Saturday, May 31, 2014

A Final Thought for the Month

As CF Awareness Month comes to a close, I want to offer a final thought that I feel is important for people to remember.

CF patients may not “look that sick,” but please keep in mind that a ton of work goes into keeping our children (or themselves for adults and older teens) as healthy as they possibly can be.  CF is a progressive disease, and as another CF mom puts it, it’s a “365 days a year, 24 hours a day” thing, and “there’s no remission or vacation from CF.”  Family, friends, loved ones, and the CF care team are in it for the patient so they can have a full life, with as many typical experiences as they can.  So while we do have a month where advocacy and awareness happen more frequently, remember that for those living with the disease and for the families affected by it, awareness and advocacy also happens throughout the year and doesn't end because the calendar flips to June 1.

If you would like to read more about CF or treatments, therapies, and other aspects of the disease that I didn’t touch on throughout the month, please visit and click on any of the categories at the top of the page or on the home page.

Thursday, May 29, 2014

No News Is Good News

Nah, don't panic at the title.  I just meant that not hearing from me means nothing has really changed.  Well, besides the fact that the house is in an elevated state of chaos, but that's to be expected when you're getting things ready to stage.

One of my bigger predicaments at the moment is trying to figure out where I'm going to safely store all of Judith's equipment and meds where they'll be safe and out of sight once the house goes on the market and we have showings/open houses.  Many other CF moms have recommended this Ikea cart for storing Vest equipment, nebulizer and parts, and even meds that don't need refrigeration.  I absolutely love the concept, but am still debating if I want to shell out $15 in shipping so I don't have to drive super long to get one in-store.  That, and I'm not 100% sure that this cart will work with our SmartVest, since most of the people who are using them have Hill-Rom or Respritech vests.  In any case, I know I need to get this vest on wheels at some point, because the generator is so darn HEAVY.  And if it helps with storage, well, that's a definite bonus right now.

I did manage to clean out my closet over the weekend, and almost 6 hours later that sucker looks immaculate.  I haven't gotten rid of a lot of stuff since high school, so some of it was at least 12 years old.  9 trash bags of clothes and about 4 of shoes were donated, and earlier in the week I took a huge bag to the recycling center to donate since I knew the other places wouldn't accept it due to it's condition.  When he saw my closet, John actually said to me, "Is that seriously all you wear?!"  I think he was shocked, because my closet was literally jammed full of stuff, and now there's barely anything in there.  I seriously regret not doing that sooner, because I could've had an awesomely neat closet for the last 7 years!

Next on my list is Judith's room, and I know this is going to be a problem.  Toys will be going into storage, and I'm facing reality that it's time to sell all of her smaller clothes.  Certain things I'm definitely keeping, like all of her smocked and monogrammed outfits, most of her 24m/2T clothes since I can use them on my puppets if I get back into puppet ministry, and all of her preemie/NB clothes since she can use them as doll clothes (and I frankly have too much of an attachment to them to get rid of them anyway), but the rest will be sorted and either sold when we have a yard sale, or donated.  I'm anticipating that Judith is going to be upset by the amount of toys that will need to be stored, but realistically she doesn't play with a lot of them right now anyway, so rotating is going to be good for her... whether she wants to acknowledge that or not!

Kalydeco, VX-809, & VX-661

In January 2012, the CF community was buzzing over the FDA’s expedited approval of the drug that has brought us as close to a cure as we have been: Kalydeco.  For the 4% of the CF population that carries a copy of the G551D mutation, the drug has been a major game changer.  While it’s not the cure that we are still waiting for, the promise that it is showing in the improvement in the quality of life in patients who have been on it is remarkable!

Kalydeco, or Ivacaftor if you use the generic name, is developed by Vertex Pharmaceuticals with significant funding/help from the Cystic Fibrosis Foundation, and they are currently working through various phases of the study process for a combination of Kalydeco and VX-809 or Kalydeco and VX-661.  The drug(s) help to restore CFTR function to the surface of the cell, which means that the flow of salt and other fluids to the lungs is restored, and mucus in the lungs can be thinned (  The reason that this is so revolutionary for the disease is because up until this point, treatments only targeted and treated the symptoms.  Kalydeco is the first drug to actually target and treat the underlying cause of CF.

