Kicking CF's Butt

Things are finally (almost!) sorted out with insurances, and we received our Pulmozyme yesterday! Our medicaid provider is requiring a preauthorization for coverage of the prescription, which Dr. G. warned us might happen, but I was kind of expecting our primary provider to be the ones to push back. So instead of waiting an additional 7-10 days for that to go through, and then however many additional days until Walgreens could ship the meds to us, I decided to just pay the regular copay for this month, and get started.

We have 2 doses in, and so far, so good! Judith's biggest complaint is that it "smells funny" and that she doesn't "like the way it smells." Keep in mind that this is Judith's 2nd experience with nebulized medications, and she wouldn't remember doing TOBI because she was too young. Since she takes her albuterol via inhaler and spacer, the smell is slightly different. Honestly, though, I'm not sure if she's really smelling the pulmozyme, because I caught some of the mist coming out of the mask and it didn't smell funky to me, or if she's actually smelling the plastic from the mask and other parts. Regardless, she's not physically fighting me to try and not do it; verbally, through many protests, yes, but that's so typically Judith.

I'm eagerly waiting to see the difference this drug is going to make for her! After the exacerbations last cold/flu season, I'm really hoping that this will help her clear the crud out of her lungs easier.

Quarterly Clinic Visit

Judith's big visit was last Tuesday, and it's the one where we do all of our annual labs: blood draws, chest x-ray, etc., along with the typical quarterly stuff like cultures. I was pleased at how well she handled the visit, particularly since she had a busy day at school in the morning! A quick nap in the car while we were traveling to clinic probably helped her mood, as did a good lunch at the "local" deli chain that we love.

I was glad to see that Judith finally gained some weight; she gained about a pound and a half, which is good because she didn't gain between January and the end of April. She's still nowhere close to where they want her to be since her BMI is around 19%, but it's a start. Part of the reason that this makes me nervous, despite her gains this quarter, is that when she gets sick again, she's not going to have a lot of reserves to fall back on. There are other factors at play besides the CF that I'm sure aren't helping the situation: her ADHD, and her inheriting John's freakishly high metabolism (which I would love to be able to bottle, because I'd make a really, really nice profit selling it!).

She gained a few fractions of an inch in height, and is up to about 40.8". So no huge growth spurt... yet!

I'm waiting for her final culture results to come back, and they should be in by tomorrow. We're going to do a stool study again, because her patterns are really whacked out even with the miralax, so we want to make sure her pancreas is still functioning properly or see if she needs to start taking some enzymes.

We are starting her on some nebulized meds, which I am actually very pleased to start. We decided to start with pulmozyme first, partly because hypertonic saline is super irritating (which I knew for a long time), but also because she's not used to nebulizing meds on a daily basis, so this will be an adjustment. Her albuterol is taken via inhaler and spacer, and we only used our neb once for a 28 day cycle of TOBI when she was about 16 months old. Pulmozyme is done once a day, and from what I understand it's a quicker neb, so it's a perfect way to get her started on the nebulizing routine. Since pulmozyme is a mucolytic, it will help thin and break down the super thick mucus in her lungs, and help her cough it up easier. By starting this, and starting it now, we're hoping that this will help cut down on the number of exacerbations she has this coming cold and flu season, and I'm hoping that this will help clear things out enough that, combined with the albuterol to open the airways, we can avoid steroids... or at least oral steroids!

And this brings me to the next finding from our annual visit: Judith's x-ray results. Up until now, every report has indicated that things looked perfectly normal; no signs of damage or mucus buildups. While this isn't totally unexpected, and I was already figuring something was going to start appearing after the number of exacerbations this past winter, Judith is now showing signs of possible damage, and is showing an increase in mucus that's building up and blocking the airways. The mucus buildup makes sense, because she's had a bit of a cough that appears a few times a day, and has been around for at least a month. We're still trying to figure out if some of it could be allergy related. Anyway, it's a bummer that we're now seeing signs of damage, and I was hoping we could go longer without that reality setting in. At the same time, I realize we're extremely lucky that we've gone this long without any signs of damage. So now that it's here, our goal for Judith has to be tweaked a bit; instead of working to maintain no damage, we're now trying to keep it from progressing if at all possible, because the less damage she has once the corrector drugs are available, the better.

So overall, results from the visit really aren't that bad! We have some routine changes to make, once CVS gets the pulmozyme to us, and our days are going to get slightly crazier, but we'll adjust quickly.

Orkambi Is Approved!

It's a joyous, and also a bittersweet, day in the CF community again. Today, the FDA approved the latest corrector drug, Orkambi, for CF patients 12 and older with 2 copies of the F508del mutation!

