You are here

Personalized medicine in advanced cystic fibrosis

Edward F Mckone

Summary

 

Clinical trials in patients with cystic fibrosis typically include patients with mild to moderate lung disease as determined by percent predicted FEV1 (ppFEV1). With improvements in ppFEV1 being the most common clinical trial primary endpoint, the rationale is that patients with very mild cystic fibrosis might not show a ppFEV1 change as disease burden is low and there might be a ceiling effect if lung function is in the normal range. Likewise, those with severe cystic fibrosis lung disease might not show a change in ppFEV1 because of irreversible lung fibrosis leading to fixed airflow obstruction.

Search form


E-Alert

Sign up to the CF Resource Center e-mail alert and enter a raffle to win the Elsevier Text book “Clinical Respiratory Medicine”


Visit the Journal Website


What do you think?

Please take a moment to fill out our reader survey.

Read more