New Health Challenges in the Post-CFTR Modulator Era

The remarkable progress in CF care has also revealed new challenges. As individuals with CF live longer, clinicians are seeing higher frequencies of complications and other down- stream health concerns that rarely emerged in the past and are now focused on aging-related diseases and maintaining their overall health.

Karen McCoy, MD, pediatric pulmonologist and renowned expert in cystic fibrosis, describes the shift in clinical concerns “For many years, everything we did was aimed at keeping children with CF from losing weight. Now, for some patients, we’re counseling about obesity. That is a sea change.”

Improved nutritional status and decreased energy expenditure appear to be driving weight gain in some patients. Pregnancy is also becoming increasingly common, having risen from 200 to 300 per year between 2010 and 2019 to over 600 per year since 2020, explains Dr. McCoy.

“We’re seeing more young women with CF planning pregnancies or becoming pregnant because they finally feel well enough to do so,” Dr. McCoy says.

She notes that with longer life expectancy also comes conditions that historically emerged later, such as diabetes and cardiovascular disease. But the complication with the most import is colon cancer.

“Colon cancer is significantly more common in people with CF than in the general population,” emphasizes Dr. McCoy. “As a result, we begin screening decades earlier than we would in individuals without CF. For patients who have undergone a lung or other solid-organ transplant and require immunosuppression, we start even earlier, often in their 20s, because the immune system plays a critical role in protecting against cancer.”

Clinicians are now updating care models to meet the needs of a generation experiencing both unprecedented health and newly emerging risks, as scientists continue to develop new insights and potential new treatments.

This article appeared in the 2026 Spring/Summer issue. Download the issue here.

Image credit: Nationwide Children’s