My Experience with Cystic Fibrosis
Mist tents, respirator vests, and a life-altering drug
I was born in 1967 in Germany to Army parents and the doctors there did not know what was wrong with me. Like many children in those days, diseases were not detected in utero or even immediately after. We came back from Germany in 1970, and I continued to have digestive and weight problems, leading to an incorrect diagnosis of celiac disease.
At the age of 7, in 1974, the doctors suspected cystic fibrosis (CF),caused by a mutation of the cystic fibrosis transmembrane conductance (CFTR) protein. When this is dysfunctional, chloride cannot move to the cell surface, which causes mucus in various organs to become thick and sticky. CF affects digestion, fertility, the ability to gain weight, and lungs- with symptoms of shortness of breath and coughing, along with frequent lung infections.
The most definitive test at that time was the sweat test. As I recall, they basically put me in a plastic bag up to my neck, to collect the sweat I produced. CF patients have 2-5 times more chloride in our sweat than others. When we sweat, chloride crystals form on our skin. This test led doctors at Nationwide Children’s Hospital in Columbus to the correct diagnosis: CF.
Once diagnosed, the treatments began. A big part of CF is the mucus in my lungs. My body is unable to remove it on its own, so my parents had to perform postural drainage “PD”. I would lie on a tilted board twice a day, and my parents would take turns ‘clapping’ my back, side, and chest to knock the mucus loose to help my body cough it out. My PD treatments stopped once I was able to achieve a satisfactory lung function test (FEV1) at my quarterly doctor visits.
Another treatment was the use of a mist tent to provide a moist breathing environment while I slept. It caused very damp disgusting bedding! This only lasted a few years as it was determined that this treatment also increased bacterial infections.
In addition to breathing issues, I have had three surgeries for nasal polyps, a common occurrence for almost half of CF patients.
As a child, medical and support staff thought beginning a camp for CF patients would 1) allow us to meet and get to know others with the disease, and 2) give us something to look forward to since our life expectancy was not promising. Although I attended for 9 years, the camps were eventually determined to do more harm than good because we were passing our resistant strains to one another. This fact was the basis of the movie “Five Feet Apart”- though I’ve not seen it and don’t plan to do so.
Medications began when the diagnosis was made. Since my pancreas does not function as it should, I began taking pancrease enzymes with every meal. This helped with digestion and weight gain. A few years later, I began taking Prilosec for acid reflux, a side effect from the enzymes. I began taking aerosolized medications which would be deposited directly into my lungs. In addition, the CF life expectancy began to increase, which meant CFers needed a way to get the PD therapy on their own.
Moving the mucus
In my 20’s, I began using the Vest. Worn like a lifejacket, the Vest is connected to an air compressor by two hoses. These hoses alternately inflate and deflate the vest to cause a ‘shaking’ of my chest to work the mucus loose. As the Vest is working, I take Albuterol, 7% Sodium Chloride, and Pulmozyme, in a nebulizer– all to knock the mucus loose. During the 40-minute treatment, the machine stops every 10 minutes so I can do a deep cough a few times in hopes of getting the now-loosened mucus out.
As more research was done, more maintenance therapies were introduced. Research indicated taking Azithromycin three days a week would keep lung infection flare-ups at bay. All flare-ups of lung issues would require more Vest/aerosol treatments. Then came the idea of month-on/month-off aerosol antibiotics with the same purpose. Altogether, my daily treatments take almost an hour to do and with an increased infection, I need to do the treatments twice a day.
Coping with lung infections and meds
Months after I was married, the antibiotics I was taking couldn’t keep me from having a flare-up. One symptom of lung infection flare-ups is hard coughing, which can, and in one instance did, result in coughing up blood. I grew an infection that caused me to hard cough more than normal which then caused a tear in a blood vessel in my lungs. I underwent a procedure to close the tear, during which a worse tear occurred. I was rushed to ICU and given a small dose of Nipride to lower my blood pressure to slow the leak…. That’s when we all found out I’m sensitive to Nipride- my blood pressure did not slowly lower, it dropped, and I became a great shade of gray! The staff at Nationwide Children’s Hospital decided not to give me that medication ever again! The leak closed itself and I was released from the hospital a few days later.
I was sent home, with a PICC line to receive IV antibiotics. Thus far, I have had three PICC lines, or Peripherally inserted central catheter lines. Having the line inserted requires a three-day hospital stay for observation. The line is inserted through my arm and runs to my chest to deposit the medication directly into my heart. The very first time Michelle dosed me, she used the bolus technique she had been trained for at Charles River. We learned NOT to do that on a human as I was immediately lightheaded. From then on, I did all the dosing myself!
A clinical trial and a way forward
In addition to the illness-causing hospital visits, I have had the opportunity to participate in several clinical trials. Among them was a 30-day oral dose of a three-drug combination therapy that targets my particular mutation. There were many blood draws on the day of the first dose and a pre-dose and post-dose FEV1 test. All those blood draws made my BP rise a bit as I hate needles. This was a double-blind placebo test in which the nurses administering the drugs don’t even know which patients get the drug and which get the placebo. Within 2 hours of the first dose, my lung function increased 15%. There was no denying I was on the drug!
I was later asked to participate in the next trial which was a 6-month double blind study with no improvement in my lung function. We were disappointed to realize I was on the placebo. My last dose on that trial was Dec 27, 2018. Then I was asked to participate on one final trial for this medication; my first dose was on Dec 28, 2018 and we all knew it was the real thing!
Five months later, just three days before my birthday, the drug was approved. Because of this new drug, my life expectancy is greatly increased. I no longer have to worry that if I catch a cold, I will have to take antibiotics and do more treatments. In fact, for the first time that I can recall, I have fought off a cold without additional treatments or meds - TWICE!
With this new drug, I can breathe easier, I don’t feel like there’s an obstruction if I breathe deeply. I no longer cough up mucus when I’m doing my aerosol treatments.
Doctors have already removed the month-on month-off antibiotic from my treatment regimen and I look forward to discontinuing more of my meds and aerosols in the following months. By discontinuing a few of the aerosols, I can use the time for something else.
In 2017, I went to the funeral of a friend of mine from the old CF camps. He had had a lung transplant 4 years earlier and passed away due to complications at the age of 47.
As I get older, I no longer have to worry if that could happen to me as my lung function eventually deteriorates, since my new therapies correct the CF gene.
This new drug fixes how my body sweats so I don’t actually have salt crystals forming on my skin anymore.
Thanks to the scientists who developed this drug, and thanks to the scientists at Charles River, my lung function (FEV1) has risen from 68% to the low 90’s. I look forward to many years of worrying less and less.
Aaron Edwards is a Mold Machine Technician at Gorman-Rupp Industries, Bellville, Ohio who resides with his wife Michelle in Mansfield, Ohio. Together they enjoy spending time with Dugout (their Norfolk Terrier), camping, and serving in their church. When he isn’t working, Aaron enjoys working in his Lego room (1/2 their basement!). Michelle has worked in Charles River’s Ashland site since 1992, presently in SEND.