Improving lung function, survival seen in CF in Europe over 10 years

But most gains were in wealthier countries, with new treatments, study finds

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Between 2011 and 2021, improving lung function and better survival rates were consistently seen among people with cystic fibrosis (CF) in Europe, particularly in wealthier countries and with the introduction of new treatments, according to a study that tracked more than 45,000 CF patients across the continent over the decade.

However, urgent action is needed to address disparities in treatment for CF and related disease outcomes, especially in lower-income countries where such improvements have not been observed, the researchers noted.

“This pan-European analysis of [patient registry] annual data … demonstrates a consistent improvement in pulmonary function … and survival over the last decade, which began even before … highly effective [disease-modifying therapies] were available,” the scientists wrote.

However, the team noted that “a less optimistic picture was observed in low-income counties” where “improvement in disease outcomes … was only minimal.”

The study, “Cystic fibrosis in Europe: improved lung function & longevity: reasons for cautious optimism, but challenges remain,” was published in the European Respiratory Journal.

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Availability of Kaftrio tied to noticeable improvements in patients

While the prognosis for people with CF has improved over recent decades, the genetic disorder remains a chronic condition, and patients will generally require lifelong care to help them stay healthy and manage disease symptoms and complications.

For many people with CF, the disease prognosis is closely linked to how well their lungs are working. To see how lung function varies across different economic levels within Europe, a team of researchers from throughout the region drew on data from the European Cystic Fibrosis Society Patient Registry. The registry includes data from CF patients from more than 40 countries.

The team collected data annually from 2011 to 2021 from a total of 47,621 people with CF, ages 6 to 60, from across the continent. The researchers then divided those nations based on their gross national income per capita, a measure of wealth based on a country’s final income in a year divided by its population.

Over the decade through to 2021, there was a consistent improvement in lung function, as measured by an increase in forced expiratory volume in one second (FEV1), which is the maximum amount of air that can be breathed out in one second, among patients. These benefits were seen even in individuals with chronic infection with Pseudomonas aeruginosa bacteria, CF-related diabetes, or undernutrition.

In 2021, when Kaftrio (elexacaftor, tezacaftor, and ivacaftor; sold as Trikafta in the U.S.) was already available, the improvement was even more noticeable. However, it was limited to those who are eligible for treatment — that is, those who carry at least one copy of the disease-causing F508del mutation. 

That year, nearly half (about 42%) of people with CF carried two copies of F508del — the most common CF-causing mutation. Another quarter of patients (about 26%) carried one copy of F508del and a minimal function mutation, or a mutation in which the resulting CFTR protein works minimally. A further 15% carried at least one residual function mutation.

“The notable improvement observed [in people with CF] carrying the F508del mutation emphasizes the need to develop treatments for all CF mutations,” the researchers wrote.

Over the years, the number of adults with CF increased by about 60%, from 13,312 in 2011 to 21,168 in 2021. They also became older; this was particularly true in countries with middle to high gross national income, where the number of adults with CF almost doubled.

“Together with the improved overall survival probability according to age and year of follow-up,” the researchers wrote, “this is a sign of increased longevity of [people with CF] starting already before [Kaftrio] was available.”

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Researchers call for improved CF services in low-income countries

In lower-income countries, lung function did not improve as much over the years. Moreover, there was a “sharper decline” of FEV1 as people with CF became older. The sharpest increase was seen in middle- to high-income countries between 2020 and 2021.

“It is interesting to see that among children there is a gradual increase of FEV1 values, without big differences between 2020 and 2021, which is likely due to lower percentage of pediatric [patients with CF] taking [Kaftrio],” the researchers wrote.

For comparison, the researchers noted that, in 2021, nearly half of people with CF in middle- to high-income countries were being treated with Kaftrio. Meanwhile, in low-income countries, except Greece, the use of this medication was “almost null,” the team wrote.

National and international advocacy efforts can raise awareness among policymakers and mobilize support for improving CF services in low-income countries. … Empowering individuals with CF and their families through education and support programs is vital.

This suggests that while progress is being made in wealthier European countries, urgent action is needed in poorer countries where similar access to treatment and improvement in outcomes is not being seen.

“The results of the current study should inform health authorities of countries with disparities in health outcomes. Increasing awareness and education about CF among healthcare providers, policymakers, and the public is essential,” the researchers concluded.

The team called for immediate action to be taken to bring awareness to the effects of poor socioeconomic status on patient outcomes in CF.

“National and international advocacy efforts can raise awareness among policymakers and mobilize support for improving CF services in low-income countries,” the researchers wrote, adding that “empowering individuals with CF and their families through education and support programs is vital.”