Currently, Kalydeco is approved for patients with a copy of G551D who are age 6 and older.  Studies are underway for patients with a copy of G551D who are ages 2-6, and it is hoped that approval for that age group will happen soon.

VX-809 and VX-661 are not approved by the FDA yet, as they are still going through various phases of the study process.  Phase 3 studies are currently underway for the Kalydeco/VX-809 combination for patients who are homozygous F508del (or those who have 2 copies of that mutation), and Phase 2 studies are underway for heterozygous F508del patients (or those that have 1 copy of that mutation and 1 copy of another mutation).  It is expected that approval for the homozygous F508del patients will happen in the next few years, and there is hope that the heterozygous F508del population will not take too much longer to get approval.  Last time I checked on VX-661, I think I remember reading that it was heading to Phase 2 trials, and was showing more promise than VX-809.  It would target the same populations as VX-809.

The reason VX-809/VX-661 has so many people on edge waiting for the studies to finish and approval to happen is because these drugs target a huge portion of the CF population; approximately 90% or more of the CF population carries at least 1 copy of F508del.  Once VX-809/VX-661 are approved for both the homozygous and heterozygous population, a vast majority of CF patients will hopefully see a dramatic improvement, and have a chance at living a longer, healthier life than they would have without these drugs.

In the first paragraph, I mentioned that the CFF provided a lot of support in various ways through funding, which includes a $75 million investment for Kalydeco alone (  This is why so many of us reach out and ask for donations and support for the Great Strides walks!  Because CF is an orphan disease, government funding is practically, if not totally, nonexistent.  Our fundraising efforts as families, friends, and others who care about the disease, is what is funding these studies for our loved ones.  It’s why we’re moving closer to a cure!  $.90 of every $1 raised goes back to the CFF, which in turn goes into the research for treatments like Kalydeco.

The drug doesn’t come without a hefty price tag: 1 year of Kalydeco can cost approximately $300,000.  I’m hearing reports from other CF parents in the US that they are not having to pay a lot out of pocket for the drug and their copays aren’t too bad, but the extremely high costs is a reason why other countries around the world have not yet approved Kalydeco for their patients.  In Australia, for example, they are still fighting the government to approve Kalydeco for patients with a copy of G551D.  My fear is that once VX-809/VX-661 gain FDA approval, patients in countries that are struggling to get Kalydeco are going to be going through additional battles to get the new combination drugs approved, even though a huge portion of the CF population would benefit.


Tuesday, May 27, 2014

Various Tests and Procedures

CF patients regularly go through certain tests to get a better idea about what’s going on with their bodies, and sometimes have to go through other tests to get a deeper look into a system:

Throat/Sputum Cultures:  These are done at every clinic visit, and sometimes happen more often if the need arises (i.e. - after finishing a round of TOBI to see if a pseudomonas infection was eradicated).  It tells the CF team what, if anything, is growing in the patient’s lungs.

X-rays:  Patients have annual x-rays done of their chest to check for damage and mucus, and can sometimes need them more often if it’s needed.  They can also x-ray the bowels to check for blockages.

Blood Work:  Like many people without CF, CF patients get annual blood work to check for a host of things, including vitamin levels, blood count, etc.

Pulmonary Function Tests (PFTs):  At a certain age, sometimes as early as 4 or as late as 6 depending on the clinic and child, CF patients perform these tests regularly.  It measures how well the lungs take in and release air, and can also measure how well they move gasses such as oxygen throughout the blood stream (

Bronchoscopy: Used to get a better look into the lungs, a scope is inserted into the lungs to see what’s going on inside.  A biopsy can also be taken during the procedure if necessary (

Sometimes other tests are also needed that I have not mentioned in this list.

