Biologics Provide Opportunity to Manage Moderate to Severe Pediatric Asthma, Improve QOL

The health burdens for pediatric patients with moderate to severe asthma are significant, as are the burdens placed on the child’s parent or caregiver, explained experts during a panel at Asembia’s Specialty Pharmacy Summit, held May 2-5 in Las Vegas, Nevada.

Pediatric patients with asthma often present with chest pain or chest tightness, wheezing, and cough, which could be nocturnal, recur seasonally, be in response to a specific exposure, or last more than 3 weeks, explained Carrie Gatzke, PharmD, CSP, manager, outpatient and specialty pharmacy services, Driscoll Children's Hospital - Driscoll Health Pharmacy.

While wheezing is “considered the hallmark” of pediatric asthma, many patients present with just a cough, she noted.

These patients often go to the doctor more, have higher hospitalization rates, and are seen more in urgent care centers, said B. Louise Giles, MD, FRCPC, FAAP, associate professor and director, Pediatric Asthma Program, The University of Chicago. And there is substantial cost, reaching into the billions, for society to manage childhood asthma.

The estimated annual spend for asthma is $6 billion, said Kelly Bridgers Short, PharmD, MBA, BCPS, manager of pharmacy operations, Optum Infusion Pharmacy, and up to 80% of that is for care such as emergency department visits, hospitalizations, and outpatient visits.

While asthma may improve in puberty, it is considered a lifelong disease, and children with uncontrolled asthma will have ongoing asthma as they age, Giles explained. Whereas boys do tend to see their asthma improve a little, girls’ asthma gets a bit worse at the onset of puberty. Still, asthma will require lifelong medications and all the costs associated with management.

For children, asthma can lead to missed school days in addition to health care costs. Nearly 14 million school days are missed each year due to asthma, said Bridgers Short. These school days often translate to absenteeism from work for caregivers who need to stay home when the child stays home.

As a result, the indirect costs of pediatric asthma are much higher than the adult direct costs, said Bridgers Short.

Although asthma mortality has declined, the United States is still not doing as well in this area as other countries, such as Canada, the United Kingdom, and Australia, said Giles. Where she practices, in the South Side of Chicago, there is high morbidity and mortality for patients with asthma and “it’s not unheard of for us to hear about deaths due to asthma.”

Patients who have persistent uncontrolled asthma despite high-dose inhaled corticosteroids and oral therapies might be candidates for biologic therapy. So far, there are no biologics approved for children under the age of 6 years. There are 3 FDA-approved biologics for children 6 years and older with moderate to severe asthma: dupilumab, mepolizumab, and omalizumab.

The approval for dupilumab was based on findings from the Liberty Asthma VOYAGE trial, which compared dupilumab with standard of care. The trial found that children with high eosinophilic counts had a reduction in symptoms and patients had improved lung function.

“So, I have to say that this is a preference of mine, if I have a child who's got lower lung function, to try to get that up,” said Giles.

Patients on dupilumab receive a loading dose followed up by doses either every 2 or 4 weeks. These doses can be self-administered at home, so the child doesn’t have to go into a medical facility to receive the injection. Common adverse events are injection site reactions, eosinophilia, and an increased risk for viral respiratory tract infections.

Mepolizumab was approved as an add-on maintenance therapy for patients with severe asthma. The common adverse effects include headache, injection site reaction, back pain, and fatigue, explained Gratzke. The therapy is delivered via subcutaneous injection and given every 4 weeks for patients between the ages of 6 and 11 years.

Finally, omalizumab was the first biologic that came to market, and it was approved for moderate to severe persistent asthma. Giles noted that with all the trials looking at omalizumab, there hasn’t been evidence that there is improved lung function. “But, certainly, there has been evidence to show that there's a decreased burden of asthma symptoms with this group,” she said.

There is a high risk of developing anaphylaxis and hives with omalizumab injections. As a result, up until recently, omalizumab had to be given in a facility that could deal with an anaphylactic reaction. Giles noted that there has been talk about trying to get omalizumab as an at-home treatment like dupilumab, but “the jury's still out whether a lot of clinicians will go down that route because of the risk of anaphylaxis in those kids.”

During the question-and-answer portion, one attendee asked about how to overcome resistance from caregivers who don’t want to administer biologics. Giles noted that fear is often the reason caregivers don’t want biologics. One way for practitioners to overcome this is by having a good relationship with the child and the caregivers and being honest about the adverse effects.

The discussion around biologics can often bring up adherence issues or proper inhalation technique, she added. When biologics are mentioned, parents might say that they’ll do a better job, “and then you know that adherence is a problem,” Giles said.

However, the introduction of biologics presents a more personalized treatment plan to address the cause of damage and inflammation. She said dupilumab was a “game changer” because of the improved lung function that hasn’t been seen with other biologics.

“Certainly, the biologics have the potential for those who qualify to have better asthma control, therefore, they have an improved quality of life and less burden of disease on what they do from a day-to-day basis,” Giles said.