Prenatal therapy for cystic fibrosis improves lifelong health outcomes

This Cystic Fibrosis Awareness Month, Children's Hospital Colorado (Children's Colorado) is highlighting a growing shift in care: starting cystic fibrosis treatment before birth to improve lifelong health outcomes and reduce hospital stays. Through a highly coordinated, multidisciplinary approach, Children's Colorado's Breathing Institute and Colorado Fetal Care Center teams have cared for 10 families through prenatal therapy for patients showing signs of cystic fibrosis in utero. To date, no other healthcare system in the United States has cared for this number of patients prenatally, positioning Children's Colorado among the most experienced of its kind in the world.

The 10 babies born with Cystic fibrosis (CF) following prenatal treatment at Children's Colorado had early signs of meconium ileus, a serious bowel blockage that often requires immediate surgery after birth. Those that received early, prenatal intervention resolved this blockage prior to delivery and showed significant signs of improved pancreatic function. As a result, neonatal intensive care unit (NICU) stays were significantly reduced compared to patients who did not receive prenatal treatment and were born with the bowel blockage.

CF is a genetic disease that affects the lungs, digestive system and other organs. It causes the body to produce thick, sticky mucus that can clog the lungs and pancreas, making it difficult to breathe and absorb nutrients. Twenty years ago, people with CF had an average life expectancy of 36. Today, children born with CF are expected to live into their 60s, reflecting steady progress driven by earlier intervention and improved treatments, like ETI therapy.

ETI therapy is a cystic fibrosis transmembrane conductance regulator modulator made up of three medications. The combination of medications improves the movement of salt and water in cells, reducing the buildup of thick mucus. At Children's Colorado, care teams are expanding access to this therapy during pregnancy, allowing treatment to begin before a baby is born.

Prenatal treatment gives families an option they've never had before: the ability to take action during pregnancy. Instead of waiting to respond to symptoms after birth, we can start supporting a baby's health earlier and partner with families every step of the way."

Michael Zaretsky, MD, director of research, Colorado Fetal Care Center

Additionally, researchers and clinicians have found exposure to treatment before birth and in early infancy supports healthier cell function during a critical window of lung development. Babies exposed during this period have shown lower‑than‑expected sweat chloride levels - a well-established marker linked to lung health. Continuing treatment after birth may help preserve those gains during infancy, when lungs are especially vulnerable to inflammation and infection.

"In cystic fibrosis, lung damage often begins very early in life," said Jordana Hoppe, MD, medical director of the pulmonary clinic at Children's Colorado. "ETI therapy has given our patients the option to shift their child's CF care from reactive to preventative, offering the incredible potential for lifelong breathing benefits."

Prenatal treatment also allows care to continue seamlessly after birth. ETI therapy can be passed through breast milk until an infant is able to take the medication independently.

Approximately 1,000 people are diagnosed with CF annually, and 75% of those diagnoses are confirmed before age two. Since the severity of CF increases over time, early intervention is key to helping infants and toddlers manage their symptoms into adulthood. The results of prenatal ETI therapy demonstrate a new and innovative care model available to families at risk for CF. Children's Colorado remains dedicated to this multidisciplinary approach and research collaboration on a local and national level to enhance the clinical experience and patient outcomes.