North American Cystic Fibrosis Conference

North American Cystic Fibrosis Conference

Source:

Merlo C, et al. Poster 178. Presented at: North American Cystic Fibrosis Conference; Nov. 2-5, 2021 (virtual meeting).

Disclosures: Merlo reports receiving funding from Vertex Pharmaceuticals.
November 09, 2021
1 min read
Save

Ivacaftor has long-term impact on survival, lung transplant in patients with cystic fibrosis

Source:

Merlo C, et al. Poster 178. Presented at: North American Cystic Fibrosis Conference; Nov. 2-5, 2021 (virtual meeting).

Disclosures: Merlo reports receiving funding from Vertex Pharmaceuticals.
You've successfully added to your alerts. You will receive an email when new content is published.

Click Here to Manage Email Alerts

We were unable to process your request. Please try again later. If you continue to have this issue please contact customerservice@slackinc.com.

Patients with cystic fibrosis with CFTR gating mutations aged 6 years and older treated with ivacaftor had lower rates of mortality, lung transplant and pulmonary exacerbations over a mean follow-up of 6 years, according to new data.

“There have been studies in the past using the U.S. CF Foundation Patient Registry, the UK CF Registry and the CF Registry of Ireland that have looked at clinical outcomes over a shorter period of time,” Christian Merlo, MD, MPH, associate professor of medicine and epidemiology at Johns Hopkins University School of Medicine, said during a presentation at the North American Cystic Fibrosis Conference. “This current study was designed to evaluate the effect of ivacaftor over a longer time period with a larger number of study subjects using the U.S. CF Foundation Patient Registry by comparing people with cystic fibrosis treated with ivacaftor to a comparator cohort of people with cystic fibrosis not eligible for treatment with ivacaftor.”

Lungs
Source: Adobe Stock.

The study included 736 participants (mean age, 20.2 years; 48% women) with cystic fibrosis and with CFTR gating mutations who initiated ivacaftor (Kalydeco, Vertex Pharmaceuticals) from January 2012 to December 2021. These participants were matched with 733 participants with cystic fibrosis (mean age, 20.2 years; 47.5% women) with F508del mutation and a minimal function mutation who were ineligible for ivacaftor.

Maximum follow-up was 7.9 years and mean follow-up was 6 years.

During follow-up, patients in the ivacaftor group had a higher probability of survival and remaining free from lung transplant compared with the comparator group. Patients in the ivacaftor group had a lower adjusted hazard of overall mortality (HR = 0.22; 95% CI, 0.09-0.45) and lung transplant (HR = 0.11; 95% CI, 0.02-0.28) compared with the comparator group.

Researchers also observed higher percent predicted FEV1 (84.04 vs. 76.02), BMI (24.30 kg/m3 vs. 22.92 kg/m3) and BMI z-scores (0.31 vs. –0.03) in the ivacaftor group compared with the comparator group.

In other results, the also ivacaftor group had a 50% reduction in all-cause hospitalization rates and a 51% reduction in pulmonary exacerbation rates.

“This study adds to the growing evidence supporting the long-term impact of CFTR modulation with ivacaftor on survival and clinical outcomes,” the researchers concluded.

Reference: