those answers!  Again, I wish I could write more this evening, but I only have a few minutes left to get ready for the "CF Prom" tonight!  One last important little tidbit that I forgot to mention earlier is that I asked Dr. Accurso why we saw Brady's sweat chloride first drop from 105 mmol/L down to 48 mmol/L, and then saw a further decrease down to 17 mmol/L.  He asked me if Brady had eaten pizza or some other super salty food before his 48mmol/L result.  I questioned why that would matter and he revealed to me that salt intake can affect sweat chloride results.  The take home point is super excessive sodium intake can artificially increase the sweat chloride score and should be avoided just prior to the test (like the day before).  I'm not suggesting that you eliminate salt intake--rather, adjust to more "normal" dietary sodium rather than the stardard high salt intake typically recommended for CFers to get a true measurement.  That was news to me!

Our flight leaves tomorrow morning at 7:30 a.m.!  I miss Brady so bad!! 


  1. Thank you Rebecca, another interesting and exciting blog entry.

  2. Safe travels and thanks for the super update. Can't wait to hear more!