Thursday, March 8, 2012

Brady 2.0

Brady saw his CF specialist again this morning.  It has been one month since he began Kalydeco.  We repeated blood work and the sweat test last week, so we could discuss the results with his Dr. today.  KREM2 news came with us to the appointment also, to get some footage of the Dr. examining Brady and ask him a few questions about Kalydeco and CF.   It is ALWAYS a busy day in the CF clinic and they were all very accommodating to the news crew.  We brought treats and coffee for the clinic team to try and balance out how annoying we can be sometimes…Anyway, I want to revisit Brady’s health statistics and see how things have changed in the last month.  The entire “Baseline” entry is here:

Cough Day 1: None.

Cough Day 27: None.  We honestly haven’t noticed any lung clearance, which to me is a good sign that his lungs actually ARE in good shape. 

Chest X-ray: His last chest X-ray looked totally normal, but was taken several months ago. 

Day 27: We’ve decided, with Brady’s Dr., to do a “low dose” chest CT scan on Brady in one month to best assess his lung health.  Brady’s Dr. said that since Kalydeco is preventative in nature, that he would feel most comfortable “challenging” Brady’s lungs by removing some of his breathing treatments and VEST if he also had a CT scan showing minimal permanent damage in the smaller airways, that are tougher to see on a regular X-ray.  I’m anxious about this because I attended a talk at the NACFC about how much damage CT scans can reveal on INFANTS with CF.  Cystic fibrosis can progress in the smaller airways in the absence of symptoms.  This silent progression is the reason that we elected to begin Pulmozyme and Hypertonic Saline so young with Brady.  I hope it has gone a long way toward helping him maintain healthy airways.  Of course, I will report back the results of the CT scan in 1 more month.  Brady’s lungs sounded clear today.

Sweat Chloride Day 1: 105mmol/L

Sweat Chloride Day 20: 48 mmol/L  Brady’s Dr. is pretty mild mannered, but he was excited to talk about this!  We discussed the average drops in the trial and then agreed that even if these huge drops are expected…it doesn’t make it any less amazing when you see it with your own eyes!

Liver Enzymes Day 1: AST—36, ALT—37

Liver Enzymes Day 22: AST—37, ALT—34  We are so relieved to see these numbers holding steady within the normal range.  Elevated liver enzymes were one of the more serious side effects of Kalydeco in trials, so we are checking Brady’s numbers monthly for 2 more months.  These numbers indicate that we aren’t overdosing him and that his liver is processing this medicine without issues. 

Weight Day 1: 47 lbs

Weight Day 27: 48 lbs  Brady is over the 90th percentile for weight-for-age  today.  This is fantastic news.  I see some changes to Brady’s diet on the horizon. 

Height Day 1: 42 ½ in.

Height Day 27: 43 in.

Blood Oxygen Sat Day 1: 99%

Blood Oxygen Sat Day 27: 100%

Sinuses Day 1: Aggressive Polyp growth.  We were doing an irrigation treatment with a steroid solution (Betamethasone) 3 times a day to keep the inflammation down as much as we could. 

Sinuses Day 27: Clear Sinuses!  Within 3 days of beginning Kalydeco, he was breathing freely through his nose, not snoring at night, and making comments about smells.  Under the direction of Brady’s Ear/Nose/Throat specialist, we reduced the sinus rinses to 2X/day and then to once a day before stopping altogether.  He has now gone over 2 weeks without a steroid rinse and is still breathing freely through his nose!  This has been the most remarkable change for Brady since beginning Kalydeco. Oral Prednisone is the only thing that has ever worked to shrink his polyps down to the point that he could breathe like this through his nose in the past.  Brady sort of hated doing his “Nasatouch” machine and still makes comments to me that he “doesn’t want to do his nose medicine today.”  Let me tell you that it is so awesome to be able to respond, “We don’t need to do it today.  Don’t worry.  Go play.” 

Pancreatic Function Day 1: 3 Zenpep per meal, 2 per snack

Pancreatic Function Day 27: 3 Zenpep per meal, 2 per snack.  We have stopped giving him any extra beads with meals, and give him the “snack” dose more often now.  His poo has been really “normal” looking and is less stinky.  He is also down to 1-2 bowel movements a day from 2-3.  He seems to be absorbing his calories and nutrients better even though he still needs enzymes.  Getting rid of the thick sticky mucus in the GI tract must be helping him gain weight.

