40% of patients on CFTR modulators see inhaled medicines as less important

About 40% of people with cystic fibrosis (CF) taking CFTR modulators, a class of medication used to treat the genetic disorder, consider inhaled medicines less important after starting these meds, according to a U.K. survey.

Many people attributed this change to improved health, fewer respiratory symptoms, or a lack of noticeable benefits from inhaled medicines. Others welcomed the opportunity to reduce the time and effort spent managing their disease.

By contrast, just more than half of respondents continued to view inhaled medicines as equally or even more important, citing their ongoing benefits and the desire to maintain their health over time.

“The impact of [CFTR modulators] on the perceived importance of inhaled medicines is variable amongst [people with CF],” researchers wrote. “Whilst population-level evidence regarding the clinical impact of reducing inhaled medicines regimens for [people with CF] taking [CFTR modulators] is awaited, clinicians should be aware of the variation in patient perspective during the process of shared decision-making and individualisation of care.”

The study, “Perspectives on inhaled medicines in the CFTR modulator era: A national survey of adults with cystic fibrosis,” was published in the Journal of Cystic Fibrosis.

CFTR modulators have transformed CF care

CF is caused by mutations in the CFTR gene, which trigger thick, sticky mucus to build up in the lungs and other organs, leading to breathing difficulties, recurrent infections, and progressive lung damage.

Treatment for CF traditionally involves several daily therapies, including inhaled medicines that help thin and clear mucus, as well as inhaled antibiotics to treat persistent or recurrent lung infections. Although effective, these treatments can be time-consuming and burdensome.

The introduction of CFTR modulators, particularly the oral triple-combination therapy Kaftrio (elexacaftor, tezacaftor, and ivacaftor), sold as Trikafta in the U.S., has transformed CF care. By boosting the function of the faulty CFTR protein, these treatments have substantially improved patients’ health, raising questions about the continued need for established inhaled therapies.

Previous research found that people taking Kaftrio who had relatively well-preserved lung function could stop certain inhaled mucus-thinning medicines without experiencing a short-term decline in lung function. However, the long-term effects remain uncertain, and some patients have already reduced their inhaled meds without consulting their care teams.

‘Since taking Kaftrio, I don’t cough’

To better understand patients’ perspectives, researchers surveyed 414 people with CF, ages 16 and older, receiving care at 17 U.K. adult CF centers between July 12 and Aug. 15, 2024. Most were prescribed CFTR modulators (83.1%) and/or inhaled medicines (77.5%).

The online survey explored how important participants considered inhaled medicines on a scale from zero to 100, whether their views had changed since starting modulators, and what influenced those perceptions.

Overall, participants prescribed CFTR modulators rated inhaled medicines as less important than those not prescribed modulators, with median scores of 72 versus 88 out of 100, respectively.

Among those prescribed CFTR modulators, 40.1% of participants considered inhaled medicines less important than before treatment, while 48.3% viewed them as equally important and 2.3% as more important. The remaining 9.3% did not answer.

When I stop taking [inhaled medicines] for over 5 days, I can feel my chest getting tight and I can’t breathe so well.

For many, the shift reflected improvements in their health, including less coughing, reduced sputum (phlegm) production, and fewer respiratory flare-ups.

“Since taking Kaftrio, I don’t cough … I feel fitter than ever,” one participant said. “In the past I used [Pulmozyme (dornase alfa)] to help clear my chest. There is no longer anything to clear.”

Others questioned the need for medicines that no longer seemed to offer noticeable benefits or preferred to use them only when feeling unwell. Some also viewed CFTR modulators as a more effective, convenient alternative to inhaled therapies.

“I feel there are maybe better ways to treat patients in modern ways that are cheaper and more practical for the patient to carry out around normal life,” one respondent said.

By contrast, some participants continued to experience clear benefits from inhaled medicines, particularly those with existing lung damage or ongoing infections.

“When I stop taking them for over 5 days, I can feel my chest getting tight and I can’t breathe so well,” one respondent said.

Some view CFTR modulators as addition to existing treatments

Some also distinguished between treatments, questioning the need for mucus-thinning medicines while continuing to value inhaled antibiotics. Others viewed CFTR modulators as an addition to existing treatments rather than a replacement. Advice from CF care teams and concerns about long-term health further reinforced the importance of continuing treatment.

“Kaftrio has made a … significant improvement for my overall well-being,” a patient said. “I am determined to maintain my high level of adherence to both oral and [inhaled] medications to ensure I am as well as possible as an older person with CF.”

Factors respondents said influenced their inhaled medicine use included established treatment routines, perceived benefits, overall health, treatment burden, how they felt after taking their medicines, and relationships with their CF care teams.

“Clinicians should consider these factors when engaging in shared decision-making consultations with [people with CF], whilst an evidence base for the role of inhaled medicines for [people with CF] taking [CFTR modulators] is being established,” the researchers concluded.