Am I Going To Die From Cystic Fibrosis?

am-i-going-to-die

Asking that question is one of my earliest memories. I was never under any delusion on what the outcome would be, rather it was a matter of when. Throughout my childhood the median survival age gradually lifted, always staying a step ahead of me. For most of my adult life, the median survival has been 37.

An odd little number that I never paid much attention to. Ironically enough, 37 marked for me, the beginning of swift and rapid decline with a double lung transplant the only hope left.

Fortunately, right before CF won the war it had waged on my body, a lifeline was offered which I grasped with both hands and my life was saved. Life has a different perspective post-transplant. It’s an enormous gift, one I struggle to find the right words to explain and thank you never seems adequate.

I’m always aware that a transplant is trade and not a cure. I have traded one set of health problems for another. The side effects from the anti-rejection medication are waging their own war on my body. I will always be a person with a chronic illness and that’s okay.

But here’s not what’s not okay. In the last few years there have been significant breakthroughs in the treatment of CF. First came Kalydeco, targeting a specific gene mutation with fantastic results. Kalydeco was followed by Orkambi – which targets the F508 gene. My gene. The most common mutation of CF.

Clinical trials show Orkambi can slow the progression of CF. Lung damage is lessened, both hospitalisations and the need for antibiotics are reduced. More importantly, people with CF taking Orkambi have a better quality of life.

Neither Kalydeco and Orkambi are a cure for CF. But they are a huge leap in that direction. This isn’t a band-aid treatment, these medications are the first to treat the underlying cause of CF – rather than the symptoms.

Which all sounds pretty fabulous right? It’s a game changer.

Except there’s a little hiccup. While the FDA in the United States has approved Orkambi, the Australian government has not.

Yep, you read that right. This life-changing medication still needs to be approved.
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As I’m post-transplant, Orkambi isn’t an option for me – but while we wait for Orkambi to be approved, people with CF get sicker, get listed for transplant or die. I can not imagine how frustrating it must be to know there was a medication that could help prolong my life, only to be told I could not access it.

You can find out more about Orkambi and the struggle to get it listed on the PBS here.

 

 

Guest Post: Helen’s Story

I can’t recall the day or the month when the doctor finally gave a diagnosis, “Your daughter has Cystic Fibrosis.”

Relief flooded over me. We had a diagnosis and an explanation for the malady of symptoms that had plagued my child since she’d been born. Surely now, things would start to get better.

A treatment schedule was planned, an appointment to learn physio would come in the mail and I was sent to the pharmacy to fill my scripts.

After spending a three-hour wait to see the consultant, the appointment itself was very brief. Although the doctor told me my daughter would not have a normal life expectancy, I did not digest the ramifications of this at the time.

I was a new mum, dealing with a seriously ill child and my first pressing thought was of the need to get to pharmacy to fill these scripts before commuting home in the peak hour traffic.

Information about Cystic Fibrosis was scarcer than hen’s teeth in the early seventies, no one we knew had ever heard of this illness that I struggled to pronounce. My sister and I scoured libraries for articles and the little information we gleaned usually consisted of only a few paragraphs. The main gist of these articles was that it was a childhood illness.

Helen and Sandi 1972

Helen and Sandi 1972

Each clinic visit I tried to glean a little more information, and as three months would pass between visits I had plenty of time to frame questions. What was lacking was an open doctor, even after several visits she either lacked the courage or could not be bothered to take the time to explain to me that Cystic Fibrosis is a progressive illness. I still assumed, or had the perception, that because this treatment regime my daughter was on was working, it meant her illness could be managed.

On one visit, I was at the door about to leave when I asked, “If Sandi has children, will they have Cystic Fibrosis too?”

I can’t recall her exact answer, but the dispirited look in her eyes awoke a need in me to be given the full picture. So after waiting three months for my next appointment I asked outright what the prognosis was, and this time she finally answered.

But it wasn’t the answer I wanted. At best, if we followed the treatment regime religiously, my daughter had a 50% chance of living to thirteen.

Just after that, I had an extraordinary moment. I picked up the Daily Newspaper and read an article about a girl in Western Australia who suffered from Cystic Fibrosis. To see and read of another child with this same unpronounceable condition was mind boggling. At that time, we didn’t have a phone, so I drove to the Post Office to use the public phone and made contact with the people from the news article.

That phone call led me to a group of parents with children who suffered from Cystic Fibrosis. I learned more from them, than I had from the doctor.

Finally, I was not alone.

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help-conquer-cf Sandi’s Note:
This group of parents met regularly, in the beginning, they were a support network for each other.
In 1976 they founded Cystic Fibrosis Western Australia.
CFWA celebrates its 40th anniversary this year – they’ve come a long way!