Breathing treatments and Airway Clearance Day 1: 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.

Breathing Treatments and Airway Clearance Day 27: Morning--Albuterol and Hypersal via nebulizer followed by 20 minutes on his VEST. Evening—Albuterol and Pulmozyme via neb. followed by 20 minutes in the VEST.  *Just today we have decided with his Dr. to omit the evening Hypersal treatment and do Pulmozyme only in the evening.  This may seem like a small reduction…but any reduction in breathing treatments is so awesome in the CF world!  Typically treatments are added continually as patients age with CF…dropping treatments is a new concept!  I also directly asked Brady’s Dr. if he felt like there was potential to no longer need any breathing treatments or VEST treatments at some point in the near future and he said he thought it was a “reasonable goal.” 

Meds Day 1: Source CF vitamins, Prevacid, Singulair, Ursodiol, Periactin, Zenpep, and started Kalydeco!

Meds Day 27: Same.  The only change we have made to his regular meds is that we no longer give him Miralax.  We used to give him around a ¼ cap or so daily to keep him from getting blockages. 

Forehead Lick Day 1: Salty

Forehead Lick Day 27: Not nearly as salty.  We noticed this change about 3 days after he began taking Kalydeco.  His sweat chloride data backs me up on this! 

Overall, we couldn’t be more pleased with the results we have seen so far and have not noticed any negative side effects.  Brady seems to be a ball of energy since beginning Kalydeco.  He has always been energetic…but he just has a little extra zip.  It might have something to do with the fact that he is sleeping better at night.  He used to struggle to breathe through his nose at night and would snore and snort himself awake several times a night.  Now his breathing is completely silent and effortless at night.  Several times I’ve had to touch his chest to make sure he was breathing AT ALL because it was so quiet. This is such a dream come true.  I almost started crying during the Dr. appointment today when we discussed the future of stepping him down from breathing treatments.  Dropping just one saline treatment a day seems so awesome to us.  His Dr. also mentioned that he thought it would be safer to test out how his lungs respond to dropping treatments when we get a little further from the cold and flu season.  There are a lot of bad bugs going around right now and we are not in the habit of taking risks!  We are more than happy to hold steady with this small drop in treatments for another month and then do the chest CT scan and go from there.  It feels like every day that passes, we get a little closer to realizing the dream of healthy breathing without machines and meds.  What an absolute dream come true!  I’m really excited to finish the interview with KREM2 and see what kind of news piece they put together.  Brady’s Dr. gave them some really optimistic, positive material to work with!!  I talked a lot about our upcoming local CF events and think the reporter might even cover some of those on the news so that is great!  More exciting updates to come!


  1. I cried through your who post! Amazing! I am so happy for you and all others getting to use Kalydeco. My son is 8 and had DDF508. Even though this doesn't help us right now, it is the first glimmer of hope I have actually started to hold on to in 8 yrs!

    1. Ah I am crying too!! Your decision to feel HOPEFUL is what makes this blog WORTH IT for me! I promise to tell the truth and I believe with my whole heart that this disease is going to be a topic for medical historians very soon! Open your heart to the possibilities that await you in the near future! I realize it is almost to good to be true...but it IS TRUE! Keep up those treatments because those lungs are going to need to be ready for the new generation of CF treatments!

  2. Fantastic news for you.
    I guess the larger than normal drop in chloride levels is because of Df508, and that kalydeco is helping poorly folding CFTR proteins to transport chloride. Our kids have a frameshift mutation as well as G551D, so I expect slightly less of an improvement.
    It's also great to see that the dosage appears ok for 4.5 year olds. It might be an ok dosage for even younger kids, which would be great for us.

    1. The average drop in the trial for 6-12 yr olds was 54 mmol/L. Brady's sweat test score dropped 57 mmol/L, which was just slightly above the average. We are so relieved that the dosage seems safe for him. I've heard of children as young as 2 1/2 yrs old that have started treatment with Kalydeco! So far so good!

  3. So very happy for you. I too believe CF will become a medical condition of the past. Since the day my grandson was born I have had a dream where I take him to see a doctor and he gives me some pills and says "Take these three times a day for 2 weeks."

    That's it ... pills for two weeks, my dream.

    And now to find a nice soft tissue to wipe away these very happy tears with <3