Why We Took Our Son Off Trikafta

Even though our son had cystic fibrosis, we found hope in the drug Trikafta®. Side effects forced us to make the difficult decision to take him off the medication.

July 2, 2024 | 7 min read
Carolanne Cimino
Carolanne Cimino
Carolanne Cimino's two-year-old son holding Easter baskets in their backyard

When we received our son's heartbreaking diagnosis of cystic fibrosis in 2021, we were offered reassurance. Bellamy’s life would be different than those who came before him. The disease could now be treated by a class of drugs called CFTR modulators. Trikafta® became a beacon of hope in an intense sea of darkness. It has been hailed as a medical miracle for our CF community. And although its effects have been miraculous for many, it was not our miracle.

It feels like yesterday, when I was snuggling my newborn and scrolling through endless social media testimonials from adults with CF; there were countless stories of critically ill adults who were given a second chance at life thanks to Trikafta. Organs were saved, and feeding tubes were removed. The hope in the CF community was palpable. Trikafta gives futures that were only dreams before; it has truly changed the face of this devastating disease. I clung to the possibilities of Trikafta — and arguably still do — because it is the most effective drug on the market for my son's mutations, and it can give him a chance at a more “normal” life. 

Now, the positive feedback from Trikafta was abundant. Initially, only the vast benefits of the drug were thrust into the limelight — rightfully so. However, as time went on, and the new drug became slightly less “new,” individuals reported devastating side effects. When I read about patients' less-than-ideal experiences with Trikafta, I wanted to shy away. I wanted to bury my head in the sand and push the very possibility that this could be happening far from my mind. This protective instinct contributes to what I find to be a less-than-accepting climate surrounding the drug's side effects. But, every medication has side effects — an all-too-familiar situation for the chronically ill. 

I heavily researched Trikafta in the years prior to its approval by the FDA for my son's age bracket. The clinical trials were astounding and this medication does wonders for so many with CF. However, I surprisingly noticed a pretty significant difference in the direct patient testimonials and experiences shared on social media compared to the reports in the clinical trials. As time went on, I began to hear more stories of adverse reactions and intense side effects, and I started to grow increasingly concerned. Trikafta was allegedly causing significant psychological effects in some. With my son being just a toddler, I was terrified about these possible effects on his developing brain. Being faced with heavy decisions comes with the territory of being a CF mom, and the weight of that responsibility never gets lighter. However, we really felt like we owed it to our son to try Trikafta. The promise of a longer, better life is all we want for him, and it was worth the risk. 

Nothing in medicine is one-size-fits-all, and that is especially true with a disease like cystic fibrosis. Our care team is phenomenal and has been the biggest asset to us in navigating my initial hesitation about such a novel drug as Trikafta.  
 

I knew from the beginning we wanted to start our son on a lower dose. Lower doses have been found to be highly effective and produce great clinical outcomes, and we were supported by our care team to proceed with this approach. The purpose (the way I saw it) of starting low and slow, was to mitigate some of the side effects the drug might produce. We also switched the morning and evening doses, after the initial reactions we saw.

Now, I know I don't have to preface our experience with this, but I do think it is helpful. Our son is such a beautiful soul. His temperament is uncharacteristically calm for a toddler, he is smiley and bright, and the happiest boy. Of course, he has big toddler feelings, as they all do, but my goodness he is a shining star in our eyes. Seeing that light be stolen little by little in the two months following his first dose of Trikafta was demoralizing. I don't think one can ever really understand how hard it is to be forced to choose between a promised dream of added years versus your child's emotional well-being.

Within the first week, we started to notice an increase in crying, and measurably so. He went from being happy and content to having inconsolable outbursts and sobbing for large portions of the day. He became aggressive, and I could tell he was struggling so much. It affected his ability to eat, play, and explore the world the way a toddler is meant to. My sweet boy was stressed and erratic. As a second-time mom, I have experienced the lovely ins and outs of a 2-year-old’s emotions. What we were experiencing was absolutely and undeniably not normal. While the effects eventually became unbearable for him, it was largely cumulative. We noticed some of the effects early on, but they were tolerable at first. As time went on, things worsened and progressed, and we knew we couldn't put him through these side effects any longer.

Our care team had us document a baseline behavioral survey before starting Trikafta, and we established a personalized baseline of sorts. I am endlessly grateful that our team recognizes that patients' experiences are valid and legitimate.

Behavioral side effects in a 2-year-old cystic fibrosis patient are inherently complex. The nature of this age results in even the most obviously abnormal behavioral disturbances being met with, “Is it just typical toddler behavior?” A mama's gut instinct is real, especially when it comes to our medical kiddos. Even the climate around Trikafta, as intimidating as it may feel, will not prevent me from sharing what happened to my son. After two intense months on the drug, we made the decision to pull him off of Trikafta.

I didn't even realize how much Trikafta took from him until I saw him come off of it. He was filled with joy again, he talked more, his curiosity returned; he no longer spent large portions of the day crying. It’s like a dark fog lifted off of our baby — and our family. I felt guilty for not pulling him off of it sooner, but when the effects were gradual, it was easy to second guess.
 

Of course, we were sad that Trikafta was not our son's miracle, but more than anything we were glad to have our happy boy back. Bellamy not being able to tolerate Trikafta would have been my absolute worst nightmare if you asked me two years ago. This experience has given us a new perspective, however, and further echoes the fact that about 10% to 15% of the CF community can't take these miracle medications at all or can’t tolerate them. We know how lucky we are to have Orkambi® to fall back on, and the promise of new modulators in the pipeline for our son in a few years. CF has a strange way of making me feel so grateful, but also so desperate for better.

Interested in sharing your story? The CF Community Blog wants to hear from you.

This site contains general information about cystic fibrosis, as well as personal insight from the CF community. Opinions and experiences shared by members of our community, including but not limited to people with CF and their families, belong solely to the blog post author and do not represent those of the Cystic Fibrosis Foundation, unless explicitly stated. In addition, the site is not intended as a substitute for treatment advice from a medical professional. Consult your doctor before making any changes to your treatment.

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Topics
Caring for a Child With CF | CFTR Modulators
Carolanne Cimino

Carolanne is a Midwest crunchy mama who planted roots in Nebraska with her fiancé and their two boys. She is a stay-at-home mom, and motherhood has always been her calling. Carolanne is a health food lover, and you can frequently find her and her family cooking and enjoying healthy dishes together. In addition, she loves connecting with the CF community, and you can follow her journey as a medical mom on Instagram.

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