Life Expectancy of CF Children Surpasses Age 50 in UK for 1st Time

Marta Figueiredo, PhD avatar

by Marta Figueiredo, PhD |

Share this article:

Share article via email
self-screening tool | Cystic Fibrosis News Today | illustration of researcher taking patient information

People with cystic fibrosis (CF) in the U.K. are now living longer, with the life expectancy of children born today surpassing 50 years for the first time, according to a newly published U.K. CF Registry annual data report.

The report covered 2020, and the researchers noted: “In this extraordinary year, we saw that people with CF are living longer lives.”

One “unequivocal positive” for patient treatment, and potentially for increased life expectancy, was the introduction, late last year, of the modulator therapy Kaftrio (ivacaftor/tezacaftor/elexacaftor), the investigators said.

“Although it’s too early to clearly measure the long-term impact, there are positive markers already that we hope to see report upon with firmer evidence in future reports,” Rebecca Cosgriff, director of data and quality improvement for the Cystic Fibrosis Trust, said in a press release.

Recommended Reading
CFTR modulators | Cystic Fibrosis News Today | illustration marking rare disease patients

Lung and Nutritional Health, Like CF Mutation, Can Affect Lifespan

Sponsored and hosted by the trust since 2007, the U.K. CF Registry collects demographic, treatment, and clinical data from consenting people with CF, with a goal toward improving patient health.

It does so by helping patients and their families understand the disease and make informed decisions, providing relevant data to clinical and research teams to improve care, and monitoring the safety and effectiveness of new treatments.

The report, available in an “at-a-glance” two-page handout and a 92-page full version, “covers a year of challenge and change for people with cystic fibrosis, dominated by the impact of the COVID-19 pandemic and the initial stages of the roll-out of Kaftrio,” David Ramsden, CEO of the Cystic Fibrosis Trust, said in the report.

“In this different year, the way in which people with CF used health services changed, with fewer in-person clinic attendances and traditional annual reviews recorded than normal (2020 91.6%, 2019 96%),” Ramsden said.

Access to public inpatient services fell by around 40%, the data show, while the proportion of virtual appointments recorded increased significantly from 4% in 2019 to 42% in 2020.

As such, whether and to what extent these changes in health services contributed to some of the report findings remain unclear. Future reports will help clarify whether 2020 trends continue or not.

According to the report, the number of CF patients in the U.K. slightly increased to 10,837 in 2020 from 10,655 in 2019 and their median age remained 21 years — a mark first reached in the prior year. In 2010, the median age was 18.

Notably, “for the first time, the median predicted survival age for children born today has surpassed 50 years,” Cosgriff said, with Siobhán B. Carr, MD, chair of the registry’s steering committee, adding that the increased life expectancy “appears to now show a progressive improvement.”

The actual life expectancy figure, 50.6 years, was based on data collected between 2016 and 2020, which also showed that male patients are expected to live six more years than their female counterparts — a median of 53.1 versus 47 years. There are now 145 people over the age of 60 living with CF in the U.K., the report showed

The 97 people with CF who died in 2020 had a median age of 36 years.

Similar to 2019 data, 30% of CF patients were diagnosed via newborn screening, by age 1. Individuals younger than 16 received a CF diagnosis at a median age of 22 days, and 14.7% of adults in the registry were diagnosed at age 16 or older.

The proportion of patients using mucus thinners grew to 40.3% in 2020 from 11.3% in 2010 for hypertonic saline or Bronchitol (mannitol), and to 69.6% from 42.7% in 2010 for Pulmozyme (Dornase alpha, or DNase).

During 2020, there was a pronounced reduction in the number of patients both evaluated for and receiving lung transplants, relative to the trends seen in prior years. Particularly, transplant evaluations were reported for 175 people in 2020 and 241 in 2019, with 12 patients receiving a bilateral lung transplant in 2020 versus 49 in the previous year.

This greater drop in lung transplants may be due, in part, to the availability of Vertex Pharmaceuticals’ Kaftrio “initially becoming available for people with severe disease on a managed access agreement followed by a UK wide roll out in late 2020,” Carr wrote in a steering committee summary included in the full report.

Recommended Reading
exhaustion, shame, Banner for

My Life (and Death) Is More Than Statistics and Dead Dreams

Kaftrio, known as Trikafta in the U.S., is an approved CFTR modulator for CF that works by correcting the function of the faulty CFTR protein in CF.

Early access to Kaftrio was supported by the U.K. CF Registry, which monitored its real-world impact; it was rolled out to more than 2,700 patients in the fall of 2020.

The report also showed a reduction in the rates of chronic Pseudomonas aeruginosa infection in people ages 16 and older, from 39.4% in 2019 to 31.9% in 2020. However, this drop, according to Cosgriff, “might be because it wasn’t possible to do as many tests for it [in 2020] as in previous years.”

In addition, the median age of patients with chronic P. aeruginosa in 2020 was 31 years, compared with 25 years in 2010; 91.4% of adults with such infection were on inhaled antibiotic therapy relative to 78.8% in 2010.

Moreover, 39.2% of CF patients received at least one course of into-the-blood (intravenous) antibiotics during this year, which reflects a 5.3% reduction from 2019, when the rate was 44.5%. While this is consistent with a trend in reduction over the last 10 years, this pronounced drop “was likely influenced by U.K. government COVID-19 lockdowns and less respiratory infections circulating,” Cosgriff added.

A total of 217 CF patients (113 males and 104 females) in the U.K. were reported to have COVID-19 last year, with one-quarter of them having been hospitalized. Five were admitted to intensive care units and less than five were thought to have died from the virus.

“Like much of what happened in 2020, figures in this report must be viewed in context of a year when the ‘normal’ rules of life did not apply,” Cosgriff noted.