‘Now I will be able to become a doctor’

Nikki Scheen (13) has cystic fibrosis. A comparison of her life before and after June 2015 couldn’t be starker, both for Nikki, her two younger sisters and her parents. They’re all still adjusting to the situation.

“Our social life always used to be fairly low-key”, says Anne-Martine, Nikki’s mother. “Birthdays, parties and suchlike were never really our thing. The risk of Nikki catching a bug somewhere and getting ill was simply too great. And besides, she was usually too tired for anything like that anyway. Feeling a little under the weather, like a normal person would, is not something Nikki ever experienced. The last time she was ill was early last year, and it took her months to recover from that.”

Nikki: “I used to be tired all the time. I went to school tired, arrived back home tired, went to bed tired and woke up tired. I could never really do very much. Weekends used to be my time to get some rest. During holidays we could only ever do one activity a day, anything more than that would have been too tiring.”

“Nikki not only had low energy, she frequently used to be ill as well. The standard procedure was admission to hospital with high doses of antibiotics. The mucus build-up in the lungs caused by CF is an ideal environment for bacteria to multiply. And due to the defective mucus production and clearance mechanisms, coupled with the generally poor health all CF patients have, it can take a really long time to recover from such infections.”

We’re looking forward to new and even better CF drugs becoming available

In June of last year, Kalydeco became commercially available. This drug has helped about 5 percent of CF patients. Nikki is one of those 5 percent. Her lung capacity has improved from 50 percent to 90 percent. “One day, Nikki ran outside into the garden. That was on the third day after starting the treatment. We watched our girl jumping on the trampoline with our mouths open. Was that really our daughter?”

“We’re looking forward to new and even better CF drugs becoming available. Nikki is doing well at the moment, but what will things be like 10 years from now? Also, the efficacy of the drugs varies from patient to patient: there are almost 2,000 different genetic mutations, and no drug can undo the damage in the form of scar tissue your body develops over the years.”

“As a kid, all you know is what you’re used to and what your life is like. CF and all the associated discomforts used to be Nikki’s reality. Seeing her go about her life like a normal 13-year-old is such a huge change. And a permanent change, too. Only now is she starting to think about the things she likes and wants. Meeting friends after school, going to parties. ‘Now I WILL be able to become a doctor’, she told us recently. And for our family as well the impact has been tremendous. I can work away from home now, and we can start building a social life. Next summer we’re all going to Sri Lanka. Who would ever have thought that?”