I used to spend a lot of time with my grandmother in large bingo halls surrounded by die-hard bingo fanatics. If you have ever had the joy of playing bingo at your local fire station or VFW, then you know there's a period where you can see the next ball to be called, but it doesn't count until the bingo caller says the words. The anticipation when you know you are about to have bingo is exhilarating, but you also have this moment of doubt. Heaven forbid you call bingo when you do not, in fact, have bingo. Waiting for the Trikafta® approval for ages 6-11 is a lot like waiting for your turn to call bingo. I can see that our turn is coming, and I feel all the hope and joy that comes with that. After all, I have been waiting for this moment since the first modulator hit the market in 2012. Despite this, I never expected to also feel the apprehension about entering the unknown world of highly effective modulator therapy.
As a parent to two children with CF, there is great pressure on me to ensure my kids stay as healthy as possible and to make the most informed decisions about their treatment plans. Since the approval of Kalydeco®, I have tried to manage my own expectations of what these new therapies could mean for my children. I have stalked social media to learn as much as I can from those who have been able to start their own therapies. I have read countless journal articles and blogs to understand the good, the bad, and the ugly. There have been many before us who have started these new therapies, including the amazing people who enrolled in the clinical trials, and I'm blessed to be able to learn from their experiences to help inform me on what a “new CF” may look like for my kids.
Despite the incredible feeling of relief and gratitude that we finally have something to help treat the underlying cause of CF for us, I am also terrified that somehow I will make the wrong decision here.
As I watch for the news of the much-anticipated U.S. Food and Drug Administration approval of Trikafta for ages 6-11, I can't help the negative scenarios from creeping into my mind: Will this work for my kids? Will they suffer from side effects? Will they have to stop this therapy and bring us back to when something didn't work for them? With all those worries comes the guilt that I am actually questioning this gift we are about to receive. It was not too long ago that the thought of a therapy that would work for my children was too far in the future to seem real -- or worse -- might not occur in their lifetime. And I know there are so many who don't have the options to take modulators and who continue to feel that the “new CF” we are starting to hear about is just outside of their reach. I wrestle with how to resolve the odd juxtaposition of hope, anxiety, and guilt that I now find myself grappling with.
I would have never imagined I would question myself like this when it came to my children initiating modulators. I have prayed every day for something to help my children. But, as the parent, my kids' health and wellness fall squarely on my shoulders. These new therapies do not come without risks. Modulators are a new kind of therapy, and I worry about what it means for my kids to be on these types of drugs for decades. It is also very difficult to trade an unknown CF for the one we have learned to manage and live with, especially when your children are relatively healthy. Ultimately, I'm responsible for teaching my children that it's OK to be cautious of a new therapy, to learn to advocate for themselves, and that it's important to listen to their bodies and tell me and their care team when they feel different -- both the good and the bad. And, just with all other therapies, we need to carefully weigh the risks and benefits through open and honest conversations with our care team. Despite this feeling of hope mixed with anxiety, I have nothing but gratitude for my kids to have a chance to try this new therapy. We are so close to being able to yell, “Bingo!”
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