Multiple biologic treatments safe, effective in children with asthma

Key takeaways:

• Omalizumab reduced exacerbations by 93% over 6 years.
• Dupilumab significantly improved quality of life.
• Tezepelumab, mepolizumab and benralizumab also offer benefits.

WASHINGTON — Physicians have a variety of safe and effective biologic choices for treating children with asthma, according to a presentation at the American Academy of Allergy, Asthma & Immunology Annual Meeting.

“This has revolutionized how we manage asthma,” Eric Schauberger, DO, PhD, assistant professor, University of Wisconsin-Madison, said during his presentation. “We now have access to these fantastic medications.”

These treatments address high type 2, eosinophilic, allergic and non-type 2 asthma alike, he continued.

“Really what’s new here, though, is the long-term safety and efficacy data that has now been trickling down from all these extension studies in the so called ‘real life, real world’ uses of medications,” Schauberger said.

In fact, Schauberger said that the safety of these medications is a common topic discussed among physicians and families, such as whether these biologics are safer than just taking steroids.

Calling it the “granddaddy of them all,” Schauberger said that omalizumab (Xolair; Genentech, Novartis) targets children age 6 years and older with severe persistent asthma whose symptoms have been inadequately controlled with inhaled corticosteroids (ICS).

These patients also have had a positive skin test or in vitro reactivity to a perennial aeroallergen, he said.

Noting a 2023 study from Spain, Schauberger said that omalizumab reduced exacerbations by 96% and decreased the direct costs of asthma by 93% over 6 years in children with severe pediatric asthma.

“However, the drug is not free,” he said, adding that omalizumab increased costs by a factor of 2.5 to three, although overall the treatment was cost-effective due to the reductions in exacerbations.

Savings in year 1 with omalizumab included 2,107 euro per avoided exacerbation, 2,059 euro per 0.5-point improvement in Asthma Control Questionnaire score and 3,141 euro per three-point improvement in Childhood Asthma Control Test score.

Schauberger also noted that the study did not account for attributable costs such as missed work and school, which would have added to the biologic’s cost-effectiveness.

Studies of dupilumab (Dupixent; Regeneron, Sanofi), which targets patients aged 6 years and older with moderate-to-severe asthma characterized by an eosinophilic phenotype and dependence on oral corticosteroids, also yielded positive results, Schauberger said.

For example, the Liberty Asthma Voyage study found reduced exacerbations in children aged 6 to 11 years with type 2 asthma whether or not they had evidence of allergic asthma, with similar trends in changes in their lung function.

“Then there was significant improvement in asthma control observed in patients with evidence of allergic asthma, but not in those without,” Schauberger said. “So even if you’re not allergic, it still does help, but not as much.”

A post hoc analysis of the Liberty Asthma Voyage study also found significantly improved quality of life for these children and their caregivers.

“We also take care of the parents, the families as well,” Schauberger said.

In the Liberty Asthma Venture study, dupilumab led to reductions in asthma exacerbation rates and improvements in lung function, asthma control and health-related quality of life in adults and adolescents who did or did not have evidence of allergic asthma.

“So, a lot of good data basically showing definitely some improvement in not only managing the asthma outcomes from the biomarkers we’re looking at, but also in the quality of life of the patients and their families,” Schauberger said.

Approved in 2021, tezepelumab (Tezspire; Amgen, AstraZeneca) treats patients aged 12 years and older with severe asthma that has been inadequately controlled even with high ICS doses combined with other treatment for maintenance.

Adolescents and adults with severe, uncontrolled asthma in the Destination study tolerated treatment with tezepelumab well and experienced reductions in their annualized asthma exacerbation rates over 104 weeks, compared with placebo.

“That’s 2 years of good data there,” Schauberger said.

Additionally, Schauberger cited mepolizumab (Nucala, GSK) for children aged 6 years and older with severe eosinophilic asthma and benralizumab (Fasenra, AstraZeneca) for children aged 12 years and older with severe asthma and an eosinophilic phenotype, although the FDA has since approved benralizumab for children aged 6 years and older as well.

Considering these choices, Schauberger said that “choosing the appropriate medication on typing is, no doubt, very important.”

“As we get more data, we’re going to figure out which of these medications is going to work best for the patients,” Schauberger said.