Mental health of concern for many with CF, despite CFTR modulators
CF Foundation survey finds 23% of patients noting mental well-being as issue
While physical health and life quality gains can be evident in people using CFTR modulators, a group of disease-modifying therapies for cystic fibrosis (CF), their mental well-being needs more attention, according to the Wellness in the Modulator Era (Well-ME) study.
Nearly 1 in every 4 patients reported poorer mental health despite treatment, findings that may help “lay the groundwork for establishing future research priorities, policy efforts, and communications in areas that improve well-being,” the researchers, all based in the U.S., wrote.
The study, “Wellness in the modulator era: An observational study of the impact of CFTR modulator therapy on the well-being of people with cystic fibrosis,” was published in the Journal of Cystic Fibrosis.
900 adult patients and caregivers of children with CF responded to survey
CF is caused by mutations in the CFTR gene that lead to no or faulty production of the CFTR protein. This protein is important for controlling the movement of salts and water in and out of cells. When it doesn’t work as intended, thick mucus builds in organs and disease symptoms result.
CFTR modulators, designed to correct the faulty CFTR protein and widely available, have brought unprecedented clinical benefits to patients. However, not much is known about how these treatments affect overall well-being.
In a 2022 survey conducted by the CF Foundation, 580 adults and 320 caregivers of children with CF were asked questions including their modulator use, health status, care experiences, daily life, and emotional, physical, and social well-being. A goal was to determine if differences in wellness existed among patients currently on a CFTR modulator and those who had stopped taking or had never taken a CFTR modulator.
Compared with 2022 data from the CF Foundation patient registry, which the researchers used to check how well survey respondents represented the broader CF community, patients in the Well-ME study were older — mean age of 29.7 years, compared with 24.3 in the registry — and more likely to report poorer lung function or having had a lung transplant.
Consistent with registry data, 665 (74%) respondents were currently taking a CFTR modulator, mostly Trikafta, a triple combination of elexacaftor, tezacaftor, and ivacaftor; 51 others (6%) were no longer using a CFTR modulator, and 184 (20%) had never used a modulator.
Hospitalizations, serious infections more common when modulator not in use
Dose adjustments were common, with 127 (19%) having changed the dose in their initial prescription. Nearly two-thirds (65%) of patients who stopped treatment did so due to side effects, and half (50%) of those never prescribed a CFTR modulator were not eligible for the treatment.
Patients currently or no longer taking a CFTR modulator were older than those who had never used such a medication. Those no longer or never on a modulator were more likely to have been admitted to the hospital or to require intravenous (into-the-vein) antibiotics to treat an infection in the previous year than those currently on such treatment.
Adults using a CFTR modulator reported better physical health, according to the Patient-Reported Outcomes Measurement Information System (PROMIS) Global 10 physical health scores, compared with those who had stopped or never taken such a medication. They also had better PROMIS mental health scores than did patients never using a modulator.
Caregivers reported similar health-related quality of life regardless of whether the children were currently on treatment, had stopped, or had never taken a CFTR modulator, suggesting the effect on children’s well-being is less clear.
Anxiety, depression, cognitive issues were chief mental health concerns voiced
Most patients on a CFTR modulator reported better overall health (85%), quality of life (79%), and physical well-being (69%) since starting treatment. Nearly half (47%) also reported a more favorable care experience (47%).
Both people still on or previously using a modulator “reported positive experiences focused on physical well-being … improved lung function or breathing, including reductions in coughing, sputum production, and … improved physical symptoms or functioning, such as energy, sinus pain, and ability to exercise,” the researchers wrote.
Negative physical experiences reported by current or previous users ranged from weight gain to gastrointestinal issues like bloating and constipation.
While most patients (77%) considered that modulator treatment “met expectations,” nearly one-quarter (23%) reported a worsening in mental well-being since starting treatment, and about 20% identified fears associated with daily life or financial needs, such as out-of-pocket medical expenses.
Common negative mental health experiences noted by current or previous users were “anxiety, depression, and cognitive issues” like memory problems, the researchers stated.
Future hopes voiced by one-third of respondents focused on improved care and physical well-being (overall health), followed by 25% who voiced hopes for a better daily life, financial well-being (employment or attending school), or social well-being (starting a family).
“To our knowledge, our study is the first to show that perceptions of physical and mental [health-related quality of life] (as defined by PROMIS-10) among [adults with CF] taking modulator therapies … are similar to the US general population,” the researchers concluded, and its “findings provide evidence of the potential positive impact of modulator therapies on well-being in adults.”
But “financial and mental well-being remain significant challenges … with up to a quarter of all respondents reporting worsening mental health and 16% reporting worsening financial well-being,” they added.
In addition, “more support may be needed for those not using or ineligible for modulator therapy,” the researchers wrote.
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