Sunday, May 25, 2014

Tune Ups and Clean Outs

Sometimes CF patients need additional help to clear up a complication that’s causing big problems, either over time or that pops up suddenly.  Sometimes measures are taken to try to clear up the problem at home, but when that doesn’t work, admission to the hospital happens so other interventions can be implemented.

During “tune ups,” CF patients often go into the hospital for up to 2 weeks for IV antibiotics, and an increased inhaled medication plan to get them back to a place where they have better lung functions and health.  IV medicines are administered through the patient’s PICC line or port, and sometimes patients can finish their course of antibiotics at home.

During a clean out, the patient often needs stronger medicines than what were tried at home to clear a bowel blockage.  An enema like Golytely can be administered through an NG tube, or through the g-tube if the patient has one.  If you remember one of my really long posts from August of 2013, Judith went through an inpatient clean out after developing a large blockage.

Friday, May 23, 2014

TOBI and Cayston

Both TOBI and Cayston are inhaled antibiotics that are used to treat CF patients who are infected with pseudomonas aeruginosa.  Cayston is the newer antibiotic that hit the market back in 2010, and even more recently there was a new form of TOBI released that comes in a podhaler which older CF patients can use instead of nebulizing TOBI.

Some patients who are colonized with pseudomonas or have recurring infections might alternate between TOBI and Cayston to try to eradicate the infections, or may do a plan with just TOBI that could be 1 month on TOBI and 1 month off TOBI, repeating it as necessary.  Like so many other aspects of this disease, that will vary on the individual and the course of action their CF team recommends.

We experienced our first 28 day cycle of TOBI when Judith was 16 months old and she had her first positive pseudomonas culture.  The nebulized treatment took us about 20-25 minutes to complete, twice a day.  You need to do treatments with inhaled antibiotics after airway clearance therapies, because you don’t want them to be coughed out of the lungs.  The whole point is to get them to stay in there and fight the bacteria, so it is crucial to do this as one of the last parts of your treatment time block.


Wednesday, May 21, 2014


Mucolytics like Pulmozyme are used by CF patients to thin the mucus in the lungs, and make it easier to cough it out (  The CFF website says:
    Pulmozyme acts like “scissors” in the mucus. It cuts up the DNA strands outside the cell that can make CF mucus thick and sticky. This DNA is from the white blood cells that work to fight lung infections. Even if a person with CF has not had a lung infection, the white blood cell DNA is still in the mucus (

Like hypertonic saline and albuterol, this is another nebulized medicine that I often hear other CF parents talk about.  Judith is not on it yet (nor is she on hypertonic saline), so our experience with both this and hypertonic saline is limited.


Monday, May 19, 2014

Hypertonic Saline

Many CF patients inhale hypertonic saline during their airway clearance time.  The purpose of the hypertonic saline is to draw more water into the airways to make it easier to cough out the mucus (


Saturday, May 17, 2014


Bronchodilators, like Albuterol, are used by CF patients to help open the airways.  Many patients receive the medicines through their nebulizers, but some may use inhalers instead.  The theory is that by opening the airways, it could help the patient cough up the thick, sticky mucus that needs to be cleared out of the lungs (


Friday, May 16, 2014

It's Chaotic Around Here

I promise I haven't fallen off the face of the earth!  I really hope you've been enjoying my series this month, but I know I've been slacking with other posts.  Within the last 5 days, though, our family has been in the throes of chaos, because we made a pretty big decision Monday evening, and something that was talked about for the last 9 months or so is coming to fruition.

We're going to put our house on the market, and start the search for a new home!

John and I have had a pipe dream of moving into a home with 3 bedrooms and even central air eventually, and into a home that's more central to everything since where we're currently living isn't central at all.  The area we're looking at is going to be much closer to a lot of things for us, and we can cut back on commute times for things like visits to my parents and trips to CF clinic.  We've been in our current home for 7 years, and were thinking about refinancing, but ultimately it's going to be better for us to just sell and purchase something else rather than refinancing and waiting longer to sell.

