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Personalized medicine in advanced cystic fibrosis
Edward F Mckone
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.