#NACFC2022 – Trikafta Patients Report Better Quality of Life
About 25% of patients said they experienced a deterioration in mental health
People with cystic fibrosis (CF) tend to report improvements in health-related quality of life associated with treatment with Trikafta. Reports of worse mental health were common, however, and were identified as an area of need.
That’s according to data shared by Aricca Van Citters, from The Dartmouth Institute for Health Policy & Clinical Practice, at the 2022 North American Cystic Fibrosis Conference (NACFC). The study, “Highly-effective modulator therapies: Impact on the well-being of people living with cystic fibrosis and implications for the CF care model,” was funded by the CF Foundation.
CF is caused by mutations that impair the function of CFTR, a protein that helps regulate the movement of water and salt molecules into and out of cells. CFTR modulators are a recently developed class of therapies that can improve the protein’s function in those with CF caused by specific mutations.
Clinical trials have shown CFTR modulator treatment can improve clinical measures in eligible CF patients. A team of scientists in the U.S. focused on how one modulator therapy — the triple-combination treatment Trikafta (elexacaftor/tezacaftor/ivacaftor) — affects patients’ health-related quality of life (HRQoL).
Working with the CF Foundation, the team distributed surveys to adults with CF and caregivers of children with the disease. The surveys, co-designed by patients, caregivers, clinicians, and researchers, were distributed in the summer and completed by more than 1,125 people. Among the respondents, 72% were adults and 28% were caregivers of children with CF. The vast majority (95%) were white, 61% were female.
About two-thirds of the patients were taking CFTR modulators at the time of the survey. Most were specifically on the triple combination therapy Trikafta. An additional 10% had taken a modulator in the past but weren’t currently on one, while the remaining 26% had never been on modulator therapy.
I don’t feel the ‘sick’ person anymore; I can enjoy life and not constantly cough and be aware of everyone looking at me, especially now in the time of [COVID-19]
Adults with CF and taking Trikafta scored better on physical and mental HRQoL compared to those who’d previously taken, were not on a modulator, or were on a different modulator (namely Orkambi, Symdeko, or Kalydeco). The differences were of at least 3 points, the threshold of a clinically meaningful difference.
Children with CF on modulator therapy had better global well-being compared with those who had never been on modulator treatment or who previously had. Caregivers of children on modulator therapy also reported that the children had fewer problems in daily life due to fatigue or pain.
Those on Trikafta reported improvements on multiple domains of well-being — physical well-being, experience of care, social well-being, and overall health.
“Physical and social well-being and burden of care have improved on [Trikafta],” Van Citters said.
Mental health changes with Trikafta
Some patients on Trikafta reported improvements in mental health aspects such as body image, sleep difficulties, and having a sense of optimism.
“I don’t feel the ‘sick’ person anymore; I can enjoy life and not constantly cough and be aware of everyone looking at me, especially now in the time of [COVID-19],” one participant said.
About one-quarter of those on Trikafta reported a deterioration of mental health, however. The domains showing deterioration included anxiety, agitation, thinking clearly, stress, body image, and depression.
“Increased anxiety, uncertainty about how I will cope with and use the extra time that I seem to have been given,” one participant said. “[I have] no patience for anyone. I just blow up … Patients should be made aware of this. It should be discussed in clinic before you start,” another said.
Mental health “was the second most frequently mentioned area of need,” Van Citters said. Also mentioned as areas of need were financial and insurance issues, as well as the importance of finding a cure for CF.
The survey’s findings lay the groundwork for research priorities, future policy efforts, and communications, according to Van Citters.
Trikafta, Orkambi, Symdeko, and Kalydeco all are sold by Vertex Pharmaceuticals, which was not involved in this study.
Note: The Cystic Fibrosis News Today team is providing in-depth coverage of the 2022 North American Cystic Fibrosis Conference (NACFC) Nov. 3-5. Go here to see the latest stories from the conference.