Of course being in a home for so long means we also have 7 years of stuff accumulated on top of items John had to bring to our house when my father-in-law retired and he and my mother-in-law had to move out of the parsonage.  I knew there was a lot of stuff hanging around everywhere, but now that we're starting to box things up and creating storage space in the basement so we can stage the rest of the house I'm seeing how much there really is.  And it's pretty darn overwhelming.  At least this is forcing us to finally do the big purge of stuff that we'll never use or wear again, and in a way it's also giving me the final nudges I need to break the sentimental ties to Judith's baby stuff because now I just want it out of here so we don't have to worry about it anymore!

Naturally we're stressed, because we're pushing ourselves as much as we can to get things presentable so we can get the house on the market ASAP, but at the same time we're really excited and are antsy to start looking at potential homes.

Thursday, May 15, 2014

Salt & Nutrition

I’ve heard other CF parents talk about dirty looks and even snarky comments they get from strangers when they see them salting their young CF child’s food or drink.  We’ve been on the receiving end of some strange looks ourselves, and from an outside perspective it really can look like the parents are starting their child down the path to hypertension rather early.  Trust me, though, we wouldn’t be doing this if it wasn’t necessary.

CF patients shed more salt than people without CF thanks to the malfunctions in the CFTR (which I still don’t completely understand because it’s pretty complicated), and as a result that salt needs to be replaced to keep the system in balance.  Dehydration can also occur faster because of this, which is often why CF patients will down beverages like Gatorade or other electrolyte-replacement drinks.  Sort of like the dosing with enzymes, how much salt each patient needs to replace will vary, and can even vary by day.  For Judith, I tend to give her less salt over the winter because she’s not sweating as much, and will often eat enough salty foods to compensate.  Over the summer, I do add extra salt to her food since she’s sweating more, and I want to make sure that she’s getting enough to replace what she’s losing.

Nutrition for CF patients is also important, because good weight gain and a high BMI are directly related to good lung health and pulmonary function.  Because of the absorption issues that many CF patients face, extra calories are needed to help the patient gain weight and get to or exceed the “optimum” BMI of 50%.  How many calories a CF patient should consume each day is going to vary depending on the individual, but a ballpark figure that I often hear mentioned is about 2,000 calories each day.  Again, that number will vary; some people will not need that many calories, while some might need more than that.


Tuesday, May 13, 2014

CF Specific Vitamins

People with CF typically need additional vitamins to help maintain their health, and to make sure they’re getting the necessary nutrients/vitamins that they may not be absorbing (  Everyone, CF or no CF, needs vitamins to stay healthy, but because CF causes vitamins to not be absorbed properly it’s important that CF patients do take a supplement if they cannot get enough individual vitamins from other sources.  Pharmaceutical companies work to create special vitamins that have the extra nutrients CF patients need, and there are some different choices on the market.  AquaDEKS, which is what Judith was taking previously, is an example of one of the vitamins on the market.


Sunday, May 11, 2014

Pancreatic Enzymes

When people think about CF, they typically think of the respiratory side of the disease, but that’s only part of the condition.  The digestive system is also heavily involved, and the vast majority of patients are pancreatic insufficient, meaning their pancreas doesn’t function properly because of the thick, sticky CF mucus clogging it up.  It is believed that over 90% of CF patients are pancreatic insufficient and need enzymes to properly digest and absorb nutrients from food.  So far, Judith is one of the very lucky minority that is pancreatic sufficient and does not need to take enzymes, although we did give them briefly early on while we were trying to figure out some GI issues.

As mentioned earlier, pancreatic enzymes help CF patients absorb nutrients, to digest carbs, proteins, and fats, and to help them gain weight.  Without enzymes, patients who are pancreatic insufficient will suffer from malabsorption and will not be able to gain weight (as a side note, some patients who are pancreatic sufficient, like Judith, may still have problems with weight gain because of the disease, so weight gain issues aren’t necessarily isolated to those who are pancreatic insufficient).

