The Latest Treatment Approval for Pediatric Pulmonary Fibrosis

Pulmonary fibrosis in children is a serious and often unrecognized condition that currently has no treatment. But researchers at Children’s Hospital Colorado are changing that.

In the United States, OFEV (nintedanib) is clinically approved for use in adults with pulmonary fibrosis. Unlike some conditions, pulmonary fibrosis involves the expertise of many disciplines, bringing specialists from radiology, pulmonology, rheumatology and pathology together to find a treatment option. Our multidisciplinary team was part of a groundbreaking trial that not only laid the groundwork for future complex trials, but also potential treatment for children with pediatric pulmonary fibrosis.

As a lead enrollment site, Children’s Colorado welcomed children from around the country to Colorado to participate. In this double-blind placebo-controlled international trial, pulmonologist and lead coordinator for the trial Robin Deterding, MD, worked alongside radiologist Jason Weinman, MD, and pulmonologist Emily DeBoer, MD, and providers in over 50 countries, to get OFEV approved for pediatric patients in Europe. It was a true team effort with Drs. DeBoer and Weinman developing much needed protocols for diagnosing pediatric patients.

"Dr. DeBoer and I have worked and continue to develop diagnostic criteria on imaging as wall as educate radiologist and clinicians on the importance of recognizing and reporting these findings," Dr. Weinman says.

“This trial allows us to understand children with this rare disease rather than trying to apply the results from studies that have been performed in adults with pulmonary fibrosis," Dr. DeBoer says.

Although the Federal Drug Administration (FDA) requires more investigation to approve in the U.S., the European Medicine’s Agencies (EMA) approved OFEV for treatment of pulmonary fibrosis in children.

“For our first trial, I'm very proud of what we accomplished,” Dr. Deterding says. “It moved the field forward significantly. With Jason and Emily's work by defining pulmonary fibrosis, we now have pediatric radiologists around the world that can say, ‘this child has pulmonary fibrosis’ and give them a diagnosis.”

In the past, pulmonary fibrosis wasn’t readily diagnosed, but with this successful trial, not only are children receiving the diagnosis they deserve, but there’s treatment available to them.

“It created new definitions around the world that are going to impact children and increase the recognition of this very significant disease process with high morbidity and mortality,” Dr. Deterding says.