This is the outcome we were expecting, and yet still hoping for, after the affirmative nod from the panel a few months ago.

Now that it's approved for the 12 and older set, they can start the trials in the 6-12 set, and after that, 2-6. It's an exciting time for sure!

Just like the emotions I had a few months ago, I can't help but feel a bit of sadness for Judith, and everyone else who is still waiting for our turn. It will come, but how long are we going to have to wait? We're getting closer, and now more than ever, I'm feeling more of an urgency to keep her as healthy as absolutely possible so she can get as much of the benefits from the drugs that she can.

Still, I rejoice with the rest of the community. I am happy for my friends whose loved one will be able to start taking this drug now, and for those who will be able to start this drug once the trials for the younger age groups are completed and approved.

Getting Ready For School

No, you read that title right. Yes, it's the middle of summer, so don't check your calendar thinking time flew by faster than you realized! Judith is starting a special class in exactly 1 week for some other things that are going on, and now we're working to gather everything she's going to need.

When we wrote her IEP, we worked on an individualized health plan that acts kind of like a rider (but kind of functions like a 504 plan, just carrying more weight since it's attached to the IEP), and we have a list of accommodations in there that Judith's teachers will need to follow. Since she's only in preschool, and it's only 1 day a week right now, there aren't a ton of things that need to be covered. Next year, though, as we transition to kindergarten, the accommodations are certainly going to increase and some will need to be changed.

But I digress. For those that know me in real life, you know I tend to be a bit of a pack mule when it comes to going anywhere, particularly with Judith in tow. I've certainly worked on that, and have improved with some of the day-to-day stuff, but for other things, I always tend to lean more toward being over-prepared, just in case. Since this is Judith's first formal schooling experience, I'm understandably a bit nervous and anxious about how the day is going to go, and want to make sure I don't forget something that she's going to need.

About a month ago, I got our team to write a prescription for a new spacer so I could leave the spare in her backpack in case she would need it. Getting an extra inhaler wasn't an issue, because I always keep 2 backups in the house in case of an emergency. Since Judith is still pancreatic sufficient, we don't have to worry about sending enzymes with her, nor do we have to worry about the staff being trained to administer them. They're used to and able to administer rescue inhalers for kids with asthma and other respiratory conditions, but the enzymes could have been a potential issue if we were on them. But the point is moot since we're not. Medication-wise, we're set. All I have to do is label everything, then toss it into her backpack so we're ready to go.

I have a bunch of new water bottles for her to carry with her, and they're all leak proof. I remembered that I'm going to have to send a couple cans of pediasure with her every day, and a spare outfit in case of accidents. I'm planning to get another travel toothbrush holder after seeing a pin on Pinterest showing how you can use it as a travel straw caddy (and I must say it works like a charm!) so Miss Picky can have the "bendy" straws that she likes.

The last thing that I realized I need to send is the Teacher's Guide to CF that the CFF publishes. Her health plan does detail certain aspects, and I know a few things are doubled on both documents, but the odds of Judith's teachers having experience with another child with CF are pretty low. I know how overwhelming it can be when you have a child come into your classroom with a complex medical condition, and all of a sudden you have a huge list of accommodations to remember, so I'm hoping the guide will help by giving them a quick document to look at if they are unsure about something.

Judith is really excited to be starting! For over a year, she's made comments about the school bus, saying how she wants to ride it, and now she's going to get her chance. She has some hard work ahead of her, but I'm hoping that this class will help eliminate some of gaps that she has with same age peers, and she'll be better prepared for kindergarten in the fall of 2016.

Hot, Hot, Hot!

It's that dreaded time of year again. If you've followed my blog for a while, or if you've known me for a while, you know that summer weather isn't exactly one of my favorite things in the world. Heat and humidity make me cranky! And the older Judith gets, the more obvious it's becoming that she takes after me with that. John didn't mind it up until recently (and he'll still try to tell you otherwise, but he's not fooling anyone because he'll complain a lot in the moment!), but the heat and especially the humidity can take a toll on Judith.

I do think that a lot of this is CF related. Some people with CF are fine with temperature extremes, on both ends of the spectrum, but Judith doesn't do well with either. She'll complain that the cold hurts her lungs, but in the summer she's not as quick to say that it hurts or bothers her. Instead, she shows other signs, like getting very cranky, and not being as active as she normally is.

Yesterday, we were under air quality alerts, or a "code orange," for poor air quality, and Philly and the surrounding suburbs had some level of heat advisory because of the heat index. For anyone, it's so important to stay well hydrated when the weather gets this hot. For a person with CF, it becomes doubly important not only to stay hydrated, but to add salt to their diet to replace what they're losing through sweat.