Enzymes are taken with meals, and while there are some exceptions of foods that do not require enzymes, most do require them.  Dosing depends on the patient, and the CF team will help determine the proper amount, but a lot of parents and patients also get really good at adjusting the dosing depending on what was consumed.

For more information on pancreatic enzymes, you can read the informational pamphlet from the Cystic Fibrosis Foundation:


Friday, May 9, 2014

Airway Clearance

Airway clearance is a major part of CF care and maintenance.  There are various ways that patients and their caregivers can administer the clearance therapies, and some of the techniques used can vary by country.  Some techniques can be done by the patient (if they’re old enough to take charge of their care), while others require someone else to assist them.

The whole point of airway clearance is to loosen and move the abnormally thick, sticky mucus out of the lungs so patients can (hopefully) breathe better.  Getting all of the mucus out can also help reduce the severity of lung infections (

One of the first airway clearance techniques parents of infants use on their child after diagnosis is manual chest physiotherapy, or CPT.  The parent or caregiver can use a cupped hand or a special percussion cup to clap over the lungs in specific zones for a set amount of time.  This is the method we used with Judith for almost 2 years until she was big enough to qualify for another method of airway clearance.

In the US, inflatable vests are a popular and common form of airway clearance, called High-frequency chest wall oscillation, or HFCWO.  I see 3 brands mentioned most often among other CF parents: The Vest by Hill Rom, Smartvest by Electromed, and the InCourage by RespirTech.  The patient puts on a cloth vest that has an inflatable bladder inside, connects the vest to a generator with a hose (or 2 hoses depending on the system), then starts the generator for the specific amount of time that the CF team recommends.  As patients get older, they will often let the vest run for a few minutes, set the program to automatically pause so they can huff cough to expel the loosened mucus, and repeat for the duration of the treatment.  Judith isn’t old enough to understand how to huff cough yet, so her treatments run straight through.  Our team wants her to do 30 minutes twice a day, which seems to fit with the average of 20-30 minutes that I’ve heard about from other CF parents.  Vest systems are hard to come by for patients outside of the US because other countries often will not pay for the systems, so if a patient wants one they often have to pay out of pocket for a new system or a used system.  There is some debate about whether or not the vest systems do a better job than manual CPT or other methods.

Another technique that older patients can use is Oscillating Positive Expiratory Pressure (Oscillating PEP).  To do Oscillating PEP, the patient blows into a device that then vibrates the large and small airways to help loosen the mucus (  2 devices I’ve heard mentioned frequently for Oscillating PEP are the Flutter and Acapella, but there are other brands available.

During normal treatment plans, most patients perform airway clearance twice a day, but some may do it more frequently depending on their individual case.  During sick plan, patients are often performing airway clearance at least 3-4 times a day to help get rid of excess mucus that’s building up, and to hopefully keep more serious infections from setting up camp in the lungs.  Airway clearance can be pretty time consuming, particularly during sick plan, but it’s necessary to do it regularly.

There are other forms of airway clearance that I didn’t cover, and if you would like to read more about them, you can find them by following the source link at the bottom of this post.


Wednesday, May 7, 2014

What Is The Pipeline?

Sometimes you may hear parents/caregivers and healthcare professionals talk about medicines that are “coming down the pipeline.”  But what exactly is it?

Basically, it’s the list of the latest drugs that researchers are working on to help fight the disease.  There’s an interactive page on the CFF website that shows you what stage of development/study the drugs are in, and you can track the progress there.

Interested in seeing some of the drugs that are in the hands of patients or what stage of a study something is in?  Check out the pipeline by following this link:

Monday, May 5, 2014

A Look Into Our Daily Routine

The CF team is one part of the core care facilitators for CF patients.  The patients themselves and their parents/caregivers or spouse/significant others are the other part of that core.  Compliance is huge for patients to be able to stay as healthy as they possibly can, to try to ward off as many bigger complications for as long as they can, and to try to live a long, full life.

