Preparing for Trikafta

With the FDA approval of Trikafta for kids 2-5 years old, our daughter will be starting the medication earlier than expected. She turns six in July so we were already on track to start, but we will happily take the additional time, even if it's just a few weeks.

Lots of prep work was involved to get us to this moment. At her most recent CF clinic appointment, she did additional blood work and will have to continue doing that quarterly during the first year she is on Trikafta to monitor her liver levels.

How we got started with Trikafta

We met with our clinic pharmacist, who went over how the drug works, dosage instructions, ways it differs from her current modulator (for example: different medicines for the am/pm due to the functionality of the modulator, different procedures in case we forgot to take the medicine at night or in the morning) and ways they are similar (must be taken with fat, both within a similar 10-12 hour time frame, and she will initially take it as a powder).

Additionally, our social worker sent us a mental health questionnaire to fill out prior to Margo beginning the medicine. With any medicine, there are many side effects, and with Trikafta, some issues with anxiety presented themselves with adults. Because kids ages 2-5 already experience a wide range of emotions and often aren’t able to explain in words how they are feeling, this will give our health care team some qualitative data should any issues arise. If we notice any adverse effects, physical or emotional, we would discuss them with our team and decide to either lower the dose or go off Trikafta and return to Orkambi, which she is currently doing well on. I really appreciated this part of our conversation. Because of how excited the CF community is for Trikafta, ourselves included, I think having to go off of it would be extremely disappointing. However, after talking to our pharmacist about it, I feel informed and will be alert. I wouldn’t want Margo to experience any adverse side effects, particularly anxiety, so knowing that going off of Trikafta wouldn’t be “failing,” particularly since she has responded so well to Orkambi. I have high hopes this won’t be the case.

My husband and I have been traveling so we will order and start the medicine once we return and feel settled back into our routine. Trikafta also comes in a tablet form once she turns six but they are bigger than her pancreatic enzymes so we want to practice with her on how to swallow larger pills.

Very hopeful

After we found out about the approval and started prepping for her to begin, I thought about something that happened in 2019, shortly after the medicine was approved for adults. We were visiting NYC and we were at a playground in Central Park— Margo was two years old at the time. I overheard two moms talking about a new drug for cystic fibrosis that was just approved and how it would be life changing for those who could start taking it. I am naturally curious and was being extremely cautious since our kids were playing in close quarters at time (I wasn’t sure if one of their children had CF), so I introduced myself and explained my daughter had CF. One of the women said her husband has CF and he just received and took his first dose of Trikafta. It was a truly life-changing moment for him and their family and I was blown away by her story. We both got teary eyed— she was truly joyful and so hopeful for him and for Margo. In the busyness of our lives, I forgot about that moment, so remembering it brought a familiar sense of joy and hope for all the 2-5 year olds now eligible to start the potentially life-changing drug.