People with CF need additional salt as it is, but during the summer months, many have to up their intake more to make sure they keep their salt levels in balance. Dehydration can happen rapidly for a person with CF due to the rapid loss of salt.

We've received some strange looks in the past when we've salted Judith's food or even her formula. I'm sure there have been some who have judged us heavily. That's simply a matter of people not knowing that A.) she has CF and B.) they aren't aware of the loss of salt that happens in people with CF. After 4 years of doing this, I've become a pro at ignoring people's judgment, as long as they don't make a snide comment to my face. Anyway, the past few days have been rough, because the heat has really wiped Judith out, and I had to up her salt intake through salty snacks because she's at a point where she won't always drink beverages with salt in them. It's challenging, because she won't always want to eat those salty snacks, and a great go-to beverage, Gatorade, is something she rejects.

We're going to be starting a new adventure in a few weeks, and Judith will be going to a special preschool class 1 day a week. Since this will be the first time she's in a formal schooling setting, I'm starting to worry that she will be too distracted to remember to drink enough in the hot and humid weather. I'm sure her teachers will be on top of it, per her health plan, but there's that mother's worry that is going to stay for a bit. I'm hoping that a new, fun container will entice her to drink more so she stays hydrated!

Super Judith

Earlier this week, we got a pleasant surprise in the mail. Something really cool, from an awesome organization!

It's a custom superhero cape from TinySuperheroes!

TinySuperheroes will send capes to children with illnesses or disabilities, and will also send capes to siblings (Super Sidekicks) to help empower them in their journey!

Judith is in love with her cape, and it's her new favorite accessory! She was so excited to open the package, and that excitement escalated when she saw what it was. She's been zooming around the house often while wearing it, and even wears it during treatments.

She wasn't sure if she could even wear it with her vest at first, but I told her that this was something that goes over top of everything, and wouldn't cause a problem with getting her vest on. I also told her that she could wear her cape during vest treatments to give some extra power to get rid of the "icky sticky mucus" that is in her lungs, and she was sold on wearing it.

We are so grateful to TinySuperheroes, for their mission to empower kids, and especially those with special needs! This cape is bringing a smile and some sunshine into Judith's life, and the timing is perfect because things have been a bit rough here lately, and she will have some additional challenges going forward (which I will briefly discuss at a later time once I feel ready to post about it).

Thank you, TinySuperheroes!

Great News from the FDA!

This afternoon, the FDA Advisory Panel recommended approval of the latest CF corrector drug, formerly VX-809/lumicaftor combined with Kalydeco, now known by it's official brand name, Orkambi! The final vote was 12 yes, 1 no. This is not the official and final approval, as the final word is expected to come on July 5th. But I learned today that the FDA uses the panel's recommendations to make their final decision, and they typically go with what the panel recommends. So while we still have to wait a couple months to find out if the portion of the CF population that carry 2 copies of DF508 and are ages 12 and older will soon have their magic pills in hand, we're significantly closer than we were before, and the remaining wait isn't much longer.

Orkambi's results weren't what we were hoping for, and during the studies it didn't show the improvements that Kalydeco had on patients with the G551 mutation. But there is enough improvement to slow the progression of the disease, which means it will buy more time for the double DF508 population, and Vertex can keep the momentum going on other corrector drugs like VX-661. And once this is approved, they can start the studies for the ages 6-12 group, and then the 2-6 group.

It's a bittersweet day, and July 5th will be bittersweet as well. If you've followed us for a while, you know I've talked about these drugs before, and have also mentioned that Judith only has 1 copy of DF508. Orkambi won't help Judith. The recommendation of approval, and (hopefully!) the final approval of Orkambi is another reminder that we're still waiting for our turn, waiting for our magic pills. All while we have to do whatever we can to keep her as healthy as possible as the disease slowly progresses. Our turn will come. I don't know when, but we'll get there. The waiting game absolutely stinks, but someday we'll join the ranks of CF patients who have a corrector drug that will benefit them.

At the same time, I'm rejoicing for my many friends whose loved ones will benefit from Orkambi, and any future correctors that are better! A chunk of my friends have children who are under the age of 12, so they won't have full access right away (but might be able to get it off-label), but it may not take long until the studies in the other 2 age groups are completed, and their children can start taking Orkambi.

I'd be lying if I said I wasn't tense today. I knew the panel recommendations were coming, but after a link to a live blog was posted in a group I participate in, I knew I'd be glued to my computer when I could. I literally let out a huge sigh of relief seeing the final vote results! Now to wait for the FDA's final decision...