Compliance can be challenging at all stages of a CF patient’s life.  One benefit I recently discovered is being able to get a glimpse into potential future battles we may have with Judith; I’m part of a Facebook group for CF parents, and the children’s ages vary greatly.  I’ll often read through posts about the challenges in dealing with teens, and file the tips away in the back of my mind in case we have the same problems in the future.  Of course there are challenges with kids Judith’s age as well, and believe me there are days when I want to pull my hair out trying to keep her compliant when she is trying her hardest to get out of doing a treatment!

One thing that helps keep us in compliance is sticking with a solid routine.  Our daily routine is strict, but also allows for wiggle room depending on what’s going on that day.  A general day for us looks like this:

Wake up, have a can of Pediasure in a straw cup, mixed with the day’s dose of Miralax
Random “get ready for the day” stuff that everyone does
Have breakfast with another can of Pediasure; take vitamin
Play time for about 60-90 minutes
Vest, Albuterol
Snack and more play time or other activities
Lunch with another can of Pediasure
Yet another can of Pediasure, then about 30 minutes of play time
Vest, Albuterol
Bath, bedtime routine, bed

Most of that is nothing out of the ordinary for a preschooler, and in fact the majority of Judith’s day consists of very typical activities.  The differences are in the treatments.  We’re fortunate because she is not on as many medicines as a lot of other CF patients, so it significantly cuts back on treatment times.  However, The Vest alone takes a full hour out of each and every day, and that’s when she isn’t sick.  Sometimes it can be hard and even annoying trying to plan appointments and activities around her treatment times and accounting for them, but it’s something you get used to.  Now, when Judith’s on “sick plan,” things are even trickier.  Treatments will at least double, which means she’s spending a good 2 hours every day on her Vest, and taking puffs of her Albuterol every 4 hours (again, though, the time is significantly less than it is for some patients because we use the inhaler with her, and don’t nebulize it).  Antibiotics are also added, which can often be a good 15 minute battle depending on what form they come in.  If she has a pseudomonas infection, TOBI would add an additional 40 minutes (approximately) to the plan.  I also know that once we add hypertonic saline and pulmozyme to her daily meds, treatment times are going to go up even more.

It’s a lot, and it’s overwhelming, especially if she’s sick.  We’re hoping that by fostering good compliance strategies now, it will help with the compliance battles we may end up facing as she gets older.

Saturday, May 3, 2014

The Cystic Fibrosis Care Team

I frequently post about Judith’s clinic visits, or calling clinic to discuss a problem or to get answers that I have for questions that pop up in between scheduled appointments.  Having a specialized care team that knows the ins and outs of CF is crucial to getting the best care possible for a CF patient.  We’re very lucky to live halfway between 2 great CF clinics and have a choice about where Judith gets her care based on her needs at the time.

There are many players in the CF care team: pulmonologists; gastroenterologists; ear, nose, throat specialists; dieticians; respiratory therapists; nurses; and social workers to name a few.  Not all CF patients see all of these specialist, some do see all of them, and some have other specialists that they need to see as well (like transplant specialists if they are a candidate).  Judith’s current CF team, for example, consists of her pulmonologist, dietician, CF nurses, her social worker, and the respiratory therapists when we need to see them.

Since CF is a rare and tricky disease, getting established with a team at a CFF accredited care center is key.  There are over 110 centers in the CFF network, and not only do they provide care for patients, they also help with the research and studies!


Thursday, May 1, 2014

May Is Cystic Fibrosis Awareness Month

Each year, I like to try to do a mini-series with various tidbits of information to hopefully help people understand a little more about CF because I know that it’s a disease that people still do not know a lot about, and often what little knowledge they do have is often outdated.  Naturally, May is the perfect time to do this since it’s CF Awareness Month!

This year, I thought it would be fun to introduce people to some of the basics in CF care.  There are a lot of things that I won’t be able to cover for various reasons, but I’m going to touch on some of the treatments, medicines, etc. that are more common and even standard for CF patients across the board.  As you are reading the posts, though, remember that every CF patient is different, and they may not need to take a certain medication/have a certain treatment!